Archive for the ‘Gene Therapy Research’ Category

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As researchers around the world race under immense pressure to develop a COVID-19 vaccine, a controversial approach could potentially help get them there faster but it's incredibly risky.

The process is known as a human challenge study, and it involves intentionally infecting willing volunteers with the coronavirus that causes COVID-19 in order to test the effectiveness of potential vaccines and treatments against it.

"These are very powerful studies that could make a difference, especially since we don't know a lot about the novel coronavirus," said Seema Shah, a medical ethicist at Northwestern University and Lurie Children's Hospital of Chicago.

"They could clarify what's happening in infections with people who are not symptomatic and people who have more severe disease even if they don't have underlying conditions that put them at higher risk."

Shah said COVID-19 human challenge studies could also help identify people who have developed immunity to the coronavirus, while also helping to narrow down the growing list of potential vaccines and treatments for patients.

"If there were a couple of vaccine candidates that had gotten through safety testing and there was a question about which one of those vaccines was more likely to work, a human challenge study could be a quick way to pick the best vaccine of the candidates," she said.

"It could also be useful to study whether the vaccine itself causes different kinds of harm."

The WHOsays any potential vaccine is still at least a year away, but human challenge trials could accelerate theprocess because of the time they save in the clinical trial phase.

Typically, researchers inject thousands of study participants with a vaccine or placebo and wait for symptoms to develop an approach that can take months or years after vaccine development.

Human challenge studies instead vaccinatea small group of people and then intentionally infectthem with the virus, saving critical research time, especially during a global pandemic.

But despite the potential benefits of a controlled human challenge study on COVID-19 patients, experts say the controversial approach is an ethical minefield that could have disastrous consequences if not handled carefully.

Human challenge studies typically recruit young, healthy volunteers in an effort to keep the risk of severe medical complications low.

Thousands of potential volunteers have already pledged to participate in human challenge trials on a website called 1DaySooner, but no such studies are yet underway.

Yet given what we know and don't know about the different ways in which COVID-19 attacks the human body even in young, healthy people, how do we effectively inform participants who may be unknowingly putting themselves at severe risk?

"We know that younger people tend to tolerate COVID-19 as an illness better, but what would worry me about that is there's a lot that is still unknown," said Kerry Bowman, a bioethicist and professor of global health at the University of Toronto.

He said the potential for COVID-19 patients of all ages to face long-term health implications and even death from a virus we still know so little aboutcalls into question how truly informed participants could be on the risks of a human challenge study.

"Do you truly have an informed decision?" he said. "You have consent, but is itreally well-informed? Do people fully understand? Because if we don't understand the virus itself, I wonder about the quality of informed consent that you can ask of people."

In a new paper published in the journal Science Thursday, Shah and a team of international researchers outline an ethical framework for how human challenge studies could be effectively used to combat COVID-19.

The researchers supportdeveloping a "challenge strain" of the coronavirus a stabilizedversion of the one thatis circulating worldwide to potentially infect participants, but stopped short of advocating for the work to proceed.

"The pandemic has affected just about everyone in the world in various ways, so the potential amount of social value is unprecedented here," Shah said.

"That's why our group concluded it's really important to give challenge studies a hard look and potentially invest in laying the groundwork for doing them.

"But then make that judgment call about whether and how to do them at a later date when they're ready to go."

The World Health Organization released specific criteria this week outlining its recommended approach to conducting human challenge studies, without advocating for or against them.

Among those recommendations is a need for "strong scientific justification" for the studies, ensuring that the potential benefits outweigh risksand that the selection of participants should be done with "rigorous" informed consent.

"The overarching, really important one is to minimize risk to participants as much as possible," said bioethicist Dr. Ross Upshur, of the University of Toronto's Dalla Lana School of Public Health, who helped work on the WHO guidelines.

"You need to make sure that people understand what's being proposed:what they're going to be doing;how they're going to be managed in this situation;how their safety and their well-being is going to be protected.

"But it's also incumbent on the researchers to outline all of the uncertainties,because we may not be able to actually quantify some of those risks."

Timothy Caulfield, a Canada Research Chair in health law and policy at the University of Alberta who has researched human challenge studies, said those ethical dilemmas have historically plagued this approach.

"I understand the desire to use human challenge trials, especially in this context, because people are desperate to get a vaccine quickly, not just for clinical reasons, but also for economic and social reasons," he said.

"So the pressure is intense, but the exact reason that we have research ethics guidelines is to protect research participants."

Caulfield said the damage that could be done if a human challenge trial were to go awry would be devastating.

He points to the ethics scandal involvingJesse Gelsinger, a teenager who died in a clinical trial for gene therapy in 1999, as an example of a failed human study that set the research ethics field back immensely.

Gelsingerhad a genetic disease called ornithine transcarbamylase (OTC) that he controlled through diet and medication, but after enrolling in the trialhe was injected with an experimental therapy and died a few days later.

"Just imagine the impact that it could have on vaccine research, especially in this environment where there's so much uncertainty," Caulfieldsaid.

"If it doesn't go well and if we cut corners on research ethics standards, it could end up backfiring and being really problematic."

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Could intentionally infecting volunteers with COVID-19 help find a cure sooner? - CBC.ca

Global Gene Therapy for Inherited Genetic Disorder Market statistical report provides a wide-ranging research on the key players and in-depth insights which includes the competitiveness of the trending players. This Market research report that evaluates its current value, size, performance and statistics. The report is an important dynamic of the market and gives an idea of the types, the process, and value chain that has been included in the report.

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BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, and Spark Therapeutics.

Key questions answered in this report:

Geographically, regions such as North America, Europe, Asia-Pacific (APAC), Middle East & Africa and Latin America can be segmented on the basis of the global Gene Therapy for Inherited Genetic Disorder Market. Market drivers, restraints, and opportunities have been evaluated to explain the anticipated nature of investments and its impact on the global market in terms of future prospects. The report provides qualitative as well as quantitative researched data of the global Market.

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Table of Contents

Global Gene Therapy for Inherited Genetic Disorder Market Research Report

Chapter 1 Gene Therapy for Inherited Genetic Disorder Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Continue TOC

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How COVID-19 Impacting On Gene Therapy for Inherited Genetic Disorder Market Trends, Drivers, Strategies, Segmentation Application with Top Key...

SDMR International recently published a report on the Global Viral Vectors and Plasmid DNA Manufacturing Market, which offers an in-depth overview of the factors that are impacting the state and progress of the worldwide business. The research report incorporates all the vital data that will help you updated with the latest market trends and get ahead in the competition in the various market segments and the leading geographies studied in the report. The study gives valuable insights into the future progress of the market and all essential aspects of the Viral Vectors and Plasmid DNA Manufacturing market for the forecast period from 2020 to 2026.

The Viral Vectors and Plasmid DNA Manufacturing Market research report gives the latest market information, highlighting the product range and services offered in the market. The report provides key facts and figures relating to the market status, size, share, and growth aspects of the Viral Vectors and Plasmid DNA Manufacturing industry. The research comprises of elaborate profiles of the leading companies operating in the global sector by taking into account their gross revenue, total sales, market share, and competitive landscape.

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Scope of the Report:The all-encompassing research weighs up on various aspects including but not limited to important industry definition, product applications, and product types. The pro-active approach towards analysis of investment feasibility, significant return on investment, supply chain management, import and export status, consumption volume and end-use offers more value to the overall statistics on the Industrial Antifungal Agents market. All factors that help business owners identify the next leg for growth are presented through self-explanatory resources such as charts, tables, and graphic images.

Prominent players profiled in the study:BioRelianceCobra BiologicsOxford BioMedicaUniQureFinVectorMolMedMassBiologicsRichter-HelmFUJIFILM Diosynth BiotechnologiesLonzaAldevronEurogentec

Market Synopsis:

The report includes details about important products, revenue, production, and the business of top industry players. The report helps the companies to understand the threats and challenges in front of the businesses. This is a well-established, and precisely formulated report acknowledges major Viral Vectors and Plasmid DNA Manufacturing industry vendors, key regions, demand & supply, applications, technology, revenue cost, and challenges. According to the report, it will be expected to gain a healthy CAGR by 2020-2026. Additionally, it focuses on manufacturing analysis including the raw materials, cost structure, process, operations, and manufacturing cost strategies. Sub-segments are the report covered along with the clear definition, sales, value, market share, volume, market competition landscape, SWOT, and development plans during the forecast period.

In market segmentation by Types of the Viral Vectors and Plasmid DNA Manufacturing , the report covers the following uses-Plasmid DNAViral Vectors

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Take a look at some of the important sections of the report

Market Overview: Readers are informed about the scope of the global Viral Vectors and Plasmid DNA Manufacturing market and different products offered therein. The section also gives a glimpse of all of the segments studied in the report with their consumption and production growth rate comparisons. In addition, it provides statistics related to market size, revenue, and production.

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Countries mapped in the study:

Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, China, Colombia, Japan, United States, Mexico, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, Taiwan, South Korea, Singapore, India, and New Zealand.

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Covid-19 impact on Viral Vectors and Plasmid DNA Manufacturing Market Research Report Analysis and Forecast till 2026 | Cell and Gene Therapy...

BURLINGTON, Mass., May 05, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) will present positive data from two studies of FX201, an investigational gene therapy for osteoarthritis (OA), at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place virtually May 12-15, 2020. The abstracts were published in the May supplement of Molecular Therapy.

The data from the rodent model served as a basis for the FX201 IND and reinforce our belief that FX201 holds the potential to become a transformative therapy for OA, saidMichael Clayman, M.D., President and Chief Executive Officer of Flexion. Further, we are delighted to be presenting manufacturing studies at ASGCT which demonstrate a capable Good Manufacturing Process (GMP) to support the production of FX201 for our clinical trials.

Establishing the Efficacy, Safety, and Biodistribution of FX201, a Helper-Dependent Adenoviral Gene Therapy for the Treatment of Osteoarthritis, in a Rat Model (Abstract 747)

Flexion assessed the efficacy of HDAd-rat-IL-1Ra, the rat surrogate of FX201, in a rodent surgical model of OA. Efficacy was assessed after a single intra-articular administration one week post-surgery via histopathology at Week 12 using a semi-quantitative microscopic grading system (Osteoarthritis Research Society International [OARSI] score) for OA-related cartilage, synovium, and bone changes. In addition, two Good Laboratory Practice (GLP) studies were performed in a rodent model of OA to evaluate the safety and biodistribution of FX201 and the rat surrogate (a helper-dependent adenovirus vector with a transgene encoding rat variant of IL-1Ra) administered four weeks post-surgery. Key study findings include:

Development of a Highly Productive and Reproducible Manufacturing Process for FX201, a Novel Helper-Dependent Adenovirus-Based Gene Therapy for Osteoarthritis (Abstract 1273)

Using a fit-for-purpose manufacturing process suitable for early development, Flexion successfully produced four batches of drug substance, which will enable GLP toxicology, pharmacology, and GMP clinical studies. Key findings include:

About FX201FX201 is a locally administered gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector, designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of osteoarthritis (OA). By persistently suppressing inflammation, Flexion believes FX201 holds the potential to both reduce OA pain and modify the disease.

About Osteoarthritis (OA) of the KneeOA, also known as degenerative joint disease, affects more than 30 million Americans and accounts for more than $185 billion in annual expenditures. In 2017, approximately 15 million Americans were diagnosed with OA of the knee and the average age of physician-diagnosed knee OA has fallen by 16 years, from 72 in the 1990s to 56 in the 2010s. The prevalence of OA is expected to continue to increase as a result of aging, obesity and sports injuries. Each year, approximately five million OA patients receive either a corticosteroid (immediate-release or extended-release) or hyaluronic acid intra-articular injection to manage their knee pain.

About Flexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis, the most common form of arthritis. The company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking Statements This release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; expected increases in the rate of individuals with OA of the knee; and the potential therapeutic and other benefits of FX201, are forward looking statements. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risks related to clinical trials, including potential delays, safety issues or negative results; risks related to key employees, markets, economic conditions, and health care reform; and other risks and uncertainties described in our filings with theSecurities and Exchange Commission(SEC), including under the heading "Risk Factors" in our Annual Report on Form 10-Kfiled with theSEConMarch 12, 2020and subsequent filings with theSEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contacts:

Scott YoungVice President, Corporate Communications & Investor RelationsFlexion Therapeutics, Inc.T: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor Relations Flexion Therapeutics, Inc.T: 781-305-7137jdowns@flexiontherapeutics.com

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Flexion Therapeutics Announces Virtual Poster Presentations for FX201, an Intra-Articular Gene Therapy Candidate for the Treatment of Osteoarthritis,...

Shares of Orchard Therapeutics fell more than 13% in premarket trading after the company announced a new strategic plan that includes the shifting of its clinical focus, the shuttering of a proposed manufacturing facility in California and the termination of 25% of company staff.

On Thursday, London-based Orchard announced the new plan that will shift the companys clinical priorities away from some of its long-time focuses, including a stem cell gene therapy for the treatment of severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) and other ultra-rare conditions. Instead, Orchard said it will now focus its development priorities on more common conditions that include Crohns disease, a type of dementia and some neurometabolic disorders. Among those are the development of a treatment for metachromatic leukodystrophy (MLD), a rare and life-threatening inherited metabolic disease. Another priority will be its treatment for Wiskott-Aldrich syndrome (WAS), a life-threatening immune disorder. Orchard will also continue to develop its treatments for mucopolysaccharidosis types I and IIIa.

The shifts in Orchards plans were announced a couple of months following a management change. In March, company founder and Chief Scientific Officer Bobby Gaspar took over the helm of the company from Mark Rothera, who led Orchard since 2017.

I feel privileged to lead Orchard as we embark on this new chapter, which is rooted in fulfilling the powerful possibilities for HSC gene therapies beyond ultra-rare diseases, Gaspar said in a statement. Moving forward, we are focusing on advancing therapies for high need and high-value diseases, and our work in neurometabolic disorders is a clear example of this. Were also excited to announce new research programs which we believe will demonstrate the breadth of the HSC platform approach.

Orchard said it intends to adjust its manufacturing plans to focus on the expected regulatory approvals of OTL-200, the MLD treatment and its WAS drug, OTL-103. The company hopes to obtain approval in the European Union for OTL-200 for the treatment of MLD in the second half of 2020 and launch in the first half of 2021. Orchard is also preparing a Biologics License Application for its WAS treatment in both the U.S. and EU in 2021.

Orchard said it also intends to stop its plans to build a gene therapy manufacturing facility in Fremont, Calif. That project was first announced in 2018.

The culling of one-fourth of its employees is expected to save the company approximately $125 million through the end of 2021. That will extend the companys existing cash runway into 2022, Orchard said. Cash, cash equivalents and investments as of March 31, were $263.9 million compared to $325.0 million as of Dec. 31, 2019.

Frank Thomas, Orchards chief operations officer, called the strategic shift and layoffs necessary steps for the company to achieve its objectives by matching our attention and resources to a set of core imperatives for the business.

I believe that these are necessary steps, especially in light of the current environment in which we are operating, with focused investments in areas such as commercial and manufacturing operations supporting the needs we have now without a near-term dependence on the capital markets, Thomas said in a statement.

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Orchard Therapeutics Cuts 25% of Staff as it Shifts Clinical Focus - BioSpace

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO) and Magenta Therapeutics (Nasdaq: MGTA) today announced a research and clinical collaboration agreement to evaluate the potential utility of MGTA-117, Magentas novel targeted antibody-drug conjugate (ADC) for conditioning patients before they receive one of AVROBIOs investigational lentiviral gene therapies.

The collaboration will combine AVROBIOs leadership in lentiviral gene therapies with Magentas expertise in ADC-based conditioning and is expected to further the two companies shared mission to enable patients to live free from disease. Under the collaboration, AVROBIO and Magenta will jointly evaluate MGTA-117 in conjunction with one or more of AVROBIOs investigational gene therapies. Magenta will retain all commercial rights to MGTA-117. AVROBIO will retain all commercial rights to its gene therapies and will be responsible for the clinical trial costs related to the evaluation of MGTA-117 with AVROBIOs gene therapies.

This agreement with Magenta springs from our strategic focus on maintaining technology leadership in gene therapy, said Geoff MacKay, AVROBIOs president and CEO. AVROBIO has always led by investing early in technological innovations that further the field of lentiviral gene therapy, such as plato, our proprietary platform designed to optimize the safety, potency and durability of our investigational lentiviral gene therapies. Were continually assessing new technologies that could be complementary to our plato platform to sustain our cutting-edge advantage and continue to evolve platos capabilities.

We believe targeted ADCs represent the next generation of medicines to prepare patients for gene therapy or transplant in a targeted, precise way. AVROBIOs investigational gene therapies complement our platform as well as our focus and commitment to patients. This partnership will allow Magenta to validate our conditioning platform in lentiviral gene therapy applications, said Jason Gardner, D.Phil., president and chief executive officer, Magenta Therapeutics. Weve selected ADCs as the preferred modality for our conditioning programs, as we believe they offer the most promising option for more patients. We have optimized our ADCs for gene therapy and transplant settings and look forward to collaborating with AVROBIO to evaluate MGTA-117 in specific gene therapy settings. Magenta will continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases.

MGTA-117, Magentas most advanced conditioning program, is a CD117-targeted antibody engineered for the transplant setting and conjugated to amanitin, a toxin in-licensed from Heidelberg Pharma. It is designed to precisely deplete only hematopoietic stem and progenitor cells and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models, suggesting that it may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in humans. Magenta plans to complete IND-enabling studies this year.

AVROBIO currently uses a personalized conditioning regimen with precision dosing of busulfan, an extensively validated conditioning agent generally considered to be the gold standard for ex vivo lentiviral gene therapy, based on decades of general use and administration to hundreds of patients treated with lentiviral gene therapy candidates. The treating clinician uses therapeutic drug monitoring (TDM) to evaluate how quickly the patient metabolizes busulfan and adjusts the dose regimen accordingly with the goal of achieving the optimum result. AVROBIO has reported early clinical data with this precision conditioning regimen with TDM in its own clinical trials, adding to a body of data that suggest busulfan can effectively clear space in the patients bone marrow, where stem cells engraft, produce generations of daughter cells carrying the therapeutic gene and make the functional protein the patient needs to maintain cellular health.

About AVROBIO

Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients. Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com. Follow Magenta on Twitter: @magentatx.

AVROBIO Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, the expected safety profile of our investigational gene therapies, and the potential and expected benefits of MGTA-117, Magentas investigational antibody-drug conjugate, including the ability of MGTA-117 to deplete hematopoietic stem and progenitor cells in order to clear space in bone marrow to support long-term engraftment in humans, as well as MGTA-117s potential application to AVROBIOs investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that AVROBIO may not realize the intended benefit of MGTA-117 with respect to AVROBIOs investigational gene therapies, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Magenta Therapeutics Forward Looking Statements

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including, without limitation, statements regarding the research and clinical collaboration agreement between Magenta and AVROBIO, including the timing, progress and success of the collaboration contemplated under the agreement, the successful evaluation MGTA-117 in conjunction with one or more of AVROBIOs investigational gene therapies under the agreement, the anticipated cost allocation and other commercial terms under the agreement, Magentas strategy and business plan, as well as the future development, manufacture and commercialization between AVROBIO and Magenta. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation, risks set forth under the caption Risk Factors in Magentas most recent Annual Report on Form 10-K, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission, as well as risks, uncertainties and assumptions regarding the impact of the COVID-19 pandemic to Magentas business, operations, strategy, goals and anticipated timelines, including, without limitation, Magentas ongoing and planned preclinical activities, ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, timelines for regulatory submissions and financial position. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200506005265/en/

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AVROBIO and Magenta Therapeutics Announce Collaboration to Evaluate Targeted Antibody-Drug Conjugate as a Potential Conditioning Regimen for...

Rockville May 9, 2020 (Thomson StreetEvents) -- Edited Transcript of Regenxbio Inc earnings conference call or presentation Thursday, May 7, 2020 at 8:30:00pm GMT

* Kenneth T. Mills

REGENXBIO Inc. - CEO, President & Director

* Patrick J. Christmas

REGENXBIO Inc. - Senior VP & Chief Legal Officer

REGENXBIO Inc. - Senior VP & Chief Medical Officer

* Vittal K. Vasista

REGENXBIO Inc. - Senior VP & CFO

Chardan Capital Markets, LLC, Research Division - Director of Research & Head of Healthcare Research

SVB Leerink LLC, Research Division - MD of Genetic Medicines & Senior Research Analyst

Good afternoon, and welcome to the REGENXBIO First Quarter 2020 Earnings Conference Call. (Operator Instructions) As a reminder, this conference call is being recorded. I would now like to turn the call over to Mr. Patrick Christmas, Senior Vice President and General Counsel for REGENXBIO. You may begin.

Patrick J. Christmas, REGENXBIO Inc. - Senior VP & Chief Legal Officer [2]

Good afternoon, and thank you for joining us today. With us are Ken Mills, REGENXBIO's President and Chief Executive Officer; Dr. Steve Pakola, our Chief Medical Officer; and Vit Vasista, our Chief Financial Officer.

Earlier this afternoon, REGENXBIO released financial and operating results for the first quarter ended March 31, 2020. The press release reporting our financial results is available on our website at http://www.regenxbio.com.

Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development plans. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend and other words of similar meaning. Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties. These risks are described in the Risk Factors and the Management's Discussion and Analysis sections of REGENXBIO's annual report on Form 10-K for the full year ended December 31, 2019, and comparable sections of REGENXBIO's other filings, which are on file with the Securities and Exchange Commission and available on the SEC's website.

Any information we provide on this conference call is provided only as of the date of this call, May 7, 2020. And we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially.

I would now like to turn the call over to Ken.

Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [3]

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. On today's conference call, we'll provide a recap of our recent progress, advancing and expanding the NAV Technology Platform as well as expected future milestones. Steve will provide an update on our clinical programs, and Vit will provide an update on the financial results for the first quarter of 2020. Then we'll open the call for questions.

First, I want to take a moment to say that I hope everyone is healthy, staying safe during the COVID-19 pandemic. At REGENXBIO, we've made some changes to our business operations in order to support the health and safety of our employees and the community, and we're fortunate that we've been able to successfully advance our business during this time. As always, and especially throughout the past few months, our overall focus remains on the important goal of improving lives through the curative potential of gene therapy, and I'm grateful that our team is dedicated to this pursuit even in these challenging times. Along these lines, we've made important progress in recent months at REGENXBIO as we continue to see the remarkably consistent and durable effects of gene therapy treatment, patients with severe wet AMD.

If you haven't done so already, I highly recommend referring back to our webcast that we hosted on April 22, in which several leading retina specialists joined us to provide their perspectives on our recently announced data. Our RGX-314 gene therapy is designed to enable sustained production of an anti-VEGF antibody fragment in the eye, and we've now demonstrated stable and consistent results out to 2 years in the third dose cohort. We believe this is the longest time line of continuous therapeutic effects demonstrated in wet AMD patients from a single administration of an anti-VEGF treatment.

We also provided additional data from the fifth cohort, which received a higher dose of RGX-314 and in which 73% of patients remain anti-VEGF injection-free 9 months after a onetime administration of RGX-314. In our program, we're thinking carefully of all aspects of clinical management, not just anti-VEGF injections, are cognizant of the variables that might impact patient care and vision. And Steve will provide more details on these results and next steps for the program in his remarks.

Beyond RGX-314, we've continued to drive our internal gene therapy pipeline forward. As we previously announced in February, we presented encouraging initial data at the WORLDSymposium from Cohort 1, the Phase I/II trial for MPS II, and we look forward to providing additional data from these patients in mid-2020. We've begun enrolling patients in Cohort 2, where they're receiving a higher dose of RGX-121 and look forward to providing interim data on Cohort 2 in the second half of 2020.

We anticipate several other important updates this year including from our Phase I/II trial of RGX-501 for the treatment of HoFH, our Phase I/II trial of RGX-111 for the treatment of MPS-I and our RGX-181 program for the treatment of CLN2 disease, as well as our research programs in hereditary angioedema, neurodegenerative and neuromuscular diseases.

Lastly, construction of our GMP production facility here in Rockville continues, and the facility is expected to be operational in 2021. We expect the facility to enable us to strategically scale production while continuing to ensure high quality for patients. So with that, I'll turn the call over to Steve for a clinical and regulatory update.

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Stephen Pakola, REGENXBIO Inc. - Senior VP & Chief Medical Officer [4]

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Thanks, Ken. As you've mentioned, last month, we announced updated data from our Phase I/IIA study of RGX-314 for the treatment of wet AMD. We reported that the gene therapy continued to be well tolerated at all dose levels. And there were no reports of ocular inflammation beyond what is expected following routine vitrectomy. The latest efficacy update was focused on the 2-year data from Cohort 3 of the study. Patients in this cohort received [6 C] 10 genome copies per eye. And now at 2 years after administration of RGX-314, they have demonstrated markedly improved visual acuity and stable retinal thickness as well as significantly reduced need for anti-VEGF intraocular injections and stable protein expression.

50% of patients within this cohort did not receive any anti-VEGF injections over the full 2 years of the study. And one additional subject did not receive injections starting 9 months after the administration of RGX-314. We saw an impressive improvement in visual acuity with an increase of 14 letters in both the full cohort as well as the 4 patients who did not receive anti-VEGF injections in the second year of the study. This improved vision and durability of anti-VEGF activity is particularly meaningful, as real-world evidence has shown us that patients commonly lose vision over time, even with current standard of care.

And finally, Cohort 3 showed consistent protein production over 2 years, giving us confidence that the transduced cells in the retina have been producing the RGX-314 protein at a steady rate throughout the study. We also provided an interim update from Cohort 5. 73% of patients were anti-VEGF injection free over 9 months. We are very pleased with these results and look forward to additional efficacy data at the 1-year time point. We will use this data to then finalize the design of the pivotal program for RGX-314, which we expect to initiate in the second half of 2020. We are also planning to start trials of RGX-314 using the in-office suprachoroidal delivery approach in 2020 and for both wet AMD and diabetic retinopathy. We look forward to providing additional information about these trials over the coming months.

Turning to our rare disease portfolio. Data thus far from our Phase I/II trial of RGX-121 has been encouraging as patients in the first cohort demonstrated consistent and sustained reduction in heparan sulfate in the CSF, and available data support early signs of neurocognitive stability. We look forward to providing additional data from these 3 patients in mid-2020. Meanwhile, enrollment in Cohort 2 at a higher dose level continues and is expected to be complete in the first half of 2020, with interim data expected in the second half of 2020.

Recruitment screening and additional site activations are ongoing in our Phase I/II clinical trial evaluating RGX-111 for the treatment of MPS I. Recruitment in this trial had been previously focused on an initial patient over the age of 18, but the protocol was recently amended to allow enrollment of patients as young as 4 months of age.

We expect to provide a program update in the second half of 2020. We also anticipate updates from our Phase I/II trial of RGX-501 for the treatment of HoFH in the first half of this year. We have several other study programs headed towards the clinic, including the HAE and neuromuscular programs, and I look forward to providing additional details in the coming months. With that, I turn the call back over to Ken. Ken?

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [5]

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Thanks for that summary, Steve. REGENX has an extensive footprint in the gene therapy space, and we're always purposeful in developing partnerships with key players in the space to continue to realize the potential of NAV Technology. Our NAV Technology is currently being applied in 1 marketed product and more than 20 additional partnered product candidates.

We continue to track the positive progress of Novartis' ZOLGENSMA, which uses the NAV AAV9 vector. Novartis has stated that they're treating about 100 patients per quarter in the U.S. based on their current launch. We're encouraged by the success that the Novartis team in reaching patients. I believe that this is among the most successful launches of gene therapy so far and believe that it demonstrates the transformational impact that NAV Technology can have on the treatment of genetic diseases with significant unmet needs.

We were also pleased to see the positive regulatory developments in Japan and Europe this quarter, which signal additional validation of the technology across the globe. And importantly, for the entire gene therapy field, Novartis recently published additional detailed data for the intrathecal delivery of ZOLGENSMA, which has shown improvement in motor function and achievement of motor skills following treatment. I'm excited for the additional progress to come from this program.

In addition, we recently announced another exclusive worldwide license agreement with Ultragenyx, extending our companies' existing gene therapy partnership. This agreement will enable the Ultragenyx team to apply our NAV technology, AAV8 and AAV9 vectors to the development of a new gene therapy for a rare metabolic disorder, and provides further validation of the breadth and depth of our intellectual property portfolio.

Throughout the remainder of this year, we also anticipate regulatory updates from our partners like Audentes Therapeutics, now part of Astellas, for their gene therapy candidate for x-linked myotubular myopathy, which uses our NAV AAV8 vector. The promising milestones and achievements from our partners, as well as the progress in our own internal pipeline, provide additional validation the proprietary NAV Technology platform and further demonstrate the transformational impact that can come from a onetime administration of gene therapy.

With that summary, I want to turn the call over to Vit for a review of our financials.

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Vittal K. Vasista, REGENXBIO Inc. - Senior VP & CFO [6]

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Thank you, Ken. REGENXBIO ended the quarter on March 31, 2020 with cash, cash equivalents and marketable securities totaling $356.6 million compared to $400 million as of December 31, 2019. The decrease was primarily attributable to net cash used in operating activities of $35.6 million and cash used to purchase property and equipment of $4.6 million.

Revenues were $17.6 million for the 3 months ended March 31, 2020, compared to $900,000 for the same period in 2019. The increase was primarily attributable to $10 million of royalty revenue recognized on net sales of ZOLGENSMA in the first quarter of 2020 (inaudible) $7.2 million of license revenue recognized from new licensing granted to Ultragenyx during the period. Commercial sales of ZOLGENSMA commenced in the second quarter of 2019. The REGENXBIO is eligible to receive a milestone payment of $80 million from AveXis upon the achievement of $1 billion in cumulative net sales of ZOLGENSMA. As of the end of the first quarter of 2020, they have reported over $530 million in net sales, so we are more than halfway to that milestone.

Research and development expenses were $37 million for the 3 months ended March 31, 2020, compared to $25.2 million for the same period in 2019. The increase was primarily attributable to personnel-related costs as a result of increased headcount, laboratory and facility costs, expenses associated with conducting clinical trials for our lead product candidates, and externally sourced services for preclinical, regulatory and manufacturing-related activities.

General and administrative expenses were $14.8 million for the 3 months ended March 31, 2020, compared to $11.6 million for the same period in 2019. The increase was primarily attributable to personnel-related costs as a result of increased headcount, and professional fees for advisory and other services.

Net loss was $40 million or $1.08 basic and diluted net loss per share for the 3 months of March 31, 2020, compared to a net loss of $32.2 million or $0.89 basic and diluted net loss per share for the same period in 2019. As of March 31, 2020, we had approximately 37.2 million common shares outstanding.

Based on our current operating plan, we expect the balance in cash, cash equivalents and marketable securities of $356.6 million to fund the completion of our internal manufacturing capabilities and clinical advancement of our product candidates into 2022. With that, I will turn the call back to Ken to provide final thoughts.

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [7]

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Thanks for that update, Vit. So next week is the American Society of Gene and Cell Therapy Conference. And we've announced a number of scientific posters and presentations that will be shared. Our research and development team continues to demonstrate a deep and impressive knowledge in AAV discovery, characterization, delivery and significant experience and expertise in process development, production at large scale. So we look forward to participating with our industry partners, friends and colleagues, highlighting some of the work next week as well as continuing to share new data from our ongoing research throughout 2020.

Finally, as I've worked with our team to continue to pursue our mission for patients, balance the challenges and dealing with the COVID-19 pandemic, I've also reflected more on how the potential gene therapy treatments to help ease the burden on the medical community while also protecting patients, families, caregivers, even larger communities from certain risks involved current treatment options during events like what we're experiencing with this pandemic. There are many patient populations that rely on traveling, regular access (inaudible) medical care facilities for injections or infusions that are important, aesthetically necessary, sight-saving or life-saving medicines. Single administration gene therapy treatments can deliver important alternatives in moments where travel and access is restricted. Health care resources are limited, and product supplies may be threatened. Against the background of a pandemic that's taxing the global health care resources at unprecedented levels, it's even more apparent to me that our single-administration treatments have the potential to also create safer and more robust system for care.

The medical community and the biotech financial community should be doing everything possible to support and expedite single administration treatments, especially for large and at-risk populations. We continue to make strong progress in advancing key programs, broadening our internal care pipeline. After more than a decade of steadfast effort and focus, we remain dedicated and committed to improving lives through the curative potential of gene therapy.

With that, we're happy to turn the call over for questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) And your first question comes from Gena Wang with Barclays.

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Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [2]

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First related to resolution on the latest update in data was really impressive, especially after the update from other competitors. So another thing I wanted to ask you is, regarding the suprachoroidal data and the initiation first half this year, and also the clinical data, first cohort data by the end of this year. Just wondering what kind of data should we expect will you be presenting at the end of this year? Regarding the patient numbers, if we understand correctly, it's 6 patients? And what other data will you be able to share with us?

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Stephen Pakola, REGENXBIO Inc. - Senior VP & Chief Medical Officer [3]

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Gena, Steve here. Thanks for the questions. Yes, as you mentioned, our guidance is we continue to target starting our suprachoroidal delivery development program, first with a wet AMD and to kick that study off by the end of the first half of this year. And we still are guiding towards having interim initial data at the end of the year. We haven't actually disclosed how many patients we have in the study and other details, and we look forward to providing that later. But certainly, we stand by our guidance of giving an update based on some of the data that we'll have as of the end of the year. One of the nice things of the wet AMD indication is we already have a good handle on wet AMD in terms of looking at the appropriate types of endpoints, both anatomic, functional and treatment burden endpoints that we've talked about in the past and very recently with our recent data update in our subretinal delivery program. So we have a good handle on those types of endpoints. So I think you can envision similar types of data readouts.

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Operator [4]

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Your next question comes from Gbola Amusa with Chardan.

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Gbolahan Amusa, Chardan Capital Markets, LLC, Research Division - Director of Research & Head of Healthcare Research [5]

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Congrats as well on the 2-year durability data for 314. I had a couple of questions on the potential for inflammation for your in-office suprachoroidal approach. Obviously, there's a lot of benefit in going in the office, but some would argue there's greater potential for inflammation with that approach. So 1 -- 2 questions is, do you agree with that assessment? And then second, given that we've seen very recent and very early results with intravitreal gene therapy using expanded courses of prophylactic steroids addressing issues, maybe addressing thoroughly issues of inflammation with the (inaudible) capsid and wet AMD and also RP. Do those results go early? Do they motivate you to use an extended course of prophylactic steroids for your SCS microinjector programs? So those 2 questions then very quickly. Novartis just said that there's a multibillion-dollar potential for ZOLGENSMA, again, and this has updated guidance, and you got obviously up to 10% of that. What -- could you cover what they said [invites public forms] on the timing of the ramp towards that guidance?

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [6]

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Steve, maybe I'll let you start with the 314 questions.

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Stephen Pakola, REGENXBIO Inc. - Senior VP & Chief Medical Officer [7]

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Okay. That makes sense. Gbola, that's a great question. Suprachoroidal delivery is a different route of administration than subretinal. Subretinal is the gold standard. That's where we have the most efficacy and safety and clear demonstration that there isn't inflammation associated with subretinal gene therapy. And that's obviously very excited about for our lead program with subretinal delivery. Suprachoroidal, the in-office potential delivery there, where we still are anatomically delivering very close to the target issue of the RPE and the photoreceptors, but you raise a legitimate question. What do we know about immune privilege or lack thereof and the potential for inflammation? Historically, there's been inflammation seen, an immune response with suprachoroidal delivery of earlier generation AAV vectors such as AAV2 and AAV5. One of the things that got us very excited about suprachoroidal delivery with our proprietary NAV technology is actual preclinical data in both small and large animal models, where we've seen no inflammation with suprachoroidal delivery of AAV8 vector including RGX-314 in multiple studies. So that gives us (inaudible) that perhaps there is less of a risk of inflammation with [suprachoroidal] delivery than exists with intravitreal, for example.

So with intravitreal, we know historically -- universally really, with preclinical and clinical experiments, that at the doses that you have to give with either AAV2 or other vectors, that you have to give a high enough dose to have fusion to get to the back of the eye, the target tissue and through the internal limiting membrane barrier, that invariably at doses where you get good transduction, you also see immune-mediated inflammation. And we continue to see that, frankly, validated in any preclinical experiments or clinical data that's come out with intravitreal administration.

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Kenneth T. Mills, REGENXBIO Inc. - CEO, President & Director [8]

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Gbola, just turning to the question about ZOLGENSMA. I think what we've seen most recently between Novartis' update at their last earnings call, and then they had an update around new data that was presented at MDA, is that for the IV route of administration, that's, of course, currently approved and being marketed, facilitating the sales numbers that we're seeing based on the U.S. approval, there's been some regulatory events in Japan and Europe. Specifically, they pointed to that indicate that reimbursement is coming in other jurisdictions as early as the end of the first half of this year. In addition, with respect to the new intrathecal data that we mentioned earlier in the call, that there was an update on the strong study showing improved outcomes as described, and there's been guidance from Novartis that a BLA could be filed or equivalents, I guess, worldwide, as early as the second half of 2020 or into 2021. So we continue to be really encouraged by the data, by what we're seeing in terms of the uptake of the use of gene therapy. Again, I think we're looking at what must mean among, if not the, most successful launch for gene therapy to date and are liking to see the regulatory and commercial milestones that are emerging.

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Operator [9]

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Your next question comes from Mani Foroohar with SVB Leerink.

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Mani Foroohar, SVB Leerink LLC, Research Division - MD of Genetic Medicines & Senior Research Analyst [10]

Follow this link:
Edited Transcript of RGNX earnings conference call or presentation 7-May-20 8:30pm GMT - Yahoo Finance

ReportsnReports always aims at offering their clients an in-depth analysis and the best research material of the various market. This report on the global Regenerative Medicine Market is committed fulfilling the requirements of the clients by giving them thorough insights into the market. An exclusive data offered in this report is collected by research and industry experts team. Regenerative Medicine Market market spread across 137 Pages, profiling 10 companies and supported with tables and figures.

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#Top Key Players in the Regenerative Medicine Market include are Organogenesis Inc. (U.S.), Osiris Therapeutics, Inc. (U.S.), Vericel Corporation (U.S.), Stryker Corporation (U.S.), and NuVasive, Inc. (U.S.). The key players in the acellular products segment are Medtronic (Ireland), Acelity (KCI Concepts) (U.S.), Integra Life Sciences (U.S.), Cook Biotech Inc. (U.S.), and C.R. Bard (U.S.).

On the basis of applications, the segments are orthopedic & musculoskeletal disorders, dermatology, cardiology, diabetes, central nervous system diseases, and other applications. Oncology is the fastest growing application market. Increasing cancer incidence, rich product pipeline and increasing demand for cancer treatments fuelling the growth of the market.

Geographically, the Regenerative medicine market is dominated by North America, followed by Europe, Asia-Pacific, and the Rest of the World (RoW). Growth in the North American segment is primarily driven by rapidly increasing aging population and increase in chronic diseases are the major drivers for developed economies like the U.S.

#Target Audience for Regenerative Medicine Market: Healthcare providers & clinical experts,Service providers,Therapeutic companies,Contract manufacturers,Academic research institutes.

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List of Tables:

Table 1 Few Important Clinical Studies in the Regenerative Medicine: Market

Table 2 Cancer Incidence and Mortality, By Region, 20122035

Table 3 Funding for Regenerative Medicine Research By the Nih Under the 21st Century Cures Act

Table 4 Nih Funding for Regenerative Medicine Research in the Us, 20142016 (USD Million)

Table 5 California Institute of Regenerative Medicine Funding for Regenerative Medicine Research

Table 6 Number of Companies Operating in the Regenerative Medicine: Market, By Region (20142018)

Table 7 Market, By Type, 20172024 (USD Billion)

Table 8 Few of the Commercialized Cell-Based Immunotherapy & Cell Therapy Products

Table 9 Cell-Based Immunotherapy & Cell Therapy Products Market, By Region, 20172024 (USD Billion)

Table 10 Cell-Based Immunotherapy & Cell Therapy Products Market, By Type, 20172024 (USD Billion)

Table 11 Cell-Based Allogeneic Regenerative Medicine

Table 12 Allogeneic Products Market, By Region, 20172024 (USD Billion)

Table 13 Cell-Based Autologous Regenerative Medicine

Table 14 Autologous Products Market, By Region, 20172024 (USD Billion)

Table 15 Few of the Commercialized Tissue-Engineered Products

.and more

The rest is here:
Regenerative Medicine Market by Type [Cell-Based Immunotherapy & Cell Therapy (Allogeneic & Autologous Products), Tissue Engineering, Gene...

Lately, there has been remarkable shift in the therapeutics method. People are inclining towards personalized medication rather than pharmaceutical treatment methods. This is result of advancements in biological therapies. As a result, advanced therapy medicinal products market (ATMP) is emerging. Such products provide solution for conditions with no therapeutic alternatives. This is a key factor driving growth of the advanced therapy medicinal products market across the globe.

At present, regulations for ATMPs is at its budding stage. Extensive research activities has been going on, which is resulting in Investigational New Drug (IND) applications.

The upcoming advanced therapy medicinal products market analysis report provides insight about the upcoming trends and restraining factors likely to shape growth of the market during forecast period (2019-2029). The report also provides a comprehensive analysis of the key companies of the market and offers details about the capacities and competencies of these companies. The market report also focusses on the markets competitive landscape and provides detail of the product portfolio of various companies.

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Advanced Therapy Medicinal Products Market: Competitive Analysis

At present, the advanced therapy medicinal products market is growing at a lucrative rate. This growth rate is attributed to recent approval of various advanced therapy medicinal products. Post success of approved products, stakeholders are investing at enormously in clinical trials of advanced therapy medicinal products.

On the other hand, companies operating in the market are adopting various strategies to accelerate the product manufacturing rate. While most of the companies are relying on in-house production of therapies, few players such as Contract Manufacturing Organizations (CMOs) are opting for third-party service providers.

Upsurge in demand for gene therapy has resulted in widening of drug development landscape and rise in the number of new entrants. However, there is lack of production capabilities.

Also, several companies are strengthening their foothold in the global market by strategic alliances and acquiring small CAR T-cell therapy developers.

Some of the key players operating in the advanced therapy medicinal products market are-

Advanced Therapy Medicinal Products Market: Key Trends

Despite high cost of the products, the market is expanding at lucrative rate. The growth rate is attributed to various health benefits provided by new classes of therapies.

Registering clinical benefits and efficiency of the products, stakeholders in the market are developing new strategies to overcome the challenged and boost application of advanced therapy medicinal products

Advanced Therapy Medicinal Products Market: Regional Outlook

Availability of significant number of FDA approved advanced therapy medicinal products in the U.S. has accounted for the prominent share of North America region, in terms of revenue. Recently, approval of products such as Yescarta, Zolgensma, and Kymriah has led to prominent investment in the U.S. advanced therapy medicinal products market.

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Europe hold second-largest pharmaceutical market space across the globe. In coming years, cell therapy developers are anticipated to hold prominent share in the Europes drug revenue. Moreover, several academic institutes in Europe are conducting extensive research in early-stage cell therapy. This factor is likely to fuel the regional revenue contribution.

Meanwhile, global manufacturing companies operating in the market are enhancing their reach across Europe. This, in turn, may drive growth in the regional market.

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Advanced Therapy Medicinal Products Market: Key Challenges and Winning Imperatives - BioSpace

As the number of thalassemia patients is increasing in India, need for a prevention and control program is felt. With preventive health checks not being the norm in India, people suffering from thalassemia are unknowingly passing on this genetic disorder to their children. The need of the hour is to have a national policy on thalassemia which will help in not just creating awareness about the disease but also ensure strategies to prevent its spread, diagnosis, and treatment for all. Even though we have the highest number of thalassemia patients in South Asia, the promising treatment modalities like bone marrow transplant (BMT) and gene therapy have their own share of risks and shortcomings due to a lack of incentives and support for the research studies.

To save valuable lives, setting up of small or micro blood donation camps or mobile vans can be done following the social distancing norms in the areas of green zones across the country with the help of various welfare organizations. Making appeals through social media platforms to healthy people to come out in numbers and donate blood is one way to create an informed society.

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How COVID-19 is endangering the lives of Thalassemia patients - National Herald

BURLINGTON, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) today reported financial results and recent business highlights for the quarter ended March 31, 2020.

We were very pleased with ZILRETTAs trajectory earlier in the first quarter of 2020; however, in March we began to experience impacts from the COVID-19 pandemic, said Michael Clayman, M.D., President and Chief Executive Officer of Flexion Therapeutics. The pandemic negatively affected our customers ability to treat patients as well as our first-quarter sales, and we believe that COVID-19 will adversely impact revenue for the remainder of the year. That said, our field teams have been utilizing various technologies to engage in conversations with prescribing physicians, and our confidence in ZILRETTAs long-term potential is undiminished. In fact, given the cancellations and postponements of total knee replacement surgeries, we believe ZILRETTA has the potential to play an increasingly prominent role in a post-pandemic environment. However, since that time horizon is uncertain, we have taken meaningful actions to reduce our operating expenses.

Dr. Clayman added, We recently made the strategic decision to discontinue our Phase 2 trial investigating ZILRETTA in shoulder OA and adhesive capsulitis. Given the small number of patients enrolled in that trial, the uncertainty around when we will be able to restart it, as well as the costs required to maintain the study in an inactive status, we believe terminating the trial is the most responsible action at this time. Moving forward, we will look to leverage our learnings from the study and potentially incorporate them into a new trial design to advance ZILRETTA in these indications. We continue to plan to reinitiate the FX201 single ascending dose trial as soon as feasible. Ultimately, we are committed to ensuring Flexion emerges from the crisis in a position of strength and that we are ready to quickly reaccelerate our commercial and R&D activities.

First-Quarter Results & Financial HighlightsThe Company reported a net loss of $36.8 million for the first quarter of 2020, compared to a net loss of $41.5 million for the same period of 2019. Net sales of ZILRETTA were $20.1 million and $10.6 million for the three months ended March 31, 2020 and 2019, respectively. Cost of sales was $2.3 million and $1.8 million for the three months ended March 31, 2020 and 2019, respectively.

Research and development expenses were $21.1 million and $15.4 million for the three months ended March 31, 2020 and 2019, respectively. The increase in research and development expenses of $5.7 million was primarily due to an increase of $2.2 million in salary and other employee-related costs for additional headcount and stock compensation expense, an increase in expenses related to FX201, including the payment of $2.5 million to GeneQuine for dosing the first human patient in the Phase I clinical trial, and an increase in other portfolio expenses, primarily related to the $0.5 million milestone to Xenon Pharmaceuticals, offset by a decrease of $0.5 million in development expenses for ZILRETTA due to lower clinical trial expenses during the period.

Selling, general and administrative expenses were $29.3 million and $32.2 million for the three months ended March 31, 2020 and 2019, respectively. Selling expenses were $20.5 million and $23.8 million for the three months ended March 31, 2020 and 2019, respectively. The year-over-year decrease in selling expenses of $3.3 million was primarily due to a reduction in physician and patient marketing activities. General and administrative expenses were $8.8 million and $8.4 million for the three months ended March 31, 2020 and 2019, respectively, which represents an increase of $0.4 million.

Interest income was $0.4 million and $1.0 million for the three months ended March 31, 2020 and 2019, respectively. Interest expense was $4.7 and $3.9 million for the three months ended March 31, 2020 and 2019, respectively.

As of March 31, 2020, the Company had approximately $125.2 million in cash, cash equivalents and marketable securities compared with $136.7 million as of December 31, 2019.

ZILRETTA Commercial MetricsSince the launch of ZILRETTA in November 2017 through March 31, 2020:

Reductions in Operating ExpensesIn response to the economic and business disruption caused by COVID-19, Flexion undertook prudent and disciplined steps to reduce expenses across the organization. The Company believes these steps will enhance financial flexibility and liquidity and estimates they will deliver between $43 million and $53 million in savings this year.As a result, Flexion expects full-year 2020 operating expenses (including cost of sales, research and development, and selling, general and administrative) will be in the range of $167 million to $177 million.The Company expects to achieve these cost savings through:

ZILRETTA Supply ChainThe Company remains confident in its ability to maintain adequate commercial supply of ZILRETTA and expects its current finished goods inventory will be sufficient to meet demand for at least the remainder of 2020. To avoid excess levels of inventory, Flexion is temporarily suspending manufacturing activities for ZILRETTA. Since Flexion employs a condominium model at Patheons manufacturing site, the Company has the ability to reinitiate manufacturing following three months notice to Patheon once additional supply is needed.

Recent News & Business Updates

Conference CallFlexions management will host a conference call today at 4:30 p.m. ET. A live webcast of the conference call can be accessed through the Investors tab on the Flexion Therapeutics website, and a replay will be available online after the call. For those planning to ask a question, the dial-in number for the conference call is 855-770-0022 for domestic participants and 908-982-4677 for international participants, with Conference ID # 4498458. Please dial in at least 15 minutes in advance to ensure a timely connection to the call.

Indication and Select Important Safety Information for ZILRETTA

Indication:ZILRETTA is indicated as an intra-articular injection for the management of osteoarthritis pain of the knee.

Limitation of Use: The efficacy and safety of repeat administration of ZILRETTA have not been demonstrated.

Contraindication:ZILRETTA is contraindicated in patients who are hypersensitive to triamcinolone acetonide, corticosteroids or any components of the product.

Warnings and Precautions:

Adverse Reactions:The most commonly reported adverse reactions (incidence 1%) in clinical studies included sinusitis, cough, and contusions.

Please seeZilrettaLabel.comfor full Prescribing Information.

About ZILRETTAOn October 6, 2017, ZILRETTA was approved by the U.S. FDA as the first and only extended-release intra-articular therapy for patients confronting osteoarthritis-related knee pain. ZILRETTA employs proprietary microsphere technology combining triamcinolone acetonide a commonly administered, short-acting corticosteroid with a poly lactic-co-glycolic acid (PLGA) matrix to provide extended pain relief. The pivotal Phase 3 trial on which the approval of ZILRETTA was based showed that ZILRETTA significantly reduced knee pain for 12 weeks, with some people experiencing pain relief through Week 16. Learn more at http://www.zilretta.com.

About Osteoarthritis (OA) of the KneeOA, also known as degenerative joint disease, affects more than 30 million Americans and accounts for more than $185 billion in annual expenditures. In 2018, more than 15 million Americans were diagnosed with OA of the knee and the average age of physician-diagnosed knee OA has fallen by 16 years, from 72 in the 1990s to 56 in the 2010s. The prevalence of OA is expected to continue to increase as a result of aging, obesity and sports injuries. Each year, approximately five million OA patients receive either a corticosteroid (immediate-release or extended-release) or hyaluronic acid intra-articular injection to manage their knee pain.

About FX201FX201 is a locally administered gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector, designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of osteoarthritis (OA). By persistently suppressing inflammation, Flexion believes FX201 holds the potential to both reduce OA pain and modify disease progression.

About FX301FX301 is a locally administered NaV1.7 inhibitor product candidate, known as funapide formulated for extended release in a thermosensitive hydrogel. The initial development of FX301 is intended to support administration as a peripheral nerve block for control of post-operative pain. Flexion believes FX301 has the potential to provide effective and durable pain relief while preserving motor function and anticipates initiating clinical trials in 2021.

AboutFlexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of people with musculoskeletal conditions, beginning with osteoarthritis, the most common form of arthritis. The Company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking StatementsThis release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; our expectations regarding expenses for the year endedDecember 31, 2020; expected savings from actions to reduce operating expenses; expected impacts from COVID-19 and the timing and duration of such impacts; Flexions ability to maintain commercial supply of ZILRETTA; long-term potential of ZILRETTA, including following the COVID-19 pandemic; expected clinical developments and clinical trial timelines; expected increases in the rate of individuals with OA of the knee; and the potential therapeutic and other benefits of ZILRETTA and Flexions product pipeline, are forward looking statements. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risk that we may not achieve expense expectations for 2020; the fact that the impacts and expected duration of the COVID-19 pandemic are uncertain and rapidly changing; the risk that we may not be able to successfully maintain an effective sales force to commercialize ZILRETTA; competition from alternative therapies; the risk that we may not be able to maintain and enforce our intellectual property, including intellectual property related to ZILRETTA; the risk that ZILRETTA may not be successfully commercialized or adopted; risks regarding our ability to obtain adequate reimbursement from payers for ZILRETTA; risks related to the manufacture and distribution of ZILRETTA, including our reliance on sole sources of supply and distribution; risks related to clinical trials, including potential delays, safety issues or negative results; risks related to key employees, markets, economic conditions, health care reform, prices and reimbursement rates; and other risks and uncertainties described in our filings with theSecurities and Exchange Commission(SEC), including under the heading "Risk Factors" in our Annual Report on Form 10-K for the year endedDecember 31, 2019filed with theSEConMarch 12, 2020and subsequent filings with theSEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contact:

Scott YoungVice President, Corporate Communications & Investor RelationsT: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor RelationsT: 781-305-7137jdowns@flexiontherapeutics.com

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Flexion Therapeutics Reports First-Quarter 2020 Financial Results and Recent Business Highlights - GlobeNewswire

Oncogenuity, Inc., a Fortress partner company, enters into an agreement with Columbia University to develop a broad platform technology using oligonucleotides

Initial target is KRAS-driven cancers, often considered un-druggable

Platform being explored as a treatment for coronaviruses, including COVID-19

NEW YORK, May 08, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (Fortress), an innovative biopharmaceutical company, today announced that Oncogenuity, Inc. (Oncogenuity), a new Fortress partner company, has entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers. The proprietary platform produces oligomers, now known as ONCOlogues, that are capable of binding gene sequences 1,000 times more effectively than complementary native DNA. The technology comes from the labs of Gary Schwartz, M.D., Division Chief, Hematology/Oncology, and Jeffrey Rothman, M.D., Ph.D., Assistant Professor of Medicine.

ONCOlogues are sensitive to a single base pair mismatch, resistant to degradation and use a proprietary delivery sequence to enter cells. ONCOlogues selectivity enables Oncogenuity to target genetically driven cancers caused by mutations without impacting wild-type (WT) DNA sequences, potentially limiting off-target toxicity. In addition, this allows ONCOlogues to target mutations that have historically been considered un-druggable.

Oncogenuity has established proof-of-concept in a pre-clinical setting for various cancer types. The companys most advanced program is targeting the KRAS mutation G12D, which was previously considered un-druggable and plays a significant role in various cancer types with substantial unmet need, including pancreatic and colorectal. Given the platforms ability to target any mutation, Oncogenuity will continue to evaluate other mutations simultaneously. The company anticipates additional data publications in the coming 12 months.

Additionally, Oncogenuity is exploring the platforms potential to treat coronaviruses. Coronaviruses have single-stranded RNA genomes, making them strong targets for ONCOlogues. The company is studying replacement sequences, which could help combat COVID-19 and provide proof-of-concept as a treatment for coronaviruses. These ongoing experiments would validate ONCOlogues as a possible treatment for COVID-19, as well as potentially expedite the discovery of treatments for future coronavirus outbreaks.

Lindsay A. Rosenwald, M.D., Fortress Chairman, President and Chief Executive Officer, said, We are excited to work with the excellent scientists and physicians at Columbia University again. Our last joint effort with Columbia University led to the formation of our partner company, Caelum Biosciences, Inc. (Caelum). Since formation, Caelum has raised approximately $60 million in development funding from a number of sources, with additional amounts available upon the satisfaction of certain milestones and will be initiating two registration clinical trials in the next several weeks. Building upon our success with Caelum, we are grateful to Columbia University for entrusting us to develop this highly innovative technology using oligonucleotides to target genetically driven cancers and coronaviruses. Using a targeted genetic approach to treat cancer has become essential to limiting toxicity and treating patients effectively. This technology has the potential to target mutations that have previously been considered un-druggable. Oncogenuity will aggressively pursue the development of ONCOlogues to ultimately provide patients with new, safe and effective treatment options.

Scientific Co-Founder Jeffrey Rothman, M.D., Ph.D., said, Through rigorous statistical, mechanical and molecular modeling, combined with gene sequence data, we are able to create sequence-specific, targeted therapeutics against oncogenes, which are the cause of and specific to tumor cells. Until now, achieving this goal had been considered nearly impossible. However, with these novel design features, we now have the ability to target cancer while potentially avoiding side effects, which are the main cause of dose-limitation, by design. There is much potential because we are able to target multiple genes and therefore, multiple cancers. Moreover, due to their single-strand format, application toward viral targets such as in COVID are even more facile given their easier accessibility. We are excited and determined to pursue this endeavor with Fortress Biotech and very much welcome their continued support.

About Oncogenuity, Inc.Oncogenuity, Inc. is a biopharmaceutical company focused on the development and commercialization of ONCOlogues for the treatment of genetically driven cancers and coronaviruses. Oncogenuitys lead asset targets a KRAS mutation, G12D. Oncogenuity is located in New York City and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO).

About Fortress Biotech Fortress Biotech, Inc. (Fortress) is an innovative biopharmaceutical company that was recently ranked number 10 in Deloittes 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, based on percentage of fiscal year revenue growth over a three-year period. Fortress is focused on acquiring, developing and commercializing high-potential marketed pharmaceutical products and development-stage pharmaceutical product candidates. The company has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and at partners it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value while mitigating risk for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is driven by a world-class business development team that is focused on leveraging its significant biopharmaceutical industry expertise to further expand the companys portfolio of product opportunities. Fortress has established partnerships with some of the worlds leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including Alexion Pharmaceuticals, Inc., AstraZeneca, City of Hope, Fred Hutchinson Cancer Research Center, InvaGen Pharmaceuticals Inc. (a subsidiary of Cipla Limited), St. Jude Childrens Research Hospital and Nationwide Childrens Hospital. For more information, visit http://www.fortressbiotech.com.

Forward-Looking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. As used below and throughout this press release, the words we, us and our may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; risks relating to the COVID-19 outbreak and its potential impact on our employees and consultants ability to complete work in a timely manner and on our ability to obtain additional financing on favorable terms or at all; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

Company Contacts:Jaclyn Jaffe and William BegienFortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Fortress Biotech Announces Exclusive Worldwide License Agreement With Columbia University to Develop Novel Oligonucleotide Platform for the Treatment...

Orchard Therapeutics plc (NASDAQ:ORTX) Q1 2020 Earnings Conference Call May 7, 2020 8:00 AM ET

Company Participants

Renee Leck Director-Investor Relations

Bobby Gaspar Chief Executive Officer

Frank Thomas Chief Operating Officer

Conference Call Participants

Whitney Ijem Guggenheim

Esther Rajavelu Oppenheimer

Yaron Werber Cowen

Peter Kim Barclays

Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call. [Operator Instructions] I would now like to hand off the conference over to your speaker today, Renee Leck, Director of Investor Relations. Please go ahead, maam.

Renee Leck

Thanks, Sonia. Good morning, everyone, and welcome to Orchards first quarter 2020 investor call. You can access the slides for todays call by going to the Investors section of our website, orchardtx.com.

Before we get started, Id like to remind everyone that statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, and including those set forth in our annual 10-K filed with the SEC and any other filings we may make. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

And with that, Ill turn the call over to our CEO, Bobby Gaspar.

Bobby Gaspar

Thanks, Renee. Hello, everyone, and welcome. Id like to start by first acknowledging the tremendous efforts of our organization and our partners in the health care field to ensure patients in need continue to receive care during this difficult time. Thank you, everyone.

The last few weeks have been an important period for Orchard. Since taking on the leadership, Frank and I, together with the executive team, have thought very carefully about what the new Orchard can become, how we can ensure that Orchard can fulfill its true potential and what we need to do to make that happen. When we think about our strategic vision as a company, its really all based on the potential of the hematopoietic stem cell gene therapy platform, where it can take us and the benefit it can provide for many patient populations even beyond our current portfolio of ultra-rare diseases. We have taken some bold and decisive actions that we believe will allow Orchard to achieve long-term growth and focus the company on sustainable value creation. This vision is supported by a new strategic plan that we have developed and which is built around four pillars. Each of these forms a chapter in our remarks this morning.

First, operating efficiencies. We have made a series of important changes to our operations that will enable us to sharpen our focus and more efficiently execute our strategy, which I will detail in a moment. Second is our commercial build. We are focused on establishing the right model for the diagnosis and treatment of patients undergoing HSC gene therapy, and see the true value of this approach over a series of ultra-rare products.

Third, one of the most exciting areas in gene therapy right now is the innovation taking place in manufacturing technologies that have the potential to deliver economies of scale. We want to be leaders and invest in this space, knowing that our near-term capacity needs are covered by our experienced CDMO network.

Finally, central to this strategy is prioritizing our portfolio to enable the expansion of Orchards pipeline beyond ultra-rare to less-rare indications. We are disclosing two new research programs for the first time today, and these are a genetic subset of frontotemporal dementia or FTD, and a genetic subset of Crohns disease. We believe that the biological and clinical validation that has already been shown in our ultra-rare indications allow us to expand with confidence to these larger indications.

Turning to the first chapter in our new strategic plan. We are focused on improving the operational efficiency throughout the organization. This started with an extensive evaluation over the past six weeks of each program in our portfolio using several criteria that are shown here on the left-hand side of Slide 5. We undertook an objective analysis that involved both financial metrics and strategic considerations in identifying those programs where there was high need for patients and high-value creation for shareholders. As you can imagine, these were difficult decisions given the potentially transformative nature of many of these programs. Each has value, and we intend to realize that in different ways and over different time horizons.

Today, however, we believe our resources are best focused on Metachromatic Leukodystrophy, Wiskott-Aldrich syndrome, the MPS programs and our research programs. This also means that we have a balanced portfolio with late, mid and early stage programs. The programs I havent mentioned such as OTL-101 and ADA-SCID and the transfusion-dependent, beta-thalassemia program, OTL-300, will have a reduced investment moving forward. We will look for alternative ways to realize value with those programs, including through partnerships.

So Slide 6 brings together a summary of the operational changes that weve announced today. We believe these changes were important and necessary to enable Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business. As summarized here, we expect to realize cash savings of approximately $15 million from the prioritization of our portfolio. Another $60 million in savings results from the decision to consolidate our R&D teams to one site and defer the investment in the manufacturing facility.

Finally, the more staged approach to the commercial build-out and 25% reduction to our existing workforce and future headcount planning will each yield another $25 million in savings. All of these cash savings are expected to be realized over 2020 and 2021, and result in total expected savings of $125 million over that period. With the revised plan, we now have cash runway into 2022 and no near term need to finance.

Its worth briefly mentioning that this $125 million savings is after making investments in the following key areas to support our new strategy, shown on Slide 7. In commercial, diagnostic and screening initiatives, including no-charge testing programs to help identify patients with MLD and other neurodegenerative conditions in time for treatment. In manufacturing, the technology, process innovations and efficiencies to drive scalability.

In R&D, initiatives in less-rare diseases that have the potential to fuel the companys future growth in a substantial way. This wasnt just an exercise to reduce expenses, but important decision-making to ensure our capital is deployed in a disciplined manner, while building a pipeline that can leverage our success across all phases of our business.

Now let me turn the call over to Frank to discuss additional key elements of the new plan.

Frank Thomas

Thanks, Bobby, and good morning, everyone. As you can tell from this mornings press release, we have carefully examined each aspect of our business. You heard that a moment ago from Bobby, with the way we are creating operational efficiencies, and I think you will see additional evidence in the next two sections as we summarize our latest thinking around commercial deployment and manufacturing.

Starting with commercial. We understand the importance of developing a commercial model that will demonstrate our ability to execute and bring these therapies to the market successfully. This model and the infrastructure that we build will also be leveraged for any future product launches.

As youll note from the bottom of Slide 9, each rare disease has certain dynamics that will impact the launch trajectory and speed with which we can penetrate the market. In fact, we anticipate our first two potential launches in WAS and MLD having distinct but complementary launch curves, as you can see from the illustrative diagrams.

Let me start with MLD on the left, where we expect to launch first in the EU, followed by the U.S. and then other countries around the world. We think an important inflection point on the revenue curve with MLD will come later when newborn screening is established, providing an opportunity for an acceleration in growth rate. Disease progression is a second important dynamic that will affect market penetration. Because MLD advances so rapidly, it will be important to diagnose patients early and get them treated.

For Wiskott-Aldrich syndrome, the dynamics are very different, and its reflected in the shape of the curve on the right. Unlike MLD, this disease is slower progressing and more readily diagnosed. We believe that WAS will provide an opportunity to treat a number of prevalent patients from the outset and also give us additional long-term revenue stream. This program, the BLA and MAA filings are on track for 2021.

Turning back to MLD for an update on the regulatory time line. We are on track to get a decision from the European Medicines Agency later this year, and if approved, launch in the EU in the first half of 2021. In the U.S., we recently engaged with the FDA on our planned BLA submission of OTL-200 for the treatment of MLD. The FDA has provided written feedback on the sufficiency of the companys data package, including the clinical endpoint, the natural history comparator and the CMC data package.

As a result of this feedback, we intend to file an IND later this year and also seek RMAT designation, both of which we believe will facilitate a more comprehensive dialogue to discuss the data more fully and resolve the open matters before submitting a BLA. We are committed to working closely with the agency, and well provide updated guidance on the new filing time lines for the BLA after further regulatory interaction.

On Slide 10, you can see that were tracking nicely for the launch of OTL-200 in the EU in the first half of 2021, if approved, with Germany being the first country where we expect to treat commercial patients. Many of the prelaunch activities are underway, and the team has been able to keep up momentum during the pandemic to work with key centers and progress with site qualifications. We intend to set up a network of treatment centers where MLD patients are often referred and who also have transplant expertise. These same centers can be leveraged in future launches, especially for programs in the neurometabolic franchise.

I previously mentioned the importance of diagnosis in MLD to identify patients at early stages of disease, and we are taking the necessary steps to achieve long-term success. Beyond typical disease awareness efforts, we are also looking at initiatives such as no-charge diagnostic testing with partners such as Invitae, and we are looking to facilitate newborn screening for MLD with funding of upcoming pilots in New York State and Italy that are designed to validate the assay and provide the data for wider implementation. Success in these key initiatives will support early MLD patient identification.

Coming up quickly behind MLD and the neurometabolic franchise, our two proof-of-concept programs in MPS disorders, where we have made recent progress even during this challenging period with COVID-19. For MPS-I, over the past year, weve shown promising preliminary proof-of-concept data with positive engraftment, biomarker correction and encouraging early clinical outcomes, and we are excited to announce our plan to begin a registrational trial next year, bringing this program one step closer to commercialization.

For MPS-IIIA, we announced late last month that the first patient was treated in a proof-of-concept trial at Royal Manchester Childrens Hospital, with enrollment planned to continue this year and interim data to be released in 2021.

You can see graphically on Slide 12 how the aggregation of these commercial markets lead to sustainable revenue growth. In addition, the infrastructure build is designed to provide the necessary commercial capabilities to realize the potential of the portfolio. On this slide, weve included the incidence figures for MLD and the incidence and prevalence figures for WAS to help you understand each opportunity as we see it today.

Given the dynamics at play for MLD that I described on Slide 9, we believe this opportunity should largely be tied to the incident patient population, which we believe ranges from 200 to 600 patients per year in countries where rare diseases are often reimbursed. Weve taken a more conservative view than previously on the addressable patient and market opportunity in countries such as those in the Middle East and Turkey, where the literature has a wide range of differing incident figures. Also, over time, with improved disease awareness, there may be prevalent patients identified who also could benefit from therapy. Our commercial strategy has always been and continues to be based not only on 1 product, but rather the aggregation of multiple potential products launching off one HSC gene therapy platform and infrastructure.

Turning to manufacturing. Weve also made some key changes to our approach in manufacturing and how we allocate capital in the short and mid-term. On Slide 14, youll see the main tenets of our new manufacturing strategy. First, in the near term, we plan to focus on innovative technologies to enable commercial scalability.

Second, to ensure the appropriate focus on those technologies, weve made a decision to consolidate R&D to a single site in London, which brings together our organization in a more efficient way. This will allow efforts made to improve our manufacturing processes to be quickly and easily shared and then scaled commercially to transfer to our third party manufacturers, all of whom are currently located in Europe. As part of this consolidation, we will close our California site, including the termination of the Fremont project and associated capital spend.

Third, we have strong relationships with CDMOs that will ensure supply of clinical and commercial product to satisfy near-term requirements. And longer term, we intend to identify a new site in the U.S. to eventually bring manufacturing capabilities in-house with a facility that is appropriately sized and fitted for future techniques and operations.

Slide 15 shows the three phases of our approach in manufacturing: invest, partner and build. Today, we are investing, and well continue to invest in technologies such as transduction enhancers, stable producer cell line and closed automated processing of the drug product. This will potentially reduce the amount of vector needed, drive down COGS and potentially change the way products are manufactured, making it less labor-intensive, less expensive and more consistent. In the near and mid-term, we will continue to rely on our manufacturing partners for the early planned launches in MLD and WAS. For example, MolMed has been with these programs since the beginning, and theyve been a reliable commercial partner with Strimvelis.

In addition to our existing CDMO network, we have begun to search for a drug product partner in the U.S. to complete a tech transfer and serve the U.S. market, thereby reducing scheduling challenges and creating some redundancy. And finally, over time, we plan to build in-house manufacturing capabilities closer to when there is a need for additional capacity. This enables us to explore options that are more aligned with our business in terms of scale and timing.

And with that, Ill turn the call back over to Bobby.

Bobby Gaspar

Thanks, Frank. In this section, Im going to briefly highlight the potential of HSC gene therapy to correct not only blood lineage cells, but also how through natural mechanisms, specific cell types may allow correction of disease in specific organ systems and enable expansion of our portfolio into new research indications.

As many of you know, and as shown on Slide 17, through HSC gene therapy, we are able to insert a working copy of the gene permanently into the genome of HSCs, and these genetically modified cells can lead to multiple corrected cell types in the bloodstream, including immune cells, red blood cells and platelets. In addition, HSCs can differentiate into cells of the monocyte macrophage lineage that naturally migrate into various organ systems, and thus gives us an opportunity to deliver genes and proteins directly to those organs, including the brain and the GI tract.

Within the neurometabolic space, in particular, we have understood through our preclinical and clinical programs in MLD, MPS-I and MPS-IIIA how HSC gene therapy can deliver genes and proteins to the CNS to correct neurodegeneration. Here is an example of this natural mechanism at work in Slide 18.

Data shows that there are a population of gene-modified HSCs that can naturally cross the blood-brain barrier, distribute throughout the brain, engraft as microglia and express enzyme that is taken up by neurons. We have seen this approach results in clinical benefits for patients with MLD, and we are also using the same approach for MPS-I and MPS-IIIA. Beyond this, we see that the HSC gene therapy approach could be used to deliver specific genes and proteins for other larger neurodegenerative conditions which have high unmet need.

One of the conditions we are disclosing today, and shown on Slide 19, is a specific genetic subset of frontotemporal dementia, where the underlying pathogenesis has a number of parallels with the neurometabolic conditions that we are already addressing. This program involves a broad strategic alliance with Dr. Alessandra Biffi, Boston Childrens Hospital and Padua University in Italy, to further explore the potential of ex vivo HSC gene therapy in neurometabolic and neurodegenerative conditions.

In other organ systems, such as the GI tract, there are similar mechanisms at work which are illustrated on Slide 20. Tissue resident macrophages in the gut wall are required to respond to bacterial invasion from the gut lumen and prevent infection. In certain disorders, such as X-linked chronic granulomatous disease or XCGD, defects in macrophage function results in an abnormal immune response and severe colitis.

Moving on to Slide 21. We have already seen in our XCGD program the modification of HSCs and migration of gene-modified cells into the gut can lead to resolution of colitis through presumed reconstitution of the immune response. Certain subsets of Crohns disease are also associated with mutations in genes that affect the response of macrophages to infection, and so our clinical observations that HSC gene therapy for XCGD suggest that the same approach may be applicable to this genetic subset of Crohns disease. This preclinical work is ongoing in our Orchard research laboratories.

As we advance our work in FTD and Crohns disease, and assuming we show preclinical proof-of-concept, these will become exciting opportunities for us to expand and address larger patient populations, either alone or in partnership. We believe we have truly just begun to explore the potential for HSC gene therapy in diseases such as these and others, and are excited to share more about the preclinical development of these programs later this year.

So to summarize our path forward on Slide 22, the next 12 to 18 months offers many important milestones as we continue our evolution to a commercial stage company and advance our next wave of clinical stage therapies. We anticipate approval and launch of OTL-200 for MLD in the EU, additional regulatory filings in Wiskott-Aldrich syndrome and MLD, a new registrational study next year in MPS-I, multiple clinical data readouts from our neurometabolic franchise and further detail and progress on our research programs in FTD and Crohns disease.

To wrap up our prepared remarks, we are confident that our new strategic plan and operational decisions announced today will set us on the right path to achieve long-term growth, build sustainable value and serve an even larger number of patients who could benefit from hematopoietic stem cell gene therapy.

Thank you very much. And now well use the rest of the time to answer your questions. So lets have the operator open up the line.

Question-and-Answer Session

Operator

Thank you. [Operator Instructions] And our first question comes from Whitney Ijem from Guggenheim. Your line is now open. Please go ahead.

Whitney Ijem

Hi, guys. Thanks for the question. So first, just wondering, can you give us some more color maybe on the discussions youre having with the FDA in MLD? Kind of what are they looking for? And I guess is the IND just sort of a tool to get RMAT? Or is there additional kind of clinical work you plan you think youll need to do?

Bobby Gaspar

Whitney, Bobby here. Thanks for that. In general, we cant go into all of the details, obviously, of the discussions with the FDA. But I think in the release and in the script, weve talked about the fact that theyve commented on certain endpoints, the natural history, the CMC package, et cetera. Now I think Id just like to say this is a and obviously, a very complex disease, a very ultra-rare population, we have extensive data set, and we have already filed with the EMA. Now for historical reasons, there hasnt been an IND in the U.S., and so we havent had the opportunity to discuss that data in full with the FDA.

What I can say is that we do have an extensive body of data. We want to be able to talk to the FDA and have a comprehensive dialogue to be able to explain that full data set. We feel confident that we have the endpoints that they are looking for and the data that they are asking for. But we need to have that conversation with them in order to be explain to be able to do that fully. So thats why were filing an IND filing, filing the RMAT, so we can have that dialogue. And once we can clarify those issues, then we can go ahead with submission of the BLA.

Whitney Ijem

Okay. Got it. And then just one quick follow-up on MLD. Can you remind us where you are with newborn screening, I guess, both in Europe and then in the U.S.?

Bobby Gaspar

Yes, sure. So newborn screening for MLD, I think, is an important, a very important issue, because, obviously, that means that well be able to get earlier diagnosis and have more patients be able to access therapy. So its a very important part of our kind of diagnostic initiatives in this disease.

What we have so far is that we have worked with a key scientist, where an assay has been developed, thats been published to show that there is an assay that weve done on a dry blood spot to understand the decrease in the enzyme activity and also the increase in the sulfur-type levels. And that assay is now going to be put into pilots, and we are funding a pilot in New York State, and that will start later this year. And were also looking at pilots in other states as well. Were also transferring that assay to Italy and that and were funding a program in Tuscany and in Italy where that will be rolled out. And were also looking for opportunities in other EU states as well. So Id say, there are already two that are going to start, we are looking to fund other pilots as well.

And together, that data will allow us to validate the assay but also allow wider implementation of newborn screening, and also for nomination, for example, onto the WAS panel for implementation in states in the U.S. So I say theres a lot of work going on in order to make sure that happens.

Whitney Ijem

Great. Thanks.

Operator

Thank you. And your next question comes from Esther Rajavelu from Oppenheimer. Your line is now open. Please go ahead.

Esther Rajavelu

Hey, guys. Congrats on all the changes. I guess, my first question again on MLD is Im trying to understand the duration between EU approval and NBS. I dont know if that math or if that graph was drawn to scale, but it looks like its almost a four-year lag from first approval to newborn screening. Can you help us understand the time line there?

Frank Thomas

You mean between EU and U.S. or around newborn screening or both?

Esther Rajavelu

Around newborn screening, generally, between EU approval and newborn screening.

Frank Thomas

Yes, sure. As Bobby mentioned, theres a pretty active program planned around newborn screening that I think we will expect will come over time in order to even apply for the Ross Panel, there are certain requirements that need to be met in terms of the number of patients or a number of children that have to be screened, identifying the positive patients and then you can apply on the Ross Panel.

And then from there, theres a process that you go through in the U.S., at least, on a state-by-state basis to get it added. So I think there are a number of steps along the way. We havent guided specifically on the time line, but I think there are other precedents out there that suggest that this could take years. Once we screen the once we apply for the Ross Panel to get sort of full reimbursement, but obviously, well focus on states initially after that approval that have the largest populations.

Esther Rajavelu

And my Yes, go ahead.

Bobby Gaspar

Esther for I was just going to say for the EU, obviously, were looking for approval for MLD later this year. As far as people screening in the EU is concerned, thats on a country-by-country basis, and sometimes its even certain states. But Ive worked on newborn screening for SCID, for example, in the EU. And now there are numerous countries in the EU that are screening for SCID with a number of pilots also in the pipeline as well. And so with that kind of experience, and we would be looking to kind of really facilitate that uptake in the EU and as in and in the U.S., as Frank has already mentioned.

Esther Rajavelu

Understood. And then the decision to defer CapEx, is that related to some of the time lines for U.S. versus EU approvals and the newborn screening? Or what really kind of went into that delay, given you already have some cost into that facility?

Frank Thomas

Yes. I can start, and Bobby can add on that again. I think, obviously, we continue to believe in-house manufacturing is an important capability that were going to want to have over some period of time. It really comes down to sort of when is the need for that capacity and capability relative to the various programs we have. Working with the CDMOs that we have today, we know that we have capacity for the MLD and WAS launches and for a period beyond the launch. So theres not an imminent need to secure the capacity today, and we think that deferring it makes the most sense. Well continue to work with CDMOs on those launches. We will look at bringing on a U.S. supplier for drug product to be able to more easily service the U.S. market.

And then longer term, look at, potentially, in-house manufacturing at a site and location that we think is more fitted to what the capacity needs will be. So I wouldnt say its tied to any sort of launch time lines because the plan always was to utilize CDMOs for WAS and MLD. But certainly, as those launches roll out and demand grows, our capacity needs will grow and that will be the appropriate time, we think, to make the investment.

Esther Rajavelu

Understood. Thank you very much.

Operator

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Orchard Therapeutics' (ORTX) CEO Bobby Gaspar on Q1 2020 Results - Earnings Call Transcript - Seeking Alpha

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Gene Editing Technologies Market What Factors will drive the Gene Editing Technologies Market in Upcoming Years and How it is Going to Impact on...

The Medical Research Council (MRC) and independent medical research charity LifeArc are streaming 16 million into establishing a network of gene therapy innovation hubs.

The centres will offer clinical grade viral vectors as well as translational and regulatory guidance to support academic-led patient trials of new gene therapies.

Operating as 'centrally coordinated facilities', the hubs aim to address challenges faced by academics as they seek to advance novel gene therapy research into early stage clinical trials, such as a shortage of viral vector production capacity and a complex and evolving translational pathway for gene therapies.

LifeArc and the MRC said they will create the network by providing UK-based research organisations with grants for up to five years, to support the costs associated with expanding or repurposing existing viral vector production centres.

The selected centres, or hubs, will also have access to LifeArcs translation advice and support.

We hope that through this unique collaboration with the MRC, LifeArc can offer its funding and expertise in technology transfer and translational science to support the progression of promising gene therapies, said Dr Melanie Lee, the charity's chief executive. Translation of advanced therapies will be a core focus of LifeArcs future strategy for delivering significant new patient benefits.

MRC executive chair Professor Fiona Watt added: Through this partnership, we aim to support clinical development of the most exciting gene therapy projects from the UKs world-leading academic researchers. This investment will streamline and accelerate progress towards a new generation of genetic medicines for patients.

Read more:
LifeArc, MRC create 16m fund to set up gene therapy hubs - PharmaTimes

SAN RAFAEL, Calif., May 3, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR will receive an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales. The company did not disclose financial terms. BioMarin management reiterated its 2020 GAAP net income guidance of $20 to $80 million, inclusive of this collaboration.

The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM). Additionally, the companies will collaborate on several of DiNAQOR's other pipeline programs, and BioMarin has the option to extend the license to include these additional programs on similar terms. Reflecting the long-term commitment to the collaboration, BioMarin is simultaneously investing in DiNAQOR.

"With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer at BioMarin. "This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies."

"We are thrilled to collaborate with the researchers at DiNAQOR to conduct this pioneering work on the development of gene therapies for inherited cardiomyophathies," said Lon Cardon, Chief Scientific Strategy Officer and Senior Vice President at BioMarin. "We believe there is tremendous potential in combining our experience in gene therapy research and development with DiNAQOR's in-depth knowledge of genetic heart diseases."

DiNAQOR was founded and is led by several leading pharmaceutical and biotechnology executives and academics with deep cardiology and gene therapy expertise. The company's holistic approach to gene therapy is focused on gene therapies for the heart that deliver a medical solution that can safely deliver gene therapies to the heart muscle, ensure transduction of the cardiac cells, and limit the exposure of the therapy to other organs.

"BioMarin is a global leader in rare disease research, development and commercialization, and their commitment to DiNA-001 is a powerful validation of DiNAQOR's gene therapy platform," said Dr. Johannes Holzmeister, Co-Founder, Chairman and CEO at DiNAQOR. "We believe our platform has many potential applications and this milestone agreement will enable us to invest in expanding our genetic medicine pipeline."

"Momentum for gene therapies continues to build, and BioMarin has demonstrated tremendous scientific, clinical, and manufacturing leadership and expertise in the space," said Thomas Voit, M.D., Ph.D., Co-Founder and Chief Scientific Officer at DiNAQOR and Director of the Biomedical Research Centre at the Great Ormond Street Hospital and the UCL Institute of Child Health, University College London. "We are looking forward to combining our strengths to expand the promise of gene therapy treatments by targeting the heart muscle to treat rare genetic cardiomyopathies."

About HCM and MYPBC3

Hypertrophic cardiomyopathy (HCM) is one of the most common genetic heart diseases, with about 500,000 patients diagnosed with HCM worldwide. Up to 60% of HCM cases have a genetic origin, and it is estimated that 40% of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C (MyBP-C).

HCM affects the heart muscle, causing the muscle to enlarge. HCM patients have an increased risk of developing heart failure and life-threatening arrhythmias. There are no approved pharmacological treatment options available that address the underlying disease biology of HCM and invasive surgery or heart transplantation may be the only options available for patients with advanced disease.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

About DiNAQOR

Founded in 2019, DiNAQOR AG is a global gene therapy platform company focused on advancing novel solutions for patients suffering from heart disease. The company's lead preclinical program, DiNA-001 is focused on the treatment of MYBPC3-linked cardiomyopathy. DiNAQOR is headquartered in Pfffikon, Switzerland, with additional presence in London, England and Boston, Massachusetts (US). For more information visit http://www.dinaqor.com.

Forward Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: BioMarin's expectations regarding the announced collaboration, the prospects for the lead and follow on pipeline products and it's 2020 GAAP profitability. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning the programs; the ability to manufacture the product candidates, BioMarin's revenue for 2020, especially with the possible impact of COVID-19, and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

Contacts:

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Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

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View original content:http://www.prnewswire.com/news-releases/biomarin-extends-gene-therapy-leadership-with-dinaqor-in-a-preclinical-collaboration-and-license-agreement-to-develop-gene-therapies-for-rare-genetic-cardiomyopathies-301051582.html

SOURCE BioMarin Pharmaceutical Inc.

Company Codes: NASDAQ-NMS:BMRN

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BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic...

When the axons that extend from neurons break during a spinal cord injury, the result is often a lifelong loss of motor functioning, because vital connections from the brain to other body parts cannot be restored. Now, researchers from Temple Universitys Lewis Katz School of Medicine say they may have found a way to recover some functions lost to axon breaks.

The researchers discovered that boosting levels of a protein called Lin28 in injured spinal cords of mice prompts the regrowth of axons and repairs communication between the brain and body. Lin28 also helped repair injured optic nerves in the animals, they reported in the journal Molecular Therapy.

The Temple team zeroed in on Lin28 because its a known regulator of stem cells, meaning it controls their ability to differentiate into various cells in the body. The researchers examined the effects of Lin28 on spinal cord and optic nerve injuries using two mouse models: one that was engineered to express extra Lin28 and another that was normal and was given the protein after injury via a viral vector.

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All of the mice experienced axon regeneration, the researchers reported. But they found that the best results occurred in the normal mice that received Lin28 injections post-injury. In fact, in animals with optic nerve injuries, the axons regrew to the point where they filled the entire tract of the nerve.

Lin28 treatment after injury improved coordination and sensation in the mice, the researchers reported.

"We observed a lot of axon regrowth, which could be very significant clinically, since there currently are no regenerative treatments for spinal cord injury or optic nerve injury," said senior author Shuxin Li, M.D., Ph.D., professor of anatomy and cell biology at the Lewis Katz School of Medicine, in a statement.

RELATED: Gene therapy with 'off switch' restores hand movement in rats with spinal cord injury

Lin28 is already a target of interest, though it has garnered the most attention so far in cancer research. Startup Twentyeight-Seven Therapeutics is developing a small molecule that inhibits the protein in the hopes that doing so will boost Let-7, a cancer-suppressing microRNA. The company raised more than $82 million in a series A financing last year.

Several new approaches for repairing spinal cord injuries are under investigation, most notably gene therapy. King's College researchers are working on a gene therapy that repairs axons by prompting the production of the enzyme chondroitinase. A UT Southwestern team is targeting the gene LZK to increase levels of supportive nervous system cells called astrocytes in response to spinal injuries.

The Temple team has a two-pronged approach to further developing their Lin28-directed treatment. They hope to develop a vector that can be safely delivered by injection and that would deliver the therapy directly to damaged neurons. They also plan to study other molecules in the Lin28 signaling pathway.

"Lin28 associates closely with other growth signaling molecules, and we suspect it uses multiple pathways to regulate cell growth," Li said, potentially revealing other therapeutic molecules that could further boost neuron repair.

Originally posted here:
Repairing spinal cord injuries with a protein that regulates axon regeneration - FierceBiotech

Hours after Gilead announced that an NIH trial testing their antiviral drug remdesivir in Covid-19 patients had succeeded, NIAID director Anthony Fauci sat on a couch in the Oval Office and gave the world the top-line readout.

The drug induced a 31% improvement on the primary endpoint of time to recovery: 11 days in the drug arm compared to 15 days in the placebo arm, he said, adding that patients taking the drug appeared less likely to die, with an 8% mortality rate in the drug arm compared to 11% in patients given the placebo.

The mortality data were not yet statistically significant, he cautioned but were trending in the right direction. Fauci, surrounded by President Trump, Vice President Mike Pence and several other advisors, said the news was a very optimistic sign in the hunt for treatments to fight the virus.

Although a 31% improvement doesnt seem like a knockout 100%, it is a very important proof of concept, he said. Because what it has proven, is that a drug has blocked this virus.

Fauci said more details would come and that the study would be submitted to a peer-reviewed journal. Trump, who deferred to Fauci in giving the readout, echoed Faucis commentary.

Its a beginning, that means you build on it, Trump said. But its a very positive event.

Shortly after the briefing, the New York Times reported that the FDA was preparing to issue an emergency use authorization for the drugs use in Covid-19. In an email to Endpoints News, the FDA did not confirm or deny the Times report, but a spokesperson said the agency has been engaged in sustained and ongoing discussions with Gilead Sciences regarding making remdesivir available to patients as quickly as possible, as appropriate.

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BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases - Endpoints News

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Summary

Its an understatement to say that Facebook has changed the world its created an ability for people to be transparent about their experiences, lives, and opinions, for better or worse. In the case of Homology, its for the latter. Homology, a gene therapy company whose technology has already been scrutinized by scientists as untrue, has just one product in clinical trials, HMI-102, for the rare disease phenylketonuria ("PKU"). As Med Genie reported back in January, FIXXs trial update showed interim phenylalanine (Phe) results that suggested HMI-102 was not efficacious for low and mid-dose patients. Our note today highlights a data point the company would very much like you not to know the only patient in FIXXs high dose cohort posted her results on Facebook, and they show that HMI-102 is unlikely to reach trial endpoints even at a high dose. We believe this patient was forced to take her posts down as a result, and management selectively disclosed the issue to the sell side, providing comments about the drugs safety, and conveniently ignoring the implications to efficacy and the business. Maybe they were hoping to raise capital before officially announcing trial results. Who knows? We believe that the HMI-102 program is dead in the water, and since its progress is the major driver of FIXXs value, we believe that the stock should trade to cash value, or $5.80, down 56% from the April 27th close.

Background

The best shorts are often one-trick ponies, but rarely do we find one where a single data point completely upends the long investment case. Homology Medicines, FIXX, is one of those rare finds.

Homology is a gene therapy company which has their first and lead product candidate, HMI-102, in a dose-escalation Phase 1/2 trial (pheNIX) for phenylketonuria. FIXX also has 2 IND-enabling programs and some discovery stage programs, but the HMI-102 trial is the companys main shot at viability.

PKU is a relatively rare disease, with a U.S. incidence of approximately 350 cases per year and a prevalence of just 16,500, per FIXXs 10-k. PKU is tied to mutations in the gene that control PAH, an enzyme that metabolizes phenylalanine, or Phe. The condition results in a deficiency in the enzymatic activity of PAH, causing an excess in Phe in the bloodstream that can result in intellectual disability. PKU patients are identified soon after birth, and are primarily treated with a Phe-restricted diet. FIXXs HMI-102 seeks to modify the underlying genetic cause of PKU, effectively curing the disease and allowing patients to eat normally and not experience the cognitive and metabolic issues from higher than normal Phe.

FIXXs technology, which does not use the CRISPR approach to gene therapy, has already been subject to scrutiny, with David Russell, a researcher at the University of Washington, saying, Whats surprising is this company raised so much money on something thought to be untrue in the scientific community, in a piece in MIT Technology Review.

We believe that recent revelations about the efficacy of HMI-102 support this skepticism and show that the drug is not efficacious, kicking out the one leg holding up FIXXs business, and that FIXX should trade to cash value, or $5.80 per share, down 56% from the April 27th closing price.

The pheNIX trial

The pheNIX trial for HMI-102 launched in June 2019, and its primary efficacy endpoint is two plasma Phe measurements below 360 umol/L (or 6 mg/dL) between 16 and 24 weeks after dosing. Following evaluation of the first two patients in a cohort, a decision can be made to either escalate to the next dose level, add a third patient or expand the cohort at the selected dose level.

Now before we review whats new, its helpful to note that a mouse study presented at the 21st Annual Meeting of the American Society of Gene & Cell Therapy titled Sustained Correction of Phenylketonuria by a Single Dose of AAVHSC Packaging a Human Phenylalanine Hydroxylase Transgene showed that HMI-102 showed an effect to mouse Phe levels just one week after dosing in fact, mouse Phe levels remained relatively flat after that first drop.

This would imply that the therapy shows its effect soon after dosing and the longer timelines contemplated in the study endpoints are to indicate that the effect is long lasting. Sure enough, we see a similar dynamic in the pheNIX trial data released by FIXX in December 2019. There were 3 patients examined here from the 10-k: (n=2 patients in the low-dose Cohort 1 and n=1 patient in the mid-dose Cohort 2) as of the data cutoff of December 2, 2019. A fourth patient was dosed in Cohort 2 subsequent to the data cutoff date and was therefore not included in the analysis.

In this release, we see the two patients in the low-dose cohort experienced no improvement in fasting Phe after dosing or even 12 weeks later. The cohort 2 patient, getting a mid-dose, showed an improvement in Phe level immediately after dosing, but their Phe level did not fall below the 360 umol/L threshold defined as the primary endpoint (it stayed around 500) calling into question the efficacy of the treatment, which Med Genie mentions in their piece (from the 10-k):

Damning revelations

On March 5, 2020, a woman we will call Miss A started a Facebook group (since made private or taken down on April 15, 2020) to discuss her experience getting gene therapy for PKU:

On March 9, 2020, Miss A, who lives in Normal, IL tells us shes going to Chicago, which happens to be one of the pheNIX trial sites:

The next day, Miss A provides us with enough to data to know that she is Patient 5, part of the high dose cohort 3 in FIXXs HMI-102 trial (cohort 3, mentioned by Miss A, would be the next dose up from the cohort 2, the mid-dose cohort):

Dr. Burton appears to be Dr. Barbara K. Burton, a physician focused on PKU at the Ann & Robert H. Lurie Childrens Hospital of Chicago, a trial site mentioned in the pheNIX trial description on clinicaltrials.gov:

This, taken together with the fact that Biomarins own PKU trial was in too early a stage to enroll a Cohort 3 patient in March 2020, its probably safe to say that Miss A is taking part in FIXXs pheNIX trial.

On March 11, Miss A receives her infusion:

Miss A then shares a series of updates on how she is feeling and the progress of her weekly visits post-infusion. Six days post infusion, she says she hasnt gotten any test results back, but that it may take 2 or 3 weeks to get a Phe level:

27 days post-infusion, Miss A tells a FB commenter that she wont get Phe levels till six weeks post-infusion:

And then 35 days post-infusion, on April 15, 2020, a bombshell Miss A gets her Phe levels, and at 25 mg/dL or 1497uMol/L, they are well above the 360 uMol/L endpoint threshold after 5 weeks (and after the drug typically takes effect), suggesting that HMI-102 is not efficacious even for a high dose patient:

Just a few hours after her post, Miss As entire group is either taken down or made private, perhaps at the demands of FIXX itself.

Management's disclosure problem

We believe that management was aware of this post and tried to manage the perception of it by talking to the sell side and to select investors. In fact, Oppenheimers equity sales desk was sharing the below email conversation between their analyst Matt Biegler and FIXXs Theresa McNeely to explain the price action on April 15th:

Who was Theresa planning to speak to? We know that Bairds Madhu Kumar got a call:

But when we asked Theresa ourselves, she was much less forthcoming:

It appears to us that FIXX chose to inform the sell side and potential larger investors, who appear to be selling the stock (it has dramatically underperformed biotech broadly), but not the average investor. This is a significant red flag that investors should be aware of.

Why all this matters

Because the mouse study and the Cohort 2 data showed an effect to Phe levels one week after dosing rather than a gradual reduction, we can conclude that Miss As level, at 1497 uMol/L, is probably not going to get better, unfortunately. Further, her levels several weeks into the trial are still much higher than the threshold level specified in the primary endpoint of 360 uMol/L. This means that the therapy is showing zero efficacy even for a high dose patient. This data point is damning given the size of the trial and importance of the high dose patient in light of the lack of efficacy in the low and mid-dose cohorts.

Now the sell side may parrot management and say that there is no way to know whether Miss A is who she says she is, but the evidence is certainly strong supporting her case. They may say that there was no way for her to know her Phe level, but her posts show that she expected to receive them. They may also say that the drug is safe, and that liver enzyme elevation should be expected in a therapy like HMI-102, but thats all beside the point. The point is that the Phe level Miss A received 5 weeks after infusion show that HMI-102 is not efficacious.

These results support the skepticism around FIXXs use of the AAVHSC vector in liver directed gene therapy, which, based on the results thus far, and in particular Miss As results, appear to show zero efficacy and thus makes it inferior to Biomarins AAV5 vector.

Furthermore, if management thought this information was material enough to talk to the sell side analysts covering the stock, why not put it in an 8-k or even a press release for the benefit of their entire shareholder base? Given the materiality of the information, wouldnt all shareholders have benefited from the same level of disclosure rather than be kept in the dark? This is behavior consistent with that of MDXG and ALLK, both companies with executives formerly from reputed companies who have seen their stock prices demolished.

For FIXX, we believe that this is a huge problem the HMI-102 pheNIX trial is the ONLY program in their portfolio that is in the Phase 1/2 stage, and thus the primary path to viability for the company. With this piece of data showing that HMI-102 is not efficacious, we believe that the program is likely worthless and unlikely to proceed to commercialization. While FIXX may try to apply HMI-102 to other indications, we believe that doing so would essentially restart the trial and approval clock without making up for the lost time to market from a failed PKU trial.

Furthermore, social media matters. While FIXX management may be dismissive of people posting on Facebook, these posts have value. In the case of Allakos (ALLK), Seligman Research put together a barn burner of a report which included numerous Facebook posts questioning the efficacy, safety, and trial design of ALLKs drug candidate. Since that report, ALLK stock is down approximately 51%, and ALLK actually has several later stage trials.

In the case of FIXX, we believe that HMI-102 in PKU is the ONLY path to viability given the lack of efficacy of HMI-102 at high dose, we believe that the HMI-102 program is dead, and that the stock should trade to its cash value per share, or $5.80 per share, down 56% from the April 27th close price.

Disclosure: I am/we are short FIXX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: This piece is our opinion and not an offer to buy/sell any securities. We're biased, you're biased, so do your own work and make your own decisions.

Link:
Can't FIXX This - We Believe Homology's HMI-102 Is In Trouble - Seeking Alpha

After a comprehensive analysis, Mrinsights.bizhas published a new research study titled GlobalHemophilia Gene Therapy Market Growth 2020-2025 that covers the latest and upcoming industry trends and offers a global spectrum of the Hemophilia Gene Therapy market, and future forecast from 2020 to 2025 years. The market is bifurcated into product type, application, key manufacturers and key regions and countries. The research assists users to achieve competitive leverage with acquiring and preserving market position as key aims of the program. The report expands on details pertaining to contributions by key players, demand and supply analysis as well as market share growth of the industry.

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It covers the leading manufacturers profiles involving market entry strategies, production analysis, market share, revenue forecast. In addition, the regional analysis of the industry is offered where the report delivers analytical information on regional segmentation. Top leadingcompaniesof global Hemophilia Gene Therapy market are:Spark Therapeutics, Ultragenyx, Sangamo Therapeutics, Bioverativ, Shire PLC, Freeline Therapeutics, BioMarin, uniQure

Industry Trends And Opportunities:

The report provides an investigation into the global Hemophilia Gene Therapy market status, shares, supply-demand, market drivers, challenges and opportunities, and geological areas. Key trends and development opportunities are covered in this analysis report. The report then serves information on sales and market share estimates by-product as well as a profile of the companys business.

Regional Analysis:

This research report consists of the worlds crucial region Hemophilia Gene Therapy market share, size (volume), trends including the product profit, price, value, production, capacity, capability utilization, supply, and demand and industry growth rate. It helps readers to understand strategies to make sound investments. The regions are extensively analyzed with respect to every parameter of the geographies in question, comprising: Americas (United States, Canada, Mexico, Brazil), APAC (China, Japan, Korea, Southeast Asia, India, Australia), Europe (Germany, France, UK, Italy, Russia, Spain), Middle East & Africa (Egypt, South Africa, Israel, Turkey, GCC Countries).

The Report Addresses The Following Queries Related To The Market:

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Furthermore in this report, external as well as internal factors that are supposed to affect the business positively or negatively have been investigated. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors by region on the global Hemophilia Gene Therapy market is given in the report. A further section of the report discusses expansion plans of companies, key mergers and acquisitions, funding and investment analysis, company establishment dates, revenues of manufacturers, and their areas served and manufacturing bases.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team ([emailprotected]), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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Global Hemophilia Gene Therapy Market 2020 Top Companies, Industry Demand, Business Review and Regional Analysis by 2025 Cole Reports - Cole of Duty

The Gene Therapy Market report market intelligence study intended to offer complete understanding of global market scenario with the Impact of COVID-19 (Corona Virus). It attempts to analyze the major components of the Market which have greater influence on it. This includes various elements of significant nature including market overview, segmentation, competition landscape, Market chain analysis, key players stratergyand more. Also, the report provides a 360-degree overview of global market on the basis of various analysis techniques including SWOT and Porters Five Forces. Approximations associated with the market values over the forecast period are based on empirical research and data collected through both primary and secondary sources. This might help readers to understand the strengths, opportunities, challenges and perceived threats of the market.

Based on Classification, each type is studied as Sales, Market Share (%), Revenue (Million USD), Price, Gross Margin and more similar information. The report can help to realize the market and strategize for business expansion accordingly. In the strategy analysis, it gives insights from marketing channel and market positioning to potential growth strategies, providing in-depth analysis for new entrants or exists competitors in the Gene Therapy industry.

The Gene Therapy Market report wraps:

There are 13 Chapters to thoroughly display the Gene Therapy market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

In the end, The objective of the market research report is the current status of the market and in accordance classifies it into a few objects. The report takes into consideration the first market players in every area from over the globe.

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

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Gene Therapy Market to witness impressive Growth in Production-Consumption Ratio through 2026 - Latest Herald

CAMBRIDGE, Mass., May 04, 2020 (GLOBE NEWSWIRE) -- TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage immunotherapy company developing the next generation of novel T cell therapies for patients suffering from cancer, today announced the appointment of Stephen Webster to its Board of Directors. With nearly 30 years of biotechnology industry experience in raising capital, business development transactions and operations, Mr. Webster has played important roles as the Chief Financial Officer of Spark Therapeutics, Optimer Pharmacuticals and Adolor Corporation. In connection with Mr. Websters arrival, Mitchell Finer, Ph.D., will be stepping down from the Board of Directors effective May 4, 2020 but will continue to serve TCR2 in an advisory capacity.

"We are delighted to welcome in another successful cell and gene therapy executive as Stephen Webster joins our Board of Directors. His distinguished track record of leading companies through periods of growth will prove invaluable at this moment in time as we prepare to present clinical data for our two lead programs, TC-210 and TC-110, and advance a third mono TRuC-T cell therapy towards the clinic," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "His business development transaction expertise will be particularly useful in helping us strike the right partnerships in pursuing our goal of developing innovative T cell therapies for patients suffering from cancer.

Mr. Webster served as the Chief Financial Officer of Spark Therapeutics, a publicly traded gene therapy biotechnology company, from July 2014 until its acquisition by Roche for $4.3 billion in December 2019. He was previously Senior Vice President (SVP) and Chief Financial Officer of Optimer Pharmaceuticals, a publicly traded biotechnology company, from July 2012 until its acquisition by Cubist Pharmaceuticals in October 2013. Prior to joining Optimer, Mr. Webster served as SVP and Chief Financial Officer of Adolor Corporation, a biopharmaceutical company, from 2008 until its acquisition by Cubist Pharmaceuticals in 2011. Mr. Webster also served in leadership positions in the investment banking healthcare groups of Broadpoint Capital and PaineWebber Incorporated.

Mr. Webster has served as a director of Nabriva Therapeutics AG (formerly Nabriva Therapeutics plc), a publicly traded biopharmaceutical company, since August 2016 and Viking Therapeutics, a publicly traded biopharmaceutical company, since May 2014. Mr. Webster received an A.B. in Economics from Dartmouth College and an M.B.A. in Finance from The Wharton School of the University of Pennsylvania.

I am thrilled to become a director of TCR2 Therapeutics, where there is a great opportunity to turn a distinctive TRuC-T cell platform into a series of novel treatments of cancer, said Mr. Webster. I look forward to working with the TCR2 leadership team and Board of Directors to add my business development expertise in helping the Company achieve its goal of bringing transformational therapies to people living with serious solid tumors and hematologic malignancies.

On behalf of TCR2 and the Board of Directors, I would like to thank Mitchell Finer for his many contributions to the rapid growth of our company, added Dr. Menzel. Our strategy to begin with an automated cell therapy manufacturing process benefited from working very closely with Dr. Finer, whose three decades of cell therapy manufacturing leadership provided us a significant competitive advantage in the cell therapy landscape. We look forward to continuing to benefit from his insights as he transitions from a Board member to a consultant.

About TCR2 Therapeutics

TCR2Therapeutics Inc.is a clinical-stage immunotherapy company developing the next generation of novel Tcell therapies for patients suffering from cancer.TCR2sproprietary T cell receptor (TCR) Fusion Construct Tcells (TRuC-T cells) specifically recognize and kill cancer cells by harnessing signaling from the entire TCR, independent ofhuman leukocyte antigens (HLA). In preclinical studies, TRuC-T cells have demonstrated superior anti-tumor activity compared to chimeric antigen receptor T cells (CAR-T cells), while exhibiting lower levels of cytokine release. The Companys lead TRuC-T cell product candidate targeting solid tumors, TC-210, is currently being studied in a Phase 1/2 clinical trial to treat patients with mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma. The Companys lead TRuC-T cell product candidate targeting hematological malignancies, TC-110, is currently being studied in a Phase 1/2 clinical trial to treat patients with CD19-positive adult acute lymphoblastic leukemia (aALL) and with aggressive or indolent non-Hodgkin lymphoma (NHL). For more information about TCR2, please visitwww.tcr2.com.

Forward-looking Statements

This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could", "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavor," "potential," "continue" or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include, but are not limited to, express or implied statements regarding the development of the Companys product candidates, future business plans and the therapeutic potential of its product candidates and platform.

The expressed or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities, including review under accelerated approval processes; orphan drug designation eligibility; regulatory approvals to conduct trials or to market products; TCR2s ability to maintain sufficient manufacturing capabilities to support its research, development and commercialization efforts, whether TCR2's cash resources will be sufficient to fund TCR2's foreseeable and unforeseeable operating expenses and capital expenditure requirements, the impact of the COVID-19 pandemic on TCR2s ongoing operations; and other risks set forth under the caption "Risk Factors" in TCR2s most recent Annual Report on Form 10-K, most recent Quarterly Report on Form 10-Q and its other filings with theSecurities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although TCR2believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur.

Moreover, except as required by law, neither TCR2nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investor and Media Contact:

Carl MauchDirector, Investor Relations and Corporate CommunicationsTCR2 Therapeutics Inc.(617) 949-5667carl.mauch@tcr2.com

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TCR Therapeutics Announces Veteran Finance Executive Stephen Webster Joins its Board of Directors - GlobeNewswire

The Global CRISPR technology market is expected to reach USD 1,715 million by 2023 from USD 562 million in 2018, at a CAGR of 25%

The rising funding from government and private organizations and the high adoption of CRISPR technology are major factors driving the growth of CRISPR technology market002E

How much is the CRISPR Technology Market worth?

MarketsandMarkets forecasts the CRISPR technology market is expected to reach USD 1,715 million by 2023 from USD 562 million in 2018, at a CAGR of 25% during the forecast period. The global CRISPR services market is segmented into four major regions, namely, North America, Europe, the Asia Pacific, and the Rest of the World.

In 2018, North America accounted for the largest share of this market majorly due to the rising government and private funding, presence of major pharma and gene therapy companies, and the adoption of CRISPR in a number of applications.

The CRISPR products segment is expected to command the largest share of the CRISPR Products market during the forecast period.

The CRISPR Products market, by product and service, is estimated to be dominated by the products segment in 2018. This is attributed to the fact that the CRISPR Products is being adopted quickly by academics and researchers, pharma and biotech companies.

The enzymes segment is expected to account for the largest share of the products market, being one of the key ingredients in the CRISPR process. Companies like Merck KGaA and Thermo Fisher Scientific are providing hands-on training to researchers, which will increase the demand for CRISPR products in the future.

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Biomedical applications to occupy the majority of the market, by application, and grow at the fastest rate during the forecast period.

The biomedical applications segment is projected to be the fastest-growing segment of the CRISPR services market, by application, during the forecast period. Developments in gene therapy, drug discovery, and diagnostics, due to the application of CRISPR, are driving the growth of this biomedical segment.

Many companies have also invested in drug discovery and gene therapy companies that are using CRISPR technology.

North America is expected to account for the largest market share during the forecast period.

North America is estimated to account for the largest share of the CRISPR services market in 2018. This is majorly attributed to the rising government and private funding, presence of major pharma and gene therapy companies, and the adoption of CRISPR in several applications.

Furthermore, crops that are treated with CRISPR-based gene editing are not considered as GMOs in US; this has attracted a number of agricultural companies to focus on the commercialization of CRISPR-edited crops.

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Who are the leading vendors operating in CRISPR Services Market?

Cellecta, Inc. (US), Thermo Fisher (US), GeneCopoeia, Inc. (US), Applied StemCell (US), Synthego Corporation (US), OriGene Technologies (US), Horizon Discovery (UK), Merck (Germany), and GenScript (US).

Thermo Fisher Scientific has established its presence in diversified life sciences markets; this has helped it to minimize risks and dependency on any business segment. The company has a strong product portfolio and brand image, which enables it to strengthen its position in the market.

The company has initiated a promotional campaign in which it conducts workshops and provides hands-on training to researchers in academic and research institutes working on CRISPR. This campaign is being organized to spread awareness on and promote CRISPR technology.

To speak to our analyst for a discussion on the above findings, clickSpeak to Analyst

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CRISPR Technology Market worth USD 1715 million by 2023 according to a new research report - WhaTech Technology and Markets News

Cancer Gene Therapy Marketis expected to reach 5075 million by 2026 from XX million in 2018 at CAGR of XX %.

The report study has analyzed revenue impact of covid-19 pandemic on the sales revenue of market leaders, market followers and disrupters in the report and same is reflected in our analysis.

REQUEST FOR FREE SAMPLE REPORT:https://www.maximizemarketresearch.com/request-sample/520/

Cancer Gene Therapy Research Report is a method of therapeutic delivery of genetic material into a patients cells as a drug to treat disease and compensate for abnormal genes or to make a beneficial protein. Cancer cells modify themselves (called faults or mutations) in several of their genes which make them divide very often and form a tumor. Gene therapy provides various methods by which doctors can cure cancer like:

Inactivation of a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. Replacement of mutated gene that causes disease with a healthy copy of the gene.Cancer caused 9.02 million deaths in 2017, and is expected to reach 10 million by 2026; the increased no of cancer prevalence is because of increased body mass index, low fruit and vegetable intake, lack of physical activity, increased tobacco and alcohol consumption. Also, Factors like increasing ethical acceptance of gene therapy for treatment of diseases and growing popularity of DNA vaccines, High success rate during the preclinical and clinical trial, Increase in funding for R&D in cancer gene therapy, Increase in geriatric population, favorable government regulations will fuel the global Cancer Gene therapy market. However, the high cost of gene therapy treatment and unwanted immune responses will restrain market growth.The highest revenue-generating region is North America in 2017 followed by Europe; reasons behind this increased growth rate are well-established health care facilities, high per capita health care expenditure, and extensive R&D activities for the gene therapy in the region. However, Asia Pacific is projected to expand at a moderate growth rate during the forecast period.

Key Highlights:

Assessment of market definition along with the identification of key players and an analysis of their strategies to determine the competitive outlook of the market, opportunities, drivers, restraints, and challenges for this market during the forecast period Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, growth strategies that can In-depth analysis of the industry on the basis of market segments, market dynamics, market size, competition & companies involved value chain Cancer Gene Therapy market analysis and comprehensive segmentation with respect to the therapy and geography to assist in strategic business planning Cancer Gene Therapy market Research Report analysis and forecast for five major geographies North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key countries Complete quantitative analysis of the industry from 2017 to 2026 to enable the stakeholders to capitalize on the prevailing market opportunities.For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.

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Research Methodology:

An objective of the study is to estimate the size of the Cancer Gene Therapy market for 2017 and projects its demand till 2026 with quantitative and qualitative analysis of Cancer Gene Therapy market. Industry experts have studied various industry journals, directories, have referred information available with various associations to identify, collect information and to put it in articulated format to make useful for all stake holders in the industry. Primary research has been done and various industry experts and suppliers from worlds wide have given their inputs to make the study more accurate.Key Players in the Cancer Gene Therapy Market Are:

Shenzhen Sibiono Genetech Adaptimmune Glaxosmithkline Oncogenex Pharmaceuticals Bluebird Bio, Inc. Synergene Therapeutics Shanghai Sunway Biotech Biocancell Celgene MerckKey Target Audience:

Cancer Gene Therapy Market Investors Cancer Gene Therapy Marketing Players Pharmaceutical and Biotechnology Companies Healthcare Institutions (Individual Surgeons, Medical Schools, Group Practices, Hospitals, and Governing Bodies) Diabetes Drugs Market Research Associations Diabetes drug Manufacturers & DistributorsScope of the Cancer Gene Therapy Market

Research report categorizes the Cancer Gene Therapy market based on Therapy and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Cancer Gene Therapy market with key developments in companies and market trendsCancer Gene Therapy Market, By Therapy:

Oncolytic Virotherapyo Adenoo Lentiviruso Retro Viruso Adeno Associated Viruso Herpes Simplex Viruso Alpha Viruso Vaccinia Viruso Simian Viruso Others Gene Transfero Naked Plasmid Vectoro Electroporationo Sonoportiono Magnetofectiono Gene Gun Gene-Induced Immunotherapyo Delivery of Cytokines Geneo Delivery of Tumor Antigen GeneCancer Gene Therapy Market, By Geography:

North America Europe Asia Pacific Middle East & Africa Latin America

MAJOR TOC OF THE REPORT

Chapter One: Cancer Gene Therapy Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cancer Gene Therapy Market Competition, by Players

Chapter Four: Global Cancer Gene Therapy Market Size by Regions

Chapter Five: North America Cancer Gene Therapy Revenue by Countries

Chapter Six: Europe Cancer Gene Therapy Revenue by Countries

Chapter Seven: Asia-Pacific Cancer Gene Therapy Revenue by Countries

Chapter Eight: South America Cancer Gene Therapy Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cancer Gene Therapy by Countries

Chapter Ten: Global Cancer Gene Therapy Market Segment by Type

Chapter Eleven: Global Cancer Gene Therapy Market Segment by Application

Chapter Twelve: Global Cancer Gene Therapy Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Cancer Gene Therapy Market Report at:https://www.maximizemarketresearch.com/market-report/cancer-gene-therapy-market/520/

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Maximize Market Research provides B2B and B2C market research on 20,000 high growth emerging technologies & opportunities in Chemical, Healthcare, Pharmaceuticals, Electronics & Communications, Internet of Things, Food and Beverages, Aerospace and Defense and other manufacturing sectors.

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Cancer Gene Therapy Market : Research Report - MR Invasion

The report aims to provide an overview of Global Assistive Technologies for Visual Impairment Market along with detailed segmentation of market by applications, end-users and five major geographical regions. Global Assistive Technologies for Visual Impairment market is expected to witness an aggressive growth during the forecast period.

Leading players of Assistive Technologies for Visual Impairment Market:VFO Group, TQM, Humanware, Handy Tech Elektronik GmbH, Perkins Solutions, Papenmeier, Amedia, Eurobraille, Nippon Telesoft, Brailletec, VisionCue

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The Global Assistive Technologies for Visual Impairment Market Analysis to 2025 is a specialized and in-depth study of the Assistive Technologies for Visual Impairment market with a focus on the global market trend. The report aims to provide an overview of the global Assistive Technologies for Visual Impairment market with detailed market segmentation by service, technology, industry vertical, and geography. The global Assistive Technologies for Visual Impairment market is expected to witness high growth during the forecast period.

Segmentation by Type:Braille Displays

Note Takers

Magnifiers

Braille Printers and Embossers

Braille Writers

Segmentation by Application:Blind School

Disabled Persons Federation and Hospital

Enterprises and Social Organizations

The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides an overview and forecast of the global Assistive Technologies for Visual Impairment market based on the service, technology, and industry vertical. It also provides market size and forecast till 2025 for overall Assistive Technologies for Visual Impairment market with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South America (SAM).

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The report evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend and provides exhaustive PEST analysis for all five regions.

What the report features:-

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Exclusive Research Report on Assistive Technologies for Visual Impairment Market, Size, Analytical Overview, Growth Factors, Demand and Trends...