Archive for the ‘Gene Therapy Research’ Category

In this new business intelligence Cancer Gene Therapy market report, PMR serves a platter of market forecast, structure, potential, and socioeconomic impacts associated with the global Cancer Gene Therapy market. With Porters Five Forces and DROT analyses, the research study incorporates a comprehensive evaluation of the positive and negative factors, as well as the opportunities regarding the Cancer Gene Therapy market.

With having published myriads of Cancer Gene Therapy market reports, PMR imparts its stalwartness to clients existing all over the globe. Our dedicated team of experts deliver reports with accurate data extracted from trusted sources. We ride the wave of digitalization facilitate clients with the changing trends in various industries, regions and consumers. As customer satisfaction is our top priority, our analysts are available 24/7 to provide tailored business solutions to the clients.

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The Cancer Gene Therapy market report has been fragmented into important regions that showcase worthwhile growth to the vendors Region 1 (Country 1, Country 2), region 2 (Country 1, Country 2) and region 3 (Country 1, Country 2). Each geographic segment has been assessed based on supply-demand status, distribution, and pricing. Further, the study provides information about the local distributors with which the Cancer Gene Therapy market players could create collaborations in a bid to sustain production footprint.

Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

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Global Briefing 2019 Cancer Gene Therapy Industry Analyzer Technique, Advancements, Market Size, Share, Opportunity and Trend with Growing CAGR till...

The response to the coronavirus is both impressive and thought-provoking. Governments, organisations and people around the world are taking peacetime actions not known in living memory. And rightly so. The projections of human loss the virus could cause are chilling. Nevertheless, I have not been able to stop myself wondering: Why do other crises not generate the same level of action, even when we know they are associated with equally or even more devastating projections?

I have often compared the state of our economy to a body that is suffering an advanced stage of cancer. This is, I realise, a shocking analogy to use, but bear with me: it can provide a new lens through which to look at our economy.

Imagine the cells in your body represented organisations, hard at work producing all the services and goods you need to live a healthy life. Cancerous cells, unlike healthy ones, grow and replicate as fast as they can at the expense of the body that hosts them. In our economy, growth has become just as prevalent at all levels: nationally we grow GDP, organisations maximise profits and individuals accumulate wealth and possessions.

And this has real consequences for human health. Air pollution a direct consequence of how we choose to organise our economy has overtaken smoking as a cause of global deaths. Consumerism and the addictive algorithms behind social media are eroding our mental health; worklessness and low-paid, insecure jobs are driving people to hunger, as evidenced by the huge rise in foodbanks across many countries.

What would happen if we took the same approach to the economic crisis that we are taking to the coronavirus crisis? The anti-viral response has four phases: contain the infection at source, delay its spread, conduct research to understand its behaviour, find a vaccine and mitigate the effects of the virus spreading.

When it comes to our global economy we have, sadly, long passed the contain phase. Cancer-like behaviours have been spreading for decades. In the UK, for example, the 1990s saw a wave of mutual building societies converting to shareholder-owned banks. Banking regulations that split retail and investment banking (such as the Glass-Steagall Act in the US) were repealed in multiple countries around about the same time.

Immunising organisations that operate with a different set of values is an urgent priority

Containing the economic cancer at source is no longer possible. We can no longer surgically remove the tumour because it has already spread. However, we can still protect some areas from becoming infected, those that run with a different ethos: the UKs National Health Service and its public broadcaster are two examples, the Swiss railway (SBB) is another. The co-operative movement has long conformed to values that include co-operating rather than competing with other co-operatives, democratic control and economic participation (thats profit sharing) for all members although sadly it has not been immune either, as the part-takeover of the UKs Co-operative Bank shows. Immunising organisations that operate with a different set of values whose genes have not yet suffered cancerous mutations is an urgent priority.

Delaying the spread of cancerous behaviours is the next priority. There are multiple approaches that can be taken here.

Business schools and management consultancies that educate and influence decision-makers have become amplifying feedback loops for the unfettered growth ideology. Happily, some are now starting to engage seriously with the need for new approaches to management and leadership, which will slow the spread of this kind of thinking.

Other delay mechanisms include representing workers on company boards (as happens in Germany), and publishing CEO-to-worker pay ratios (now mandatory in the UK), which can curb the growth of top salaries.

Any serious attempt to combat our economic cancer must understand its root causes. Human cancer is caused by mutations in our genes the manual for our human bodies that is stored in every one of our cells. Similarly, our economic cancer is caused by how our economic manual the values that underpin our economic and management practices have evolved.

Labs are conducting thousands of experiments as they race towards a vaccine for the coronavirus. Similarly, in every part of our economy people are trying out doing business according to different values

At this very moment, labs all around the world are conducting thousands of experiments as they race towards a vaccine for the coronavirus. Similarly, in every part of our economy people are experimenting, trying out doing business according to different values, conducting gene therapy in which they replace harmful values with healthy ones.

In Germany, Purpose is trialling new models of ownership; In the UK, Operation Upgrade is tackling company law to weaken the power of shareholders; complementary currencies such as the Swiss WIR have operated in parallel to mainstream finance for decades; Common Future is creating more regional and community based economies in the US; B Corps are testing lots of ways of using business as a force for good; and even multi-national corporations are committing to a net positive impact on our climate, Microsoft and Interface being just two examples.

Mitigation is arguably where most effort has been going for a very long time. The raison-detre of many charities and public services has, for centuries, been to mitigate the worst excesses of our current system. Looking after those who have fallen through the cracks of our welfare system, cleaning up environmental pollution or protecting endangered species are all mitigation activities.

In recent times the Deep Adaptation movement is providing more sobering advice on the consequence of climate change that is already inevitable.

Mitigation is necessary and often the only compassionate and practical course of action. But we mustnt let fighting the symptoms distract us from tackling the root cause of the disease. Unlike the coronavirus vaccine, the values and behaviours we need for a healthy economy are already among us today; but we need to spread them quickly. Values even collective ones reside in people. This is why, at On Purpose, we work with people who want to learn how to apply healthy values in the real life of their everyday work.

We mustnt let fighting the symptoms distract us from tackling the root cause of the disease

We are learning a tough lesson in how quickly a virus like corona can spread around the world. Viruses, as it happens, are also the vehicle by which gene therapists smuggle healthy genes into cancerous cells one of the newest approaches to tackle the root causes of cancer. This gives me hope that fundamental change can happen more quickly than we think. We are already seeing the rapid spread of healthy viruses from school climate strikes and veganism to the anti-plastic movement. I believe these are the trailblazers for many more gene therapies to come.

Tom Rippin is thefounder and CEO of On Purpose.

Thanks for reading Pioneers Post. As somebody working in the impact economy, you'll know that producing quality work doesn't come free. We rely on paid subscriptions and partnerships to sustain our purpose-led journalism so if you think it's worth having an independent, specialist media platform to share your news, insight and debate across the globe, please consider subscribing. You'll also be buying social: Pioneers Post is a social enterprise itself, reinvesting all profits to help you do good business, better.

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The other public health crisis: What the global economy can learn from our response to coronavirus | The Social Enterprise Magazine - Pioneers Post

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Gene therapy could dramatically alter how dozens of inherited diseases are treated. It's also transforming how the academic institutions working in this growing field move research from the laboratory to the clinic.

Private sector skepticism a decade or more ago spurred institutions like the University of Pennsylvania and Nationwide Children's Hospital to advance experimental projects much further before selling their ideas to biopharma companies a departure from the previous model of identifying a molecular target and letting industry do the heavy lifting.

As a result, university technology transfer officers are much more involved in the technical and commercial details of preclinical drug development, from assembling financing and creating private companies to building manufacturing capacity. The product is a host of new startups, such as AveXis, Spark Therapeutics and Bamboo Therapeutics, that in recent years have been swallowed up by large pharmaceutical companies.

"The old way is, 'I have a patent, I'm going to throw it over the fence to you and you throw me a sack of money,'" said John Swartley, managing director of the University of Pennsylvania's Penn Center for Innovation, in an interview. "This is completely different. This is co-development."

John Swartley

Permission granted by University of Pennsylvania

"We're directly involved over multiple years in helping to move the technology forward. And our commercialization partner is going to take it hopefully all the way to the market."

A paper published earlier this month in JAMA quantifies the shift. Together, hospitals, universities and the National Institutes of Health sponsored 206 of the 341 identified gene therapy trials that were active in 2019. Biotech and pharma companies led the remaining 135.

Measured by funding, hospitals, universities and the NIH had a hand in more than 280 of those studies, as some trials had multiple funders. Fourteen trials were funded by other federal sources or non-profit charities.

Hospitals and universities were most active in early-stage studies, with industry sponsoring only 22% of Phase 1 trials. But, in gene therapy, those initial human tests can hold more weight, as the benefits of a genetic fix can be quickly apparent.

"This is a sign that the model of drug development that was prominent in the past academia does basic science and finds some targets and then pharma develops the actual drug product is pretty different with gene therapy," one of the paper's authors, Walid Gellad, director of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh, wrote in an email to BioPharma Dive.

The changing academic model also raises questions about the rich price tags being sought by drugmakers for gene therapies, given the greater role played by universities and other non-profit entities.

"The paper, I think, informs discussions about how high prices really need to be in order to encourage private risk taking for gene therapies it may be a different number than for other drugs that have less late stage involvement by academia and NIH," wrote Gellad.

University involvement in gene therapy development was driven in part by the private sector's reluctance to get involved in a therapeutic approach perceived, until several years ago, as risky. The death of Jesse Gelsinger in a Penn gene therapy trial in 1999 inflicted severe reputational damage on the field, driving away drugmaker interest.

Scientists kept the faith, and their institutions carried the field forward for years afterward. When Swartley began working at Penn in 2007, one of his first meetings was with the university's gene therapy director James Wilson, who was in charge of the tragic trial that led to Gelsinger's death.

James Wilson

Permission granted by the University of Pennsylvania

"From an external perspective, from an industrial perspective, there was almost nothing happening," he said. "But it was evident from the kind of research that Dr. Wilson and his colleagues were sharing with us, they made a very convincing case that this was going to rapidly shift into a more of a developmental paradigm."

"They were anticipating a tremendous amount of industry interest when that shift occurred," Swartley added. "It turned out to be very prophetic."

At the University of North Carolina, the situation was similar in the early part of the 2000s. The institution reached a slightly different solution, however, spinning out companies like Asklepios BioPharmaceutical to advance gene therapy beyond the walls of the university laboratories.

"We had a lot of vector technology, but the market was not receptive to gene therapy at the time," said Kelly Parsons, associate technology commercialization director at UNC, in an interview. "We had a startup company that had to work very diligently to try to establish the merits of gene therapy."

Asklepios is still an independent company today, some of its gene therapy work having been folded into a Pfizer-owned Duchenne muscular dystrophy project that was previously developed by Bamboo Therapeutics.

But the time spent building the knowledge and expertise at universities or closely affiliated startups has been one of the reasons why big pharmas have rushed into the space. By advancing the technology, the universities reduced the risk of failure, making pharmas more willing to buy in.

"We had a recognition that if we wanted the for-profit sector and the investment sector and the [venture capital] world to give gene therapy a chance, it was important as an institution we were able to start that process of de-risking the asset," said Matthew McFarland, vice president of commercialization and industry relations at Nationwide, in an interview.

Doing so was a greater commitment than they expected. "What we did is say: 'What stage would these assets need to get to before external dollars would be interested in investing?'" he said. "And the reality is, oh my gosh, you have to de-risk it all the way to the point it's ready to go into the patients."

That included the initial Phase 1 study of the spinal muscular atrophy gene therapy now known as Zolgensma, which was licensed to AveXis and later acquired by Novartis.

More broadly, development included building production capabilities compliant with Good Manufacturing Practices, which govern quality and consistency standards for finished drug products, and a regulatory team that was able to prepare Investigational New Drug applications within the hospital's technology transfer office.

Building up manufacturing expertise has resulted in a new business for Nationwide: the for-profit Andelyn Biosciences, which will run a commercial scale gene therapy production facility.

Solving the manufacturing question is something many academic gene therapy centers are still grappling with as they near the point of handing off to private-sector partners. Biopharma companies want to have confidence that the therapies manufactured by university scientists will work as well in clinical trials and in wider use as they did in earlier study.

"There's no university that has the ability to ramp their early production manufacturing production to a level to get enough doses that industry doesn't have to recapitulate it," said Jim O'Connell, director of technology transfer at the University of Florida's UF Innovate, in an interview. "It's notorious for university labs, small molecules and others, to not be able to have their work reproduced out in the real world."

This very question may have been behind data quality issues for Zolgensma. Last summer, Novartis was chastised by the Food and Drug Administration for having submitted manipulated preclinical data, a scandal that the Swiss pharma tied to AveXis co-founder and former Nationwide trial investigator Brian Kaspar. Through his lawyer, Kaspar has denied all wrongdoing.

"Academic institutions have got to ask themselves: How far into this do we want to go?," said O'Connell. "It's going to have a whole bunch of costs that universities aren't used to taking on. How do we share the expense? How do we share the risk appropriately?"

Thorny questions notwithstanding, the increased investment has led to better returns for universities. Technology transfer offices interviewed by BioPharma Dive report the licensing deals are much richer for gene therapies that have advanced to human testing or near it money which gets returned to scientists and their departments to fund new research.

Returns aren't equally shared, however. Schools blessed with research that is sought-after by private industry flourish, while others struggle, said Lee Vinsel, a Virginia Tech assistant professor who is writing a book called "The Innovator's Delusion."

Indeed, broadly speaking, universities reported a little more than $3 billion in licensing revenue in 2017, but spent $68 billion, according to the Association of University Technology Managers. Less than 1% of licenses yielded more than $1 million in revenue.

Moreover, Vinsel argues the potential for licensing revenue incentivizes universities to only conduct research the private sector wants to license.

"One reason why we need federal funding and university research is to do basic science that corporations aren't going to pay for and do," Vinsel said. "If we tack more university research towards the profitable, who is going to do this basic work, including research that could really help society but will enrich no one?"

McFarland of Nationwide, however, points to less lucrative licenses it has signed, such as a device to prevent pressure ulcers in patients with tracheostomies, along with a mental health research and treatment facility the institution has launched, as ventures that were enabled by bigger deals like in gene therapy.

"If we can take that return and continue to foster research not only in [gene therapy] but even further spread that out and have an impact across all of research," he said.

"There are a lot of times when we're not the office of tech commercialization, but instead we're the office of tech realization, because what we go into is just about getting it out there to the public, and we're not going to get a return on it."

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Big pharma shied away from gene therapy for years. Academia picked up the slack - BioPharma Dive

Revenue from marketed dermatology products increased 85% for fourth quarter 2019 and 49% for full-year 2019 compared to 2018

NDA for IV tramadol accepted for review by FDA; PDUFA date is set for October 10, 2020

Rolling NDA submission for CUTX-101 for the treatment of Menkes disease is on track to begin in the fourth quarter of 2020

NEW YORK, March 16, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (NASDAQ: FBIO) (Fortress), an innovative biopharmaceutical company, today announced financial results and recent corporate highlights for the fourth quarter and full year ended December 31, 2019.

Fortress achieved multiple key milestones in 2019 and early 2020, including:

Lindsay A. Rosenwald, M.D., Fortress Chairman, President and Chief Executive Officer, said, We have generated significant momentum throughout 2019 and into early 2020. In order to drive our next phase of growth, our world-class business development team continues to identify and acquire high-potential marketed and development-stage assets to further expand our portfolio of product opportunities. Additionally, Fortress and our development partners continue to advance our clinical-stage programs across multiple therapeutic categories. With five commercial products and over 25 programs in development, we aim to continue to meaningfully increase value and decrease overall risk for Fortress shareholders. Looking ahead, we expect 2020 to be a record revenue-generating year and a transformational one for many of the development-stage programs across Fortress and our partner companies. Finally, we look forward to continued acquisitions of marketable dermatology drugs and in-licenses of development-stage drug candidates.

2019 and Recent Corporate Highlights1:Marketed Dermatology Products

IV Tramadol

CUTX-101

CAEL-101

MB-107 (Lentiviral Gene Therapy for XSCID)

Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

CK-101 (third-generation EGFR inhibitor)

MB-102 (CD123-targeted CAR T cell therapy)

MB-101 (IL13R2-targeted CAR T cell therapy)

MB-108 (Oncolytic Virus C134)

MB-104 (CS1-targeted CAR T cell therapy)

MB-103 (HER2-targeted CAR T cell therapy)

MB-105 (Prostate Stem Cell Antigen (PSCA)-targeted CAR T cell therapy)

MB-106 (CD20-targeted CAR T cell therapy)

BAER-101 (novel 2/3subtype-selective GABA A positive allosteric modulator [PAM])

General Corporate

Financial Results:

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 and development-stage drugs and drug 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 therapeutic 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., 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

____________________________________________1 Includes product candidates in development at Fortress, majority-owned and controlled partners and partners in which Fortress holds significant minority ownership positions. As used herein, the words we, us and our may refer to Fortress individually or together with our affiliates and partners, as dictated by context.

FORTRESS BIOTECH, INC. AND SUBSIDIARIESConsolidated Balance Sheets($ in thousands except for share and per share amounts)

FORTRESS BIOTECH, INC. AND SUBSIDIARIESConsolidated Statements of Operations($ in thousands except for share and per share amounts)

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Fortress Biotech Reports Record Fourth Quarter and Full-Year 2019 Financial Results and Recent Corporate Highlights - BioSpace

Global Cancer Gene Therapy Report available at Digits n Markets contains an overview of the Global Cancer Gene Therapy which covers market size, opportunities, trends, growth rate, and competition landscape. The Global Cancer Gene Therapy is segmented Source, Product Type Applications and regions. With forecast to 2027.

Digits n Markets has recently published a comprehensive market research report on the Global Cancer Gene Therapy that includes evaluation of market size and various segments. The competitive environment is analyzed along with study of winning strategies adopted by key players.

The report is a detailed study on the accounting Global Cancer Gene Therapy with details regarding an in-depth evaluation of the industry vertical. The study is performed taking into consideration a twofold aspect of consumption and production. Speaking of the product category, the report provides detailed product remuneration, manufacturing of the product and the gross margins of the firms manufacturing the products. With regards to the consumption, the study reveals the product consumption value and the product consumption volume along with the status of import as well as the export of the products.

The Global Cancer Gene Therapy Market Anticipated to exhibit a CAGR 35.1% during Forecast Period. 2018-2025

Avail a free sample in PDF format along with a quick look at vital report briefs:https://digitsnmarkets.com/sample/6650-global-cancer-gene-therapy-market

Key Questions Answered by the Report:

Numerous micro and macro-economic factors impacting the growth of the market are analyzed and the data is represented in a way to aid the clients to enhance their strategic decision making. Key players operating in the Global Cancer Gene Therapy are:

Table of Content

Chapter 1: IntroductionChapter 2: Executive SummaryChapter 3: Market OverviewChapter 4: Cancer Gene Therapy Market, By TypeChapter 5: Cancer Gene Therapy Market, By ApplicationChapter 6: Cancer Gene Therapy Market, By RegionChapter 7: Competition Landscape

Originally posted here:
Global Cancer Gene Therapy Market, Trends, Analysis, Opportunities, Share and Forecast 2018-2025 - Galus Australis

SAN DIEGO and PENNINGTON, N.J., March 17, 2020 /PRNewswire/ --OncoSec Medical Inc. Incorporated (NASDAQ:ONCS) (the "Company" or "OncoSec"), a company developing late-stage intratumoral cancer immunotherapies, today announced the publication of positive TAVO monotherapy data in patients with metastatic melanoma in the Annals of Oncology. The publication titled, "Intratumoral Delivery of Tavokinogene Telseplasmid Yields Systemic Immune Responses in Metastatic Melanoma Patients," features data previously highlighted at both American Association of Cancer Research (AACR) and the Melanoma Bridge annual meetings.Annals of Oncologyis the official publication of the European Society for Medical Oncology.

The complete publication in Annals of Oncology is linked here and available on OncoSec's website at https://oncosec.com/publications/.

The publication describes OncoSec's study of patients with Stage III/IV melanoma who were treated intratumorally with plasmid encoding IL-12 (tavokinogene telseplasmid or TAVO), followed by electroporation on days 1, 5, and 8 every 90 days in the main study with additional patients treated in two exploration cohorts with alternative schedules. Correlative analyses for programmed death-ligand 1 (PD-L1), flow cytometry to assess changes in immune cell subsets and analysis of intratumoral immune-related gene expression were carried out on pre-and post-treatment samples from study patients, as well as from additional patients treated during exploration of additional dosing schedules beyond the pre-specified protocol dosing schedule.Response was measured by study-specific criteria to maximize detection of latent and potentially transient immune responses in patients with multiple skin lesions.Toxicities were graded by the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).

The objective overall response rate (ORR) was 35.7% in the main study, with a complete response (CR) rate of 17.9%.The median progression-free survival in the main study was 3.7 months while the median overall survival was not reached at a median follow up of 29.7 months. A total of 46% of patients in all cohorts having both injected and uninjected lesions experienced regression of at least one of these uninjected lesions and 25% had a net regression of all untreated lesions. Transient procedural pain (n= 24, 80%) and injection site reactions were the most commonly experienced adverse events.

Transcriptomic and immunohistochemistry analysis showed that immune activation and co-stimulatory transcripts were up-regulated, with an increase of adaptive immune resistance.

The publication concluded that intratumoral TAVO was well-tolerated and led to systemic immune responses in advanced melanoma patients. While tumor regression and increased immune infiltration were observed in treated as well as untreated/distal lesions, adaptive immune resistance limited the response.

"TAVO treatment appears to drive a change in the immune microenvironment, which results in an immune response to melanoma with minimal systemic toxicity. These data demonstrate that thisin situtumor vaccination strategy may be a safe and effective approach to inducing multiple sustained, productive changes in the immune microenvironment that would be too toxic using similar systemic agents and drive significant clinical results," concluded study co-author Adil Daud, M.D., Department of Medicine, University of California, San Francisco."We look forward to continued evaluation of the TAVO approach as a monotherapy in future clinical trials."

TAVOis currently being evaluated as a combination therapy in multiple clinical trials, including KEYNOTE-695, a pivotal trial in late-stage anti-PD-1 checkpoint refractory metastatic melanoma, and two phase 2 trials, one for triple negative breast cancer (TNBC) and a second for head and neck cancer. TAVO enables the intratumoral delivery of DNA-based IL-12, a naturally occurring protein with immune-stimulating functions.OncoSec's technology, which employs electroporation, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body.Results from recently completed clinical studies of TAVOhave demonstrated a local immune response, and subsequently, a systemic effect as either a monotherapy or combination treatment approach.

"While our ongoing pivotal KEYNOTE-695 study is evaluating TAVO and KEYTRUDA combination therapy in late-stage checkpoint refractory metastatic melanoma patients and has begun to yield positive results, publication of monotherapy data with TAVO demonstrates its utility as a standalone treatment in this patient population," stated Christopher Twitty, Ph.D., OncoSec's Chief Science Officer and a co-author of the publication. "The increase in adaptive resistance observed in the tumor microenvironment, in particular PD-L1, makes TAVO a particularly well-suited partner with anti-PD-1 checkpoint therapies.We are encouraged to see such a high response rate and will continue to evaluate TAVO's utility as a monotherapy for metastatic melanoma."

Annals of Oncology is the latest among a presently growing volume of peer-reviewed journals to highlight the potential of TAVO as a novel immunotherapy. A recent publication in Cancer Immunology Research, linked here, also explored the mechanism of activation of systemic immunity in patients from the TAVO monotherapy study in metastatic melanoma patients. Additionally, Clinical Cancer Research featured TAVO monotherapy data in Merkel cell carcinoma on the cover of its February 2020 issue, linked here. You can find a list of all TAVO publications and scientific presentations at https://oncosec.com/publications/.

About OncoSec Medical IncorporatedOncoSec Medical Incorporated (the "Company," "OncoSec," "we" or "our") is a late-stage biotechnology company focused on developing cytokine-based intratumoral immunotherapies to stimulate the body's immune system to target and attack cancer. OncoSec's lead immunotherapy investigational product candidate TAVO (tavokinogene telseplasmid) enables the intratumoral delivery of DNA-based interleukin-12 (IL-12), a naturally occurring protein with immune-stimulating functions.The technology, which employs electroporation, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body. OncoSec has built a deep and diverse clinical pipeline utilizing TAVOas a potential treatment for multiple cancer indications either as a monotherapy or in combination with leading checkpoint inhibitors; with the latter potentially enabling OncoSec to address a great unmet medical need in oncology: anti-PD-1 non-responders.Results from recently completed clinical studies of TAVOhave demonstrated a local immune response, and subsequently, a systemic effect as either a monotherapy or combination treatment approach. In addition to TAVO, OncoSec is identifying and developing new DNA-encoded therapeutic candidates and tumor indications for use with its new Visceral Lesion Applicator (VLA), to target deep visceral lesions, such as liver, lung or pancreatic lesions. For more information, please visit http://www.oncosec.com.

TAVOis a trademark of OncoSec Medical Incorporated.

KEYTRUDAis a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.

Risk Factors and Forward-Looking Statements This release, as well as other information provided from time to time by the Company or its employees, may contain forward-looking statements that involve a number of risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements. Forward-looking statements provide the Company's current beliefs, expectations and intentions regarding future events and involve risks, uncertainties (some of which are beyond the Company's control) and assumptions. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. You can identify forward-looking statements by the fact that they do not relate strictly to historical or current facts. These statements may include words such as "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "should," "will" and "would" and similar expressions (including the negative of these terms). Although we believe that expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements. The Company intends these forward-looking statements to speak only at the time they are published on or as otherwise specified, and does not undertake to update or revise these statements as more information becomes available, except as required under federal securities laws and the rules and regulations of the Securities Exchange Commission ("SEC"). In particular, you should be aware that the success and timing of our clinical trials, including safety and efficacy of our product candidates, patient accrual, unexpected or expected safety events, and the usability of data generated from our trials may differ and may not meet our estimated timelines. Please refer to the risk factors and other cautionary statements provided in the Company's Annual Report on Form 10-K for the fiscal year ended July 31, 2019 and subsequent periodic and current reports filed with the SEC (each of which can be found at the SEC's websitewww.sec.gov), as well as other factors described from time to time in the Company's filings with the SEC.

Company Contact:Gem HopkinsHead of Corporate Communications858-210-7334ghopkins@oncosec.com

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SOURCE OncoSec Medical Incorporated

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OncoSec Announces Publication of Positive TAVO Monotherapy Results in Metastatic Melanoma Patients in Annals of Oncology - BioSpace

Reuben Jacob and Fiona Kellas, Maucher Jenkins share their expertise on IP strategies and considerations for gene therapy inventions.

Gene therapy enables the treatment of a disorder or disease through the insertion of a gene into a patients cells instead of using drugs or surgery.This technique involves the introduction of genetic material into cells to compensate for abnormal genes in the patient or to make protein that will be beneficial to the patient.As an example, if a mutated gene causes a protein that is necessary for the correct functioning of cells to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.Gene therapy is understood to be useful in the treatment of a range of conditions such as cancer, cystic fibrosis, muscular dystrophy and Alzheimers disease.

UK role in gene therapy techR&D

Gene therapy is considered to be very important to the future of medicine and as such, many companies are focussing their research and development into gene therapy technologies.The UK is a growing industry for research into these areas and it is anticipated that by 2035 the UK industry around cell and gene therapy technologies will be worth in the region of 10 billion.Gene therapy research is still at an early stage.Due to this length of time and the associated costs involved in developing an effective gene therapy and taking it through to approval, it will be important for companies working in this area to put into place an effective IP strategy that will provide protection for their inventions and assist them in maintaining their market position.In addition, the competitive nature of the gene therapy industry means that will be important for a company to obtain patent protection for inventions being developed, as well as reviewing the patent landscape to check that the company is free to operate in their chosen area.

What makes something patentable?

In order for an invention to be patentable, it must be new, inventive and capable of industrial application.In addition to the requirement that an invention meets the above requirements of patentability, it is also important that the invention does not contain subject matter that is excluded from patentability.One of the challenges associated with obtaining patent protection for gene therapy inventions is that the European and US patent systems include a number of exceptions to patentability that are relevant to biological material and natural products.In Europe, it is not possible to obtain patent protection for a method of treatment or surgery of the human body.Thus, the removal of cells from a patient would not be considered to be patentable in Europe.In addition, inventions relating to stem cells that are derived from the destruction of human embryos are not patentable in Europe.In the US, recent case law (Molecular Pathology v Myriad Genetics, Inc, 2013) has meant that inventions relating to natural phenomena and natural products must show characteristics that are different to their natural counterpart(s).

However, despite the above challenges, there are a number of aspects of the gene therapy technology that may be eligible for patent protection.Typically, the gene therapy procedure can involve performing the required modification procedure on cells that have been removed from a patient before reintroducing the cells into the subject to produce their modified effect.The process of modifying the cells may be patentable if it fulfils the above requirements of patentability.In addition, it may be possible to obtain protection for the methods that are used to culture, manipulate or modify the cells that are used for gene therapy.

At Maucher Jenkins, we have a team of attorneys who can provide IP advice and assistance in the area of patenting inventions involving gene therapy, molecular biology and biochemistry.

by Fiona Kellas, Reuben Jacob

16 March 2020

14:20

Original post:
Thinking out loud: IP strategies for gene therapy inventions - Med-Tech Innovation

Researchers have successfully treated cardiac dysfuntion in mice models of Barth syndrome by using a gene therapy to replace TAZ.

A new study has demonstrated the success of a gene therapy in preventing or reversing cardiac dysfunction in mice models of Barth syndrome.

The research, conducted at Boston Childrens Hospital, US, investigated the mutations in a gene called tafazzin (TAZ), which causes life-threatening heart failure, weakens the skeletal muscles, undercuts the immune response and impairs overall growth in boys.

In 2014, the scientists from the study found that TAZ is essential in causing cardiac dysfunction, using a heart-on-a-chip model. Adding the TAZ gene normalised the heart muscle cells and organised the mitochondria inside these cells.

A gene therapy delivery vector (adeno-associated virus) being taken up in the heart, as shown in green at increasing magnification (credit: adapted from Prendiville TW et al., PLoS One 2015 May 29, https://doi.org/10.1371/journal.pone.0128105).

For the current study, the researchers moved to an animal model. They used two kinds of knockout mice: one with no TAZ in any cells and the other with TAZ absent in just the heart.

Most mice with the whole-body TAZ deletion died before birth, mostly because of skeletal muscle weakness. However some survived and these mice developed progressive cardiomyopathy, in which the heart muscle enlarges and loses pumping capacity. Their hearts also showed scarring similar to human patients with dilated cardiomyopathy, the left ventricles were also dilated and thin-walled.

Mice lacking TAZ just in their cardiac tissue, which all survived to birth, showed the same features. Electron microscopy showed heart muscle tissue to be poorly organised, as were the mitochondria within the cells.

The team then used gene therapy to replace TAZ, injecting an engineered virus under the skin in newborn mice or intravenously in older mice. Treated mice with whole-body TAZ deletions were able to survive to adulthood. TAZ gene therapy also prevented cardiac dysfunction and scarring when given to newborn mice and reversed established cardiac dysfunction in older mice whether the mice had whole-body or heart-only TAZ deletions.

Further tests showed that TAZ gene therapy provided durable treatment of the animals cardiomyocytes and skeletal muscle cells, but only when at least 70 percent of heart muscle cells had taken up the gene.

The problem is that neutralising antibodies to the virus develop after the first dose, said Dr William Pu, director of Basic and Translational Cardiovascular Research at Boston Childrens. Getting enough of the muscle cells corrected in humans may be a challenge.

L-R: Mitochondria from a normal mouse, a Barth syndrome mouse treated with the viral vector only, with no gene and a Barth syndrome mouse treated with a TAZ-carrying gene therapy vector (credit: Wang S; et al., Circ Res 2020 Mar 9; https://doi.org/10.1161/CIRCRESAHA.119.315956).

Maintaining populations of gene-corrected cells is another challenge, say the researchers. While levels of the corrected TAZ gene remained fairly stable in the hearts of the treated mice, they gradually declined in skeletal muscles.

The biggest takeaway was that the gene therapy was highly effective, concluded Pu. We have some things to think about to maximise the percentage of muscle cell transduction and to make sure the gene therapy is durable, particularly in skeletal muscle.

The findings were published in Circulation Research.

See the rest here:
TAZ gene therapy reverses Barth syndrome in mice - Drug Target Review

The Center for Gene Therapy at Nationwide Childrens Hospital is working to develop childrens gene therapy treatments. Officials say the gene therapy research and clinical trials there are starting to attract companies to central Ohio.

Nationwide Childrens Hospital is in the forefront of curing several genetic childhood diseases, transforming Columbus into a major medical hub, several gene therapy experts say.

The hospital's Center for Gene Therapy at the Abigail Wexner Research Institute is working to develop treatments for children, which is attracting patients and companies to Ohio, according to officials at Nationwide Childrens and JobsOhio, the state's economic development organization.

The illnesses that were making use of in gene therapy are devastating illnesses, said Dr. Kevin Flanigan, the director of Nationwide Childrens Center for Gene Therapy. These are ones we know that children would be significantly impaired for life or die because of the disease.

Gene therapy involves altering the genes inside the patient's cells in an effort to treat or stop disease. It gives doctors the chance to treat many previously untreatable rare and genetic diseases.

Gene therapy is currently available primarily in a research setting, with only four gene therapy products approved by the U.S. Food and Drug Administration for sale in the United States. One of the four, Zolgensma, started as a clinical trial for spinal muscular atrophy at Nationwide Childrens in 2014.

The hospital is working on a handful of gene therapy treatments for various childhood diseases that affect muscle, motor or mental functions, Flanigan said.

Gene therapy presents a tremendous opportunity for our medical system, and Columbus has been a huge part of that growth thanks to the work being done at Nationwide Childrens Hospital, Edith Pfister, chairwoman of the American Society of Gene & Cell Therapys communications committee, said in an email.

The FDA approved Zolgensma, a one-time treatment that intravenously delivers the gene that is missing in children with spinal muscular atrophy, on May 24.

SMA is a progressive childhood neuromuscular disease that is caused by a mutation in a single gene that attacks nerve cells. It causes major physical limitations including the inability to breathe, swallow, talk or sit up. Children born with SMA typically die or need permanent breathing assistance by the time they turn 2 years old.

Donovan Weisgarber was diagnosed with SMA type 1 at Nationwide Childrens in November 2015 when he was 5 weeks old. His parents, Matt and Laura Weisgarber, decided to participate in a clinical trial at the hospital and Donovan received Zolgensma.

Before the treatment, Donovan was unable to swallow and had difficulty breathing. Today, the 4-year-old has doubled his life expectancy and is able to talk, sit up, roll over and hold his head up on his own. He also attends the Early Childhood Education and Family Center on Johnstown Road on the East Side, which offers services from the Franklin County Board of Developmental Disabilities.

(Gene therapy) has given us an opportunity that we otherwise wouldnt have to love Donovan and experience him, said Matt Weisgarber, 33, of northeast Columbus.

A lot of people hear Ohio and think flyover state, but now Columbus is going to be a hub of the most groundbreaking science known to mankind and thats a really cool thing, he said.

Boston Childrens Hospital and Childrens Hospital of Philadelphia also have impressive gene therapy centers, but Columbus sets itself apart from those East Coast cities, said Severina Kraner, JobsOhios health care director.

The cost to operate, manufacture and live in Ohio is cheaper than Boston and Philadelphia, putting Ohio in a position to win cell and gene therapy companies, she said.

People are being priced out of these coastal cities, Kraner said.

One of the companies who has committed to building in Columbus is Sarepta Therapeutics, a Massachusetts-based biopharmaceutical company. Sarepta signed an agreement with Nationwide Childrens in May 2019, giving the company the licensing to a gene therapy treatment that came out of hospital research for limb-girdle muscular dystrophies, a group of diseases that cause weakness and wasting of the muscles in the arms and legs.

Sarepta is scheduled to open an 85,000-square-foot Gene Therapy Center of Excellence near Nationwide Childrens Hospital in the fall to do early research for all the companys gene therapy programs. A team of about 30 employees from Sarepta is currently working at a facility at Easton Town Center.

The region has every ingredient needed for a thriving gene therapy cluster: a strong academic foundation, world-renowned research hospitals, and, now, industry investment, Louise Rodino-Klapac, Sareptas senior vice president of gene therapy, said in an email. All of these contribute to creating a pipeline of talented people who will accelerate scientific advances that help patients.

Nationwide Childrens recently also announced it will be expanding its gene therapy research by creating Andelyn BioSciences, a new for-profit subsidiary that will manufacture gene therapy products for the biotechnology and pharmaceutical industries.

Were hoping, and we have a vision, that Andelyn can help capitalize a biotechnology hub in central Ohio focused on developing and advancing gene therapies, said Dr. Dennis Durbin, Nationwide Childrens chief science officer.

Andelyn BioSciences will launch this summer and operate out of the Abigail Wexner Research Institute, 575 Children's Crossroad. Nationwide Children's is trying to secure a permanent location for Andelyn and is looking at land on Ohio State Universitys West Campus.

Gene therapy treatment, however, comes at a high price.

The manufacturer set the price of Zolgensma at more than $2.1 million. Insurers can pay $425,000 a year for five years for one treatment.

Insurance companies are used to regular installment payments, but the single-dose nature of gene therapies are adding a level of uncertainty to health insurance structures, Pfister said in an email. A one-time administration gene therapy costs less overall, but it occurs in one upfront payment.

Pfister said she is hopeful the cost of gene therapy will go down.

Currently, most of the FDA-approved gene and cell therapies are tailored for the specific patient, but theres an incredible amount of research going into standardizing the components and delivery mechanisms behind gene therapy, Pfister said in an email.

Dr. Jerry Mendell helped usher in the era of gene therapy at Nationwide Childrens when he came to the hospital in 2004.

Nationwide Childrens first gene therapy trial was in 2006 for duchenne muscular dystrophy, a rare, inherited, degenerative muscle disorder that almost exclusively affects boys.

Things have really changed significantly in the gene therapy world because of the contributions weve made here, and its been a very gratifying experience, said Mendell, the principal investigator in Nationwide Childrens Center for Gene Therapy.

Read the rest here:
Nationwide Childrens among hospitals leading the way in gene therapy - The-review

The hunt for the elixir of life is such a universal mythological trope that to talk about it in the context of science seems almost ridiculous. But breakthroughs in the last decade have made the impossible seem possible, and researchers are quickly converging on the consensus that aging may well be a disease that we can treat just as easily as any other.

Impressive results in animals that have had their lifespans boosted by up to 40 percenthave started making their way into humans. Some trials are more questionable than others, but most promisingly there seem to be multiple potential avenues, from cocktails of common drugs to gene therapies and stem cell treatments.

Stem cells are particularly promising, because they can be coaxed into becoming any kind of cell before being transplanted to treat damaged tissue. These therapies often fail to work well in older tissue, though, limiting their future use in older patients who could need them most. This appears to be because these tissues have significantly higher levels of inflammation that prevent stem cells from properly integrating.

Now Portuguese researcher Joana Neves has won the 2019 Sartorius & Science Prize for Regenerating Medicine & Cell Therapy for her discovery of a way to sidestep this roadblock and significantly increase the success of stem cell treatments.

Because of the central importance of tissue repair to all organisms, Neves assumed that many of the mechanisms behind it would be shared among all animals. So she started looking at proteins produced by immune cells in the well-known animal model of the fruit fly.

She discovered that a poorly-understood protein known as MANF (mesencephalic astrocyte-derived neurotrophic factor) plays a crucial role in reducing inflammation in fruit flies. More importantly, she found that mice and humans also produce it, and its prevalence reduces in all the species as they age, suggesting it plays a key role in limiting age-related inflammation.

That prompted her to see if introducing MANF would boost the effectiveness of stem cell treatments in older animals. She used the protein in combination with a procedure that uses stem cells to replace degenerating photoreceptors in the retina of older mice and found it greatly improved the restoration of vision.

Going further, her research team then investigated whether MANFs anti-inflammatory effects could have more general age-defying benefits. Previous research had already demonstrated that infusing old mice with blood from younger ones could reduce various signs of aging, and by carrying out similar experiments the team showed that MANF is one of the factors responsible for that outcome. They even showed that directly injecting mice with MANF could have similar effects.

Translating these ideas to treat other diseases and for use in humans will take some time, but the research chimes with work on an emerging class of drugs known as senolytics. These are drugs that kill senescent cells, which are zombie cells that become more prevalent as we age and spew out harmful chemicals that result in chronic inflammation.

Senolytics are generally seen as a broad-spectrum treatment that could help stave off multiple conditions at once, but they could also be used to create a more hospitable environment for stem cell treatments just like MANF.

There are still plenty of barriers to bringing any of these treatments to the clinic, from the difficulty of producing stem cells to the challenges of regulating treatments for aging (a condition we still dont formally class as a disease), or fighting back against the huge number of bogus treatments that threaten to undermine trust in the field. But given the huge potential for near-term impact, theres growing momentum.

Weve moved from being able to extend health and lifespan of simple organisms like yeast and worms and flies to being able to do this quite easily in animals, in mice and monkeys, David Sinclair, director of the Center for the Biology of Aging at Harvard Medical School, told the Harvard Gazette.

He added that instead of trying to treat one disease at a time, he believes we can develop medicines that will treat aging at its source, therefore having a much greater impact on health and lifespan than drugs that target single diseases.

The wheels are in motion for us to find out.

Image Credit: Monika Robak from Pixabay

See the original post:
To Turbocharge Anti-Aging Treatment, Just Add... a Protein Found in Fruit Flies? - Singularity Hub

He swipes once. Twice. Three times.

The lidocaine goes in, to freeze the skin. Then the needle.

Slowly, precisely, Rasquinha removes spinal fluid through the lumbar puncture, commonly known as a spinal tap. He then gives him the injection that Snow and his wife, Kelsie, believe is saving his life.

They believe because they want to, because they have to, and because -- against every single odd, against every single thing you've ever heard regarding amyotrophic lateral sclerosis, or ALS -- it just might be working.

Snow, a 38-year-old assistant general manager for the Calgary Flames, was diagnosed with ALS on June 17. It was not a surprise. His family has been ravaged by the familial form of the disease, with his father, two uncles and a cousin having died from ALS caused by the SOD1 genetic mutation.

ALS affects motor neurons, the cells that control muscle movement. As ALS progresses, the motor neurons die, the muscles become weaker, and eventually movement slows or becomes impossible.

It's what Snow saw happen in his right hand, how he began to suspect that the disease had come for him too. But almost immediately after his diagnosis, which usually carries a life expectancy of between six and 18 months, Snow enrolled in a phase 3 clinical trial at Sunnybrook Health Sciences Centre in Toronto for those with the SOD1 mutation, which affects 2 percent of ALS patients.

In this, he was lucky, both that it was available and that he qualified.

He has made the trip to Toronto with Kelsie every four weeks since then, though initially there was a two-in-three chance that every trip brought him an injection of tofersen, a drug that doctors and researchers hoped could slow the progression of the disease.

For the first six months of the trial, two-thirds of the study participants are given the actual drug. One-third are given a placebo.

He does not know for sure whether he was in the group given the medication or the placebo. The Snows believe he was one of the lucky ones, one of those given tofersen. They believe because they, remarkably, have not seen a progression of the disease since he entered the trial. He still does not have use of his right hand. He has use of everything else, all the things they feared might slip away from him in the weeks and months after they confirmed the diagnosis.

The injection takes two minutes, two minutes of silent meditation for Snow. He thinks about his mom, Linda, who committed suicide in 2012. "Because," as he says, "she'd be really happy and really sad if she were here. One of the things I got from her was a real joy for life. She always was happy that I was that way. So, I think about her and how I get to move on because of this." He thinks about his dad, Bob, who died of ALS in 2018, and his uncles and his cousin.

He wishes they had had the opportunity for these two minutes. For that two-in-three chance.

"I move through those thoughts fairly quickly," he says. "I usually say a prayer or two. For them. Probably in part for myself."

****

The Snows have already been at Sunnybrook for hours at this point, Feb. 20 marking their 11th visit to this hospital, his 10th lumbar puncture. At 9:15 a.m., the driver picks them up at their hotel for the 20-minute ride. When they walk in the doors at the hospital, a massive campus that looks like a small airport, Snow is on edge.

They find their way down to Room UG21, where Snow will undergo a battery of tests, all of them designed to determine whether the progress of the disease has slowed, whether it has stopped, whether it has -- God forbid -- picked up again.

They start with a detailed neurological assessment.

"Have you experienced any changes to your health since you were last here?"

"No."

"Have you noticed any difference in your speech?"

"No."

They test knowledge next, the year, the season, the province, the city. A series of words that never changes from visit to visit: apple, penny, table. He counts backward from 100 by seven.

These are the hardest weeks, when the anxiety sweeps up and the visit looms closer and the fear that "no change" might have turned into "some change" pierces the bubble they have formed around themselves.

"My bad hand, I don't have any expectations for it being better," Snow says. "It's more the opposite, that I'm always kind of concerned about and testing the good parts. The other hand, in particular. Living without one hand is not difficult. Living without two hands is difficult.

"It's testing my good hand, and then I overtire it. And then I convince myself something is wrong."

This is when Kelsie can sense his perpetual optimism faltering.

"None of the physical aspects of any of that, the lidocaine, the needle itself, I couldn't care less about those things," Snow says. "It's just the emotional anguish that you can put yourself through, with wondering, am I a little bit different? And if I'm a little bit different, what does that mean?

"Because that's totally unknown. But then you worry, that could be a slippery slope."

He does a breathing test, to determine lung capacity, and one to test muscle strength. That is the one that gives him the most anxiety, because they are testing his hand and his strength, and that is where the disease started to eat at him.

"This is our 10th time doing most of these tests," Snow says.

"And there has not been any changes," research coordinator Jahan Mookshah says.

"Those are our favorite words," Kelsie says.

Kelsie bends down and ties her husband's shoes. The testing is over, for the moment.

****

There is no blood test for ALS. It's only diagnosed by process of elimination, a factor that often can be problematic, as the disease progresses while the patient is still waiting for answers. Snow was told June 10 by an EMG technician in Calgary that it was likely ALS, a diagnosis that was confirmed a week later by Dr. Michael Benatar at the University of Miami.

It was the last answer they wanted in the world. It was a death sentence.

They crumbled, seeing an unknown present, a future robbed.

"I don't know how we did it," Snow said. "We did our days, and then we cried."

Back in Calgary, fellow assistant general manager Craig Conroy got the news on the phone from Snow, while looking at Snow's kids, Cohen and Willa, now 8 and 5, who were staying at his house while their parents were in Miami. As Conroy said, "That just breaks your heart."

But there was some good news.

Snow had the best-case scenario: a family history and a mutation of the SOD1 gene. He had pressed the issue with doctors, over and over again, after starting to feel the weakness in his right hand while lifting weights during the Stanley Cup Playoffs in April, even as he wanted to believe it was a pinched ulnar nerve, desperate for any answer but this. For Snow, that meant it was only a matter of months between the onset of symptoms and the diagnosis, as opposed to 1 1/2 to 2 years, which is the average.

Which was why when the Snows talked to Benatar on June 17, it was like being flattened and lifted up in the same moment. They confirmed Snow had ALS. They also learned there was a trial, with openings for those with a fast-progressing form of the disease, like him. There was something they could do. There was a place they could go. There was a medicine he could take, produced by Biogen, a company whose corporate headquarters are on Binney Street in Cambridge, Massachusetts, the exact street where Snow lived when he moved there to work at The Boston Globe, another career and another lifetime ago.

There was -- maybe, possibly -- hope. An impossibility in this world.

"It's just like someone believably telling you you're going to die, then telling you you might not die," Kelsie said. "You can't describe that in a more dramatic way than that. You feel like you got your life back. You've got a chance. Just a chance, right?"

Most of the time ALS is sporadic -- that is, not inherited. But in 5 to 10 percent of cases, there is a familial link, an altered gene that has folded. When Kelsie researched trials in the immediate aftermath of the diagnosis, she realized that the studies that were furthest along happened to be for SOD1, Snow's mutation, the second-leading cause of familial ALS.

They would head to Toronto every month, their kids left with a rotating selection of friends, off to get the injection that might -- or might not -- include the liquid hope that his future rested on.

They had six months in which he could have been receiving either the drug or the placebo, until last month, when they were finally assured he would be getting the medication from this point on.

They told their kids that they would have to have a summer to last a lifetime.

"Because we felt like it was maybe going to have to," Kelsie said.

They jammed in everything, all the bike rides and ice cream and pictures, the trip to Merrymeeting Lake in New Hampshire, Snow's family cabin. They jammed happiness in every moment, the tears reserved for private times, for when they couldn't hold it in any longer, wiped away when the kids approached. She wondered if he would make it to February and, if he did, whether he would want to hang around once he got there. She told him to just stay the way he was. They put one foot in front of the other and, still, they don't know exactly how.

By October, they had started to feel differently. Hopeful.

Snow got back on the ice, courtesy of a Flames equipment manager, who sewed his glove into a fist, enabling him to grip a hockey stick. He shot a puck. It rang off the crossbar. Kelsie took a video. It was evident to her that the disease had not progressed, not robbed him of any more strength.

That moment, that month, changed them.

"I felt like that was probably the first moment where I could really breathe," she said.

They went public on Dec. 18, and it ricocheted around the hockey community. Kelsie wrote a letter detailing their diagnosis and their hope, something she has continued to chronicle on her blog, kelsiesnowwrites.com, and recently, in a story that ran in Sports Illustrated.

It has become a piece of her every day, every thought, something that still fells her at a moment's notice. When she is asked if there's been a day that she hasn't thought about ALS since the diagnosis, tears start to flow down her face.

"These three letters are a part of my life forever," Kelsie says. "I sure wish they weren't. But I know that a lot of good things will come to us as a result of this. And I've seen a lot of good in a lot of people because of this.

"There's so much beauty in grief, and there's so much beauty in sadness and tragedy. Because you get to see the best in people. And that's not a small thing. But I wish I didn't have a byline in Sports Illustrated. I don't want a byline in Sports Illustrated. I don't want to be telling this story. But if this is what I'm here for, then I'm OK with that. It's not small. I know that. I know that being there for somebody you love is significant. It's enough for me."

She swipes at the tears, running her fingers underneath her eyes. She sniffles.

"This is because I know you, too," Kelsie says, starting to laugh amidst the tears. "That's the problem."

****

It has been 15 years since I met Kelsie and Chris, back when she was still Kelsie Smith and he was still a baseball writer. The summer of 2005 was one of those summers that's only possible when you are young and there is no responsibility, no ties, no worry. When a White Russian just before the bar closes seems like a good idea, and a shut-off car in a parking lot is the right place to bare a soul, for a friend to reveal she just might be falling in love.

As Kelsie texted me recently, "Honestly, best summer of my life."

They were too young, realistically, for it to work. She was 21. He was 23. They met in a bar, the White Horse Tavern, down the street from the apartment that Snow would buy in Allston, Massachusetts, and that I would later buy from him and live in for the next decade.

He was the Boston Red Sox beat writer at the Globe, she was an intern in the Globe's sports department out of the University of Kansas, and I would be hired into the sports staff that summer as a general assignment reporter.

They got engaged the next summer, and married in December 2007, on a frigid, 9-degree day in St. Paul, Minnesota, where they moved after he was hired as the director of hockey operations for the Minnesota Wild, an unorthodox move that would launch him on a career in hockey and give me a chance to succeed him on the Red Sox beat at the Globe.

She covered the Minnesota Twins for the St. Paul Pioneer Press. We spent spring trainings together in Fort Myers, Florida.

It's hard to think about that day in 2007, so many years ago now, when they promised a lifetime to each other. Because, as Kelsie said, "That I have extra months is not lost on me. I know that I've been already given a gift. It's just that I want what we all thought we were getting when we got married. That we are going to grow old together."

Kelsie has always trusted in Snow, something she has never been shy about expressing. She's always believed he would do what he said, that he would succeed despite all the odds: That he would make the unheard-of jump from baseball reporter to NHL front office member; that he would figure it out after the Wild let him go; that they could find their way on one income (first hers, then his) in a new city, in a new country with a new baby.

And he did. They did.

"It was always like, Chris will figure this out," she said. "And that's probably an unfair amount of pressure that I put on him, but I just believed in him. I've always believed in him that much."

She still does, even in a battle that, up to this point, has been unwinnable.

****

At 1 p.m., after a pizza lunch, it's time to head back to UG21 for the lumbar puncture. Snow laughs about the diet he has been instructed to stick to -- high protein, high fat, high carb -- because losing weight is a marker of the disease. It's one thing he doesn't mind.

This session, too, starts with some tests. Of reflexes. Hands. Ankles. Feet. Jaw.

"This might hurt," Rasquinha says. "Sorry."

Rasquinha flips his hands over, examines them, tells Snow to relax. This is not Snow's forte.

Snow acknowledges yet again that he cannot do anything with his right hand. That went in June, and the atrophy has set in up to his elbow. Kelsie -- or, if he's at work with the Flames, one of his colleagues -- makes sure to cut his food for him, if needed; they tie his skates and his shoes. "Tight, but not too tight," as she puts it.

Testing done, it's time for the lumbar puncture. Snow is now on the open-label extension of the trial. After this visit, he is assured that the fluid sent into his spine will be tofersen. This is a comfort, even as they believe he has been receiving the medication all along.

"All right," Snow says, "let's rock and roll."

The risks are read out, the warnings given, as they always are. The Snows know this nearly by heart. Rasquinha snaps on sterile green gloves and a baby blue face mask. The lidocaine goes in. "Mosquito bite," Rasquinha calls it. "A little burn. Sorry."

Lorne Zinman enters the room. He is part of the reason for their hope, a man who oozes sunshine as he talks, despite having devoted his life to a disease that to this point has claimed every one of its victims.

"This is exciting," says Zinman, the director of the ALS/Neuromuscular Clinic at Sunnybrook and an associate professor of neurology at the University of Toronto. "This is gene therapy for ALS. I've been talking about this for, like, two decades. The fact that it's a reality and we could be helping people like Chris, it's just the happiest thing. I always say -- apology to my children -- the happiest day of my life will be when we finally have something for this disease, because I've seen too many people go through it."

It is easy for Zinman to be joyful these days. He, like all those who work with ALS, has spent years and years in the darkness, and he somehow just might see a little light emerging, a speck that grows bigger with every day that Snow, and potentially others, are stable.

"It's been decades and decades of, really, failure after failure in ALS," Zinman said. "When I give lectures, I put up a slide and I call it the graveyard of failed trials. And it's just failure after failure. The big turning point came when we found the first gene for ALS in the early '90s."

Researchers put the gene into mice, and the mice started to show signs of disease. They thought a cure would come soon after. They were wrong.

Things started to turn when advances in gene therapy led the focus to shift. They knew SOD1 made a protein, and that the protein misfolded, leading to the killing of motor neurons that led to ALS. That became their target.

On Feb. 3, Kelsie posted a video to Twitter showing Snow lifting his right hand at his wrist. It was something he hadn't been able to do since the disease took hold. They don't know what it means, and they try not to read too much into it, but the idea of stability, let alone improvement, sometimes seems like a dream they do not want to end.

And that is what makes Zinman so excited.

"We don't usually see that in ALS trials," he said. "The objective is to slow things down, not to make things improved. It's really exciting when you hear something like that, where I'm actually able to do something that I couldn't do before. You always have to take it with a grain of salt it's just really exciting when you hear something like that."

He envisions a day when a patient like Snow or one of his family members could find out he has the SOD1 mutation, that they could start him on the drugs in the pre-symptomatic phase, as a preventative, similar to the way that doctors have been able to use PrEP to keep HIV from taking hold, or the way that doctors use vaccines to prevent diseases.

"We're diagnosing ALS about five to six times a week, and three to four of our patients die every week," Zinman said. "I've been doing this for 15 years now. I see the faces of these patients, not just them, their family members and what they went through, so to be able to offer someone something like this -- research is hope."

When Zinman and I exited the room, I later learned, Snow grew emotional. He cried. It was joy and relief and release. He had finished another session at Sunnybrook. There had been no change. He soon would be assured of getting more of the medicine that has given him more, where before, he expected there would be less and less.

Because these two minutes, every four weeks, might just be saving him.

"The first few times he was dosed I had full-on panic attacks," Kelsie said. "It was all about the fact that I didn't know what [he was getting]. They'd come in and they'd put this little Ziploc baggie on the desk in there and it had this syringe in it. I remember noticing right away that it was 15 milliliters of this clear fluid. And that was every ounce of hope I had in my life."

****

The appointment over for the month, Snow walks down the corridor, a drab, shabby, beige hallway. It is hardly inspiring, with its fluorescent lights and well-trod flooring. But Snow is nearly bouncing. This is the best he feels all month, his hopes confirmed, the medication, he assumes, flowing through his body.

"Every time I walk this hallway, it's a good feeling," he says. "Because you picture yourself being wheeled."

But he can walk. He can breathe. He can do his job, almost the same way he always has, with a few simple modifications. He wears a suit without a tie these days, because he cannot tie one and, really, who needs it? He has not yet transitioned to slip-on shoes, perhaps out of stubbornness.

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Gene therapy giving Flames executive Snow hope in ALS fight - NHL.com

MarketandResearch.biz broadcasted a new title Global Hemophilia Gene Therapy Market 2020 by Company, Regions, Type and Application, Forecast to 2025 which delivers in-depth assessment on key market trends, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players functioning in the market. Vast coverage of industry players has been analyzed and further compared with the overall sector for each component such as profit, purchases, marketing, utilities, and depreciation. The report has a segmented market, by its types and applications. All segments have analyzed completely on the basis of its production, consumption as well as revenue. It provides forecasts for the global Hemophilia Gene Therapy market from 2020 to 2025.

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Global Hemophilia Gene Therapy Market 2020 Trending Technologies, Developments, Key Players and Forecast to 2025 - Bandera County Courier

The Global Gene Therapy Industry Market analysis report published on Upmarketresearch.com is a detailed study of market size, share and dynamics covered in XX pages and is an illustrative sample demonstrating market trends. It covers the entire market with an in-depth study on revenue growth and profitability. The report also delivers on key players along with strategic standpoint pertaining to price and promotion.

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The Global Gene Therapy Industry Market report entails a comprehensive database on future market estimation based on historical data analysis. It enables the clients with quantified data for current market perusal. It is a professional and a detailed report focusing on primary and secondary drivers, market share, leading segments and regional analysis. Listed out are key players, major collaborations, merger & acquisitions along with upcoming and trending innovation. Business policies are reviewed from the techno-commercial perspective demonstrating better results. The report contains granular information & analysis pertaining to the Global Gene Therapy Industry Market size, share, growth, trends, segment and forecasts from 2020-2026.

With an all-round approach for data accumulation, the market scenarios comprise major players, cost and pricing operating in the specific geography/ies. Statistical surveying used are SWOT analysis, PESTLE analysis, predictive analysis, and real-time analytics. Graphs are clearly used to support the data format for clear understanding of facts and figures.

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About UpMarketResearch:UpMarketResearch (https://www.upmarketresearch.com) is a leading distributor of market research report with more than 800+ global clients. As a market research company, we take pride in equipping our clients with insights and data that holds the power to truly make a difference to their business. Our mission is singular and well-defined we want to help our clients envisage their business environment so that they are able to make informed, strategic and therefore successful decisions for themselves.Contact Info UpMarketResearchName Alex MathewsEmail [emailprotected]Website https://www.upmarketresearch.comAddress 500 East E Street, Ontario, CA 91764, United States.

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Global Gene Therapy Industry Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts Research - Packaging News 24

The global Cancer Gene Therapy market research report initiates a comprehensive analysis of the global Cancer Gene Therapy market. It offers an in-depth analysis of the rate of development of the market in the estimated time period. Offering a brief outline, the report explores the influencing factors and size of the global Cancer Gene Therapy market in the estimated period. It also covers the major leading factors restraining the expansion of the global Cancer Gene Therapy market. The global Cancer Gene Therapy market research report emphasizes commanding players in the market linked with their market shares.

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The report offers helpful data that discloses the foremost players in the Cancer Gene Therapy market it also discusses the revenue division, business general idea, and product contributions of the key players in the market. The research study also analyzes the growth of the well-known market performers with the help of SWOT analysis. In addition, while evaluating the growth of main market players, the research report covers their recent business developments. Furthermore, various products and segments

Table of Content

Chapter 1: IntroductionChapter 2: Executive SummaryChapter 3: Market OverviewChapter 4: Cancer Gene Therapy Market, By TypeChapter 5: Cancer Gene Therapy Market, By ApplicationChapter 6: Cancer Gene Therapy Market, By RegionChapter 7: Competition Landscape

The global Cancer Gene Therapy market is bifurcated on the basis various segments. The development of each segment is evaluated along with their predicted growth in the future. Reliable data and statistics are collected from the regulatory authorities to calculate the growth of various segments of the market. Additionally, the global Cancer Gene Therapy market is also divided on the basis of various regions such as the North America, and Europe, Asia Pacific, Latin America, and Middle East & Africa.

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The Global Cancer Gene Therapy Market Anticipated to exhibit a CAGR 32.4% during Forecast Period 2018-2025. - Galus Australis

The one-of-a-kind resource at LivingWithLynch.org offers voices of hope in the face of a Lynch syndrome diagnosis on Lynch Syndrome Awareness Day, March 22, 2020.

In November 2019, 13 Lynch-positive patients, seven women and six men, eight cancer survivors and five previvors, met for the first time in Houston at a weekend sponsored byPromega Corporation. While together they had the opportunity to talk with experts in the field of genetic counseling, oncology, research, treatment, and immunotherapy. They were also given the opportunity to connect with each other and bond over shared experiences.

Lynch syndrome is a genetic condition that increases a person's risk for certain cancers, the most common being colorectal and endometrial, said Dave Dubin, founder of AliveAndKickn, Lynch positive, and colon and kidney cancer survivor. The Living with Lynch weekend was designed to empower patients living with Lynch syndrome to be advocates for themselves, their families, and others with a shared diagnosis. Our goal is to increase awareness of this genetic condition and provide support and hope for those with the Lynch syndrome gene in their family.

The experience is captured atLivingWithLynch.org. The microsite features videos and photos of patients sharing how their Lynch syndrome diagnosis impacts them, their family, and their outlook on the future. AliveAndKickn and the Colon Cancer Coalition hope to help patients Living with Lynch make informed decisions about health and provide a community to relate to as they navigate future health needs.

The Living with Lynch weekend was an amazing and inspiring experience, says Jean Edelstein, Living with Lynch participant, Lynch positive, and a cancer previvor. As a previvor, I know what a Lynch diagnosis means for me, I understand that I have an elevated risk for many cancers in my future. This weekend and meeting these amazing advocates gave me strength to face whatever comes next in my Lynch journey.

It was a privilege to be a part of the Living with Lynch weekend, adds Sarah DeBord, stage IV colon cancer patient (non-Lynch related), communications and program manager for the Colon Cancer Coalition. To hear the stories of individuals and families impacted by Lynch syndrome has opened my eyes even wider to the realities of a hereditary cancer diagnosis. Many individuals with a Lynch mutation develop related cancers much younger than the general population. It is important for individuals to know their family history and begin screening earlier for those with a known Lynch mutation.

About the Colon Cancer CoalitionThe Colon Cancer Coalition is based in Minnesota and dedicated to encouraging screening and raising awareness of colorectal cancer. The organizations signature Get Your Rear in Gear and Tour de Tush event series are volunteer-driven in communities throughout the United States. In 2019, the Colon Cancer Coalition granted over $1 million dollars to local communities to build and sustain programs promoting early prevention, screening, as well as patient support efforts for those living with this disease. By making the words colon, colorectal and colonoscopy a part of the everyday language, we believe we can overcome the fear and decrease deaths from this largely preventable cancer. For more information visitColonCancerCoalition.org.

About AliveAndKicknAliveAndKickns mission is to improve the lives of individuals and families affected by Lynch Syndrome and associated cancers through research, education, and screening. It is the only nationally recognized patient advocacy organization to provide resources, education and awareness for patients with Lynch syndrome hereditary cancer genetic mutations. AliveAndKickn is the go-to patient advocacy resource for patients, clinicians and researchers. In launching the HEROIC patient registry, AliveAndKickn has developed the first RWE patient driven database and worked with the top Lynch syndrome researchers around the country to support and advance research in the field. To read more about AliveAndKickn, please visitwww.AliveAndKickn.org.

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AliveAndKickn and the Colon Cancer Coalition look to empower patients LIVING WITH LYNCH - Curetoday.com

Although anti-CD19 chimeric antigen receptor T-cell (CAR-T)therapy has led to dramatic results in patients with hematological malignancieswho are seemingly out of treatment options, it is far from a panacea for allpatients, as a meaningful portion have disease that either never responds toCAR-T or eventually comes back after treatment with CAR-T.

To develop drugs for these patients who have exhausted yetanother treatment option, drug developers need to know how long these patientssurvive after CAR-T therapy so that they have historical controls in thepostCAR-T setting. These controls are thought to be the most useful asbenchmarks for future trials.

Not a lot is known as to what happens in the patients forwhom CAR-T therapy does not work, James Gerson, MD, assistant professor ofclinical medicine, Perelman School of Medicine, University of Pennsylvania,Philadelphia, told Cancer Therapy Advisor. Anecdotally, we say thatpatients often progress rapidly and arent able to get further therapies, butits not something theres a lot of data for.

With CAR-T therapy coming to the forefront as a treatmentoption, he said a lot of patients are going to get CAR-T therapy earlier onin their treatment course. As a result, drug discovery is most likely going tomove forward in the postCAR-T space because that will be the unmet need.

Researchers from Fred Hutchinson Cancer Research Center and Seattle Cancer Care Alliance, Washington, are helping address this unmet need, specifically for patients with chronic lymphocytic leukemia (CLL).

At the Transplantation & Cellular Therapy Meetings held in early 2020 in Orlando, Florida, these researchers reported the outcomes and characteristics of patients with CLL who received an investigational anti-CD19 CAR-T therapy during a clinical trial (ClinicalTrials.gov Identifier: NCT01865617), but either never had a response or eventually had disease relapse.1 The trial enrolled approximately 200 patients with CD19-expressing CLL, acute lymphoblastic leukemia, or non-Hodgkin lymphoma.

A total of 28 patients with CLL were included in the study,and among these, 16 (57%) had stable or progressive disease and 12 (43%)initially had a response after CAR-T therapy but then had disease relapse aftera median of 11 months.

Patients were retrospectively evaluated and found to havelived a median of 10.4 months after CAR-T therapy. Now we at least have anumber that tells you whats the expected survival if you were to have CAR T cells,said Premal Lulla, MBBS, assistant professor at the Center for Cell and GeneTherapy at Baylor College of Medicine and member of the Dan L DuncanComprehensive Cancer Center at Baylor College of Medicine, Houston, Texas,during an interview with Cancer Therapy Advisor.

Although he was not involved in the study, Dr Lulla saidlarge, multicenter analyses are needed to get a more comprehensive picture ofwhat happens to patients after CAR-T therapy fails.

The study also revealed 2 factors that were predictive ofoutcomes in this patient population. However, lead author Mazyar Shadman, MD,MPH, assistant member, clinical research division at Fred Hutchinson CancerResearch Center, Seattle, Washington, cautioned against overinterpretation ofthe data given that the study was retrospective and had a relatively smallsample size.

The first factor identified was the extent of treatment beforeCAR-T therapy. Specifically, patients whose disease progressed during treatmentwith ibrutinib and treatment with venetoclax before CAR-T therapy lived only amedian of 7 months after CAR-T therapy. In contrast, patients whose diseaseprogressed during treatment with only 1 of these drugs lived a median of 16.4months, an improvement that was determined to be statistically significant (P =.01).

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An Early Look at When CAR-T Therapy Fails Patients With CLL - Cancer Therapy Advisor

Newswise What gets the leader of the NIH jazzed?

Speaking to a packed West Pavilion auditorium March 6, Francis Collins, M.D., Ph.D., director of the National Institutes of Health, shared his picks of 10 areas of particular excitement and promise in biomedical research. (Watch the full talk here.)

In nearly every area, UAB scientists are helping to lead the way as Collins himself noted in several cases. At the conclusion of his talk, Collins addedhis advice for young scientists. Here is Collins top 10 list, annotated with some of the UAB work ongoing in each area and ways that faculty, staff and students can get involved.

1. Single-cell sequencing

[see this section of the talk here]

I am so jazzed with what has become possible with the ability to study single cells and see what they are doing, Collins said. They have been out of our reach now we have reached in. Whether you are studying rheumatoid arthritis, diabetes or the brain, you have the chance to ask each cell what it is doing.

Single-cell sequencing and UAB:Collins noted that Robert Carter, M.D., the acting director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, was a longtime faculty member at UAB (serving as director of the Division of Clinical Immunology and Rheumatology). For the past several years, UAB researchers have been studying gene expression in subpopulations of immune cells inpatients with rheumatoid arthritis.

Join in:Researchers can take advantage of the single-cell sequencing core facility in UABsComprehensive Flow Cytometry Core, directed by John Mountz, M.D., Ph.D., Goodwin-Blackburn Research Chair in Immunology and professor in the Department of Medicine Division of Clinical Immunology and Rheumatology.

Learn more:Mountz and other heavy users of single-cell sequencing explain how the techniqueslet them travel back in time and morein this UAB Reporter story.

2. New ways to see the brain

[See this section of the talk here]

The NIHsBRAIN Initiativeis making this the era where we are going to figure out how the brain works all 86 billion neurons between your ears, Collins said. The linchpin of this advance will be the development of tools to identify new brain cell types and circuits that will improve diagnosis, treatment and prevention of autism, schizophrenia, Parkinsons and other neurological conditions, he said.

Brain tech and UAB:Collins highlighted thework of BRAIN Initiative granteeHarrison Walker, M.D., an associate professor in the Department of Neurology, whose lab has been developing a more sophisticated way to understand the benefits of deep brain stimulation for people with Parkinsons and maybe other conditions, Collins said.

Join in:UABs planned new doctoral program in neuroengineering would be the first of its kind in the country.

Learn more:Find out why neuroengineering is asmart career choicein this UAB Reporter story.

3. Induced pluripotent stem (iPS) cells

[See this section of the talk here]

Researchers can now take a blood cell or skin cell and, by adding four magic genes, Collins explained, induce the cells to become stem cells. These induced pluripotent stem (iPS) cells can then in turn be differentiated into any number of different cell types, including nerve cells, heart muscle cells or pancreatic beta cells. The NIH has invested in technology to put iPS-derived cells on specialized tissue chips. Youve got you on a chip, Collins explained. Some of us dream of a day where this might be the best way to figure out whether a drug intervention is going to work for you or youre going to be one of those people that has a bad consequence.

iPS cells at UAB:Collins displayed images of thecutting-edge cardiac tissue chipdeveloped by a UAB team led by Palaniappan Sethu, Ph.D., an associate professor in the Department of Biomedical Engineering and the Division of Cardiovascular Disease. The work allows the development of cardiomyocytes that can be used to study heart failure and other conditions, Collins said.

Join in:UABs biomedical engineering department, one of the leading recipients of NIH funding nationally, is a joint department of the School of Engineering and School of Medicine. Learn more about UABsundergraduate and graduate programs in biomedical engineering, and potential careers, here.

Learn more:See howthis novel bioprinterdeveloped by UAB biomedical researchers is speeding up tissue engineering in this story from UAB News.

4. Microbiome advances

[See this section of the talk here]

We have kind of ignored the fact that we have all these microbes living on us and in us until fairly recently, Collins said. But now it is clear that we are not an organism we are a superorganism formed with the trillions of microbes present in and on our bodies, he said. This microbiome plays a significant role not just in skin and intestinal diseases but much more broadly.

Microbiome at UAB:Collins explained that work led by Casey Morrow, Ph.D., and Casey Weaver, M.D., co-directors of theMicrobiome/Gnotobiotics Shared Facility, has revealed intriguing information abouthow antibiotics affect the gut microbiome. Their approach has potential implications for understanding, preserving and improving health, Collins said.

Join in:Several ongoing clinical trials at UAB are studying the microbiome, including a studymodifying diet to improve gut microbiotaand an investigation of the microbiomes ofpostmenopausal women looking for outcomes and response to estrogen therapy.

Learn more:This UAB News storyexplains the UAB researchthat Collins highlighted.

5. Influenza vaccines

[See this section of the talk here]

Another deadly influenza outbreak is likely in the future, Collins said. What we need is not an influenza vaccine that you have to redesign every year, but something that would actually block influenza viruses, he said. Is that even possible? It just might be.

Influenza research at UAB:Were probably at least a decade away from a universal influenza vaccine. But work ongoing at UAB in the NIH-fundedAntiviral Drug Discovery and Development Center(AD3C), led by Distinguished Professor Richard Whitley, M.D., is focused on such an influenza breakthrough.

Join in:For now, the most important thing you can do to stop the flu is to get a flu vaccination. Employees can schedule afree flu vaccination here.

Learn more:Why get the flu shot? What is it like? How can you disinfect your home after the flu? Get all the information atthis comprehensive sitefrom UAB News.

6. Addiction prevention and treatment of pain

[See this section of the talk here]

The NIH has a role to play in tackling the crisis of opioid addiction and deaths, Collins said. The NIHs Helping to End Addiction Long-term (HEAL) initiative is an all-hands-on-deck effort, he said, involving almost every NIH institute and center, with the goal of uncovering new targets for preventing addiction and improving pain treatment by developing non-addictive pain medicines.

Addiction prevention at UAB:A big part of this initiative involves education to help professionals and the public understand what to do, Collins said. The NIH Centers of Excellence in Pain Education (CoEPE), including one at UAB, are hubs for the development, evaluation and distribution of pain-management curriculum resources to enhance pain education for health care professionals.

Join in:Find out how to tell if you or a loved one has a substance or alcohol use problem, connect with classes and resources or schedule an individualized assessment and treatment through theUAB Medicine Addiction Recovery Program.

Learn more:Discover some of the many ways that UAB faculty and staff aremaking an impact on the opioid crisisin this story from UAB News.

7. Cancer Immunotherapy

[See this section of the talk here]

We are all pretty darn jazzed about whats happened in the past few years in terms of developing a new modality for treating cancer we had surgery, we had radiation, we had chemotherapy, but now weve got immunotherapy, Collins said.

Educating immune system cells to go after cancer in therapies such as CAR-T cell therapy is the hottest science in cancer, he said. I would argue this is a really exciting moment where the oncologists and the immunologists together are doing amazing things.

Immunotherapy at UAB:I had to say something about immunology since Im at UAB given that Max Cooper, whojust got the Lasker Awardfor [his] B and T cell discoveries, was here, Collins said. This is a place I would hope where lots of interesting ideas are going to continue to emerge.

Join in:The ONeal Comprehensive Cancer Center at UAB is participating in a number of clinical trials of immunotherapies.Search the latest trials at the Cancer Centerhere.

Learn more:Luciano Costa, M.D., Ph.D., medical director of clinical trials at the ONeal Cancer Center, discusses the promise ofCAR-T cell therapy in this UAB MedCast podcast.

Assistant Professor Ben Larimer, Ph.D., is pursuing a new kind of PET imaging test that could give clinicians afast, accurate picture of whether immunotherapy is workingfor a patient in this UAB Reporter article.

8. Tapping the potential of precision medicine

[See this section of the talk here]

The All of Us Research Program from NIH aims to enroll a million Americans to move away from the one-size-fits-all approach to medicine and really understand individual differences, Collins said. The program, which launched in 2018 and is already one-third of the way to its enrollment goal, has a prevention rather than a disease treatment approach; it is collecting information on environmental exposures, health practices, diet, exercise and more, in addition to genetics, from those participants.

All of Us at UAB:UAB has been doing a fantastic job of enrolling participants, Collins noted. In fact, the Southern Network of the All of Us Research Program, led by UAB, has consistently been at the top in terms of nationwide enrollment, as School of Medicine Dean Selwyn Vickers, M.D., noted in introducing Collins.

Join in:Sign up forAll of Usat UAB today.

Learn more:UABs success in enrolling participants has led to anew pilot study aimed at increasing participant retention rates.

9. Rare diseases

[See this section of the talk here]

Rare Disease Day, on Feb. 29, brought together hundreds of rare disease research advocates at the NIH, Collins said. NIH needs to play a special role because many diseases are so rare that pharmaceutical companies will not focus on them, he said. We need to find answers that are scalable, so you dont have to come up with a strategy for all 6,500 rare diseases.

Rare diseases at UAB: The Undiagnosed Diseases Network, which includes aUAB siteled by Chief Genomics Officer Bruce Korf, M.D., Ph.D., is a national network that brings together experts in a wide range of conditions to help patients, Collins said.

Participants in theAlabama Genomic Health Initiative, also led by Korf, donate a small blood sample that is tested for the presence of specific genetic variants. Individuals with indications of genetic disease receive whole-genome sequencing. Collins noted that lessons from the AGHI helped guide development of the All of Us Research Program.

Collins also credited UABs Tim Townes, Ph.D., professor emeritus in the Department of Biochemistry and Molecular Genetics, for developing the most significantly accurate model of sickle cell disease in a mouse which has been a great service to the [research] community. UAB is now participating in anexciting clinical trial of a gene-editing technique to treat sickle cellalong with other new targeted therapies for the devastating blood disease.

Join in:In addition to UABs Undiagnosed Diseases Program (which requires a physician referral) and the AGHI, patients and providers can contact theUAB Precision Medicine Institute, led by Director Matt Might, Ph.D. The institute develops precisely targeted treatments based on a patients unique genetic makeup.

Learn more:Discover how UAB experts solved medical puzzles for patients by uncovering anever-before-described mutationandcracking a vomiting mysteryin these UAB News stories.

10. Diversity in the scientific workforce

[See this section of the talk here]

We know that science, like everything else, is more productive when teams are diverse than if they are all looking the same, Collins said. My number one priority as NIH director is to be sure we are doing everything we can to nurture and encourage the best and brightest to join this effort.

Research diversity at UAB:TheNeuroscience Roadmap Scholars Programat UAB, supported by an NIH R25 grant, is designed to enhance engagement and retention of under-represented graduate trainees in the neuroscience workforce. This is one of several UAB initiatives to increased under-represented groups and celebrate diversity. These include several programs from theMinority Health and Health Disparities Research Centerthat support minority students from the undergraduate level to postdocs; thePartnership Research Summer Training Program, which provides undergraduates and especially minority students with the opportunity to work in UAB cancer research labs; theDeans Excellence Award in Diversityin the School of Medicine; and the newly announcedUnderrepresented in Medicine Senior Scholarship Programfor fourth-year medical students.

Join in:The Roadmap program engages career coaches and peer-to-peer mentors to support scholars. To volunteer your expertise, contact Madison Bamman atmdbamman@uab.eduorvisit the program site.

Learn more:Farah Lubin, Ph.D., associate professor in the Department of Neurobiology and co-director of the Roadmap Scholars Program,shares the words and deeds that can save science careersin this Reporter story. In another story, Upender Manne, Ph.D., professor in the Department of Pathology and a senior scientist in the ONeal Comprehensive Cancer Center, explains how students in the Partnership Research Summer Training Program gethooked on cancer research.

Follow this link:
Looking to the future with Dr. Francis Collins - Newswise

Recently, Sangamo Therapeutics (SGMO) announced an extensive collaboration with Biogen (BIIB) to develop gene therapies for neurological diseases, comprising of Alzheimer's disease and Parkinson's disease. Sangamo is to receive a $350M from an upfront payment and proceeds from a stock purchase. The Biogen collaboration will be added to an impressive list that comprises of other big-name players such as Pfizer (PFE) and Gilead (GILD). Sangamos partnerships and collaborations are one of my primary reasons for making an early investment in Sangamo, so, I am even more bullish on SGMO following a collaboration with one of the leading neurological companies in the world.

I intend to review the details of the Biogen deal and why I am even more bullish on SGMO. In addition, take a look at the companys current market valuation in order to defend a long-term investment. Finally, I take a look at the charts to lay out a plan for starting a position in SGMO.

The Biogen collaboration will utilize Sangamo's ZFP technology to control the expression of tau and alpha-synuclein genes that are thought to be responsible for Alzheimer's, Parkinson's, and 10 other neurological targets. In return, Sangamo is expected to receive a $125M upfront payment and $225M in proceeds from a stock purchase at $9.21 per share. In addition, Sangamo is eligible for up to $2.37B in other milestone payments and could claim "high-single-digit to sub-teen double-digit royalties on net sales."

In terms of responsibilities, Sangamo will be take on the early research activities and the development of the AAV delivery vectors and ZFP-TFs. Once Sangoma has finished their preclinical work, Biogen will take over and will take the programs through the regulatory pathway and commercialization.

This collaboration is a significant event for Sangamo for a number of reasons. Personally, I find it extremely bullish that one of the worlds leading neurological companies has tapped Sangamos technology to take on some of the worlds most notorious neurological diseases. In addition, the details of the collaboration show that Biogen is dedicated to Sangamo with an impressive financial commitment and is now one a large shareholder of the company.

If these neurological programs are successful, they could be the first products that actually slow or stop the progression of these diseases with a one-time treatment. Obviously, this would be a medical breakthrough and could change the way we treat some of the worst diseases known to man.

The Sangamo-Biogen collaboration will use the company's ZFP technology to develop genomic therapeutics that switch genes on and/or off in the brain that are responsible for neurological diseases. Sangamo has announced that the ST-501 program will target tau and the ST-502 program will target alpha-synuclein. These programs will employ Sangamo's zinc finger protein transcription factors ZFP-TFs, which is a genome regulation technology that uses adeno-associated viruses AAVs.

ZFP-TF has already shown some success in neurological diseases such as Huntington's and ALS. Sangamo presented preclinical data at the companys R&D Day, which showed that more than half of the ZFP-TFs tested reduced the total alpha-synuclein levels by greater than 50% in ex-vivo cell culture system. So, it looks as if ZFP-TF could address the underlying source, by repressing the genes expression at the DNA level with a one-time administration. As a result, I am feeling optimistic that Sangamo's differentiated ZFP-TF technology will show several benefits over other therapeutic approaches.

In my previous article, I discussed how Sangamo has partnerships and collaborations with some of the most distinguished biotech and pharmaceutical companies in the world onboard (Figure 1). Perhaps the most impressive collaborations are Sangamos hemophilia A gene therapy candidate, SB-525, being partnered with Pfizer (PFE). Sangamo transferred their SB-525 IND to Pfizer, which activated a $25M milestone payment from Pfizer. Now, SB-525 will be entering its late-stage of development and could be the companys first licensed product.

Figure 1: SGMO Partnerships (Source: SGMO)

In addition, the company has a partnership with Sanofi (SNY) for sickle cell disease and beta-thalassemia. Sangamo also has collaborations with Takeda Pharmaceuticals (OTCPK:TKPHF) and Gileads (GILD) Kite Pharma. It is important to note that these partners entered agreements with Sangamo at an early stage of development and were willing to agree to big financial commitments to Sangamo in order to get a piece of the action.

Thus far, Sangamo has brought in around $700M in license fees, milestones, and equity from these partnerships or collaborations. Sangamo could pull in up to a cumulative $6.34B in potential milestone payments from these partnerships.

Looking at the names in figure 1, we can say the company has a knack for attracting and closing major deals. Not only am I bullish about the details of these partnerships, but I also bullish about managements ability and willingness to sign big-name partners. Most developmental biotech/pharma companies would kill to get one of those names associated with their company and technology. Meanwhile, Sangamo has five big names willing to make major commitments before the company has marketed therapies. These partnerships should supply significant milestone payments that can be used to fund the companys impressive pipeline and OpEx. Looking at figure 2, we can see that Street analysts expect Sangamo to start collecting on these milestone payments and eventually royalties in the coming years (Figure 2).

Figure 2: Estimated Annual Earnings (Source: Seeking Alpha)

Currently, the SGMO is fairly valued for its expected 2020 annual revenue, however, it is considerably undervalued when looking at the companys estimated ~$550M annual revenue in 2025, which is a forward price-to-sales of 1.50x. The biotech industrys average price-to-sales of 5x, so we should expect the market to start to recognize the opportunity and the share price will start to be priced in line with the rest of the industry.

Can the company hit $550M? I would point to the estimated $6.5B in potential milestone payments. The company has only collected about $700M, which leaves over $5B of potential milestones to get dispersed in the coming years, therefore, $550M in revenue isnt impossible.

What is more, the company expects their near-term cash from Biogen, combined with the company existing cash to be sufficient to potentially get through Pfizer's first BLA filing of SB-525 for Hemophilia A and beyond. So, it possible that we have little-to-no dilution in the coming quarters.

This historic market sell-off has hammered small-cap biotechs and SGMO has not been immune. Admittedly, the charts are far from looking bullish, so I am waiting for SGMO to show some signs of support before hitting the buy button.

Figure 3: SGMO Daily (Source: Trendspider)

I am looking for a double or triple bottom to ensure a level of support has been formed. Once the stock is able to clear the high of the formation before committing to a buy. Although SGMOs valuation points to the buy, I am not going to bind my cash to a ticker that is being dragged down with the rest of the market.

Disclosure: I/we have no positions in any stocks mentioned, but may initiate a long position in SGMO over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Original post:
Sangamo: Adding Another Partnership To The Bull Thesis - Seeking Alpha

SARASOTA, Fla. and NOVATO, Calif., March 16, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc.Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that it has dosed the first patient in its KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS).

The Phase 1/2 open-label, multiple-dose, dose-escalating study will enroll 20 patients to evaluate the safety, tolerability, and potential efficacy of GTX-102 in pediatric patients with Angelman syndrome. This is the first investigational study testing an antisense oligonucleotide as a potential therapy to treat AS. Further details can be referenced at: https://clinicaltrials.gov/ct2/show/NCT04259281.

Today is an important milestone with the dosing of the first patient in the KIK-AS study, stated Dr. Scott Stromatt, GeneTxs Chief Medical Officer GTX-102 has the potential to address the underlying deficiency that causes Angelman syndrome and we are excited, grateful and humbled to be leading this scientific quest. We look forward to the results of this study and sharing them with the Angelman community.

The GeneTx team has achieved a tremendous accomplishment rapidly advancing this program into the clinic, and GTX-102 may one day provide patients with Angelman syndrome with the first targeted therapy and a potentially transformative option for this devastating disease, said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

Chicagos Rush University Medical Center is the first clinical site to begin enrolling patients in the KIK-AS study, with additional sites being planned in Boston, Cincinnati, Denver, Los Angeles, New York and Ottawa, Canada.

When I held the syringe with this investigational treatment in my hand to inject it for the first time, I thought about the scientific advances in genomic and molecular medicine to produce potential treatments that bring hope of changing the disease course and ameliorating severity of symptoms in those with Angelman syndrome, said Elizabeth Berry-Kravis, site principal investigator at Rush. This is an exciting time for the field of neurodevelopmental disorders as we embark on a path to understanding the outcomes of treatments directed at correcting the underlying molecular causes of disease.

Pending additional site activation, GeneTx Biotherapeutics expects to report preliminary data from the first cohorts in the study in early 2021.

About Angelman Syndrome

Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

About GTX-102

GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expressionof UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). InAugust 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

About GeneTx Biotherapeutics

GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

AboutUltragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the Company's website atwww.ultragenyx.com.

Ultragenyx Forward-Looking Statements

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our partnership with GeneTx, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with theSecurities and Exchange CommissiononFebruary 14, 2020, and its subsequent periodic reports filed with theSecurities and Exchange Commission.

Contacts:

GeneTxPaula Evans630-639-7271Paula.Evans@GeneTxBio.com

Ultragenyx Investors & MediaDanielle Keatley415-475-6876dkeatley@ultragenyx.com

See the original post here:
GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome - BioSpace

[Editors note: 7 Biotech Stocks to Buy and Hold in 2020 was previously published in December 2019. It has since been updated to include the most relevant information available.]

If it were not for the surge in stock prices in the fourth quarter, biotechnology stocks would have ended the year in double-digit losses. Instead, they had a banner end of the year and now are suffering or succeeding based on the coronavirus and broad market pressures.

At a macroeconomic level, markets now believe the government regulators will not scrutinize drug pricing. The government is starting to realize that high healthcare costs are not due solely to rising drug prices. So, biotech companies that raise prices to offset higher research and development costs may do so in 2020.

Mega mergers in 2019, like AbbVie (NYSE:ABBV) buying Allergan (NYSE:AGN), signaled the undervaluation in the sector. In 2020, price-to-earnings valuations may expand to correct the market discount.

There are seven biotechnology stocks in 2020 that investors should hone in on. Get ready to pop these in your portfolio.

Source: Pavel Kapysh / Shutterstock.com

Biogen (NASDAQ:BIIB) rose from $220 to $300 in October when the company said it would resume filing for approval to bring its Alzheimers therapy drug to market. Then on Dec. 6, the company presented Phase 3 results for Aducanumab.

Over an 18-month period, it enrolled 3,285 patients over the two studies. The results showed that with a high dose over a long period of time, there was a meaningful slowing of decline in Alzheimers patients. And so, if the drug passes regulatory review, BIIB stock will live up to its hype.

The Aducanumab data showed mixed results. Although a higher dosage benefited patients, the side effects on the higher dosage are not clear.

Biogen reported steady revenue growth in the third quarter. Revenue grew 5% to $3.6 billion as earnings per share grew 17% to $8.39 (on a GAAP basis). Its multiple sclerosis drug portfolio is resilient. But Biogen is focused on addressing the intellectual property challenge with Tecfidera, a drug that treats relapsing forms of MS. This is offset by the launch of Vumerity, a drug also used to treat people with relapsing forms of MS.

Biogen reported revenue for Spinraza growing in the double-digits year-over-year and quarter-over-quarter. This drug treats patients with spinal muscular atrophy (SMA). Since Spinraza has a well-characterized safety profile, Biogen will run further studies to evaluate the benefits of higher doses to achieve greater efficacy.

Analysts who offer a price target on BIIB stock have an average of a $306.75 price target.

Source: Michael Vi / Shutterstock.com

Amgen (NASDAQ:AMGN) trades with analyst price targets that average $248. But at a trailing P/E of 18.6 times, the companys Otezla acquisition from Celgene (NASDAQ:CELG) is accretive to full-year results.

Last year Amgen announced a collaboration with BeiGene (NASDAQ:BGNE). Forming collaborations are in Amgens growth strategy. Since 2011, it grew by expanding to 100 countries, including China and other emerging markets. BeiGene, which represents a strategic investment in China, offers strong oncology expertise. It also has good commercial and clinical capabilities.

In 2020 and beyond, the Amgen-BeiGene collaboration may accelerate the commercialization of Amgens approved oncology products in the region. It paid $2.7 billion for a 20.5% equity stake, which was a 36% premium to BeiGenes 30-day average share price.

In Q3, Amgen reported EPS of $3.66. Revenue declined 2.9% to $5.7 billion. Despite a global sales decline, Amgen reported double-digit sales for a multitude of drugs. If generic competition lessens in 2020 and drug pricing improves, Amgen stock could continue trending higher.

Source: Casimiro PT / Shutterstock.com

Gilead Sciences (NASDAQ:GILD) surged early as a coronavirus darling before getting sucked back down into range by the prevailing market forces. And as investors wait for the stock to build an uptrend, they may collect a dividend that yields around 4%.

The U.S. approved Gileads Descovy for PrEP (pre-exposure prophylaxis). This drug is indicated to reduce the risk of sexually acquired HIV-1. Filgotinib was validated in the European Union and the company submitted a new drug application in Japan. Filgotinib is a JAK1 inhibitor that treats rheumatoid arthritis. If approved, it will compete with AbbVies (NYSE:ABBV) Humira.

Setting aside Gileads viability as a coronavirus vaccine leader, investors should consider Gilead for 2020 because of its strong cash flow generation. It produced $2.6 billion in cash from operations in the third quarter and now has $25.1 billion in cash and investments.

That healthy cash flow allowed the company to easily spend on R&D. It paid $5.5 billion for global research collaboration activities and it also invested in Galapagos (NASDAQ:GLPG). Galapagos gives Gilead access to many compounds, including six molecules that are in clinical trials. For example, Gilead gains rights to a Phase 3 candidate that treats idiopathic pulmonary fibrosis. GLPG1972 is a Phase 2b candidate that treats osteoarthritis.

Gilead could grow its revenue beyond the 12.7% rate. If its purchase of Kite Pharmaceuticals yields new products brought to market, the stock is set to blast off in late 2020 and beyond.

Source: Shutterstock

Regeneron (NASDAQ:REGN), which is getting better known for its blockbuster drug Dupixent, broke out of a downtrend late last year and hasnt stopped growing.

The company reported an EPS of $6.67 as revenue soared 23.1% year-over-year to $2.1 billion. Markets incorrectly worried over generic pressure on Eylea, which treats advanced wet age-related macular degeneration. Eylea sales grew 14% to $1.9 billion.

Dupixent has a growing addressable market. As it gains approvals worldwide, sales might even accelerate. In 2020, Regeneron will continue investing in researching activities for the drug to have additional indications.

For example, late this year it will have data in combination with Aimmunes (NASDAQ:AIMT) AR101 for treating peanut allergies. It will also have a data readout for its interleukin-33 (IL-33) antibody called Regeneron 3500 for treating atopic dermatitis and COPD (chronic obstructive pulmonary disease).

Regeneron continues to develop drugs in the oncology market. It launched Libtayo, an anti-PD-1 therapy that treats cutaneous squamous cell carcinoma. It also has new data from Libtayo in treating lung cancer.

At a P/E near 20 times, Regeneron still has plenty of upside potential. Even if the companys revenue were to slow to the 4% range in the next five years, the stock is still worth around $526.

Source: Shutterstock

Crispr Therapeutics (NASDAQ:CRSP), which is based in Switzerland, almost doubled in 2019. So after that performance, why should investors expect any more upside in 2020?

CRSP reported encouraging results for a potential immune-evasive cell replacement therapy for diabetes. From its press release, it said: The data demonstrate that the CyT49 pluripotent stem cell line, which has been shown to be amenable to efficient scaling and differentiation, can be successfully edited with CRISPR.

The results are further proof that regenerative medicine and gene editing may lead to cures in various diseases. Crispr is currently focusing on chronic diseases like diabetes.

Optimism for CRSP stock is so strong that a 4.3 million stock offering at $64.50 barely hurt the stock. The stock sale will add $274.1 million of cash, which Crispr may use to fund its ongoing clinical studies in sickle cell disease and beta-thalassemia.

Digging into the details, Crispr said that it successfully treated its first patient in the CLIMB SCD-121 study. The neutrophil engraftment had 46.6% hemoglobin F four months later. That suggests a curative response. Since the study is ongoing, the company will continue to inform investors of the safety profile of the treatment and its efficacy.

Crispr is ushering in an innovative form of therapy through gene editing. And markets like what the future holds.

Source: Shutterstock

Editas Medicine (NASDAQ:EDIT) has negligible revenue and is losing money, but its collaboration with Celgene gives it the resources in advancing its pipeline.

Editas reported a $70 million payment from its Celgene collaboration. This payment is in recognition of the work it did so far. It also includes contributions it will make to the collaboration. Its first patient will receive a doss of EDIT-101 by early 2020. This medicine treats subjects suffering from LCA10 or Leber congenital amaurosis. In Q3, Editas presented data for its USH2A study. It produced up to 60% corrected gene expression.

Looking ahead, additional collaborations with Celgene could give EDIT stock a lift. On its conference call, the company said:

All of that work is rolled into the new collaboration with Celgene. As you can imagine, as a leader and we believe the leader in T cell medicines for oncology including arguably the best CD19 and the best BCMA CAR-T programs that have a strong interest in maintaining their leadership position and gene editing is certainly an important part of what are likely to be the next generation of T cell medicines for oncology.

Editas collaboration with Celgene continues to progress nicely. Wall Street, which has a $42.50 price target, is also optimistic that the company will reward investors in 2020.

Source: Shutterstock

Innoviva (NASDAQ:INVA) has been trading sidewas all year until the recent mass-sell-off.

Last year, Innoviva booked $69.2 million in gross royalty revenues from GlaxoSmithKline(NYSE:GSK). This included royalties of $46.4 million connected to sales of Relvar/Breo Ellipta. This drug is a combination inhaled corticosteroid that treats patients with COPD (chronic obstructive pulmonary disease). Anoro Ellipta, which treats COPD and is taken once daily, brought in royalties of $11.6 million.

The company had $2.8 million in legal and related fees. Theravance Biopharma (NASDAQ:TBPH) initiated an arbitration. In the final decision, the Theravance Respiratory Company would reimburse those legal costs.

Global Relvar/Breo Ellipta sales fell 10% globally and by 32% in the U.S. Pricing discounts hurt revenue, and that revenue was partially offset by volume growth. Market share gains in various European markets and in Japan offset the overall revenue declines. Anoro Ellipta net sales grew 18% globally and were up 17% in the U.S. The negative impact of higher rebates was offset by higher sales volumes.

For 2020, Innoviva has a lower operating cost basis, helped by ending its Brisbane office lease. So, if the company reduces rebate offers and continues to grow its sales volume, revenue and profits may rebound.

As of this writing, Chris Lau held shares of INVA.

Here is the original post:
7 Biotech Stocks to Buy and Hold in 2020 - Investorplace.com

HOUSTON--(BUSINESS WIRE)--Kuur Therapeutics today announced the launch of its new business in partnership with Baylor College of Medicine and Baylors Center for Cell and Gene Therapy. Houston-based Kuur Therapeutics will advance the work of its predecessor, Cell Medica, to develop anti-cancer therapies using its innovative chimeric antigen receptor natural killer T cell (CAR-NKT) therapy platform.

IP Group, Baylor College of Medicine and Schroeder Adveq are investing to support two phase 1 studies: GINAKIT 2 (autologous CAR-NKT cells in neuroblastoma) and ANCHOR (allogeneic CAR-NKT cells in CD19 malignancy). The ongoing GINAKIT2 study is now enrolling patients at the third dose level and the ANCHOR study IND has recently been approved by the FDA, with first patient treatment expected in 1H 2020. The funding will also support the preclinical development of an allogeneic CAR-NKT product for treatment of hepatocellular carcinoma, ahead of an IND submission anticipated in 1H 2021.

In conjunction with the new investment, Kevin S Boyle, Sr, was named CEO of Kuur, succeeding Chris Nowers. Mr Boyle joined Cell Medica as CFO in February 2018. Kevin previously held senior finance roles at both NASDAQ-listed and private equity backed companies. He is an accomplished capital markets professional, having raised over $2.0 billion in equity and debt capital.

Kuurs novel CAR-NKT platform is a next-generation technology of engineered immune cells with enhanced functions for the treatment of hematological and solid tumors. It utilizes the unique properties of NKT cells, a specialized type of innate lymphocyte, which shares properties with both T and NK cells. This platform, developed in the laboratory of Baylor Principal Investigator Leonid Metelitsa, is exclusively licensed to Kuur by its partner and collaborator, Baylor College of Medicine.

Annalisa Jenkins, Chair of Kuurs Board, said: We are fortunate to have Kevin step up to the role of CEO. He will act as a change agent, leading the company during a crucial period for our clinical trials and working to secure the additional capital required to progress our two lead CAR-NKT products through the clinic.

The Board would also like to thank Chris for his exceptional leadership during a transition period that has resulted in a company on the right path forward for its investors, with a focus on its important collaborations.

Kevin S Boyle, Sr, Kuurs CEO, said: I am excited to lead Kuur Therapeutics at such a pivotal moment. We are making final preparations to take our off-the-shelf program into the clinic and believe the allogeneic approach holds huge promise for unlocking the potential of CAR therapies for large patient populations. Compared with patient-specific autologous CAR products, it is immediately available for treatment and less expensive to manufacture.

Leonid S. Metelitsa, BCM Principal Investigator, said: My goal is to make a difference in the lives of cancer patients, especially children, and Im excited to be working with the Kuur team to make this goal a reality. I believe that the NKT-cell platform technology, developed in my laboratory and progressed to first-in-human clinical testing in close collaboration with colleagues at BCM and Texas Childrens Hospital, offers a unique route to next-generation off-the-shelf CAR therapies for a broad range of malignancies.

- ENDS -

Notes to Editors

About Kuur Therapeutics

Kuur Therapeutics, headquartered in Houston, is a clinical-stage biotechnology company focused on transforming the treatment of solid and hematological cancers by developing next generation chimeric antigen receptor-natural killer T cell (CAR-NKT) therapies. Developing a portfolio of primarily allogeneic therapies, the companys revolutionary platform spanning both hematological and solid tumors is being created in partnership with Baylor College of Medicine and Baylors Center for Cell and Gene Therapy.

For further information, please visit http://www.kuurtx.com. Follow Kuur Therapeutics on LinkedIn

About Kuurs CAR-NKT cell technology

One of the challenges with allogeneic therapies is that infusing a patient with donor-derived lymphocytes can induce graft versus host disease (GvHD), a potentially life-threatening condition in which the infused cells recognize the patients tissues as foreign. The NKT cells used in Cell Medicas CAR-NKT platform have an invariant T cell receptor (iTCR) that does not distinguish between self- and non-self tissues, making them unlikely to induce GvHD when given to another person. Kuurs CAR-NKT cells are also engineered to secrete IL-15, to prolong persistence and enhance anti-tumor activity.

Baylor has previously reported evidence of clinical activity and a good safety profile in neuroblastoma patients treated at low dose levels of autologous CAR-NKT cells at the 2019 American Society for Gene and Cell Therapy conference.

About Baylor College of Medicine

Baylor College of Medicine (www.bcm.edu) in Houston is recognized as a premier academic health sciences center and is known for excellence in education, research and patient care. It is the only private medical school in the greater southwest and is ranked 16th among medical schools for research and 5th for primary care by U.S. News & World Report. Baylor is listed 21st among all U.S. medical schools for National Institutes of Health funding and number one in Texas. Located in the Texas Medical Center, Baylor has affiliations with seven teaching hospitals and jointly owns and operates Baylor St. Lukes Medical Center, part of CHI St. Lukes Health. Currently, Baylor trains more than 3,000 medical, graduate, nurse anesthesia, physician assistant and orthotics students, as well as residents and post-doctoral fellows.

Follow Baylor College of Medicine on Facebook (http://www.facebook.com/BaylorCollegeOfMedicine) and Twitter (http://twitter.com/BCMHouston).

About the Baylor College of Medicine Center for Cell and Gene Therapy

The Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital, and Houston Methodist Hospital in Houston, Texas is led by Helen E Heslop, MD, DSc (Hon), Director and Malcolm K Brenner, MD, PhD, Founding Director. The Center for Cell and Gene Therapy provides an infrastructure to rapidly translate novel cell and gene therapy protocols from the laboratory to the clinic. The comprehensive approach of the center brings a wide variety of scientists and clinicians together to develop strategies for the treatment of cancer, HIV and cardiovascular disease. Patient facilities include the adult stem cell transplant unit at Houston Methodist Hospital and the pediatric stem cell transplant unit at Texas Children's Hospital.

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Kuur Therapeutics Launches to Develop and Commercialize Off-the-shelf CAR-NKT Cell Therapies Targeting Hematological and Solid Tumors - Business Wire

Several government organizations and private institutions are investing in the regenerative medicine industry, for research and development. New development is undertaken as per the rules and regulations standardized by organizations like Food and Drug Administration (FDA) in the USA, the European Medicines Agency (EMA) in Europe and other regional authorities.

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From being able to identify and heal life threatening diseases, to their prevention, the health and medicine sector has witnessed a revolutionary development over the years, with regenerative medicine being a major innovation.

The average life expectancy of a human being has increased, thus giving rise to the geriatric population. That, coupled with the increasing number of neurodegenerative, orthopedic, oncological, genetic and chronic disorders, constitutes the major factors that are fuelling the growth of the regenerative medicine market size. It has provided a tremendous opportunity to manufacturers, to satisfy this demand of the market. They are collaborating with leading research specialists, labs, pharmaceutical and biotechnology companies, and investors, towards the invention of more regenerative medicines, like gene therapy, nanotechnology, and tissue engineering. The regenerative medicine industry is evolving as one of the most cost-effective and definitive healthcare solutions. Regenerative medicine product analysis shows cell therapy fetching the highest revenue in the forecast period followed by tissue engineering, gene therapy and small molecules and biologics.

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North America is expected to hold the highest regenerative medicine market share which invests in oncology and stem cell research. Presence of organizations such as National Cancer Institute, Alliance for Regenerative Medicine, National Institutes of Health and International Society for stem cell research supports the research and development of regenerative medicine which is accelerating the growth in this region. The Asia Pacific region is expected to witness the fastest growth during the forecast period, owing to the population growth in this region. Countries like China and Japan are introducing flexible regulations towards the clinical adoption of regenerative medicine techniques. The government is also actively participating in funding towards the infrastructure required for the research and development. Thus, boosting the global regenerative medicine market further.

What to expect from the upcoming report on Global Regenerative Medicine Market:

Growth projections of the global regenerative medicine market during the forecast period (2018-2025).

-Areas of growth and concern in the field of regenerative medicine.

Economic factors likely to influence the market.

Trends, drivers, opportunities, restraints, and challenges in the development and use of regenerative medicine.

The involvement of government and other private organizations that are elevating the research and development of regenerative medicine.

Analysis of the regional demographics affecting the market.

Information about the competitors in the market, and the initiatives taken by them to improve this market.

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Regenerative Medicine Market 2020 Global Share, Growth, Size, Opportunities, Trends, Regional Overview, Leading Company Analysis, And Key Country...

New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.

A new report from Americas Biopharmaceutical Companies has revealed that there are 362 cell and gene therapies in development in the US. Roughly a third of the therapies, 132, are potential treatments for rare diseases.

The research also highlights that the rate of R&D in this field is growing, as in 2018, a Pharmaceutical Research and Manufacturers of America (PhRMA) report on the cell and gene therapy pipeline found 289 therapies in clinical development in the US.

There are currently nine cell or gene therapy products approved by the US Food and Drug Administration (FDA).

Cell and gene therapies represent two overlapping fields of biomedical research with similar aims, which target DNA or RNA inside or outside the body. Gene therapies use genetic material, or DNA, to alter a patients cells and treat an inherited or acquired disease, whereas cell therapy is the infusion or transplantation of whole cells into a patient for the treatment of an inherited or acquired disease.

According to the report, the novel cell and gene therapies range from early to late stages of clinical development and are focused on a variety of diseases and conditions from cancer, genetic disorders and neurologic conditions.

Some of the cell and gene therapies in the pipeline include:

Another finding highlighted by the report is the 60 RNA therapeutics in development. Whilst not a kind of cell or gene therapy,RNA interference (RNAi) and antisense RNA use a genes DNA sequence to turn it off or modify the gene expression. So, these treatments can potentially inhibit the mechanism of disease-causing proteins.

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Almost 400 cell and gene therapies in US pipeline, finds report - European Pharmaceutical Review

Barth syndromeis a rare metabolic disease caused by mutation of a gene calledtafazzinorTAZ. It can cause life-threatening heart failure and also weakens the skeletal muscles, undercuts the immune response, and impairs overall growth. Because Barth syndrome is X-linked, it almost always occurs in boys. There is no cure or specific treatment.

In 2014, to get a better understanding of the disease,William Pu, MD, and colleagues at Boston Childrens Hospital collaborated with the Wyss Institute to create a beatingheart on a chip model of Barth syndrome. The model used heart-muscle cells with theTAZmutation, derived from patients own skin cells.It showedthatTAZis truly at the heart of cardiac dysfunction: the heart muscle cells did not assemble normally, mitochondria inside the cells were disorganized, and heart tissue contracted weakly. Adding a healthyTAZgene normalized these features, suggesting that gene replacement therapy could be a viable treatment.

But to fully capture Barth syndrome and its whole-body effects, Pu and colleagues needed an animal model. The animal model was a hurdle in the field for a long time, says Pu, director of Basic and Translational Cardiovascular Research at Boston Childrens and a member of the Harvard Stem Cell Institute. Efforts to make a mouse model using traditional methods had been unsuccessful.

As described in the journalCirculation Research, most mice with the whole-bodyTAZdeletion died before birth, apparently because of skeletal muscle weakness. But some survived, and these mice developed progressive cardiomyopathy, in which the heart muscle enlarges and loses pumping capacity. Their hearts also showed scarring, and, similar to human patients with dilatedcardiomyopathy, the hearts left ventricle was dilated and thin-walled.

Mice lackingTAZjust in their cardiac tissue, which all survived to birth, showed the same features. Electron microscopy showed heart muscle tissue to be poorly organized, as were the mitochondria within the cells.

Pu, Wang, and colleagues then used gene therapy to replaceTAZ, injecting an engineered virus under the skin (in newborn mice) or intravenously (in older mice). Treated mice with whole-bodyTAZdeletions were able to survive to adulthood.TAZgene therapy also prevented cardiac dysfunction and scarring when given to newborn mice, and reversed established cardiac dysfunction in older mice whether the mice had whole-body or heart-onlyTAZdeletions.

Thats where the challenge will lie in translating the results to humans. Simply scaling up the dose of gene therapy wont work: In large animals like us, large doses risk a dangerous inflammatory immune response. Giving multiple doses of gene therapy wont work either.

The problem is that neutralizing antibodies to the virus develop after the first dose, says Pu. Getting enough of the muscle cells corrected in humans may be a challenge.

Another challenge is maintaining populations of gene-corrected cells. While levels of the correctedTAZgene remained fairly stable in the hearts of the treated mice, they gradually declined in skeletal muscles.

The biggest takeaway was that the gene therapy was highly effective, says Pu. We have some things to think about to maximize the percentage of muscle cell transduction, and to make sure the gene therapy is durable, particularly in skeletal muscle."

Reference: Wang et al. (2020).AAV Gene Therapy Prevents and Reverses Heart Failure in A Murine Knockout Model of Barth Syndrome.Circulation Research.https://www.ahajournals.org/doi/abs/10.1161/CIRCRESAHA.119.315956.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Gene Therapy Reverses Heart Failure in Mouse Model - Technology Networks