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Archive for the ‘Gene Therapy Research’ Category

Global Cell and Gene Therapy Market, Forecast to 2025 by Product, Disease, End-user and Region – COVID-19 Updated – PRNewswire

DUBLIN, Aug. 10, 2020 /PRNewswire/ -- The "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019-2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Key Questions Answered

1. What is the cell and gene therapy market size and growth rate during the forecast period?2. What are the factors impacting the growth of the cell and gene therapy market share?3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market?4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares?5. Which product type/ end-user type/region is generating the largest revenue in the Asia-Pacific region?

The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.9% during the period 2019-2025

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe.

The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

Cell and Gene Therapy Market Segmentation

The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography.

In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product

Segmentation by Disease

Segmentation by End-user

Insights by Geography

In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 1-2 years, the number of approved gene therapies is expected to double.

The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography

Insights by Vendors

The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares.

Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors

Other Prominent Vendors

Market Dynamics

Opportunities & Trends

Growth Enablers

Growth Restraints

For more information about this report visit https://www.researchandmarkets.com/r/is5t27

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

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Global Cell and Gene Therapy Market, Forecast to 2025 by Product, Disease, End-user and Region - COVID-19 Updated - PRNewswire

112M Fund Launched to Commercialize UCLs Gene Therapy… – Labiotech.eu

The leading UK institution University College London and the firm AlbionVC have made the first closing of a 112M (100M) fund with the aim of investing in UCL research and spinouts with a focus on gene and cell therapy.

This is the second so-called UCL Technology Fund and could double the size of the first, which raised around 56M (50M) in 2016. The fund is managed by AlbionVC in collaboration with UCLs commercialization company UCL Business (UCLB). Investors in the second fund include the firm British Patient Capital and UCL itself.

While the value of the first closing was not disclosed, Anne Lane, CEO of UCLB, told me that investments from this fund have already begun, and the second close is expected within the next 12 months. The larger size of the fund than the first could also give the team more flexibility on how many investments it makes and how big they are.

We have a focus in terms of UCLs cell & gene therapy research and that is reflected in the portfolio of the fund, but no specific disease areas as such, Lane said.

Examples of investments in the pipeline for this fund include a gene therapy for an undisclosed neurometabolic disorder, a gene therapy for epilepsy, and a cell therapy for glaucoma.

One of the biggest success stories from the first fund is the gene therapy company Orchard Therapeutics, whose Nasdaq IPO raised around 290M in 2018. Another UCL gene therapy spinout, Freeline Therapeutics, is also geared to launch a Nasdaq IPO in the coming weeks.

UCL has a strong entrepreneurial scene comparable to the biotech hubs in Oxford and Cambridge. For example, UCL spinouts reportedly raised 644M (579M) in external investment between 2018 and 2019, the largest amount of any university in the country.

The European startup scene has been shaken by the Covid-19 pandemic this year, with the eurozone economy going into a deep recession. Furthermore, gene therapy developers have experienced disruption with their programs due to clinical trial delays and changes in strategy.

Regarding the effect of Covid-19 on their fundraising, Lane said that the pandemic definitely made it more challenging and took more time than expected. But UCLs research continues and we look to its research base in overcoming the challenges posed by the global pandemic.

Image from Shutterstock

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112M Fund Launched to Commercialize UCLs Gene Therapy... - Labiotech.eu

Spark Therapeutics Deepens Drug Development Expertise in Hematology and Rare Disease with Appointment of Gallia G. Levy, MD, Ph.D., as Chief Medical…

PHILADELPHIA, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the appointment of Gallia Levy, M.D., Ph.D., as chief medical officer. Dr. Levy will be responsible for strategic and operational leadership across all functions in the product development lifecycle, including setting the global development strategy for current and future pipeline programs.

We are thrilled to welcome Dr. Gallia Levy to our growing gene therapy company striving to create a world where no life is limited by genetic disease, said Jeffrey D. Marrazzo, chief executive officer, Spark Therapeutics. Dr. Levys passion for hematology and gene therapy research is immediately evident and exactly the perspective needed to achieve our goal of unlocking the full potential of gene therapy. Especially during this pivotal time in hemophilia research, Dr. Levys deep understanding of rare blood disorders and the community will help accelerate our ability to deliver potentially transformative gene therapies for hemophilia, while progressing potential gene therapies for other genetic disease across our pipeline.

Dr. Levy joins Spark Therapeutics from Genentech, also a member of the Roche Group, where she served as the Vice President and Global Head of the Rare Blood Disorders franchise in Product Development. In this role, she was responsible for the clinical development of HEMLIBRA for hemophilia A as well as treatments for other rare blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). She played a key role in the evolution of gene therapy as a new modality within the Roche Group.

Ive spent my career working to find new, innovative treatment approaches for patients affected by rare, life-altering disorders, and it is with great pride that I join the Spark team to help advance novel gene therapy programs and create next-generation solutions for patients, said Dr. Levy. Spark Therapeutics shares the same affinity for breaking barriers and putting the patient first, and I look forward to what we will achieve together.

Dr. Levy first joined Genentech in 2009, where she worked in both early and late-stage clinical development. She later moved to Portola Pharmaceuticals, where she led the clinical development program for hematology and oncology indications and returned to Genentech in 2014 to lead the hemophilia program.

Dr. Levy is board-certified in hematology and holds an M.D. and Ph.D. in Molecular and Cellular Biology from the University of Michigan. She completed her residency in internal medicine at Stanford University and a fellowship in hematology and oncology at the University of California, San Francisco. She also holds an M.S. in of Molecular and Cellular Biology from the University of Paris, VI and a B.A. from the University of California, Berkeley.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanokevin.giordano@sparktx.com(215) 294-9942

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Spark Therapeutics Deepens Drug Development Expertise in Hematology and Rare Disease with Appointment of Gallia G. Levy, MD, Ph.D., as Chief Medical...

LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the…

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter ended June 30, 2020, provided a business update and announced the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for LB-001 for the treatment of methylmalonic acidemia in pediatric patients. LogicBio released a separate press release this morning providing further details on the planned Phase 1/2 clinical design for LB-001.

We are thrilled to have received clearance to move forward with this first-in-human clinical trial with our lead product candidate, LB-001, for the treatment of methylmalonic acidemia, a life-threatening congenital genetic disease with no current therapeutic treatment options. This represents a significant milestone in our goal of bringing a treatment to MMA patients as well as for our GeneRide platform. We have maintained continuous dialogue with the centers of excellence that are planned to participate in the Phase 1/2 clinical trial, and we look forward to activating these sites as quickly as possible, said Fred Chereau, CEO of LogicBio. We have instituted systems attempting to mitigate COVID-19 dynamics on our study start-up process and, based on our best estimates, we plan to enroll our first patient in early 2021.

Commenting on the Next Generation Capsid Program, Mr. Chereau said, We are very excited about the recent advances in our novel capsid program, which has generated liver-tropic capsids intended for use in gene editing technologies such as GeneRide and other gene therapy approaches. We are focused on executing across all of our programs and look forward to sharing further details on our novel capsids in early 2021.

Appointment of Daniel Gruskin, M.D. to SVP, Head of Clinical Development

Daniel Gruskin, M.D. was appointed as SVP, head of clinical development in August 2020. Dr. Gruskin has served as interim head of clinical development of LogicBio since June 2020. In April 2020, Dr. Gruskin started consulting with the Company as a special advisor. Previously, Dr. Gruskin served in roles of increasing responsibility at Sanofi Genzyme, most recently as vice president, head of global medical affairs, rare disease, in which capacity he oversaw medical affairs, life cycle management, scientific affairs and other medical and development activities related to metabolic, rare and/or genetic diseases. Prior to his role at Sanofi Genzyme, Dr. Gruskin served as assistant professor, human genetics and pediatrics at Emory University School of Medicine, where he was also the chief of the genetics section at Childrens Healthcare of Atlanta.

Daniel has been instrumental in leading LB-001 clinical development efforts including getting the IND cleared. His deep experience in genetic medicines and metabolic diseases will serve LogicBio well as we look to execute on our goals for both the GeneRide and Next Generation Capsid platforms in search of transformative medicines, said Mr. Chereau.

Anticipated Milestones for 2020 and 2021:

Second Quarter 2020 Financial Results

Three Months Ended June 30, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms.

LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the Companys plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of its cash and cash equivalents to fund operating expenses and capital expenditure requirements. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Investors:Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Media:Stephanie SimonTen Bridge CommunicationsStephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the...

Gene Therapy Promising in BCG-Unresponsive Bladder Cancer – Medscape

More than half the patients with high-grade bacillus Calmette-Gurin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC) treated with nadofaragene firadenovec, an investigational intravesical viral gene therapy, achieved a clinical response at 3 months in a phase3 trial.

The results "provide a significant efficacy benefit that, pending regulatory approval, might offer patients with a difficult-to-treat bladder cancer a bladder-sparing alternative," said Neal Shore, MD, medical director for the Carolina Urologic Research Center in Myrtle Beach, South Carolina.

There is clearly an unmet need for new bladder-sparing treatments in these patients, said Fred Witjes, MD, from the Radboud Institute for Molecular Life Science in Nijmegen, the Netherlands, who discussed trial findings during the virtual European Association of Urology 2020 Congress.

"The drugs that we have are old and there is a limited availability for both MMC [mitomycin-C] and BCG. We need some alternatives for initial adjuvant therapy," he explained. "The unmet need is, of course, especially there in BCG-unresponsive patients or BCG-unresponsive CIS [carcinoma insitu]."

"Clinically appropriate patients with BCG-unresponsive NMIBC are currently faced with radical cystectomy," Shore explained during his presentation at the congress.

Nadofaragene firadenovec is a viral-based gene therapy that consists of a replication-deficient adenovirus that delivers the gene for interferon alpha-2b (IFN2b). When administered with the polyamide compound Syn3, the viral vector can deliver the IFN2b gene to the epithelial lining of the bladder. The gene is subsequently incorporated into cellular DNA, meaning that large amounts of the IFN2b protein can be produced locally.

For their open-label, randomized trial, Shore and his colleagues looked at 157 patients with a mean age around70 years. All participants had carcinoma insitu or high-grade Ta (noninvasive) or T1 (invasive) papillary disease with or without carcinoma insitu, and all had been unresponsive to standard intravesical treatment with BCG in the previous 12 months.

Nadofaragene firadenovec was administered once every 3 months, for up to four doses in the first year. If patients showed no signs of high-grade disease recurrence at 12 months, they were offered continued treatment.

For patients with high-grade carcinoma insitu, the complete response rate was 53.4% at 3 months and 24.3% at 12 months. For patients with papillary tumors, the response rate was 73.0% at 3 months and 44% at 12 months.

The majority of study participants (72%) received two or three courses of BCG overall; that rate was 68.3% for those with carcinoma insitu and 80.0% for those with papillary disease. Just over half the patients with carcinoma insitu were refractory to BCG, as were 70% of those with papillary disease.

Almost one third of patients will not respond to BCG, and more than 50% will experience recurrence and progression during long-term follow-up, according to results from the phase2 study of nadofaragene firadenovec, which Shore was involved in.

In that trial of 40 patients, the response rate was 30% at 12 months for those with carcinoma insitu, and durable responses were seen out to 36 months. Investigators reported no dose-limiting toxicities or immune toxicity.

In the phase3 study, treatment-emergent adverse events were experienced by 93% of participants, but the vast majority were transient and grade1 or 2 events; approximately 17% were grade3. There was one grade4 event, but this was not related to the study treatment.

The most common treatment-emergent adverse events were instillation-site discharge, reported by 33.1% of the patients; fatigue, reported by 23.6%; bladder spasm, reported by 19.7%; micturition urgency, reported by 17.8%; and hematuria, reported by 16.6%.

"Follow-up and treatment of these patients is ongoing in an extension study," Shore said.

"We do really need something new in nonmuscle invasive bladder cancer," Witjes observed. "There has to be an alternative to cystectomy and, fortunately, news is coming."

"There is a highly unmet need, but we have to realize that there is a lot in the pipeline," he explained. "We have trials with immune checkpoint blockade, vaccines, genetic therapy, and drug-delivery systems."

Nadofaragene firadenovec creates "adaptive immunity that may be lifelong," Witjes reported. "Nadofaragene firadenovec has a good basis, has consistent, good results, and it has a good safety profile. In light of current developments, I think this certainly is an interesting option."

The study was sponsored by FKD Therapies Oy and conducted in collaboration with the Society of Urologic Oncology Clinical Trials Consortium. Shore reports receiving research and consulting fees from Amgen, Astellas, Bayer, BMS, Dendreon, Fergene, Ferring, Janssen, Merck, Myovant, Nymox, Pacific Edge, Nucleix, Pfizer, Sanofi-Genzyme, Sun Pharma, and Tolmar. Witjtes reports receiving advisory or lecturer fees from multiple companies, but none relevant to his comments.

European Association of Urology (EAU) 2020 Congress. Presented July17, 2020.

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Gene Therapy Promising in BCG-Unresponsive Bladder Cancer - Medscape

Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 – PRNewswire

DUBLIN, Aug. 4, 2020 /PRNewswire/ -- The "Cell and Gene Therapy Tools, and Reagents: Global Markets" report has been added to ResearchAndMarkets.com's offering.

Gene and cell therapy are emerging as important tools to treat human health. Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers. The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene-based diseases. As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging. This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

The scope of this study encompasses an investigation of the market's cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors. This research analyzes each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years. Technological issues, including the latest trends, are discussed. The report analyzes the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

The Report Includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Market Breakdown by Region

Chapter 5 Market Breakdown by End User

Chapter 6 Government Regulations

Chapter 7 Patent Review/New Developments

Chapter 8 Analysis of Market Opportunities

Chapter 9 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/p5fqx6

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

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Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 - PRNewswire

In the face of COVID-19, cell and gene therapy space shows ‘remarkable resilience:’ report – FierceBiotech

In the early days of COVID-19, the Alliance for Regenerative Medicine (ARM) was unsure how the pandemic and its accompanying economic downturn would affect the cell and gene therapy space.

It was a really specific time when the world and the markets were clearly reeling from the first appreciation for the seriousness of COVID-19, Janet Lambert, the organizations CEO said.

Now, the numbers are inand theyre better than ever. In the first half of 2020, the regenerative medicine sector raised $10.7 billion, more than the total capital raised in 2019 and a 120% jump over the first half of 2019, ARM found in a new report titled, Innovation in the Time of COVID-19. The proceeds were shared pretty evenly between cell therapy companies ($7.5 billion) and gene and gene-modified cell therapy companies ($7.9 billion), with companies focused on tissue engineering reeling in $84 million.

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RELATED: Biotech IPO bonanza: Legend's $350M offering as Repare, Forma get in on the action

That $10.7 billion was driven by a couple of outsize deals and includes $1.4 billion raised in five IPOs, $1.6 billion in follow-on offerings and $3 billion in venture capital. Chinese CAR-T player Legend Biotech led the pack with its mammoth $487 million Wall Street debut in June, but its peers netted considerable sums too. That same month, gene therapy companies Generation Bio and Akouos raised $230 million and $244 million, respectively. In February, another gene therapy outfit, Passage Bio, raised $284 million and gene-editing biotech Beam Therapeutics bagged $207 million.

On the venture side, Sana Biotechnology scored $700 millionalmost as much as the five next largest private rounds raised by Orca Bio Elevate Bio, Legend, Freeline Therapeutics and Poseida, the report found. Like Legend, Generation Bio and Akouos also completed sizable private rounds the same year they went public.

RELATED: 'The silver lining': Biotech IPOs in the time of coronavirus

All this enthusiasm for this sector right now is evidenced by these really astonishing financing numbers I think the drivers of that enthusiasm remain in place and make me optimistic for the second half of 2020, Lambert said. We continue to see really promising clinical results. We continue to see products making it to market. We continue to see patient, regulator and payer enthusiasm for these products.

Part of that enthusiasm stems from an appreciation for the biotech sector generally, Lambert said.

Attention is being paid to what the biopharma sector can do for us all as we try to weather and get out of the pandemic, she said, echoing the sentiments of venture capitalists whove managed to raise life sciences funds in spite of the pandemic.

The other side of the equation is the nature of biotechbecause the drug development cycle is long, biotech investors arent looking for quarter-to-quarter returns, but at milestone readouts that can come more than a year after IPO, Jordan Saxe, head of healthcare listings at Nasdaq, said in a previous interview.

Biotech is actually fairly well positioned to weather these kinds of events because youre not relying on day-to-day consumer spending. Youre relying on meaningful clinical catalysts at the end of the day to really generate value, and thats still going to be there in this environment, said Jason Pitts, Ph.D., a principal at Sofinnova, in ARMs report.

RELATED: Flagship raises $1.1B to create biotechs for post-pandemic world

All this gas in the tank isnt just bankrolling existing cell and gene therapies, but also driving company formation, Lambert said. For the first time, ARM counts more than 1,000 companies working in the sector, with more than 1,000 clinical trials going on worldwide. More than half of those studies are in phase 2, with just over a third in phase 1 and the remainder in phase 3.

Of those studies, 11 are testing regenerative medicine approaches against COVID-19, with several academic research centers and biopharma companies working on new treatments to treat the disease in the short and long term.

Most of them are using cell therapies to address ARDS, or acute respiratory distress syndrome, which is a consequence of COVID-19, Lambert said. Unlike other prospects in the pipeline, such as antibodies, which could potentially be used to prevent infection as well as treat it, regenerative treatments focus on repairing damage to the lungs or other organs that patients can suffer as part of COVID-19.

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In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience:' report - FierceBiotech

Adverum Biotechnologies Announces Positive Interim Data from Cohorts 1-4 from OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for wet AMD,…

-- Continued robust treatment response from both high and low doses --

-- Long-term durability beyond 15 months from single IVT injection with zero rescue injections in Cohort 1 --

-- Well tolerated across all Cohorts; encouraging early safety data from Cohort 4 --

-- OPTIC enrollment complete; planning to start pivotal trial in wet AMD mid-2021 --

-- Company to host conference call and webcast with Key Opinion Leader Dr. Arshad Khanani today at 1:30 pm PT / 4:30 pm ET --

REDWOOD CITY, Calif., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced positive new interim data from Cohorts 1-4 of the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their wet age-related macular degeneration (AMD). The company also reported recent business progress and financial results for the second quarter ended June 30, 2020.

For the first time, interim data from all four cohorts of the OPTIC trial (July 23, 2020 cutoff date), including preliminary safety data from Cohort 4, are presented. These data further demonstrate the transformative potential of ADVM-022 to greatly reduce the treatment burden for patients with wet AMD:

OPTIC Phase 1 Clinical Trial Data:

Before: 9.6After: 0

Before: 10.0After: 1.3

N/A

1 Best corrected visual acuity (BCVA)2 Central retinal thickness (CRT)3 All patients from Cohort 1 (n=6)4 All patients from Cohort 2 (n=6) and Cohort 3 (n=9)5 Annualized rate (Before) = (number of IVTs in 12 months prior to ADVM-022) / (days from the first IVT in the past 12 months to ADVM-022 / 365.25)Annualized rate (After) = (number of aflibercept IVTs since ADVM-022) / (days from ADVM-022 to the last study follow-up / 365.25)6 Fluorescein angiography of posterior pole

Arshad M. Khanani, M.D., M.A., managing partner and director of clinical research, Sierra Eye Associates, clinical associate professor of ophthalmology, University of Nevada, and top enrolling investigator in the OPTIC trial said, In my experience the sustained anatomical treatment response following a single intravitreal injection of ADVM-022 is unprecedented, which is remarkable considering that the patients enrolled in OPTIC were difficult-to-treat and had previously required frequent injections to maintain their vision. The safety profile to date shows that ADVM-022 is well tolerated, and that the prophylactic steroid eye drop regimen has been effective at limiting early ocular inflammation. I believe that ADVM-022 has the potential to be a transformational gene therapy improving real-world outcomes for patients living with wet AMD.

Aaron Osborne, MBBS, chief medical officer of Adverum, added, We have completed dosing patients in OPTIC, with 30 patients being followed up across four cohorts, and we look forward to presenting additional data from these cohorts by the end of this year. With the exciting data presented to date from OPTIC highlighting the potential of ADVM-022 to dramatically reduce treatment burden for patients with wet AMD, we plan to seek U.S. and international regulatory authorities input as we progress towards initiating a pivotal clinical trial in mid-2021.

Laurent Fischer, M.D., chief executive officer of Adverum stated, With these impressive OPTIC data, we are now poised to move our potentially transformative therapy into pivotal trials. ADVM-022, a novel gene therapy providing continuous delivery of aflibercept, has the potential to be a one and done IVT injection that would dramatically reduce the treatment burden for the millions of patients with wet AMD and DME worldwide. As we continue to see positive data from ongoing trials, we are preparing to accelerate our development and future commercial launch plans for ADVM-022. We continue to add industry-leading talent to our team and are beginning to expand our business operations and capabilities, including clinical, regulatory, manufacturing, and pre-commercial functions. Its an exciting time at Adverum, and Im humbled by the dedication of the retina specialists, their patients and our employees for their tireless efforts to help patients with severe ocular diseases during a pandemic.

Recent ProgressClinical DevelopmentINFINITY Phase 2 Clinical Trial of ADVM-022 in Diabetic Macular Edema

Future PlansADVM-022 in wet AMD

ADVM-022 in DME

Manufacturing

COVID-19To date, Adverum has experienced limited impact from COVID-19 on its operations and ongoing clinical programs, including the OPTIC and INFINITY clinical trials. The company is continuing to monitor and attempt to address or limit the potential impacts of COVID-19 on its employees and operations, patient safety, patient enrollment, continued participation of patients already enrolled in the companys clinical studies, protocol compliance, data quality, and overall study integrity.

Financial Results for the Three Months Ended June 30, 2020

Conference Call and Webcast InformationAdverum will host a conference call and webcast today with Key Opinion Leader Dr. Arshad Khanani at 1:30 pm PT / 4:30 pm ET to present new data from the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD and provide an update on recent business progress. The live webcast will be accessible under Events and Presentations in the Investors section of the company's website. To participate in the conference call dial 1-855-327-6837 (domestic) or 1-631-891-4304 (international) and refer to the Adverum Biotechnologies Conference Call. It is recommended call participants dial in 15 minutes in advance. The archived webcast and slide presentation will be available on the Adverum website following the call and will be available for 30 days.

About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMDThis multi-center, open-label, Phase 1, dose-ranging trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. Patients received a high dose (6 x 10^11 vg/eye) of ADVM-022 in Cohort 1 (n=6) and Cohort 4 (n=9) and patients received a low dose (2 x 10^11 vg/eye) of ADVM-022 in Cohort 2 (n=6) and Cohort 3 (n=9). Patients in Cohorts 3 and 4 received six weeks of prophylactic steroid eye drops rather than 13 days of prophylactic oral steroids which were used in Cohorts 1 and 2. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as the need for anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.

Eleven leading retinal centers across the United States are participating in the OPTIC Phase 1 trial for ADVM-022. For more information, please visit https://clinicaltrials.gov/ct2/show/NCT03748784.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visit http://www.adverum.com.

Forward-looking Statements

Statements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to statements regarding: the potential for ADVM-022 in treating patients with wet AMD and DME; Adverums expectations that it will present additional data from all four cohorts of the OPTIC Phase 1 trial for ADVM-022 in wet AMD by the end of this year; Adverums expectations as to its plans to advance ADVM-022 in wet AMD by initiating a pivotal trial mid-2021 and in DME by continuing to enroll patients in the INFINITY trial, including without limitation its expected enrollment numbers for the INFINITY trial; Adverums expectations that it will present data from the INFINITY trial in the second half of 2021; Adverums expectations that it will accelerate its development, manufacturing, and commercial launch plans for ADVM-022; and Adverums expectations that its current cash position will fund its operations into 2022. All of these statements are based on certain assumptions made by Adverum on current conditions, expected future developments and other factors Adverum believes are appropriate in the circumstances. Adverum may not achieve any of these in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for preliminary or interim results of clinical trials to change as the clinical trial continues or in connection with the preparation and analysis of final results; the potential for future complications or side effects in connection with use of ADVM-022; obtaining regulatory approval for gene therapy product candidates; enrolling patients in clinical trials; reliance on third parties for conducting the OPTIC and INFINITY trials and vector production; the effects of the COVID-19 pandemic on the companys operations and on the companys ongoing clinical trials; and ability to fund operations through completion of the OPTIC and INFINITY trials and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with the SEC on August 10, 2020 under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor and Media Inquiries:Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com1-646-876-5196

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Adverum Biotechnologies Announces Positive Interim Data from Cohorts 1-4 from OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for wet AMD,...

Ocugen Receives Fourth FDA Orphan Drug Designation for the Same Product, OCU400 (AAV-NR2E3) Gene Therapy, for the Treatment of Another Key Inherited…

MALVERN, Pa., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the fourth Orphan Drug Designation (ODD) for OCU400 in the treatment of PDE6B gene mutation-associated retinal diseases. Retinitis Pigmentosa (RP) caused by PDE6B mutation is an inherited retinal dystrophy that leads to blindness by midlife and is characterized by the progressive loss of photoreceptors, with or without the loss of retinal pigment epithelium cells. At least one mutation in the PDE6B gene has been found to cause autosomal dominant congenital stationary night blindness, which is characterized by the inability to see in low light.

Ocugens Modifier Gene Therapy Platform offers a unique approach in ophthalmology by addressing multiple diseases with a single product. A novel gene therapy product candidate, OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (NHR) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis and potentially offer longer benefit, stabilizing cells and rescuing photoreceptor degeneration and vision loss. In pre-clinical studies, OCU400 has demonstrated improved vision signals in the retina where Electroretinogram response reveals rescue under both Scotopic (dim-lit) as well as Photopic (well-lit) conditions. The Company believes targeting multiple diseases with one product could also offer a smoother regulatory pathway and the ability to recover development costs over multiple therapeutic indications. Ocugen is planning to initiate two parallel Phase I/II clinical trials next year targeting two unique IRDs.

Following up on recent announcement of an ODD for RHO mutation-associated retinal degeneration and previous ODDs for both NR2E3 and CEP290 mutation-associated retinal degeneration, the ODD for PDE6B gene mutation-associated retinal degeneration continues to support Ocugens breakthrough modifier gene therapy platforms potential to treat multiple blindness diseases with a single product. RP is a group of heterogenic inherited retinal diseases associated with over 150 gene mutations, affecting over 1.5 million individuals worldwide. In addition, ~40% of RP patients cannot be genetically diagnosed, confounding the ability to develop personalized RP therapies. Traditional gene therapy or gene editing approaches may require more than 150 products to rescue these patients from vision loss. OCU400, a single product candidate, has potential to address broad-spectrum RP.

As principal investigator of numerous major clinical trials developing new medical and surgical treatments for retinal disorders, I have been on the cutting-edge of many new ophthalmology treatments. I am very encouraged by the potential for OCU400 given the uniqueness of Ocugens Modifier Gene Therapy Platform and the fact that FDA has issued four ODDs for this product. I look forward to Ocugen commencing clinical trials for OCU400 next year and the potential of helping patients by restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases including broad-spectrum RP, said Carl D. Regillo, M.D.,F.A.C.S., member of Ocugens Retina Scientific Advisory Board and Professor of Ophthalmology at the Sidney Kimmel Medical College at Thomas Jefferson University, Chief of the Retina Service at Wills Eye Hospital and founder and former director of the Wills Eye Clinical Retina Research Unit in Philadelphia.

I am thrilled to announce our fourth ODD for OCU400 from the FDA after announcing our third ODD for RHO mutation-associated retinal degeneration just a few days ago. With no approved treatments that slow or stop the progression of RP, we are dedicated to driving the development of our Modifier Gene Therapy Platform forward and potentially addressing the unmet need of multiple gene mutations, including mutations in the PDE6B gene with only one product, said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen.

The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including a seven-year period of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.

About OCU400OCU400 (AAV-hNR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration and vision loss.

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. For more information, please visit https://ocugen.com/.

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact:LaVoieHealthScienceEmmie Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042

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Ocugen Receives Fourth FDA Orphan Drug Designation for the Same Product, OCU400 (AAV-NR2E3) Gene Therapy, for the Treatment of Another Key Inherited...

Enterprise Ireland awards 1.3m to TCD and RCSI research projects – Siliconrepublic.com

Through its commercialisation fund, Enterprise Ireland is investing in three research projects from Trinity College Dublin and RSCI.

Enterprise Ireland has awarded 1.3m to three third-level research projects through its commercialisation fund. Each of the projects will receive more than 400,000 in investment so researchers can transform their ideas into commercially viable and relevant businesses.

Enterprise Ireland has been working with third-level researchers for a decade through this initiative, with the aim of bringing commercially relevant technology out of the lab and into the marketplace.Previous awardees of the commercialisation fund include AudioSourceRE, Cala Medical and Senoptica Technologies.

The agencys senior commercialisation specialist, Eithne McShane, said: The commercialisation fund is an avenue for Irelands brightest scientists to commercialise their research and bring it to the market.

At Enterprise Ireland, we recognise that funding innovation is key to ensuring that the Irish economy remains competitive on the world stage through the creation of technology-based start-up companies and the transfer of innovations developed in higher education institutions and research-performing organisations to industry in Ireland.

In the latest iteration of the commercialisation fund, two research projects from Trinity College Dublin (TCD) and one from the Royal College of Surgeons in Ireland (RCSI) will receive investment. All three projects are led by women.

The medical research being undertaken by each of their teams will be looking at issues such as infectious diseases, adult blindness and drug delivery,McShane added.

We have seen in recent months the importance of medical research and look forward to assisting each on their journey to the marketplace with the ultimate goal of improving lives.

One of the two projects from TCD is a study focusing on the development of gene therapies for common retinal disorders, led by principal investigator Prof Jane Farrar.

Farrars team is researching age-related macular degeneration (AMD), which is the most common cause of blindness in the developed world. There are two distinct forms of AMD: dry and wet. While there are several treatments for wet AMD, there are no licensed medical therapies for dry AMD, which represents about 90pc of AMD cases and affects approximately 150m people globally.

The team has developed a novel gene therapy for dry AMD, based on a yeast-derived gene encoding mitochondrial complex in an adeno-associated virus vector. A commercial case feasibility study was conducted in 2019, with backing from Enterprise Ireland.

Among the outcomes targeted through the fresh funding from Enterprise Ireland are the development of in-vitro human retinal pigment epithelial cell models and the finalisation of in-vivo preclinical data packs for therapies with associated study reports, as well as the initiation of key commercialisation activities.

Led by TCDs Prof Aisling Dunne, Adjuvenate is developing a platform solution for improved subunit vaccines. The research is being conducted in response to the rise in infectious disease and the need to develop new vaccines that are capable of eliciting effective and sustained immune responses.

Adjuvenate focuses specifically on whooping cough, which is on the rise due to the inability of current vaccines to provide sustained immunity, according to the TCD researchers. They said that new, more effective adjuvants are needed, which would enable vaccine makers to produce a sustained, lasting immune response.

The team has discovered and patented a new whooping-cough vaccine component, which may have the potential to be a third-generation standalone booster vaccine for whooping cough.

The commercialisation fund will support the further development of this novel adjuvant to develop a new vaccine. In addition, the team will continue to develop the adjuvant molecule as a novel adjuvant for combination with other new vaccines in development.

The RCSI project receiving funding is StarMat Technologies, which focuses on star-shaped polypeptide materials for biomedical applications. Led by Prof Sally Ann Cryan with Prof Andreas Heise, the team is examining ways in which its patented StarMat technology can be tailored to deliver specific drug payloads.

The technology can be integrated with medical devices when required to target specific tissues and cells, and may be particularly well suited to applications in biotherapeutic delivery in respiratory and regenerative medicine.

The team conducted a commercial feasibility study, funded by Enterprise Ireland, through which the researchers worked with multiple industry partners. Their analysis highlighted the demand for direct-to-cell delivery technologies, particularly advanced therapeutics medicinal products. This includes nucleic acid-based therapies, such as RNA-based technologies, which are being used in some of the Covid-19 vaccine technologies currently in development.

The commercialisation funding will be used will support the development ofscalable processes for drug payload incorporation, product refinement and further toxicology studies.

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Enterprise Ireland awards 1.3m to TCD and RCSI research projects - Siliconrepublic.com

This North Texas biotech startup just raised a whopping $95 million amid the pandemic – The Dallas Morning News

Dallas-based Taysha Gene Therapies has raised $95 million in new funding for development of its treatments for rare childhood diseases.

The financial boost from investors such as Fidelity Management & Research, BlackRock and GV (formerly Google Ventures) marks the startups second big investment this year. In April, Taysha wrapped up a $30 million seed round that was the North Texas regions largest funding haul in the second quarter, despite funding for startups slowing amid the pandemic.

Tayshas work is focused on finding therapies for genetic diseases that can inhibit childrens ability to perform basic functions like walking, talking, eating and even breathing.

The biotech company will use the money to bolster its work on gene therapies for rare diseases affecting the central nervous system in partnership with UT Southwestern Medical Centers Gene Therapy Program. Itll also help build a scalable manufacturing facility.

This significant investment from premier, long-term investors will allow us to advance our mission of eradicating monogenic CNS disease for the thousands of patients who suffer from these devastating disorders, Taysha founder and CEO RA Session II said in a statement.

The company plans to file four investigational new drug applications by the end of 2021, according to Session. It will begin clinical studies later this year for treatment of GM2 Gangliosidosis, a group of rare diseases in children that progressively destroy nerve cells in the brain and spinal cord.

Taysha has a pipeline of 15 gene therapy drug candidates, with options on an additional four.

We believe this financing provides significant validation of our corporate strategy and will enable us to continue to rapidly translate programs from preclinical development into the clinic, Taysha board chairman Sean Nolan said in a statement.

Session formed Taysha with investors and executives from another well-known company, AveXis, which sold to Swiss pharmaceutical giant Novartis for $8.7 billion in 2018.

UT Southwestern has been on a roll in terms of commercializing its research. In May 2019, pharmaceutical giant Merck bought cancer drug spinoff Peloton Therapeutics in a $2.2 billion deal. Last June, a Boston-based pharmaceutical company acquired another spinoff, Exonics Therapeutics, in a deal that could be worth close to $1 billion.

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This North Texas biotech startup just raised a whopping $95 million amid the pandemic - The Dallas Morning News

Advanced Wound Care Management Market: Chronic wounds to be highly lucrative segment – BioSpace

Transparency Market Research (TMR) has published a new report titled, Advanced Wound Care Management Market - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026. According to the report, theglobal advanced wound care management marketwas valued at US$ 8.5 Bn in 2016 and is projected to expand at a CAGR of 5.8% from 2018 to 2026. Surge in incidence of chronic wounds and trauma cases is anticipated to boost the demand for advanced wound care management during the forecast period. North America and Europe are projected to dominate the global advanced wound care management market owing to higher rate of adoption and awareness regarding wound care management. Asia Pacific, Latin America, and Middle East & Africa are potential markets for advanced wound care management. The market in Asia Pacific is expected to expand at a CAGR of 6.3% from 2018 to 2026.

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Cost-effective advanced wound care management to treat complex wounds to drive global market

Increase in trauma and accident cases across the globe drives the advanced wound care management market. According to the World Health Organization (WHO), road accidents account for around 1.25 million deaths across the globe each year. Additionally, the number of patients with chronic wounds is rising rapidly across the world. This is likely to increase the number of surgical procedures; consequently, propelling the global advanced wound care management market.

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Chronic wounds to be highly lucrative segment

Chronic wounds is an emerging segment of the advanced wound care management market. In the chronic wounds segment, the ulcers sub-segment holds a prominent share. Venous leg ulcers account for 80% of all leg ulcers. Moreover, the chronic wounds segment is likely to hold major share due to high cost of therapy across the globe. As per a paper published by NCBI (National Center for Biotechnology Information), in May 2014, the annual burden of venous leg ulcers in the U.S. is nearly US$ 14.9 Bn, which includes indirect costs (productivity loss) as well. The diabetic foot ulcer sub-segment of the advanced wound care products market is expanding at a significant CAGR.

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Hospitals & clinics to be notably promising segment

In terms of end-user, the global advanced wound care management market has been segmented into hospitals & clinics, ambulatory surgical centers (ASCs), home health care, and others. The hospitals & clinics segment held major share of the global market in 2017. Expansion of the segment can be attributed to the availability of multiple service options and devices and tie-ups with health care companies in order to enhance health care product and service offerings. Moreover, hospitals are the preferred choice due to availability of advanced technology and better health care services. The home health care segment is expanding at a high growth rate, especially in developed economies, due to rise in geriatric population and increasing health care infrastructure and support. Moreover, rise in demand for advanced wound care management in home care settings for geriatric patients is projected to drive the segment.

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North America expected to dominate global market

In terms of region, the global advanced wound care management market has been segmented into five major regions: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America dominated the global advanced wound care management market in 2017. The market in North America was valued at US$ 3.2 Bn in 2017 owing to a highly developed health care sector, increase in awareness among health care providers about advanced wound care management, and continuous evolution of wound care management. Advanced wound care management market offers significant growth potential in the region. The advanced wound care management market in Asia Pacific is anticipated to expand at a CAGR of 6.3% during the forecast period due to increase in awareness about advanced wound care products and expansion of the health care sector in countries such as China, Japan, and India. Moreover, technological advancements and increase in rate of adoption of advanced wound care management products are expected to propel the market in the region during the forecast period.

Smith & Nephew plc, Acelity L.P., Inc. and Mlnlycke Health Care AB anticipated to lead global market

The global advanced wound care management market is highly fragmented in terms of number of players providing different products. Key players in the global advanced wound care management market include Smith & Nephew plc, Acelity L.P., Inc., Mlnlycke Health Care AB, ConvaTec Inc., B. Braun Melsungen AG, Coloplast A/S, 3M Healthcare, Integra LifeSciences Corporation, Cardinal Health, PAUL HARTMANN AG, BSN Medical, Hollister Incorporated, Organogenesis Inc., and Medline Industries, Inc. Expansion of product portfolio through mergers and acquisitions is a key strategy followed by several global players. In December 2015, Acelity L.P., Inc. announced the acquisition of Spiracur, Inc., expanding its offering in disposable, portable, mechanical NPWT (Negative-pressure wound therapy) technology, and allowing sales and service channels to boost the expansion of the SNaP therapy system to patients and their care teams across the world who need access to NPWT devices.

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Advanced Wound Care Management Market: Chronic wounds to be highly lucrative segment - BioSpace

Global Cancer Gene Therapy Industry Analysis, Share, Growth Drivers and Future Scope – Owned

In the coming years, the cancer gene therapy market size is projected to witness considerable growth, particularly due to the surging prevalence of cancer, increasing investments in research & development (R&D) activities, improved regulatory framework, acceptance of gene therapy for the treatment of the disease, rising popularity of deoxyribo nucleic acid vaccines, and technological advancements. Clinical research laboratories, hospitals, biotechnology companies, and oncology institutes are the major end-users in the cancer gene therapy industry.

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The different therapies utilized for treatment are oncolytic virotherapy, gene transfer, and immunotherapy. Out of these, the gene transfer therapy is expected to lead the global cancer gene therapy market in the near future. There are several ways to apply gene therapy, for example, replacing absent or abnormal genes with healthy ones, changing the way genes are regulated, and introducing foreign genes in cells, which alter their function or/and survival. The gene transfer therapy further has several types, namely electroporation, magnetofection, naked/plasmid vectors, sonoportation, and gene gun.

Asia-Pacific (APAC) is projected to register the fastest growth in demand for cancer gene therapy in the coming years. The growth of the cancer gene therapy market in the region is ascribed to the improving healthcare infrastructure and increasing government initiatives. The healthcare infrastructure in the region is improving due to the rising disposable income of people in emerging economies, which, in turn, is resulting in the growing affordability of innovative treatment options. Moreover, countries including Korea and India have a large patient pool, which is further contributing to the growth of the industry.

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Some major players in the cancer gene therapy market are Cell Genesys Inc., GenVec Inc., Adaptimmune Therapeutics plc, Achieve Life Science Inc., Genelux Corporation, Advantagene Inc., and BioCanCell Ltd.

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Potential impact of Covid-19 on Gene Therapy Market Growth and Demand, Concludes Fact.MR – The Cloud Tribune

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Potential impact of Covid-19 on Gene Therapy Market Growth and Demand, Concludes Fact.MR - The Cloud Tribune

Edited Transcript of FOLD earnings conference call or presentation 10-Aug-20 12:30pm GMT – Yahoo Finance

Cranbury Aug 10, 2020 (Thomson StreetEvents) -- Edited Transcript of Amicus Therapeutics Inc earnings conference call or presentation Monday, August 10, 2020 at 12:30:00pm GMT

Amicus Therapeutics, Inc. - Director of IR

* Bradley L. Campbell

Amicus Therapeutics, Inc. - President, COO & Director

* Daphne E. Quimi

Amicus Therapeutics, Inc. - CFO

* Jeffrey P. Castelli

Amicus Therapeutics, Inc. - Chief Development Officer

* John F. Crowley

Amicus Therapeutics, Inc. - Chairman & CEO

* Debjit D. Chattopadhyay

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

Robert W. Baird & Co. Incorporated, Research Division - Senior Research Analyst

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics, Inc.'s Second Quarter 2020 Financial Results Conference Call and Webcast. (Operator Instructions) As a reminder, this conference call is being recorded.

I'd now like to turn the conference over to your host, Mr. Andrew Faughnan, Director of Investor Relations. You may begin.

Andrew Faughnan, Amicus Therapeutics, Inc. - Director of IR [2]

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2020 financial results and corporate highlights.

Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer; Bradley Campbell, President and Chief Operating Officer; Daphne Quimi, Chief Financial Officer; and Dr. Jeff Castelli, Chief Development Officer.

As referenced on Slide 2, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved. Any or all of the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof.

All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and Risk Factors section of our annual report on Form 10-K for the year ended December 31, 2019, and the quarterly report on Form 10-Q for the quarter ended June 30, 2020, to be filed later today with the Securities and Exchange Commission.

At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer. John?

John F. Crowley, Amicus Therapeutics, Inc. - Chairman & CEO [3]

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Great. Thank you, Andrew. Good morning, and welcome, everyone, to our second quarter 2020 results conference call.

As we did last quarter, let me start by stating that I hope everybody and their families are well and safe.

Our leadership team at Amicus continues to emphasize a range of programs and initiatives to protect and support our global workforce during this ongoing pandemic. While adapting to all of the changes brought about by the global pandemic, for Amicus, the first half of 2020 has been a period of excellent growth and execution across all aspects of our business, including science, clinical, regulatory, and as you see, our commercial effort, as we continue to build one of the next great global biotechnology companies poised to impact people around the world living with rare diseases.

As we did in this morning's press release, I'd like to highlight several key accomplishments. First, Galafold continues its strong launch performance and remains the cornerstone of our success. With $62.4 million in second quarter revenue, the Galafold launch continues to exceed expectations. Second quarter revenue represents the performance across the global business, including new patient starts from both switched and treatment-naive patients throughout the quarter in all major regions, including those hardest hit by COVID-19.

Second, our R&D time lines remain on track. We continue to expect the Phase III PROPEL study of AT-GAA in late-onset Pompe disease to read out in the first half of 2021. Additionally, the rolling BLA submission for AT-GAA remains on schedule, and we expect the first submission later this year. Within our gene therapy pipeline, we continue to move forward our lead Batten disease programs with CLN6 and CLN3 as well as our most advanced preclinical programs in gene therapy.

And third, following our strategic financing in July, Amicus' cash position is sufficient now to achieve profitability without the need for any future diluted financings.

Our continued revenue growth, prudent expense management and growth potential has allowed us to reach this important milestone of self-sustainability as we continue to realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world.

Turning now to Slide 4. We are well on track to achieve our 5 key strategic priorities for 2020, including, first, Galafold, our precision medicine for Fabry disease. We will continue to drive Galafold to more people living with Fabry disease with a minimal variance in existing and new markets around the world. We look to achieve global product revenue this year of $250 million to $260 million.

Second, we are increasing the clinical regulatory manufacturing and pre-commercial activities and planning surrounding our Pompe program for AT-GAA as we move this important therapy toward approval.

Third, again, we are advancing our industry-leading rare disease gene therapy portfolio. Standing from our new Global Research and Gene Therapy Center of Excellence in Philadelphia, we will be advancing the clinical development, manufacturing and regulatory discussions for both our CLN6 and CLN3 Batten programs.

Fourth, in addition, we are progressing our Pompe gene therapy towards IND, and we plan to disclose up to 2 additional IND candidates this year. A lot of work is underway with our manufacturing partners with the manufacture and scale-up of the Pompe gene therapy as well as our other potential IND candidates. So again, we look forward to sharing the additional IND candidates from our Penn collaboration later this year.

Just, again, we will continue to maintain a strong financial position as we carefully manage our expenses and our investments, and we remain fully funded now to profitability.

So with that, before I turn the call over to our team to provide more detail on the strong quarter, I just wanted to take a moment to acknowledge Dr. Ted Love's retirement from the Amicus Board of Directors. Ted, as many of you know, is a remarkable leader, a great friend of mine and a fierce advocate for innovation for patients. Now Ted and I will continue to work closely together as the Chair and Vice Chair of the Emerging Companies section, on the Bio Board, and I would very personally like to thank him for his nearly decade of service on our Board. He has helped to put Amicus in a much stronger position. So thank you, Ted.

So with that, let me turn the call now over to Bradley Campbell, our President and Chief Operating Officer, to further highlight the strong Galafold performance. Brad?

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Bradley L. Campbell, Amicus Therapeutics, Inc. - President, COO & Director [4]

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Great. Thanks, John. Good morning, everyone. As John mentioned, I'll now walk you through in more detail our Galafold performance for the quarter, and let me start on the continued growth of Galafold revenue in the second quarter of 2020 here on Slide 6. And this is where we give our global snapshot of the Galafold commercial progress.

For the second quarter, as John mentioned, total product revenue was $62.4 million, driven by strong patient demand, favorable reimbursement dynamics and business continuity even during the worst quarter of COVID so far. And importantly, our global compliance and adherence rates continue to exceed 90%.

The geographic breakdown of revenue during the quarter was $41.5 million or 67% of revenue generated outside the United States, and the remaining $20.8 million or 33% coming from within the United States. On a sequential basis, you'll note that ex-U. S., quarterly revenue did decline slightly from Q1 to Q2 due to the timing of orders. However, we continue to see strong growth in patients added during the second quarter, and that strength has continued now into the first month of Q3, so we expect to finish the year around 70-30 ex-U. S. to U.S. sales. And as a reminder, we have now over 4 countries around the world with regulatory approvals, and commercial sales now in over 30 of those countries. And this expanding global footprint is important not just to support the continued growth of patients with access to Galafold, but it really lays a strong foundation that is highly leverageable to support the potential launch of AT-GAA for Pompe and our future products as well.

One other interesting aspect of the expanding market access to Galafold, which is highlighted on this slide, and this is quite unique, is that we now have added over 1,000 additional amenable mutations to the European label. The previous 300 mutations were all associated with specific patients who have been identified with those mutations. These new mutations represent nearly all of the possible missense mutations in the GLA gene that are amenable to Galafold, as determined by our amenability assay. So if the physician now diagnoses a new patient with one of these mutations, they'll no longer have to wait for us to characterize the mutation and run it through the amenability assay, a process which previously could take several months. Now the physician can simply refer to the website and check the amenability of the mutation, and the patient can get immediate access to Galafold. Again, a really exciting application and a really unique application of the pharmacogenetic aspects of this medicine, and one that we're looking to apply to other geographies as well.

Turning now to Slide 7. We've had a very strong quarter even in the face of the COVID-19 pandemic. Our global supply chain remains fully intact. Our customers have great confidence that they can access Galafold, and our field team has been able to achieve a significant portion of their pre-COVID touch points through digital, telephonic and other means of interacting with their physicians. All of this has led to continued uptake with new patient starts all throughout the crisis in both switch and treatment-naive patients. Even in the hardest hit countries like the U.K., France, Italy, Spain, Japan, U.S. and many others, we are able to bring on patients to Galafold throughout the quarter.

And it's important to note that on a country-by-country basis, as local conditions allow, and in a very carefully prescribed manner, we're now allowing our field organization to go back out to visit their physicians and key accounts. We believe that these dynamics may continue to provide some tailwinds, even as the evolving global pandemic inevitably has caused some headwinds, which will continue to closely monitor as the full impact of the pandemic currently remains unknown.

From a numbers perspective, you can see that the second quarter sales increased 41% from second quarter 2019, which does include a 2% negative impact from foreign exchange. So from a true operational perspective, sales increased by 43% compared to last year.

On the left-hand side, we show our quarterly performance over the past several quarters. And as we've mentioned on previous calls, while we continue to expect strong growth quarter-to-quarter, due to a variety of factors, the rate of growth is typically nonlinear. Looking to the back half of the year, as we've seen in many years past, we expect a higher quarter-on-quarter growth in Q4 as compared to Q3, which means we expect a larger portion of the revenue in the second half of the year will fall into the fourth quarter. But overall, as we've just finished the 7th month of the year, here in July, we continue to be very confident in our guidance of $250 million to $260 million in full year global sales.

Now on Slide 8, with several years of performance behind us and now launched at most major markets around the world, we can confidently say we are on a path to that $500 million of sales opportunity in 2023.

As I've outlined previously, to get to that $500 million, we expect a 5-year average CAGR of about 40% from 2018 to 2023, and we expect to generate $1 billion in cumulative revenue between 2020 and 2022 alone, which goes a long way towards funding our R&D and OpEx over that period.

We also have even further confidence now in the $1 billion revenue opportunity at peak as we continue to see significant growth in the Fabry market globally, driven by continued diagnosis from high-risk screening, newborn screening and other diagnostic initiatives, in addition to penetration into the diagnosed untreated population.

As we have orphan exclusivity in the U.S. and Europe, in addition to our multiple orange book-listed patents that give us IP coverage into the late 2030s, so we have lots of opportunity to provide access to Galafold for a long period to come.

With that, let me hand the call now over to Dr. Jeff Castelli, our Chief Development Officer, who will further highlight our lead pipeline programs. Jeff?

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Jeffrey P. Castelli, Amicus Therapeutics, Inc. - Chief Development Officer [5]

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Yes. Thanks, Bradley, and good morning, everyone.

Moving on to our R&D updates on Slide 10. We wanted to remind everyone of our highly differentiated Pompe therapy, AT-GAA, and its mechanism of action. AT-GAA is our novel next-generation therapy consisting of ATB 200 or alglucosidase alfa, an investigational human recombinant GAA enzyme designed to target muscle cells throughout the body, administered via IV infusions, combined with AT2221, an orally administered enzyme stabilizer. AT2221 is administered shortly before the infusion of ATB200 begins and is intended to bind and stabilize ATB200 in circulation, allowing more active enzyme to be taken up into cells and deliver to lysosomes. The combination of ERT and enzyme stabilizer is one major distinction from the standard of care and other treatments in development for Pompe.

The other and, we believe, potentially more impactful distinction is the unique carbohydrate profile of the enzyme itself. Dr. Hung Do, our Chief Science Officer, and his team have been working over the past decade to develop a Pompe ERT, which improved binding the target receptors for efficient uptake in the cells, while importantly retaining the ability of the ERT to be processed by those cells after it's taken up to the mature, more active form of GAA.

With that in mind, and now turning to Slide 11, we would like to highlight a recent publication in the Molecular Therapy - Methods & Clinical Development journal, where Dr. Nina Raben's team at the National Institute of Health published findings from an independent preclinical study, building upon previous work with AT-GAA that showed additional improvements with longer-term treatment to knock out mice. The studies show that AT-GAA was successful in completely reversing excess glycogen simulation, restoring muscle strength and significantly improving a cascade of secondary abnormality. Including the elimination or reduction of autophagic buildup in muscle fibers, improvements in markers of lysosomal damage and in cell signaling. These are particularly exciting findings as these impacts have not been observed in previously disclosed studies.

Finally, on Slide 12, we want to remind everyone of the integrity of our Pompe Phase III PROPEL clinical trial. We reiterate that steady time lines remain on track, with data expected in the first half of '20 -- I think first half of 2021. To date, more than 97% of the 2,800 plus planned infusions and off-study assessments for the ongoing PROPEL study have been completed on schedule. Additionally, we continue to enroll patients in our pediatric studies and advanced manufacturing to support a 2021 BLA and MAA.

We want to express thanks to all Pompe clinical study participants and their families, to all our investigators and their staff and to our cross-functional Amicus Pompe team for their collective unwavering commitment and supportive efforts during this unprecedented time.

Moving on now to Slides 14 and 15. I'll briefly highlight here our industry-leading portfolio of gene therapies for rare diseases. During this time of COVID, we've been able to maintain our critical science and lead programs across the gene therapy portfolio, including CLN6 and CLN3 Batten disease as well as the Pompe and Fabry gene therapy programs with University of Pennsylvania.

Starting with our Batten disease franchise in CLN6, we previously reported positive interim data in our clinical study that demonstrates meaningful impact of our AAV gene therapy in this extremely devastating form of Batten disease. In October, at the virtual Child Neurology Society Annual Meeting, we're expecting additional data from the CLN6 Phase I/II study. We continue to advance regulatory discussions to finalize the clinical and regulatory path, and expect to provide an update in early 2021.

We believe this initial CLN6 data provide an important readthrough for our clinical study in CLN3 Batten disease, which is the most common form of childhood neuro generation. We plan to report initial data from the ongoing Phase I/II CLN3 study early next year based on scheduling changes for some of the major gene therapy conferences. Concurrent with the data, early next year, we will provide the expected regulatory path for the CLN3 program.

We continue to make great progress in all of our commercial manufacturing process for both CLN6 and CLN3, and we remain on track to begin dosing additional patients in both of these programs next year.

Moving on to Slide 16. I would like to remind everyone of the research collaboration with the University of Pennsylvania, which will be an important driver of growth for Amicus in the future. This collaboration with the Wilson Lab at Penn combines Amicus' protein engineering and glycobiology expertise with Penn's gene transfer technologies and expertise to deliver novel gene therapies that are designed for optimal cellular uptake, targeting, dosing, safety and manufacturability. As part of this collaboration, Amicus has rights to over 50 different diseases, in addition to the active ongoing preclinical programs.

Several Amicus presentations were given at the American Society of Gene & Cell Therapy Annual Meeting back in the second quarter. This data was highlighted by our Pompe gene therapy results, which showed the Amicus engineered GAA protein had improved targeting and clearance of glycogen storage in Pompe diseased minds. Additionally, preliminary data in nonhuman primates suggested therapeutically relevant expression levels in target organ, which is a key factor as we move to larger species.

We remain very encouraged by this data and continue to progress our Pompe gene therapy towards an IND. We also continue to make progress across our preclinical gene therapy programs and look to disclose up to 2 additional IND candidates later this year.

With that, I would like now to turn the call over to Daphne to review our financial results, guidance and outlook. Daphne?

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Daphne E. Quimi, Amicus Therapeutics, Inc. - CFO [6]

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Thank you, Jeff, and good morning, everyone.

Our financial overview begins on Slide 18 with our income statement for the quarter ended June 30, 2020.

For the second quarter, we achieved Galafold revenue of $62.4 million, which is a 41% increase over the second quarter of 2019. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 43%, offset by a negative currency impact of 2%. Cost of goods sold as a percentage of net sales was 10.8% in the second quarter as compared to 12.1% for the prior year period. Cost of goods sold as a percent of revenue was favorable as Galafold revenue continues to grow in the United States, where we do not owe royalties; as well as in other countries, where we are subject to lower royalties.

Total operating expenses were $107 million in the second quarter of 2020, which decreased as compared to $115.2 million in the second quarter of 2019. On a non-GAAP basis, total operating expenses were $95.9 million in the second quarter of 2020 as compared to $103.6 million in the second quarter of 2019. The decrease in research and development costs reflects decreased travel and third-party costs, offset by continued investments to support the PROPEL study and in our gene therapy pipeline.

Our investment in research and development includes the impact of the implementation of the cost reduction measures that were previously announced, as does the decrease in selling, general and administrative expenses.

Due to the timing of expenses, the non-GAAP operating expense is expected to increase in the third quarter of 2020 as compared to the second quarter of 2020. We define non-GAAP operating expense as research and development and SG&A expenses, excluding share-based compensation, changes in fair value of contingent consideration and depreciation.

Net loss for the second quarter was $52.5 million or $0.20 per share as compared to net loss of $84.6 million or $0.36 per share for the prior year period. As of June 30, 2020, we had approximately 257 million shares outstanding.

Turning now to Slide 19. As John mentioned, following our $400 million senior secured term loan facility, we are now on a clear path to profitability without the need for any future dilutive financing. As we initially laid out last year at our Analyst Day, we have achieved this milestone by our continued revenue growth with Galafold as well as driving efficiencies, cost savings and careful expense management. Securing this financing with market setting turns gives us a strong financial platform to advance both patient and Amicus shareholder interest. Going forward, again, to emphasize, we expect total non-GAAP operating expenses in 2020 to remain relatively flat from 2019 as we, first, leverage the commercial infrastructure that is already in place for the AT-GAA launch and other products in our pipeline; secondly, we transitioned the costs associated with the development of AT-GAA to multiple gene therapy programs in our pipeline; and third, maintain financial discipline while meeting our objective.

To reiterate, all high priority research programs in gene therapy are moving ahead on schedule, especially in CLN3, CLN6, Pompe and Fabry, and we continue to fully support the work with Jim Wilson and Penn.

A few comments about our cash position and 2020 financial guidance. Cash, cash equivalents and marketable securities were $309.6 million at June 30, 2020 compared to $452.7 million at December 31, 2019. Following the receipt of net proceeds from the July debt facility and retiring the prior term loan of $150 million, Amicus has a cash position of over $530 million as of July 31. We are reaffirming our full year Galafold revenue guidance of $250 million to $260 million in addition to our non-GAAP operating expense guidance of $410 million to $420 million.

And with that, let me turn the call back to John for closing remarks.

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman & CEO [7]

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Great. Thank you, Daphne, Jeff and Bradley.

So as you can see, we have been relentlessly focused on performance across the business despite all of the unprecedented change and challenges that have been brought about by the global pandemic. We have a great global team of passionate entrepreneurs at Amicus, who have led and will continue to lead us through this. I am confident that as the world emerges from this crisis, Amicus will emerge even stronger.

So with that, operator, we're happy to take any questions.

================================================================================

Questions and Answers

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Operator [1]

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(Operator Instructions) And your first question comes from the line of Anupam Rama from JPMorgan.

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Anupam Rama, JPMorgan Chase & Co, Research Division - VP and Analyst [2]

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Just a quick question on the PROPEL study. For those small number of patients, who did not get an on-time infusion -- schedule an infusion, do these patients ultimately get an infusion outside of the window? Or was it just a skip infusion, and then they got the next one?

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Edited Transcript of FOLD earnings conference call or presentation 10-Aug-20 12:30pm GMT - Yahoo Finance

Selecta Biosciences Inc (NASDAQ:SELB) Will Advance Gene Therapy Program In H1 2021 In MMA: Closes SLA For SEL-212 With Sobi – BP Journal

Selecta Biosciences Inc (NASDAQ:SELB) posts a loss of $24.1 million in Q2 2020. Its general and administrative expenses in Q2 2020 are $5.6 million, whereas R & D spending surged to $10.7 million.

President of Selecta, Carsten Brunn, said it is a transformative period for the company. It is strengthening its leadership position in immune tolerance. To unlock its ImmTOR immune tolerance platforms full potential, the company entered a pact with Sobi to get funding. The funding will optimize the safety and efficacy of biologics and enable the redosing of gene therapies. It will use funds for developing innovative immunotherapies for the treatment of autoimmune diseases.

Selecta is committed to developing SEL-212. As per the terms of the pact, Sobi is responsible for commercial, regulatory, and development activities of SEL-212. In alliance with Sobi, the company will initiate Phase 3 clinical trials in Q3 2020 and report topline data of Phase 2 COMPARE trial. Sobi will bear all the expenses for the Phase 3 clinical trial. It will provide $100 million as an initial payment to Selecta.

Selecta will also advance its gene therapy in MMA (methylmalonic academia) in H1 2021 to the clinic. It also prepares for IND submission for autoimmune disease treatment in 2021.

Selecta inked an RLOA (Research License and Option Agreement) with Sarepta to provide its ImmTOR for curing certain neuromuscular diseases. Sarepta will use ImmTOR to treat LGMDs (limb-girdle muscular dystrophies) and DMD (Duchenne muscular dystrophy).

Sarepta will engage in the investigational study of gene therapies and ImmTOR to reduce the creation of neutralizing antibodies. Under the terms of the agreement, Selecta already received initial payment and expects to get fees for certain pre-clinical milestones.

Selecta confirmed Peter G. Traber as a full-time Chief Medical Officer with effect from August 1, 2020, from the current position of interim CMO. Peter brings significant experience in academia, biotech, and pharma companies. He is responsible for overseeing clinical strategy and development, program management, medical affairs, and clinical and scientific advisory services for business development.

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Global Hemophilia Gene Therapy Market 2020 Trends Analysis and Coronavirus (COVID-19) Effect Analysis | Key Players Market With COVID-19 Impact…

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Global Hemophilia Gene Therapy Market 2020 Trends Analysis and Coronavirus (COVID-19) Effect Analysis | Key Players Market With COVID-19 Impact...

Novartis and a string of high profile backers fund next-gen Treg cell therapy startup GentiBio – FierceBiotech

OrbiMed, Novartis Venture Fund and RA Capital Management have joined forces to fund and help launch GentiBio.

The Boston, Massachusetts-based biotech starts life with a $20 million seed funding with an aim of creating new engineered regulatory T cells to deliver immune tolerizing meds for autoimmune, allergic and inflammatory diseases.

GentiBio lands with tech out of the Seattle Childrens Research Institute, Benaroya Research Institute and MIGAL Galilee Research Institute.

September 2-3, 2020 Live, Online Course: Biopharma Revenue Forecasting that Drives Decision Making and Investments

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The biotech was in fact co-founded by a team of scientists from these three institutes to restore immune tolerance using Treg cell therapy engineered with novel technologies that direct potent EngTregs to specific tissues damaged by abnormal immune responses, said the biotech in a statement.

The hope is that this tech will overcome many of the current limitations of Tregs therapeutics, including rarity and plasticity of endogenous Tregs populations.

Regulatory T cells, or Tregs, play a key role in modulating other cells in the immune system so they don't attack the bodys own tissues. The failure of Tregs can lead to autoimmune diseases.

In a June paper published in the Science Translational Medicine journal, the team from the Seattle Childrens Research Institute and the Benaroya Research Institute made Treg-like cells by gene editing ordinary T cells to express Foxp3 and paired them with antigen-specific T cell receptors. The engineered cells prevented harmful autoimmune response in Type 1 diabetes in mice.

GentiBio is focused on addressing the technical bottlenecks that have throttled Treg therapeutics, and we are thrilled to exclusively in-license a portfolio of unprecedented breadth from Seattle Childrens and Benaroya Research Institute in the U.S. and MIGAL Galilee Research Institute in Israel, said Adel Nada, M.D., co-founder and CEO of GentiBio.

The technologies licensed from these premier research institutions are mature and well-differentiated, and will be further optimized in sponsored research collaborations with the scientific teams that discovered them to advance novel and potent therapeutics with the potential to treat and cure serious autoimmune and inflammatory diseases.

Tregs are rare cells within the immune system and current therapies that source Tregs cells from the blood stream can be costly and cumbersome. In contrast, Seattle Childrens unique technology enables the generation of engineered regulatory T cells, or EngTregs, from the more abundant CD4+ cell population, addressing a critical manufacturing shortcoming for this novel treatment, said David Rawlings, M.D., director, center for immunity and immunotherapies, Seattle Childrens Research Institute and scientific co-founder of GentiBio.

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Novartis and a string of high profile backers fund next-gen Treg cell therapy startup GentiBio - FierceBiotech

Research shows promising results for LHON gene therapy – Ophthalmology Times

This article was reviewed by Alvin Luk, PhD, MBA, CCRA

Results from two investigator-initiated studies demonstrate that a single intravitreal injection of rAAV2-ND4 (Neurophth Therapeutics Inc.) is associated with long-term safety and durable efficacy for improving vision in patients with Leber hereditary optic neuropathy (LHON), according to Alvin Luk, PhD, MBA, CCRA.

The research is comprised of a single center study including 9 patients followed for 75 to 90 months and a multicenter, international trial including 159 patients followed for up to 12 months, with observation ongoing.

Related: TANGO: Helping target genes produce more protein

Across the entire cohort, there were no serious or severe adverse events, and efficacy data showed that a majority of patients benefited with significant and sustained improvement in BCVA.

LHON is a rare inherited visual disorder that leads to bilateral vision loss and for which there is currently no effective treatment, said Luk. These 2 studies contain the largest and longest follow-up of patients treated withthis gene therapy. Based on the results, we are very excited about its potential impact for restoring vision and greater independence for patients with LHON.

Luk, the CEO at Neurophth Therapeutics Inc., noted gene therapy also is offering a positive impact on treatment.

The gene therapy restores function of the mitochondrial respiratory chain in retinal ganglion cells by delivering the NADH ubiquinone oxidoreductase subunit 4 (ND4) gene.

A series of preclinical studies confirmed that the intravitreal treatment resulted in targeted gene delivery and provided evidence of its safety.

Related: Gene therapy zeroes in as LHON treatment

Importantly, the confocal microscopy analysis confirms that rAAV2-ND4 reaches the targeted areas in the eyes where the pathological changes of LHON occur, Luk said. Therefore, providing support to the hypothesis that treatment with rAAV2-ND4 may alleviate the underlying cause of the vision loss in patients with LHON.

The clinical studies of rAAV2-ND4 gene therapy reported by Luk have been led by Bin Li, MD, PhD, Tongji Hospital, Huazhong University of Science and Technology, Wuhan, China.

The first study was initiated in 2011. Known as SEE4 LHON (NCT01267422), it included 3 patients aged younger than 12 years who received 0.5 x 1010 vector genome (vg)/eye and six patients aged older than 12 years who were treated with a dose of 1.0 x 1010vg/eye.

According to investigators, the injections were given into 1 eye in an outpatient procedure and had a volume of 0.05 mL.

Although planned follow-up was initially for 12 months, 8 of the 9 patients have continued follow-up until today, Luk said.

Related: Study targets ocular damage from chronic intravitreal injections

According to Luk, there have been no late toxicities noted nor any abnormalities in intraocular pressure (IOP) in extensive laboratory testing, which includes assessments of hepatic, renal, and immune function.

Efficacy is being evaluated with measurement of the logarithm of the minimum angle of resolution (logMAR) in best-corrected visual acuity (BCVA).

Luk pointed out that at 3 months post-injection, 8 patients (89%) demonstrated improvement from baseline, and 6 of 9 patients (67%) maintained improvement at 3 years.

They also noted that the BCVA response was maintained at month 70 by 5 of the 6 responders who achieved a mean BCVA gain of 0.68 logMAR.

Patients in this study also benefited with some improvement in the untreated eye, Luk said. This kind of bilateral response was also seen in the second larger trial of this therapy and is consistent with observations by other groups working on gene therapy for this inherited eye disease.

Based on the encouraging results of SEE4LHON, a larger scale study named 4-HOPE was launched in 2017.

Related: Research targets precision dosing for gene, cell therapy

All patients were treated at three investigational sites in China and 10 patients are continuing follow-up at their local centers in Argentina.

Luk pointed out that the study enrolled 159 patients aged 6 years or older who received a unilateral injection with 1.0 x 1010 vg/eye.

The safety review showed there were no drug-related adverse events. Ocular hypertension was the most common adverse event that patients experienced, but it is related to the course of oral steroid treatment that is given in conjunction with the injection, Luk explained.

The IOP elevations are generally mild and resolve spontaneously once the steroid treatment is ended, he said.

Of the 159 enrolled patients, 106 had data available from a 12-month follow-up visit. Of the 106 patients, Luk noted that 63% showed an improvement from baseline BCVA with an average gain of 0.3 logMAR.

Related: Greater IOP-lowering with iStent inject

It is important to point out that the patients enrolled in this study represent a heterogenous group with a wide range of ages, time since diagnosis, and baseline BCVA values, Luk concluded. We would not be surprised to see even better efficacy results in a cohort enrolled using narrower inclusion criteria.

Luk noted some of the patients in the study also benefited with bilateral BCVA improvement.

Taking improvements ofinjected and noninjected eyes into consideration from baseline to 12 months post treatment, 43.7% patients who classified as legally blind by World Health Organizationcriteria ( > 1.3 logMAR) were recovered to low (> 0.5 to 1.3 logMAR) or normal ( 0.5 logMAR) vision.Read more by Lynda Charters

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Alvin Luk, PhD, MBA, CCRAe:alvin.luk@neuropth.comLuk is an employee of Neurophth Therapeutics Inc, but has no other relevant financial interests to disclose.

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Research shows promising results for LHON gene therapy - Ophthalmology Times

Taysha Gene Therapies Announces Oversubscribed $95 Million Series B Financing to Bolster Initial Clinical Studies in GM2 Gangliosidosis and Advance…

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease, today announced that it has closed an oversubscribed $95 million Series B financing with a premier syndicate of life science investors, led by Fidelity Management & Research Company LLC. Additional new investors include funds and accounts managed by BlackRock, GV (formerly Google Ventures), Invus, Casdin Capital, Franklin Templeton, Octagon Capital, Perceptive Advisors LLC, Sands Capital, ArrowMark Partners and Venrock Healthcare Capital Partners. Also participating in the round were founding investors PBM Capital and Nolan Capital. Proceeds from the Series B financing will be used to advance the initial cohort of lead programs into the clinic, accelerate progress on anticipated IND submissions, build a commercially scalable GMP manufacturing facility and continue development of the companys extensive portfolio of potentially curative gene therapies in partnership with the UT Southwestern Gene Therapy Program.

This significant investment from premier, long-term investors will allow us to advance our mission of eradicating monogenic CNS disease for the thousands of patients who suffer from these devastating disorders, said RA Session II, President, CEO and Founder of Taysha. We remain on track and expect to file four Investigational New Drug (IND) applications by the end of 2021, with TSHA-101 initiating clinical studies later this year for the treatment of GM2 Gangliosidosis.

Taysha is currently developing a deep and sustainable pipeline of 17 gene therapy product candidates, with exclusive options to acquire four additional programs across three distinct franchises, including neurodegenerative diseases, neurodevelopmental disorders and genetic forms of epilepsy. TSHA-101 is expected to initiate clinical studies later this year for the treatment of GM2 Gangliosidosis, followed by TSHA-102 for the treatment of Rett syndrome, TSHA-103 for the treatment of SLC6A1 haploinsufficiency disorder and TSHA-104 for the treatment of SURF1 deficiency. Taysha expects to file INDs for each of these four product candidates by the end of 2021.

We have brought together experts in gene therapy with leading healthcare and institutional investors to create a company that is uniquely positioned to advance the development of potentially curative gene therapies for CNS disease in rare and large patient populations, said Sean Nolan, Chairman of the Board of Taysha. We believe this financing provides significant validation of our corporate strategy and will enable us to continue to rapidly translate programs from preclinical development into the clinic.

About Taysha Gene Therapies

Taysha Gene Therapies is a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease. We are focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. We were founded in partnership with The University of Texas Southwestern Medical Center, or UT Southwestern, to develop and commercialize transformative gene therapy treatments. Together with UT Southwestern, we are advancing a deep and sustainable product portfolio of 17 gene therapy product candidates, with exclusive options to acquire four additional development programs. By combining our management teams proven experience in gene therapy drug development and commercialization with UT Southwesterns world-class gene therapy research capabilities, we believe we have created a powerful engine to develop transformative therapies to dramatically improve patients lives. More information is available at http://www.tayshagtx.com.

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2020 Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations" report has been added to ResearchAndMarkets.com's offering.

This research identifies some of the key developments across CAR-T cell therapies and provides insights across technological, IP, and investment landscapes. The study also provides an analysis of the competitive landscape while highlighting the key growth opportunities within the CAR-T cell therapy platform.

Companies Mentioned

Key Topics Covered:

1.0 Executive Summary

1.1 Research Focus: Emerging Technologies Enabling chimeric antigen receptor (CAR) T-cell Therapies

1.2 Analysis Framework: The Author's Core Value

1.3 Research Methodology: Five Steps Toward Success

1.4 Key Findings of Technology Breakthrough Driving Sepsis Diagnosis

2.0 Technology Snapshot

2.1 Rising Pace of Cell and Gene Therapy Approvals

2.2 Regulatory and Ethical Perspectives on Gene Therapy

2.3 Rising Demand for Precision Medicine Strategies

2.4 Manufacturing Continues to be the Key Bottle Neck

2.5 II Generation Chimeric Antigen ReceptorS Likely to Dominate the Cell Therapy Landscape in the Future

2.6 CD-19 is the Most Common Target Antigen for Allogeneic CAR-T Therapies

3.0 Emerging Patent Landscape

3.1 Steady Increase in Patent Grants for CAR-T Cell Therapies

3.2 University of Pennsylvania and Novartis Lead the Patent Landscape for CAR-T Cell Therapies

3.3 China and the US Lead the Patent Landscape for CAR-T Cell Therapies

3.4 Snapshot of Key Patent Grants: Novartis

3.5 Snapshot of Key Patent Grants: Cellectis and BlueBird Bio

4.0 Analysis of the Investment Landscape

4.1 Key M&A Trends Across the Global Life Sciences Sector

4.2 Gene Therapy - Venture Capital Funding Assessment

4.3 Gene Therapy - Big Pharma In-licensing Deals Assessment

4.4 Strategic Insights: Cell Therapies and Gene Therapies, Viral Vector CMOS

5. Analysis of the Competitive Landscape

5.1 Allogene Therapeutics

5.2 Precision BioSciences Inc.

5.3 CRISPR Therapeutics AG

5.4 Cellectis S.A.

5.5 Celyad

5.6 Bristol-Myers Squibb (BMS)

5.7 Gilead

5.8 Novartis

5.9 BlueBird Bio

5.10 Summary of the Scoring Methodology

5.11 Competitive Analysis of CAR-T Participants

6.0 CAR-T Cell Therapies: Growth Opportunity Universe

6.1 Growth Opportunity: CAR-T for Solid tumors, 2020

7.0 Industry Influencers

7.1 Key Contacts

7.2 Legal Disclaimer

For more information about this report visit https://www.researchandmarkets.com/r/ndt6sc.

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2020 Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations - ResearchAndMarkets.com - Business Wire

Global Gene Therapy in Oncology Market 2020 Growth Statistics, New Opportunities, Competitive Outloo – PharmiWeb.com

Researchstore.bizhas published the latest research study onGlobal Gene Therapy in Oncology Market 2020 by Company, Type and Application, Forecast to 2025that presents a complete overview of the market with a detailed description of the global market. The report provides complete information about the advancing market trade and business data. The report highlights the dynamics of the market such as internal and external driving forces, restraining factors, risks, challenges, threats, and opportunities. The complete view linked with the progress of this globalGene Therapy in Oncologymarket by the significant players involved in this business. Analysts of this research report predict the financial attributes such as investment, pricing structures along with the profit margin.

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The report delivers brief information on the competitors and the specific growth opportunities with key market drivers. Complete market analysis is given by segmenting the report by companies, region, type, and applications in the report. Top players also analyzed by splitting the globalGene Therapy in Oncologymarket by product type and applications/end industries. The overall report encompasses many aspects of the industry like market size, market status, market trends, and forecast. Additionally, development trends, competitive landscape analysis, and key regions development status has been demonstrated. It also focuses on a product analysis, application analysis, competitive strategies, and strategies impacting the industry. An expert and in-depth analysis of key business trends and future market development prospects, key drivers and restraints, profiles of major market players, and forecasting for 2020 to 2025 time-period has been given.

The report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution. With mergers and acquisitions and fast building of product portfolio, key players in the globalGene Therapy in Oncologymarket are analyzed to take charge of a leading share. Some of the tough competitors in the global market areBristol-Myers Squibb, Editas Medicine, Amgen, Cold Genesys, CRISPR Therapeutics, Advantagene, Idera Pharmaceuticals, Bio-Path Holdings, AstraZeneca, Geron Corp, Mologen AG, Oncotelic, Intellia Therapeutics, Sillajen Biotherapeutics, Oncolytics Biotech, Merck, Johnson & Johnson, Shenzhen SiBiono GeneTech, Oncosec, Marsala Biotech, Tocagen, UniQure, Ziopharm Oncology.

Geographically, this market report studies the following key geographical regions:North America (United States, Canada and Mexico), Europe (Germany, France, United Kingdom, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), Middle East & Africa (Saudi Arabia, UAE, Egypt and South Africa)

Moreover, the report explains the summary of all products and major changes occurring currently in the globalGene Therapy in Oncologyindustry. Later, production and production value estimates by type, estimates of key producers, and production and production volume estimates by region added in the research report. The noticeable feature of this market covered here includes numerous dynamic and static aspects of the businesses. The report comprises of the regional development status of the regions. This report assesses data type concerning various regions includes capacity, production, market share, price, revenue, cost, gross, gross margin, growth rate, consumption, import, export, etc.

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Table of Contents:1 Market Overview2 Company Profiles3 Market Competition, by Players4 North America Market Size and Forecast by Countries5 Europe Market Size and Forecast by Countries6 Asia-Pacific Market Size and Forecast by Countries7 South America Market Size and Forecast by Countries8 Middle East & Africa Market Size and Forecast by Countries9 Market Size Segment by Type10 Market Size Segment by Application11 Research Findings and Conclusion12 Appendix

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Global Gene Therapy in Oncology Market 2020 Growth Statistics, New Opportunities, Competitive Outloo - PharmiWeb.com

Gyroscope Announces Appointment of Leaders in Retinal Disease, Gene Therapy and the Complement System to Its Clinical and Scientific Advisory Boards -…

Aug. 4, 2020 06:00 UTC

LONDON--(BUSINESS WIRE)-- Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced the appointment of leading experts in retinal disease, gene therapy and the complement system to its Clinical and Scientific Advisory Boards. The newly appointed boards will help guide the development of the company's lead investigational gene therapy, GT005, a one-time therapy being developed to potentially slow the progression of dry age-related macular degeneration (AMD), as well as novel gene therapy delivery systems and additional pipeline candidates.

This is an exciting time for Gyroscope and we are honoured to have a group of highly esteemed leaders in retinal surgery, ophthalmology and gene therapy join our Clinical Advisory Board, said Nadia Waheed, M.D., Chief Medical Officer of Gyroscope. We look forward to working with these leaders as we advance the clinical development of GT005 and our proprietary delivery system, with the ultimate goal of helping preserve the sight of millions of people who suffer from vision loss as a result of dry AMD.

We have embarked upon an ambitious journey exploring the potential of gene therapy beyond rare disease and building a pipeline of potential medicines for one of the worlds leading causes of blindness, said Jane Hughes, Ph.D., Chief Scientific Officer of Gyroscope. We are excited to welcome our Scientific Advisory Board members to Gyroscope and to benefit from the insights of some of the worlds leading experts as we advance our understanding of the role of the complement system in retinal diseases.

Members of the Gyroscope Therapeutics Clinical Advisory Board include:

Professor Jacque Duncan, M.D. Professor of Ophthalmology at the University of California, San Francisco School of Medicine.

Jeffrey Heier, M.D. Co-President and Medical Director, Director of the Vitreoretinal Service and Director of Retina Research at Ophthalmic Consultants of Boston.

Professor Allen Ho, M.D. Professor of Ophthalmology at Sidney Kimmel Medical College of Thomas Jefferson University, and Attending Surgeon and Director of Retina Research at Wills Eye Hospital in Philadelphia.

Professor Nancy Holekamp, M.D. Director of Retina Services at Pepose Vision Institute and Professor of Clinical Ophthalmology at the Washington University School of Medicine in St. Louis.

Arshad Khanani, M.D., M.A. Managing Partner, Director of Clinical Research and Director of Fellowship at Sierra Eye Associates, and Clinical Associate Professor at the University of Nevada, Reno School of Medicine.

Professor Robert MacLaren Professor of Ophthalmology at the University of Oxford in the United Kingdom, Consultant Ophthalmologist at the Oxford Eye Hospital, Honorary Professor of Ophthalmology at the UCL Institute of Ophthalmology, Honorary Consultant Vitreoretinal Surgeon at Moorfields Eye Hospital, and an NIHR Senior Investigator. Professor MacLaren is also a member of the Scientific Advisory Board.

Professor Sir Keith Peters, M.D. Senior Consultant to The Francis Crick Institute in London and Regius Professor of Physic Emeritus at the University of Cambridge in the United Kingdom. Professor Peters is also a member of the Scientific Advisory Board.

Professor Hendrik Scholl, M.D. Founder and Director of the Institute of Molecular and Clinical Ophthalmology Basel in Switzerland, and Professor and Chairman of the Department of Ophthalmology at the University of Basel.

Professor David Steel, M.D., MBBS, FRCOphth Consultant Ophthalmologist at Sunderland Eye Infirmary in the United Kingdom and Honorary Professor of Retinal Surgery at Newcastle University in Newcastle upon Tyne, United Kingdom. Professor Steel is also a member of the Scientific Advisory Board.

Professor Bernhard Weber, Ph.D. Head of Institute of Human Genetics, Head of Institute of Clinical Human Genetics, and Director of the Diagnostics Unit for DNA Testing and Vice President of Research of the University of Regensburg, Germany.

Charles Wykoff, M.D., Ph.D. Director of Research at Retina Consultants of Houston, Deputy Chair for Ophthalmology at Blanton Eye Institute, and Clinical Associate Professor of Ophthalmology Weill Cornell Medical College at Houston Methodist Hospital.

Members of the Gyroscope Scientific Advisory Board include:

Professor Alberto Auricchio, M.D. Professor of Medical Genetics at the Department of Advanced Biomedicine, Federico II University in Naples, and Coordinator of the Molecular Therapy Program at Telethon Institute of Genetics and Medicine (TIGEM) in Pozzuoli (NA), in Italy.

Professor Pete Coffey, DPhil Theme Lead of Development, Ageing and Disease at University College Londons Institute of Ophthalmology and the Co-Executive Director of Translation at the University of California Santa Barbaras Center for Stem Cell Biology and Engineering.

Professor Claire Harris Professor of Molecular Immunology at Newcastle University.

Professor David Kavanagh, Ph.D., FRCP Professor of Complement Therapeutics at the National Renal Complement Therapeutics Centre (NRCTC), Newcastle University.

Professor Sir Peter Lachmann, FRS, FMEDSCI Emeritus Sheila Joan Smith Professor of Immunology, University of Cambridge.

Professor Robert MacLaren Professor MacLaren is also a member of the Clinical Advisory Board.

Professor Sir Keith Peters, M.D. Professor Peters is also a member of the Clinical Advisory Board.

Professor Matthew Pickering, Ph.D., M.B., B.S. Professor of Rheumatology, Imperial College LondonHonorary Consultant Rheumatologist, Imperial College Healthcare NHS Trust, and Wellcome Trust Senior Fellow in Clinical Science.

Professor David Steel, M.D., MBBS, FRCOphth Professor Steel is also a member of the Clinical Advisory Board.

Professor Timothy Stout, M.D. Sid W. Richardson Professor and Margarett Root Brown Chair of the Department of Ophthalmology, and Director of the Cullen Eye Institute at Baylor College of Medicine in Houston.

Full biographies for members of the Gyroscope Advisory Boards are available at http://www.gyroscopetx.com.

About Gyroscope Therapeutics: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing and delivering gene therapy beyond rare disease to treat a leading cause of blindness, dry age-related macular degeneration (AMD). Currently, there are no approved treatments for dry AMD.

Our investigational gene therapy, GT005, is designed to restore balance to a part of our immune system called the complement system. An overactive complement system leads to inflammation that damages healthy eye tissues. Our ultimate goal is to slow the progression of dry AMD. Patients in our Phase I/II clinical trial, known as the FOCUS study, receive a single dose of GT005 through an injection under their retina.

Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform, and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information, visit http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200803005822/en/

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Gyroscope Announces Appointment of Leaders in Retinal Disease, Gene Therapy and the Complement System to Its Clinical and Scientific Advisory Boards -...

Of mice and memories: Gene therapy study returns function in Alzheimer’s mice – ABC News

Norman Swan: Alzheimer's disease is the commonest form of dementia, and the results of billions of dollars of research investment have been bitterly disappointing in terms of finding an effective treatment. However, experiments on mice by a group from the Dementia Research Centre at Macquarie University have come up with some promising findings. Professor Lars Ittner is the director of the centre. Welcome to the Health Report Lars.

Lars Ittner: Hello, how are you?

Norman Swan: Now, essentially you are targeting an enzyme which protects nerve cells in the brain from the toxic effects of amyloid beta and that's one of the two main substances which accumulate in Alzheimer's disease. What did you actually do in this study?

Lars Ittner: So in this we found prior to this study that this enzyme activity that protects the brain from Alzheimer's disease is actually lost in Alzheimer's disease. And we devised a gene therapy to replace the enzyme activity and bring the enzyme back into the brain cells.

Norman Swan: And these are mice which replicate Alzheimer's disease in some shape or form.

Lars Ittner: Yes, they are, so we genetically engineered them to develop Alzheimer's disease.

Norman Swan: And they were showing signs of memory and thinking problems?

Lars Ittner: Yes, so their ability to form memory and then store the memory over longer terms is compromised.

Norman Swan: And this was the gene for this enzyme?

Lars Ittner: So we brought backit's called the P38 gamma gene, which we brought back into the brains of these mice and that restored their ability to form memory.

Norman Swan: So you actually got healing?

Lars Ittner: So we were quite surprised because when you set out with these type of studies you expect at most that you stop the progression. But yes, we got far more than we set out for.

Norman Swan: People have tried gene therapy before from Parkinson's disease and other things, and it's quite hard to get the gene therapy into the brain. And of course Alzheimer's disease is quite widespread as opposed to Parkinson's disease. How do you get the gene therapy in reliably?

Lars Ittner: So from the early days of gene therapy done in Parkinson's disease, the vehicles that are used to bring the genes into organisms or in the brain in particular have improved, so these days we use modified viruses that we take advantage of their ability to infect brain cells, and they then deliver the genes for us.

Norman Swan: In the right place. Were there any side effects?

Lars Ittner: So we did toxicity studies as part of our study, and then you use incredibly high amounts of the virus, and we did not see long-term side-effects.

Norman Swan: How do you getthere's something called the bloodbrain barrier, the brain is a protected organ and it's quite hard for things to get into the brain because of this barrier, how did you get beyond that with these gene therapies?

Lars Ittner: So with the mice we can take advantage of a modified virus which has been selected to actually passage this naturally, but in humans you would do a single injection, it's like a lumbar puncture, it's at the base of your neck, and it's directly into the liquid around the brain, so you basically mechanically bypass the bloodbrain barrier.

Norman Swan: There have been very disappointing results. I mean, what happens in mice particularly in Alzheimer's disease does not necessarily happen in humans, and there's not a single amyloid beta therapy that has had much effect on the brains of the people with Alzheimer's disease. Why do you think this one might work in humans when others haven't?

Lars Ittner: So the problem with the amyloid beta is that it is now understood that this is a disease inducing pathology but is not required for the progression of the disease, and we are targeting here actually the tau protein specifically which is

Norman Swan: It's the other thing that

Lars Ittner: Exactly, and that is responsible for the progression of the disease, so it's actually moving away from the amyloid beta as a drug target which has failed in the past.

Norman Swan: Now, with COVID-19 around we are getting used to the language of clinical trials and accelerating trials. When are you ready to go to phase 1 which would be a safety trial in humans?

Lars Ittner: So preclinical experiments have actually been completed for this particular study, and the next step are in fact phase 1 clinical trials, and we are currently working with Macquarie University and their commercialisation arm to find the right partner to move forward into clinical trials.

Norman Swan: Fascinating. Well, we'll follow that up when you do. Thanks for joining us.

Lars Ittner: It was my pleasure.

Norman Swan: Professor Lars Ittner is director of the Dementia Research Centre at Macquarie University.

You've been listening to the Health Report, I'm Norman Swan, and I'd really enjoy your company next week.

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Of mice and memories: Gene therapy study returns function in Alzheimer's mice - ABC News

Detailed Information on Gene Therapy Market 2020 | Covid-19 Impact Analysis | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio,…

Global Gene Therapy Market report forecast to 2026 investigate the Impact of COVID-19 on Industry further market size, manufactures, types, applications and key regions like North America, Europe, Asia Pacific, Central & South America and Middle East & Africa, focuses on the consumption of Gene Therapy in these regions. This report also studies the global Gene Therapy market share, competition landscape, status share, growth rate, future trends, market drivers, opportunities and challenges, sales channels and distributors.

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Leading Players from the market are covered in this report- Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon

Impact of Covid-19 on Gene Therapy Industry 2020

Gene Therapy Market report analyses the impact of Coronavirus (COVID-19) on the Gene Therapy industry. Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Gene Therapy market in 2020.

The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; emergency declared in many countries; massive slowing of the supply chain; stock market unpredictability; falling business assurance, growing panic among the population, and uncertainty about future.

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Market Segments:

Based on Types, the Gene Therapy Market is Classsified as Ex vivo, In vivo

Based on Application, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share and growth rate for each application, including Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others

Gene Therapy Market Report Provides Comprehensive Analysis as Following:

Study on Table of Contents:

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