Archive for the ‘Gene Therapy Research’ Category

Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that it has submitted the Marketing Authorisation Application (MAA) for its lead product LUMEVOQ to the European Medicines Agency (EMA), seeking approval for the treatment of patients with vision loss due to Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene.

This first regulatory submission for GenSight is a major milestone in our progression from a pure research organization to one with commercial capabilities. It validates a technology platform that has the potential to address the high unmet medical needs of patients suffering from a range of rare diseases. I would like to thank all GenSight employees and partners whose motivation, focus and effort made this submission possible, said Bernard Gilly, Co-founder and Chief Executive Officer of GenSight Biologics.

LHON is a rare, mitochondrial genetic disease, mainly affecting young males. The ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There continues to be a high unmet medical need for the 800-1200 new LHON patients in Europe and the U.S. each year, particularly those who are struck blind in their prime working years.

Lenadogene nolparvovec (tradename: LUMEVOQ) is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2), containing a cDNA encoding the human wild-type mitochondrial NADH dehydrogenase 4 protein (ND4), which has been specifically developed for the treatment of LHON associated with mutation in the ND4 gene. It received orphan drug designation status for the treatment of LHON from the EMA in 2011 and from the U.S. Food and Drug Administration (FDA) in 2013.

GenSight submitted the MAA based on the benefit-risk balance established by results from a Phase-I/IIa study (CLIN-01), two pivotal Phase-III efficacy studies (CLIN-03A: RESCUE, and CLIN-03B: REVERSE) and the long-term follow up study of RESCUE and REVERSE (CLIN 06 readout at Year 3 post injection). To demonstrate the efficacy of LUMEVOQ in the context of a contralateral effect, the Company used a statistics-based indirect comparison methodology to assess the visual outcomes in LUMEVOQ-treated patients (from LUMEVOQ efficacy studies) against those in untreated patients from Natural History studies and GenSights REALITY Natural History Registry.

GenSight expects to submit the Biologics License Application (BLA) for LUMEVOQ to the FDA in H2 2021. First-in-human data from GenSights second clinical stage program, GS030, are expected to be available in H2 2021.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to the eye by intravitreal injection to offer patients a sustainable functional visual recovery.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200914005857/en/

GenSight BiologicsChief Financial Officer

Thomas Gidointgidoin@gensight-biologics.com+33 (0)1 76 21 72 20

LifeSci AdvisorsInvestor Relations

Guillaume van Renterghem

gvanrenterghem@lifesciadvisors.com+33 (0)6 69 99 37 83

RooneyPartnersMedia Relations

Marion Janic

mjanic@rooneyco.com+1-212-223-4017

Orpheon FinanceRetail Investors

James Palmerj.palmer@orpheonfinance.com+33 (0)7 60 92 77 74

Excerpt from:
GenSight Biologics Submits EU Marketing Authorisation Application for LUMEVOQ Gene Therapy to Treat Vision Loss Due to Leber Hereditary Optic...

When a researchers talk about gene editing, theyre usually thinking about several steps. First you need to zero in on the defective gene; then, depending on the need, youd want to knock out, replace or insert genetic material.

CRISPR/Cas9 technologies have transformed the field by making a breakthrough for the first problem. Inducing double-stranded DNA breaks, or achieving single-letter changes as base editing allows, have promising applications in multiple diseases that are starting to get tested in humans.

But Versant Ventures, one of the pioneering investors in the space, still sees a gap. And theyre teaming up with a group of prominent Stanford researchers plus a seasoned biotech exec to fill it.

Achieving high-efficiency targeted gene integration has been a critical objective of gene editing for more than 15 years, but only now is this technologically possible, Jerel Davis, Versants Vancouver-based managing director, said.

Graphite Bio has $45 million to start things off, but the money matters little when compared to the rich brain bank its drawing from.

Matthew Porteus, an academic founder of CRISPR Therapeutics, is lending the tech platform to create this next-gen play alongside gene therapy expert Maria Grazia Roncarolo.

Drawing from research work led by Danny Dever while a postdoc at Porteus lab, Graphites big promise is to increase integration efficiency from less than 1% to greater than 50% across diverse genetic lesions in a wide range of cell types.

Specifically, Dever and Porteus showed that they could correct the sickle globin gene in patient-derived hematopoietic stem cells ex vivo by combining Cas9 ribonucleoproteins with a donor molecule that serves as a template, delivered in recombinant adeno-associated viral vectors of serotype 6 (rAAV6).

That could make for a much more scalable replacement for transplants, they wrote in a 2017 paper describing mouse studies.

Notably, we devise an enrichment paradigm to purify a population of HSPCs with >90% targeted integration, they wrote. We also show efficient correction of the SCD-causing E6V mutation in patient-derived HSPCs that after differentiation into erythrocytes, express adult -globin (HbA) mRNA, confirming intact transcriptional regulation of edited HBB alleles.

Chief executive Josh Lehrer is leading the charge to start Phase I for this program in early 2021, bringing all the sickle cell knowledge and experience from a six-year run as Global Blood Therapeutics, most recently as CMO. More preclinical therapies are in the works for unnamed serious diseases. Samsara BioCapital is joining Versant for the launch round and sending Abe Bassan to the board, which also features Davis and Carlo Rizzuto from Versant.

Read more here:
Versant teams up with Stanford gene editing experts on a $45M next-gen play marrying CRISPR and AAV to fix sickle cell - Endpoints News

David Dismuke, Ph.D., joins as Chief Technical Officer, Christopher Shilling as Vice President of Regulatory Affairs and Quality Assurance, and Christina Perry as Vice President of Finance and Operations.All three leaders will be in place in September and join Forge's executive leadership team.

"The addition of David, Chris, and Christina to the Forge leadership team brings immeasurable value to the Forge team and expands our team of gene therapy experts," said Timothy J. Miller, Ph.D., Forge's Co-Founder, President & CEO. "We believe that our accomplished and passionate team, with demonstrated experience in manufacturing and developing gene therapy products, is the foundation of fulfilling our mission to accelerate transformative medicines to reach those who need them most."

David Dismuke joins Forge with more than 15 years of experience in large-scale manufacturing. He is an authority in the bioprocessing and design of gene therapy vectors and has led CMC operations in the large-scale manufacturing of pre-clinical and clinical-grade AAV vectors for more than 10 years. Prior to joining Forge, David was the Vice President of Manufacturing at StrideBio where he directed the development of manufacturing and analytical processes for AAV vectors that utilize novel capsids. In addition, he led the design of therapeutic and reporter transgenes and innovative molecular enhancements to improve AAV production and therapeutic function.He was also previously the Head of Vector Production at Voyager Therapeutics where he led teams in the manufacturing and analytical testing of AAV using the baculovirus/Sf9 production system.Prior to Voyager, David was the Director of the UNC Vector Core, where he oversaw GMP operations as well as the production of research-grade vectors.He earned his PhD from Vanderbilt University, focusing on the molecular biology and lifecycle of HIV-1, and then performed his postdoctoral research at UNC Chapel Hill.

Christopher Shilling joins Forge as Vice President of Regulatory Affairs and Quality. He has over 15 years of experience in development of novel gene therapies for rare and severe disorders. Christopher is an experienced leader in gene therapy regulatory affairs, pharmacology, toxicology, and project management focused on developing strategies for early phase clinical trials in support of a variety of transformative therapeutics for pediatric and rare diseases. Prior to joining Forge, Christopher started the Drug and Device Development program at Nationwide Children's Hospital which was instrumental in gaining acceptance from regulators for over twenty first-in-human gene therapy clinical trials of novel biologic products, a dozen orphan drug designations, and two fast track designations. He received his Bachelor of Science degree in biology and a Master of Science degree in Pathology both from the Ohio State University.

Christina Perry joins Forge Biologics as the Vice President of Finance and Operations. She spent the last several years as the CFO of Drive Capital where she built out all accounting and finance operations, back-office needs, and investor relations and reporting. Christina managed complex entity legal structures and has had extensive exposure to equity and debt financings.Over the last 15+ years Christina has worked with public and private companies and startups across varying industries, developing operational processes to scale with high growth. She began her career at Deloitte, is a licensed CPA in the state of Ohio, earned her Bachelor of Business Administration at the University of Notre Dame and a Master of Accountancy from Miami University.

These additions to the Forge Biologics leadership team continue Forge's momentum within the biotechnology industry in Columbus Ohio, bringing positive impact to both Ohio and the global rare disease community.

About Forge BiologicsThe mission of Forge Biologics is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, referred to as "The Hearth", to serve as their headquarters. The Hearth will be home to a custom-designed cGMP facility, dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit https://www.forgebiologics.com.

Media Inquiries:Dan SalvoForge Biologics Director of Communications and Community Development[emailprotected]

Business Inquiries:Erandi De Silva, Ph.D.Forge BiologicsCo-Founder and Vice President of Product Development[emailprotected]

SOURCE Forge Biologics

https://forgebiologics.com

The rest is here:
Forge Biologics Strengthens Gene Therapy Leadership with Key Hires in AAV Manufacturing, Regulatory Affairs and Finance to Support Strategic Growth -...

DUBLIN--(BUSINESS WIRE)--The "Viral Vector & Plasmid DNA Manufacturing Market Research Report by Type, by Application, by End User - Global Forecast to 2025 - Cumulative Impact of COVID-19" report has been added to ResearchAndMarkets.com's offering.

The Global Viral Vector & Plasmid DNA Manufacturing Market is expected to grow from USD 551.56 Million in 2019 to USD 2,101.82 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 24.97%.

This research report categorizes the Viral Vector & Plasmid DNA Manufacturing to forecast the revenues and analyze the trends in each of the following sub-markets:

The FPNV Positioning Matrix evaluates and categorizes the vendors in the Viral Vector & Plasmid DNA Manufacturing Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

Cumulative Impact of COVID-19:

COVID-19 is an incomparable global public health emergency that has affected almost every industry so far and the long-term effects projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlaying COVID-19 issues and potential paths forward. The report is delivering insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecast, considering the COVID-19 impact on the market.

The report provides insights on the following pointers:

1. Market Penetration: Provides comprehensive information offered by the key players

2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets

3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments

4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players

5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:

1. What is the market size and forecast of the Global Viral Vector & Plasmid DNA Manufacturing Market?

2. What are the inhibiting factors and impact of COVID-19 shaping the Global Viral Vector & Plasmid DNA Manufacturing Market during the forecast period?

3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Viral Vector & Plasmid DNA Manufacturing Market?

4. What is the competitive strategic window for opportunities in the Global Viral Vector & Plasmid DNA Manufacturing Market?

5. What are the technology trends and regulatory frameworks in the Global Viral Vector & Plasmid DNA Manufacturing Market?

6. What are the modes and strategic moves considered suitable for entering the Global Viral Vector & Plasmid DNA Manufacturing Market?

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/w76mxp

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Global Viral Vector & Plasmid DNA Manufacturing Market Analysis and Forecasts to 2025 - Cumulative Impact of COVID-19 - ResearchAndMarkets.com -...

More than 1 million Americans are diagnosed with a chronic brain disorder each year, yet effective treatments for most complex brain disorders are inadequate or even nonexistent.

A major new research effort at the McGovern Institute for Brain Research at MIT aims to change how we treat brain disorders by developing innovative molecular tools that precisely target dysfunctional genetic, molecular, and circuit pathways.

The K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience was established at MIT through a $28 million gift from philanthropist Lisa Yang and MIT alumnus Hock Tan 75. Yang is a former investment banker who has devoted much of her time to advocacy for individuals with disabilities and autism spectrum disorders. Tan is president and CEO of Broadcom, a global technology infrastructure company.This latest gift brings Yang and Tans total philanthropy to MIT to more than $72 million.

In the best MIT spirit, Lisa and Hock have always focused their generosity on insights that lead to real impact," says MIT President L. Rafael Reif. Scientifically, we stand at a moment when the tools and insights to make progress against major brain disorders are finally within reach. By accelerating the development of promising treatments, the new center opens the door to a hopeful new future for all those who suffer from these disorders and those who love them. I am deeply grateful to Lisa and Hock for making MIT the home of this pivotal research.

Engineering with precision

Research at the K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience will initially focus on three major lines of investigation: genetic engineering using CRISPR tools, delivery of genetic and molecular cargo across the blood-brain barrier, and the translation of basic research into the clinical setting. The center will serve as a hub for researchers with backgrounds ranging from biological engineering and genetics to computer science and medicine.

Developing the next generation of molecular therapeutics demands collaboration among researchers with diverse backgrounds, says Robert Desimone, McGovern Institute director and the Doris and Don Berkey Professor of Neuroscience at MIT. I am confident that the multidisciplinary expertise convened by this center will revolutionize how we improve our health and fight disease in the coming decade. Although our initial focus will be on the brain and its relationship to the body, many of the new therapies could have other health applications.

There are an estimated 19,000 to 22,000 genes in the human genome and a third of those genes are active in the brain the highest proportion of genes expressed in any part of the body. Variations in genetic code have been linked to many complex brain disorders, including depression and Parkinsons disease. Emerging genetic technologies, such as the CRISPR gene editing platform pioneered by McGovern Investigator Feng Zhang, hold great potential in both targeting and fixing these errant genes. But the safe and effective delivery of this genetic cargo to the brain remains a challenge.

Researchers within the new Yang-Tan Center will improve and fine-tune CRISPR gene therapies and develop innovative ways of delivering gene therapy cargo into the brain and other organs. In addition, the center will leverage newly developed single-cell analysis technologies that are revealing cellular targets for modulating brain functions with unprecedented precision, opening the door for noninvasive neuromodulation as well as the development of medicines. The center will also focus on developing novel engineering approaches to delivering small molecules and proteins from the bloodstream into the brain. Desimone will direct the center and some of the initial research initiatives will be led by associate professor of materials science and engineering Polina Anikeeva; Ed Boyden, the Y. Eva Tan Professor in Neurotechnology at MIT; Guoping Feng, the James W. (1963) and Patricia T. Poitras Professor of Brain and Cognitive Sciences at MIT; and Feng Zhang, James and Patricia Poitras Professor of Neuroscience at MIT.

Building a research hub

My goal in creating this center is to cement the Cambridge and Boston region as the global epicenter of next-generation therapeutics research. The novel ideas I have seen undertaken at MITs McGovern Institute and Broad Institute of MIT and Harvard leave no doubt in my mind that major therapeutic breakthroughs for mental illness, neurodegenerative disease, autism, and epilepsy are just around the corner, says Yang.

Center funding will also be earmarked to create the Y. Eva Tan Fellows program, named for Tan and Yangs daughter Eva, which will support fellowships for young neuroscientists and engineers eager to design revolutionary treatments for human diseases.

We want to build a strong pipeline for tomorrows scientists and neuroengineers, explains Hock Tan. We depend on the next generation of bright young minds to help improve the lives of people suffering from chronic illnesses, and I can think of no better place to provide the very best education and training than MIT.

The molecular therapeutics center is the second research center established by Yang and Tan at MIT. In 2017, they launched the Hock E. Tan and K. Lisa Yang Center for Autism Research, and, two years later, they created a sister center at Harvard Medical School, with the unique strengths of each institution converging toward a shared goal: understanding the basic biology of autism and how genetic and environmental influences converge to give rise to the condition, then translating those insights into novel treatment approaches.

All tools developed at the molecular therapeutics center will be shared globally with academic and clinical researchers with the goal of bringing one or more novel molecular tools to human clinical trials by 2025.

We are hopeful that our centers, located in the heart of the Cambridge-Boston biotech ecosystem, will spur further innovation and fuel critical new insights to our understanding of health and disease, says Yang.

Originally posted here:
New molecular therapeutics center established at MIT's McGovern Institute - MIT News

IndustryGrowthInsights (IGI), one of the worlds prominent market research firms has announced a novel report on Global Gene Therapy Market. The report contains vital insights on the market which will support the clients to make the right business decisions. This research will help both existing and new aspirants for Gene Therapy market to figure out and study the market needs, market size, and competition. The report incorporates data regarding the supply and demand situation, the competitive scenario, and the challenges for market growth, market opportunities, and the threats faced by key players during the forecast period of 2020-2026.

Request Free Sample Report @ https://industrygrowthinsights.com/request-sample/?reportId=168032

Impact of COVID-19 on Gene Therapy Market

The report also includes the impact of ongoing global crisis i.e. COVID-19 on the Gene Therapy market and what the future holds for it. It provides an analysis of the effects of the pandemic on the global economy. The outbreak has directly disturbed the demand and supply chain. The report also analyzes the financial impact on firms and financial markets. IndustryGrowthInsights (IGI) has gathered insights from several delegates of the industry and got involved in the primary and secondary research to provide the clients with data and strategies to combat the market challenges during and after COVID-19 pandemic.

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Industry experts and research analysts have worked extensively to prepare the research report which will help you to give that extra edge in the competitive market. The market research report can be customized according to you to your needs. This means that IndustryGrowthInsights (IGI) can cover a particular product, application, or can provide a detailed analysis in the report. You can also purchase a separate report for a specific region.

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Some of the major companies that are covered in this report:

Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.AdvantageneGene Therap

*Note: Additional companies can be included on request

The market scenario is likely to be fairly competitive. To analyze any market with simplicity the market is fragmented into the following segments:

By Application:

CancerMonogenicInfectious diseaseCardiovascular diseaseOther

By Type:

Ex vivoIn VivoGene Therap

By Geographical Regions

Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa

Segmenting the market into smaller components helps in analyzing the dynamics of the market with more clarity. Another key component that is included in the report is the regional analysis to assess the global presence of the Gene Therapy market. You can also opt for a yearly subscription of all the updates on the Gene Therapy market.

Request a sample before buying this report @ https://industrygrowthinsights.com/request-sample/?reportId=168032

Below is the TOC of the report:

Executive Summary

Assumptions and Acronyms Used

Research Methodology

Gene Therapy Market Overview

Gene Therapy Supply Chain Analysis

Gene Therapy Pricing Analysis

Global Gene Therapy Market Analysis and Forecast by Type

Global Gene Therapy Market Analysis and Forecast by Application

Global Gene Therapy Market Analysis and Forecast by Sales Channel

Global Gene Therapy Market Analysis and Forecast by Region

North America Gene Therapy Market Analysis and Forecast

Latin America Gene Therapy Market Analysis and Forecast

Europe Gene Therapy Market Analysis and Forecast

Asia Pacific Gene Therapy Market Analysis and Forecast

Middle East & Africa Gene Therapy Market Analysis and Forecast

Competition Landscape

If you have any questions on this report, feel free to reach us! @ https://industrygrowthinsights.com/enquiry-before-buying/?reportId=168032

About IndustryGrowthInsights (IGI):

IndustryGrowthInsights (IGI) has a vast experience in making customized market research reports in various industry verticals. Our motto is to provide complete client satisfaction. We cover in-depth market analysis, which consists of stipulating lucrative business strategies, especially for the new entrants and the emerging players of the market. We make sure that each report goes through intensive primary, secondary research, interviews, and consumer surveys before final dispatch.

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Impact Of Covid-19 On Gene Therapy Market 2020 Analysis By Geographical Regions, Type And Application Till 2027 With Top Key Players: Bluebird Bio,...

NEW YORK--(BUSINESS WIRE)--As part of a two-day virtual Investor Day, Pfizer Inc. (NYSE: PFE) provided an extensive overview of pipeline advances and shared updates on the Companys efforts to battle the COVID-19 pandemic on multiple fronts, including new data on the BNT162b2 vaccine candidate being developed in collaboration with BioNTech SE. The pipeline updates contribute to the Companys expectation of at least a 6% revenue CAGR over the next five years, as well as delivery of longer-term topline growth beyond that period.

Pfizers goal of delivering up to 25 breakthroughs to patients by the year 2025 has 38 such opportunities to draw from as of today, including the companys 20-valent pneumococcal conjugate vaccine candidate (20vPnC). On a non-risk adjusted basis, these opportunities collectively represent more than $15 billion (excluding 20vPnC) in potential incremental revenue for Pfizer from 2020 to 2025, as well as aggregate peak annual sales potential of $35 billion to $40 billion (including 20vPnC). If successful, the Companys COVID-19 programs would be incremental to these estimates.

Pfizers purpose Breakthroughs that change patients lives has never been more relevant, and our R&D pipeline has never been more dynamic, said Dr. Albert Bourla, Pfizer Chairman and CEO. I am proud of the truly transformational science that our research and clinical teams are bringing to the fight against disease, as well as the unprecedented speed with which we are advancing our clinical programs in the battle against COVID-19. In the coming months and years, I look forward to the new Pfizer continuing to demonstrate the agility and innovative spirit of a biotech combined with the scale of Big Pharma. With the depth and breadth of our current portfolio, the tremendous potential of our pipeline and scientific engine, and the power of our culture of innovation, we are poised to continue delivering meaningful value to patients by addressing some of the worlds most difficult health challenges.

UPDATES ON COVID-19 DEVELOPMENT PROGRAMS

Pfizer announced several key advances in its efforts to protect humankind from the COVID-19 pandemic and prepare the pharmaceutical industry to better respond to future global health crises.

BNT162 mRNA-based Vaccine Program

Pfizer and BioNTech shared several updates from their BNT162 mRNA-based vaccine program against SARS-CoV-2, the virus that causes COVID-19 disease, including:

Protease Inhibitor Program

The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814. Of note,

THERAPEUTIC AREAS OF FOCUS

Pfizer shared significant research advances across its various therapeutic areas including candidates with blockbuster potential expected to launch by 2025.

Vaccines

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Updates on these late-stage clinical development programs include:

Rare Disease

Pfizers Rare Disease late-stage pipeline currently includes three gene therapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. Key updates include:

Oncology

Pfizers Oncology pipeline has the potential to deliver up to 14 approvals expected by the end of 2025 and the potential for 24 new molecular entities in the clinic by the end of 2021. Key updates included, for the first time, early-stage opportunities obtained from the 2019 acquisition of Array BioPharma:

Inflammation and Immunology

The Inflammation & Immunology pipeline is focused on patients with autoimmune and chronic inflammatory diseases across rheumatology, gastroenterology and dermatology, with five distinct immuno-kinases, in oral and topical formulations, studied for potential treatment of 10 diseases, and three additional novel biologics in Phase 2 studies. Key updates included:

Internal Medicine

The Internal Medicine pipeline addresses the increasing global burden of cardiometabolic disease, with nine investigational medicines in active clinical studies and additional therapies in the pre-clinical pipeline. Key updates included:

To access a replay of the webcast, including audio, video and presentation slides, visit our web site at http://www.pfizer.com/investors.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice: The information contained in this release is as of September 15, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release or the webcast as the result of new information or future events or developments.

This release and the webcast contain forward-looking information about Pfizers anticipated operating and financial performance, business plans and prospects, Pfizers pipeline portfolio (including anticipated regulatory submissions, data read-outs, study starts, approvals, revenue contributions and market opportunities), and our efforts to respond to COVID-19, including our investigational vaccine candidate against SARS-CoV-2 and our investigational protease inhibitor, including their potential benefits, among other things, that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications, biologics license applications and/or emergency use authorization applications may be filed in any jurisdictions for any potential indication for Pfizers product candidates; whether and when any such applications that may be filed for any of Pfizers product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether any such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Pfizers product candidates, including development of products or therapies by other companies; manufacturing capabilities or capacity; uncertainties regarding the ability to obtain recommendations from vaccine technical committees and other public health authorities and uncertainties regarding the commercial impact of any such recommendations; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

More:
Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas - Business Wire

Here are the top biotech companies you will find in Oxford, a city with an old scientific tradition and an enormous output of biotech applications.

Oxford is well known for its university, one of the oldest in Europe and considered to be one of the best in the world. Recently, the University of Oxford has been the center of attention thanks to an experimental Covid-19 vaccine that the university is developing in partnership with big pharmaceutical companies. If successful, the vaccine, already in phase III testing, could be one of the first to get approval for this new disease.

The University of Oxford has hosted thousands of bright minds over the years. Thanks to its emphasis on technology transfer, the university has also helped a large number of them turn their ideas into successful spinout companies. The environment created around the university has also attracted many talents and businesses to the city, making it the ideal melting pot for new and promising ideas.

Biotech is one of the fortes of the innovation seen in the city of Oxford. So we consulted with local experts to put together a list of the most remarkable companies in the city, be it for their size, innovation, or influence in the sector.

Founded in 2008, Immunocore is one of just a few private biotech companies in Europe that are estimated to be worth over 1B. The company is tackling multiple forms of cancer as well as infectious and autoimmune diseases using T-cell receptor (TCR) technology. TCRs are proteins on the surface of immune T cells that are responsible for identifying a threat that must be destroyed, such as cancerous or infected cells. Immunocore aims to patients with engineered TCRs to circumvent the mechanisms by which these threats evade the immune system, restoring its ability to fight disease.

The company is collaborating on several projects with Genentech, AstraZeneca, Eli Lilly, and GSK. Its most advanced program is a treatment for uveal melanoma that is currently in phase III trials. Other programs target solid tumors, hepatitis, HIV, and type 1 diabetes.

Adaptimmune Therapeutics was founded at the same time as Immunocore with the goal of exploiting TCR technology in the form of T-cell therapy. The company engineers the TCRs naturally present on the patients own immune T cells to improve their ability to identify cancerous cells.

Adaptimmune is now getting ready to start late-stage clinical trials in multiple cancer types. Thanks to a deal with Astellas Pharma, the company is also gearing up to start clinical testing of a version of its T-cell therapy that doesnt require engineering each dose individually for each patient, using donor cells instead. Adaptimmune also has several partnerships with companies including GSK, Noile-Immune Biotech, and Alpine Immune Sciences.

Oxford Biodynamics was spun out of Oxford University in 2007 with the goal of developing liquid biopsy tests that can perform a diagnosis from just a drop of blood. The company specializes in epigenetics, that is changes to the structure of our DNA that determines which genes are switched on or off.

Oxford Biodynamics works in a wide range of indications, including cancer, diabetes, Alzheimers, multiple sclerosis, and rheumatoid arthritis among many others. The tests are not only designed to diagnose a disease; they can also be made to determine which patients are going to benefit the most from a specific drug, and how likely the disease is to progress faster or relapse.

With these tests, the company is supporting the development of personalized medicine approaches and helping drug developers increase their chances of succeeding in clinical trials. Partners include big pharma such as Pfizer and EMD, as well as universities and research institutes.

Chronos Therapeutics started out in 2009 as a spinout of the University of Oxford with the goal of developing drugs for age-related conditions. The companys lead program targets amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, for which there are no treatments able to stop its progression.

Over time, the company has expanded its portfolio into other indications, particularly those that affect the brain, by acquiring assets from third parties. They include programs targeting fatigue caused by multiple sclerosis, addictive behaviors such as binge eating and alcohol use disorder, and post-traumatic stress disorder.

Evox Therapeutics is developing a drug delivery technology based on exosomes nanoparticles that our bodies naturally use to transport molecules. The company engineers exosomes to carry different types of drugs, such as proteins, RNA, or small drugs. The key advantage is that these natural carriers are able to reach targets that conventional drug delivery systems cant, such as the brain.

Founded in 2016, the company has signed big deals with Takeda and Eli Lilly. Its programs are all in preclinical testing and mostly target rare diseases. One of them targets the rare liver disorder argininosuccinic aciduria and is scheduled to enter clinical trials in 2021.

Exscientia is a pioneer in the application of artificial intelligence to drug discovery. The company uses AI to identify potential drug candidates and optimize their structure to maximize their chances of success in clinical trials. The goal is to speed up and reduce the costs of the drug discovery process.

Earlier this year, the companys drug candidate for obsessive-compulsive disorder became one of the first AI-designed drugs to enter clinical trials. While a drug typically takes five years from identification until clinical trials, this one did so in just a year.

Exscientia is partnered with Bayer, BMS, Sanofi, and GSk among others. In the wake of the Covid-19 pandemic, the company set out to go through a database of 15,000 approved and investigational drugs that had already passed safety testing to find candidates that can then be fast-tracked to clinical testing in Covid-19 patients.

Oxford Biomedica was set up in 1995 as a developer and provider of lentiviral vectors for gene and cell therapy. These vectors allow the permanent introduction of a desired DNA sequence into a target cell, be it in the test tube or directly in the patients body.

The technology of Oxford Biomedica is regularly used by companies such as Novartis, Sanofi, Boehringer Ingelheim, Imperial Innovations, and Orchard Therapeutics. Notably, the vectors developed by the company are used in Novartis Kymriah, the first CAR T-cell therapy approved in Europe and the US as a cancer treatment.

Oxford Biomedica also has a preclinical pipeline of proprietary programs in a wide range of applications, including cancer, eye disease, ALS, and liver disease. Last year, the company struck a deal with Microsoft to reduce the complexity and costs of gene and cell therapy manufacturing using artificial intelligence.

Founded in 2016, Arctoris aims to bring the benefits of automation to cancer research. Through the companys services, a researcher could just order an experiment online and spend their valuable time designing experiments and analyzing results rather than performing the repetitive tasks needed to complete them.

Arctoris aims to contribute to reducing the costs of drug discovery, which are increasing every year as treatments become personalized and results more difficult to replicate. In the context of the Covid-19 pandemic, Arctoris has established assays that allow scientists the possibility of running Covid-19 experiments remotely.

Founded in 2015, Orbit Discovery is a drug discovery company focusing on the identification of peptide drugs. The company has developed a technology that significantly improves on conventional methods of drug screening such as phage display or mRNA display.

The technology consists of fusing peptides to the DNA sequence encoding them and presenting them to live cells. This method allows the screening of peptide targets that were previously missed by other technologies, and to study their effect on live cells to better predict their function.

The company has already identified several candidates in the areas of cardiology, immunology, and cancer, and is working with partners such as Zealand Pharma in their preclinical development.

OxStem is a drug discovery company with an unusual approach to stem cell treatments. Instead of using stem cells as a therapy, the firm focuses on developing small molecule drugs that can reprogram the patients own stem cells to treat a wide range of diseases related to aging, including cancer and diabetes as well as neurological, cardiovascular, and ocular conditions.

Founded in 2013, the strategy of OxStem is to spin out companies that specialize in each disease area to focus on the development of the drugs found by the parent company, with five subsidiaries set up so far.

Originally posted here:
The Top 10 Biotech Companies Brewing at... - Labiotech.eu

This month, Tmunity Therapeutics is dosing the first patients who have enrolled in an early clinical trial testing an engineered CAR-T immune cell therapy for metastatic prostate cancer. The trial's startis notable, and not just because it's been challenging to recruit patients for cancer trials during the COVID-19 pandemic. The experimental CAR-T is among the first cell therapies to target a solid tumoran enemy that was once considered too difficult to tackle with that type of treatment.

As the first FDA-approved CAR-T cell therapies start to take hold in the blood cancer sphere, a second wave of cell therapies for cancer is following close behind, promising to offer even more choices for patients. In addition to personalized CAR-T therapies for solid tumors, the pipeline includes off-the-shelf CAR-Ts for blood cancers and cell therapies that harness the bodys innate ability to kill cancer.

The CAR-T treatments on the marketNovartis Kymriah, and Yescarta and Tecartus from Gileads Kite Pharmaall target CD19 on the surface of B cells that drive some blood cancers. The treatments involve removing T cells from individual patients and engineering them to be able to recognize and kill their cancers.

ESMO Post Show: Highlights From the Virtual Conference

Cancer experts and pharma execs will break down the headline-making data from ESMO, sharing their insights and analysis around the conferences most closely watched studies. This discussion will examine how groundbreaking research unveiled over the weekend will change clinical practice and prime drugs for key new indications, and panelists will fill you in on the need-to-know takeaways from oncologys hottest fields.

Much of the work on next-gen cell therapies for cancer revolvesaround finding new targets for the engineered immune cells to home in on, as well as improving the therapies to make them more efficient and to reduce the risk of dangerous immune side effects. Theres a lot of activity in this field and different strategies that I think will prove to be effective in the next few years, said Dan Kaufman, M.D., Ph.D., a hematologist and professor of medicine at the University of California, San Diego in an interview.

RELATED: Andreessen Horowitz leads $75M round for Tmunity as it eyes phase 2 studies for 2021

Tmunitys prostate cancer CAR-T is engineered to target prostate-specific membrane antigen, a protein thats often overexpressed by prostate cancer cells, as well as transforming growth factor (TGF) beta, which can promote tumor growth.

Normally, TGF beta binds to T cells in a way that prevents them from mounting an immune attack, Tmunity CEO Usman Oz Azam, M.D., said in an interview. The company's engineering process changes the TGF beta receptor so it cant interfere with the T cells' work.

Its a combination of targeting the antigen but also giving the CAR-T cells a chance to persist and endure, which is where the TGF beta component comes in, he said. The prostate cancer CAR-T is now in a phase 1 trial at six clinical sites in the U.S.

Like the three FDA-approved CAR-Ts, Tmunitys prostate cancer therapy is made from patients own immune cells, a time-consuming and expensive process. Several companies are trying to avoid those costswith off-the-shelf, or allogeneic CAR-Ts. These cell therapies are made from donor cells and engineered so that they dont cause the rejection reaction known as graft-versus-host disease (GVHD).

Allogene, co-founded by Kite pioneer Arie Belldegrun, is one of the pioneers in off-the-shelf CAR-T, and in May it unveiled early data from a trial of its CD19-targeted candidate, ALLO-501, in patients with lymphoma. The company reported a 63% response rate.

RELATED: ASCO: Allogene reports 63% response rate in early trial of off-the-shelf CAR-T in lymphoma

Off-the-shelf CAR-T cells are genetically engineered to avoid GVHD. Precision Biosciences, another player working on a CD19-directedoff-the-shelf CAR-T, developed a technology that removes the TCR gene, which causes GVHD, and inserts the CAR (chimeric antigen receptor) all in one stepan approach that could offer some advantages, Precision CEOMatt Kane said in an interview.

With the first generation of CAR-T cells, the CAR randomly goes into the genome of cells, and as a result they can express at different levels of potency. We insert the CAR into one location, which takes away that variability, Kane said. The company expects to share clinical trial data late this year or in early 2021.

The off-the-shelf CAR-T field has hit some obstacles, though.Cellectis is developing an anti-CS1 CAR-T for multiple myeloma thats also designed for off-the-shelf use, but the FDA put a phase 1 trial on hold after one patient on the higher dose being tested developed cytokine release syndrome (CRS)and died of cardiac arrest.

The company is reviewing the case, and once Cellectis provides feedback to the FDA, the agency will have 30 days to decide whether to restart the trial, Cellectis CEO Andr Choulika, Ph.D., said in an interview.

Another potential option for off-the-shelf cell therapies for cancer is to engineer them not with T cells, but with different residents of the immune system known as natural killer or NK cells. These cells can be taken from donors and given to patients without causing GVHD, and they have an innate ability to recognize and destroy cancers. But there typically arent enough NK cellsto infiltrate tumors effectively, and the cells tend to be short-lived.

Nkarta is developing NK cell therapies that are designed to address both those shortcomings. Nkartas cells are engineered so they have an enhanced ability to recognize eight ligands that are upregulated in several blood cancers and solid tumors. The cells also express interleukin-15 (IL-15), a cytokine growth factor.

"Were able to maintain the growth of the cells for a long period of time, said James Trager, Ph.D., chief scientific officer of Nkarta, in an interview. The company recently raised $114 million in a series B round and expects to move its lead candidate, NKX101, into clinical trials by year-end.

RELATED: Nkarta nets $114M to push natural killer cell therapies into the clinic

Several NK cell developers are following close on Nkartas heals. Fate Therapeutics is working on both off-the-shelf CAR-T and NK therapies and recently signed a $50 million co-development deal with Johnson & Johnson.

Two startups are hoping to harness the power of anti-cancer drugs to enhance the potency of NK cells. Artiva debuted in June with $78 million in series A financing and a plan to test its NK cells in combination with anti-cancer monoclonal antibodies.

Acepodia was founded in 2016 by Patrick Yang, Ph.D., a former exec at CAR-T maker Juno Therapeutics, and is now pursuing NK cell therapies conjugated with therapeutic antibodies. Its lead program combines an NK cell with a monoclonal antibody targeting HER2, an antigen found in breast, gastric and other solid tumor types.

Meanwhile, Gilead is in on the NK act as well, building on its strong position in CAR-T therapies. In April, it penned a partnership with Australia-based oNKo-innate, which is using genomic screening to find pathways that boost the ability of NK cells to combat tumors.

Were looking at the intracellular pathways that are either inhibiting or preventing the optimal expansion and development of an NK therapy, Peter Emtage, Ph.D., Gilead's senior vice president of cell therapy research, said in an interview. We need to identify the optimal structure that will make that NK cell the best it can be.

Gilead envisions the field of cell therapy moving beyond the single-modality approach and towarda more holistic view of treating cancer, Emtage said. We have to start thinking outside of the classical Yescarta CD19 therapy and start looking for methods to harness the endogenous immune system, so that we can make a concerted impact on the tumor.

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From natural killer cells to off-the-shelf CAR-Ts, biopharma targets cancer with next-gen cell therapies - FierceBiotech

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the biologic therapeutics market and it is poised to grow by $ 190.94 bn during 2020-2024, progressing at a CAGR of almost 12% during the forecast period. The report offers an up-to-date analysis regarding the current market scenario, latest trends and drivers, and the overall market environment.

Although the COVID-19 pandemic continues to transform the growth of various industries, the immediate impact of the outbreak is varied. While a few industries will register a drop in demand, numerous others will continue to remain unscathed and show promising growth opportunities. Technavios in-depth research has all your needs covered as our research reports include all foreseeable market scenarios, including pre- & post-COVID-19 analysis. We offer $1000 worth of FREE customization

The market is concentrated, and the degree of concentration will accelerate during the forecast period. AbbVie Inc., AstraZeneca Plc, Bristol-Myers Squibb Co., F. Hoffmann-La Roche Ltd., GlaxoSmithKline Plc, Johnson & Johnson, Merck & Co. Inc., Novartis AG, Pfizer Inc., and Sanofi are some of the major market participants. To make the most of the opportunities, market vendors should focus more on the growth prospects in the fast-growing segments, while maintaining their positions in the slow-growing segments.

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Strong R&D pipeline has been instrumental in driving the growth of the market. However, the high cost of biologics might hamper the market growth.

Technavio's custom research reports offer detailed insights on the impact of COVID-19 at an industry level, a regional level, and subsequent supply chain operations. This customized report will also help clients keep up with new product launches in direct & indirect COVID-19 related markets, upcoming vaccines and pipeline analysis, and significant developments in vendor operations and government regulations. Download a Free Sample Report on COVID-19 Impacts

Biologic Therapeutics Market 2020-2024: Segmentation

Biologic Therapeutics Market is segmented as below:

Biologic Therapeutics Market 2020-2024: Scope

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources. The biologic therapeutics market report covers the following areas:

This study identifies the introduction of biosimilars as one of the prime reasons driving the biologic therapeutics market growth during the next few years.

Technavio suggests three forecast scenarios (optimistic, probable, and pessimistic) considering the impact of COVID-19. Technavios in-depth research has direct and indirect COVID-19 impacted market research reports.

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Biologic Therapeutics Market 2020-2024: Key Highlights

Table of Contents:

Executive Summary

Market Landscape

Market Sizing

Five Forces Analysis

Market Segmentation by Product

Market segmentation by application

Customer landscape

Geographic Landscape

Vendor Landscape

Vendor Analysis

Appendix

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Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

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COVID-19 Recovery Analysis: Biologic Therapeutics Market | Strong R&D Pipeline to Boost the Market Growth | Technavio - Business Wire

Two years after acquiring the gene therapy company AveXis, Novartis has renamed it Novartis Gene Therapies to underscore the potential value of developing such treatments for genetic diseases.

This decision was based partly on the success of Zolgensma, originally developed by AveXis, as a gene therapy for spinal muscular atrophy (SMA) that is approved in the U.S., Japan, Europe, and Brazil as an intravenous infusion.

Novartis sees tremendous potential in the future of gene therapy, and weve seen the impact gene therapy can have on so many lives, Vas Narasimhan, CEO of Novartis, in a saidpress release. With the creation of Novartis Gene Therapies, we will continue to advance our gene therapy pipeline for rare genetic diseases, to accelerate the delivery of transformative innovation in areas of high unmet need, and to reimagine medicine for patients all around the world.

Zolgensma targets the underlying cause of SMA, deliveringa working copy of theSMN1gene which is mutated in SMA to motor neurons, the nerve cells that control muscle contraction.

This therapy, the worlds most costly treatment at $2.125 million for one-time use, relies on a genetically engineered virus, called adeno-associated virus (AAV) 9, to transport a workingSMN1 transgene (so-called because its DNA comes from an external source) directly to motor neurons.

In the U.S., the treatment is approved for patients with all SMA types up to the age of 2, given in a single, hourlong intravenous infusion. In Europe, it is approved for those weighing up to 21 kg (about 46 lbs), with a clinicaldiagnosisoftype 1SMA or up to three copies of theSMN2gene.

To date, 600 patients worldwide have been treated with Zolgensma, either through clinical trials, managed access programs, or its commercial availability, Novartis said in its release.

Regularly decisions are expected in Switzerland, Canada, Australia, Argentina, and South Korea this year or in early 2021, it added.

The company has placed a priority on AAV-delivered gene therapies to treat other disorders.The Novartis Gene Therapies research group is reported to be working on treatments for people with Rett syndrome, those with a genetic form of amyotrophic lateral sclerosis (ALS), and withFriedreichs ataxia.

Our patients and their families are the motivation for everything that we do, and under the banner of Novartis Gene Therapies, our dedicated team will continue to create a lifetime of possibilities to people suffering from rare genetic diseases, said David Lennon, president of Novartis Gene Therapies who formerly led AveXis. Becoming Novartis Gene Therapies symbolizes the importance of our gene therapy advances for the future of Novartis and our industry leadership at large.

By unifying the Novartis and AveXis brands, Novartis intends to establish a global presence for Novartis Gene Therapies, Zolgensma, and for potential gene therapies to come.

Total Posts: 85

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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AveXis Now Known as Novartis Gene Therapies, Focus of Continuing Work - SMA News Today

-- Webcast conference call to be held on Monday, Sept. 28, 2020 at 8:30 a.m. Eastern Time --

-- Additional poster presentations at WMS will highlight data from Sareptas RNA and gene therapy programs --

CAMBRIDGE, Mass., Sept. 14, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that new data from its most advanced gene therapy programs will be presented at the WMS25 Virtual Congress, the 25th International Annual Congress of the World Muscle Society, being held Sept. 28 Oct. 2.

Sarepta will host a webcast and conference call on Monday, Sept. 28, 2020 at 8:30 a.m. ET, to discuss the results, which include two-year functional data from Study 101 of SRP-9001 for Duchenne muscular dystrophy and 18-month functional results from Cohort 1 in the study of SRP-9003 for Limb-girdle muscular dystrophy Type 2E.

This will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentationsand will be archived there following the call for one year. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 6793650. Please specify to the operator that you would like to join the "Long-term Functional Data from Sareptas Gene Therapy Programs call.

In total, Sarepta will present 16 abstracts at this years meeting. All posters will be available on-demand throughout the Congress beginning on Monday, Sept. 28 at 7:00 a.m. EST. The full WMS25 Virtual Congress program is available here: https://www.wms2020.com/programme/.

Gene Therapy:

RNA Platform:

Natural history and other presentations:

Presentations will be archived under the events and presentations section of the Sarepta Therapeutics website at http://www.sarepta.comforone year following their presentation at WMS25.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.com

Media: Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

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Long-term functional data from Sarepta Therapeutics' Most Advanced Gene Therapy Programs to be Presented at Upcoming Annual Congress of the World...

LONDON--(BUSINESS WIRE)--The new Soft Tissue Repair Market Research from Technavio indicates Positive and Superior growth in the short term as the business impact of COVID-19 spreads.

"One of the primary growth drivers for this market is the rising incidence of accidental injuries, says a senior analyst for Healthcare at Technavio. As the markets recover Technavio expects the soft tissue repair market size to grow by USD 10.44 million during the period 2020-2024."

Get detailed insights on COVID-19 pandemic Crisis and Recovery analysis of soft tissue repair market. Download free report sample

Soft Tissue Repair Segment Highlights for 2020

Regional Analysis

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Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

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COVID-19, Soft Tissue Repair, Tissue Repair, Soft Tissue Fixation Devices and Accessories, Cell Therapy, and Tissue Scaffold

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New Soft Tissue Repair Market Research Highlights Recovery Path for Businesses from COVID-19 based on Cell Therapy and Tissue scaffold Products |...

Sep. 14 /CSRwire/ - CRB is proud to announce that theAmicus TherapeuticsResearch and Gene Therapy Center facility has wonEngineering News-Record(ENR) MidAtlantics 2020 Best Projects (Award of Merit) in the Healthcarecategory.

Two panels of industry judges reviewed more than 80 projects located throughout the region, including Delaware, Maryland, Pennsylvania, Virginia, West Virginia, and the District of Columbia. Projects were scored on the ability of the project team to overcome challenges, contribution to the industry and community, project innovation, safety, and construction/designquality.

Amicus is a biopharmaceutical company that discovers and develops medicines for rare diseases. Its global research and gene therapy center of excellence consolidates Amicus five research departments into a unified space on the top two floors of a 14-story tower. The research workplace is the convergence of technical laboratories, office, and amenity spaces. The design creates spaces that connect science, people andpurpose.

Our team provided architectural, engineering, and lab design to realize our clients vision: a standout research facility that would attract and retain top researchers, promote collaboration, and break down barriers between employees to rapidly advance these potentially life-saving therapies through the drugpipeline.

To read more about the ENR 2020 Best Projects,click here.

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Amicus Therapeutics Wins 2020 ENR Regional Award for Best Project - CSRwire.com

VANCOUVER, B.C., Sept. 14, 2020 /PRNewswire/ -- The Global Femtech Marketis expected to reach USD 60.01 Billion by 2027, according to a new report by Emergen Research. Demand for the femtech industry is motivated mainly by the growing burden of both chronic and infectious diseases among the world's female population. An increase in the number of health problems relating to women would stimulate competition for technologically innovative healthcare solutions. Growing women's emphasis on reproductive health and sexual empowerment in developing economies would further encourage development in the industry.

Increasing awareness among women of the detection and management of early illness as part of the patient care program would improve the market outlook. Various efforts by government and other agencies in developing countries to raise awareness of women's health would accelerate the development of the industry. Furthermore, an increasing tendency towards daily preventive care check-ups, as well as the advancement of user-friendly technology to track individual health problems, may prove beneficial to the developments in the women's health industry.

While more and more people today choose to be more transparent about their health concerns and treatment, in some of the lesser developed regions, women's health issues remain stigmatized. For these places, Femtech applications are likely to be favored because the scanning is less invasive and more secure. Increasing population growth is related to being one of the main factors behind the case.

Request free sample of this research report at: https://www.emergenresearch.com/request-sample/37

Key Highlights From The Report

Read more at: https://www.emergenresearch.com/industry-report/femtech-market

For the purpose of this report, Emergen Research has segmented into the Global Femtech Market on the basis of type, end-use, application, and region:

Type Outlook (Revenue: USD Billion; 2017-2027)

End Use Outlook (Revenue: USD Billion; 2017-2027)

Application Outlook (Revenue: USD Billion; 2017-2027)

Regional Outlook (Revenue, USD Billion; 2017-2027)

Find more research reports on healthcare and pharmaceuticals industry, by Emergen Research:

Regenerative Medicine MarketRegenerative Medicine Market By Product (Tools, Therapeutics), By Therapeutic Category (Musculoskeletal, Dermatology, Immunology & Inflammation, Cardiovascular), and By Applications (Wound Care, Musculoskeletal Disorders, Ocular Disorders), Forecasts to 2027

Next-Generation Sequencing MarketNext-Generation Sequencing Market by Technology (Whole Exome, Whole Genome, Others), By Workflow (Sequencing, Pre-Sequencing, Others), By Application (Consumer Genomics, HLA Typing, Others) and By End-Use (Academic, Clinical, Others), Forecasts to 2027

RFID in Healthcare MarketBy Product (Tags, Systems & Software) and By Application (Asset Tracking, Patient Tracking, Pharmaceutical Tracking, Blood Tracking, Others), Forecasts to 2027

Non-Invasive Prenatal Testing MarketBy Method, By End-Use, By Application, By Region, Forecasts to 2017-2027

Viral Vector and Plasmid Manufacturing MarketBy Vector Type (Retrovirus, Adenovirus, Others), By Workflow (Upstream, Downstream), By Disease (Cancer, Genetic Disorders, Others), By Application (Gene Therapy, Retailers) and By End-User, Forecasts to 2027

Interoperability Solutions in Healthcare MarketBy Level (Foundational, Structural, Semantic), By Product Type (Services, Solutions), and By Application (Diagnostics, Treatments, Others), Forecasts to 2027

About Emergen Research

At Emergen Research, we believe in advancing with technology. We are a growing market research and strategy consulting company with an exhaustive knowledge base of cutting-edge and potentially market-disrupting technologies that are predicted to become more prevalent in the coming decade.

With market-leading insights and an in-depth understanding of leading and niche technologies, our solutions address the most pertinent questions for your business needs. A major technological shift has been witnessed towards creating a 'Circular Economy,' fuelled by factors, such as the increased adoption of bio-based materials, along with other methods for achieving carbon neutrality. We are conversant in technologies, viz., Artificial Intelligence (AI), Augmented Reality (AR), Virtual Reality (VR), Robotic Process Automation (RPA), Smart Manufacturing, Internet of Things (IoT), Big Data Analytics, Machine learning, Nanotechnology, Edge Computing, Blockchain Technology, Cloud Computing, Vehicle Electrification, Advanced Maintenance Analytics, and Predictive Maintenance, among other prevalent and emergent technologies.

Contact Us:Eric LeeCorporate Sales SpecialistEmergen Research | Web: https://www.emergenresearch.comE-mail: [emailprotected]

Read full Press Release at :https://www.emergenresearch.com/press-release/global-femtech-market

SOURCE Emergen Research

Original post:
Femtech Market to Reach USD 60.01 Billion By 2027 | CAGR of 15.6%: Emergen Research - PRNewswire

September 14, 2020 NIH contracts to explore radiation countermeasures, Alzheimers medication, and convalescent plasma, Mission Bio gets $70M for single cell multi-omics technology, and more.

$300M: Series D for New Materials

Science and material innovation company Zymergen (Emeryville, Calif.) has announced $300M in new investment to accelerate its delivery of revolutionary, high performance materials. The investment includes initial Series D funding led by Baillie Gifford, joined by Baron Capital Group and one of the worlds largest sovereign wealth funds, as well as additional growth financing from Perceptive Advisors. A number of current investors are also returning, and Zymergen expects to raise additional capital in Q4 as part of a Series D round. Zymergen develops and launches better performing products more sustainably and for a fraction of the cost and time that it typically takes using incumbent techniques. A unique combination of biology, chemistry, machine learning and lab automation underpins Zymergens powerful proprietary platform, driving the companys ability to discover, design and commercialize never-before-seen materials.

$270M: Series C for Multiomics Blood Testing for Cancer

Freenome (South San Francisco, Calif.) announced an oversubscribed $270M Series C financing to accelerate the PREEMPT CRC clinical trial for Freenomes blood test for colorectal cancer screening and precancerous lesion detection, advance a pipeline of blood tests for both the early detection and early intervention of additional cancers, and continue building the companys proprietary multiomics platform. PREEMPT CRC is an FDA registrational study launched in May 2020 to support approval by the U.S. Food and Drug Administration (FDA) for the first front-line blood test to help the 45 million people who are currently not up-to-date on colorectal cancer screening guidelines in the U.S. The Series C financing was led by new investor Bain Capital Life Sciences and existing investor Perceptive Advisors. They were joined by a group of other new investors, including Fidelity Management & Research Company, LLC, Janus Henderson Investors, Farallon Capital Management, Rock Springs Capital, Cormorant Asset Management, EcoR1 Capital, LLC, Catalio Capital Management, and the Colorectal Cancer Alliance.

$100M: Radiation Countermeasures

SRI International (Menlo Park, Calif.) has been awarded a seven-year contract of up to $100 million from the National Institutes of Healths (NIH) National Institute of Allergy and Infectious Diseases (NIAID) to support the research and development of radiation/nuclear medical countermeasures (MCMs). Under the contract, SRI Biosciences, a division of SRI International, will provide facilities, expertise, and capabilities to advance the development of MCMs for the mitigation or treatment of acute radiation syndromes as well as the treatment of delayed effects from acute radiation exposure (ARS/DEARE) and internal radionuclide contamination. SRI researchers will also support NIAIDs ongoing biodosimetry efforts.

$74M: Five-Year NIH Grant To Test Alzheimers Meds

Alzheon (Framingham, Mass.) has announced today that the U.S. National Institute on Aging (NIA) has awarded the company a grant expected to total $47 million over 5 years to support a Phase 3 clinical study with ALZ-801, an oral agent that blocks the formation of neurotoxic soluble amyloid oligomers. The study will enroll Early AD patients, who have two copies of the apolipoprotein e4 allele (APOE4/4). AD patients with this genetic profile have been shown to have a higher risk of rapid disease progression and to be responsive to agents targeting pathogenic amyloid oligomers.

$70M: Series C for Single Cell Multi-Omics Technology

Mission Bio (South San Francisco, Calif.) announced $70 million in its Series C financing led by Novo Growth, the growth equity arm of Novo Holdings. Soleus Capital also joins the round, along with earlier investors Mayfield, Cota, and Agilent, bringing the companys total funding to more than $120 million. The funds will be used to scale its single-cell multi-omics technology, the Tapestri Platform, to expand the companys reach in more effective clinical trials for novel cancer treatments, as well as characterization for cell and gene therapy.

$50M: Decentralized Research

THREAD (Durhan, NC) received additional capital commitment of up to $50 million from strategic health care investors, Water Street Healthcare Partners and JLL Partners. The companys latest capital infusion builds on a year of significant growth and investments in its platform and services to advance decentralized research approaches for large-scale, Phase Ib - IV global clinical trials. To date, THREAD has supported more than 100 decentralized studies. The company will continue to invest in expanding and enhancing its innovative platform and supporting services.

$40M: Decentralized Clinical Trials

Science 37 (Los Angeles) has closed an oversubscribed $40 million funding round. Existing investors Lux Capital, Redmile Group, and PPD, Inc. led the round, and are joined by existing investors Novartis, Amgen, Sanofi Ventures, GV, and Glynn Capital. Notable new investors include LifeSci Ventures and Mubadala Ventures. Science 37 will use the new capital to support its rapid growth, expand its technology platform, and accelerate its global expansion effortsfurther strengthening its ability to help sponsors execute decentralized trials and enable patients to participate in trials from anywhere, without the burden of traveling to a traditional clinical site.

$34M: NIH Grant for Nationwide Study of Convalescent Plasma

Vanderbilt University Medical Center (Nashville, Tenn.) has been awarded a one-year, $34-million grant by the National Center for Advancing Translational Sciences, part of the National Institutes of Health, to conduct a nationwide study of "convalescent plasma" as a treatment for COVID-19. The randomized, controlled trial will test whether infusions of plasma, the liquid part of blood collected from COVID-19 survivors, can help other hospitalized patients with COVID-19. The study will recruit 1,000 participants in approximately 51 sites across the country. The goal is to complete enrollment by October 31, and report results by November.

$25.5M: Series B for Healthcare Interoperability

Bridge Connector (Nashville, Tenn.), an interoperability company changing the way health care communicates, today announced it has raised $25.5 million in Series B financing. The latest round, led by Axioma Ventures, was joined by all existing investors, including veteran investor Jeff Vinick, and brings Bridge Connectors total funding to over $45 million. After achieving over 1000% year-over-year growth in 2019, the investment will further support the companys increasing market share in health care interoperability and growth of Destinations, a new integration-platform-as-a-service (iPaaS) that connects health data systems using use-case-based interoperability blueprints to speed integrations with major vendors. Bridge Connector provides a suite of vendor-agnostic integration solutions and a full-service delivery model, helping health care vendors, providers, and payers more easily share data between disparate systems, such as electronic health records (EHRs) or patient engagement solutions.

$12M: Cloud Clinical Data Platform

Castor (Hoboken, New Jersey) has announced a $12 million funding round led by Two Sigma Ventures with participation from Hambrecht Ducera Growth Ventures and existing investor INKEF Capital. Castors clinical data platform that simplifies the clinical trial process, from recruitment to analysis, for researchers globally. Its used by more than 50,000 users across academia and commercial research, powering more than 4,000 studies with more than 2,000,000 enrolled patients across 90 countries. 192 medical device, biotech, and pharmaceutical companies and contract research organizations (CROs) are using Castors platform. Castor made its platform freely available for all non-profit COVID-19 research starting in February.

$11.8M: Series A for Digital Sedation

Oncomfort (Brussells), the Belgian inventor of and leader in Digital Sedationa method for relieving patients' pain and anxiety without medicationhas completed a 10 million Series A funding round co-led by two prominent institutional investors: Debiopharm and Crdit Mutuel Innovation. Oncomfort plans to use this investment to develop its innovative digital therapy solutions and accelerate its international expansion in Europe and the US. Founded in 2017, Oncomfort invented Digital Sedation, a completely new method for relieving patients' pain and anxiety through treatment with clinically proven sessions of therapeutic Virtual Reality. Since the launch of the Sedakit for Digital Sedation in Benelux and France in June 2019, over 30,000 patients have been treated and have had their pain and anxiety relieved before, during, and after interventions in many clinical fields such as anesthesia, oncology, interventional radiology and cardiology, as well as pediatrics.

$9.94M: Five Year NIH Grant for Rare Genetic Diseases

Baylor College of Medicine (Houston, Tex.) has received a five-year, $9.94 million grant from the National Institutes of Health for the new Center for Precision Medicine Models to facilitate the study of rare genetic diseases. The center will use precision animal models of a patients or group of patients specific genetic variation and study why the change causes disease and how the disease can be treated. The center will take nominations for genetic variants to model from patients, patient groups, clinicians, researchers, and NIH-funded consortiums like the Undiagnosed Diseases Network and the Centers for Mendelian Genomics. An accepted disease-causing genetic variant must previously be identified in order to be considered for modeling. After a case is submitted, the centers clinical and bioinformatics teams will independently assess the genetic variant for likelihood of causing disease. Next, the modeling team will decide if a precision model organism can be produced for that particular genetic change and whether appropriate resources are available within the center to study the disease. A final decision about whether to select the case will be made based on likely clinical benefit to the patient.

$3M: Series of Grants from Society to Improve Diagnosis in Medicine

The Society to Improve Diagnosis in Medicine (SIDM) is issuing the first of three grant rounds, totaling $3 million, as part of the organizations DxQI Seed Grant Program. The initial awards are up to $50,000 for 17 organizations testing interventions to improve the quality, accuracy, and timeliness of diagnoses. The 17 grantees awarded in this round are: Advocate Aurora Health; Atrium Health; The Atrium Health Levine Childrens Hospital; Beth Israel Deaconess Medical Center; Brigham Health/Brigham and Womens Hospital; The Johns Hopkins University School of Medicine; Kaiser Permanente East Bay; Maine Medical Center; McMaster University; MedStar Health; Northwell Health; Northwestern Memorial HealthCare; Tufts Medical Center and Floating Hospital for Children; The University of Michigan and Hurley Medical Center; The University of Texas Health Science Center at San Antonio & University Health System; The University of Pittsburgh School of Medicine, and The Veterans Education and Research Association of Michigan and VA Ann Arbor Healthcare System.

$1.5M: Regulatory Information Management Solutions

Rimsys (Pittsburgh, Penn.) closed a $1.5 million investment round, led by Allos Ventures. The financing round will support Rimsys penetration of the MedTech Regulatory Information Management market through planned expansions of its product offering, sales, and marketing execution. Rimsys solution seamlessly integrates with MedTech manufacturers existing quality management systems, product lifecycle management systems, and sales and distribution software systems. Rimsys robust digital platform, with its intuitive user interface and global intelligence, enables its customers to meet market entrance requirements and grow internationally. John McIlwraith, managing director at Allos Ventures will join Rimsys board of directors.

$1.3M: AI for Drug Repurposing for COVID-19

Relation Therapeutics (London), a drug development company driven by data science and machine learning (ML), has announced Project RE, which will apply Relation Therapeutics and its partners technology to the identification of repurposed drug combinations as potential therapeutic candidates for COVID-19. Funding for Project RE is provided by a $1.3 million grant from the Bill & Melinda Gates Foundation to Relation Therapeutics. Project RE will focus on finding therapies to tackle viral entry and replication and is co-led between Mila (Quebec AI Institute) and Relation Therapeutics, with the overall scientific direction by Mila founder Professor Yoshua Bengio. The project will also create a platform to develop therapies that appropriately modulate the immune response through distinct stages of infection with oversight from Relations Chief Medical Officer, Dr. David Roblin.

$356,000: AI for Microbiome-Disease Link

BioLizard (Ghent, Belgium) received a 300,000 research grant from Flanders Innovation & Entrepreneurship (VLAIO) to leverage its world-leading expertise in bioinformatics, AI and machine learning, to establish the causal link between the human microbiome and disease. Collecting data from the broadest set of microbes, patient background and interactions, BioLizard is building a framework using state-of-the-art bioinformatics and AI tools to evaluate causal links in the human microbiome, purely from data. This will allow evaluation of whether microbe A causes disease B or if theyre only distantly related, thereby requiring a refining of focus.

$100,000: COVID-19 Fast Grant for Enzymes Critical to SARS-CoV-2 Replication

Dr. Christopher Basler, professor and director of the Center for Microbial Pathogenesis in the Institute for Biomedical Sciences at Georgia State University, has received a $100,000 COVID-19 Fast Grant to study enzymes that are critical for the replication of SARS-CoV-2, the virus that causes COVID-19. Basler is exploring several small molecule inhibitors that would block formation of membranes needed for SARS-CoV-2 infection. These include enzymes such as VPS34, long chain fatty acyl-coA synthetase and fatty acid synthase. Drugs that affect cell membranes and lipids are being pursued as treatments for other medical conditions, including cancer, diabetes and obesity. Early data from the Basler lab suggest such drugs might be effective to slow SARS-CoV-2.

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Follow the Money: Alzheimer's Meds, Single Cell Multi-Omics Technology - Bio-IT World

While there is no cure for blindness and macular degeneration, scientists have accelerated the process to find a cure by visualizing the inner workings of the eye and its diseases at the cellular level.

In an effort led by UW Medicine, researchers successfully modified the standard process of optical coherence tomography (OCT) to detect minute changes in response to light in individual photoreceptors in the living eye.

The results were published Sept. 9 in Science Advances.

We have now accelerated the life cycle of vision restoration, said lead author Vimal Prabhu Pandiyan, a ophthalmology researcherat the University of Washington School of Medicine.

The study was fundedin partby the National Eye Institutes Audacious Goals Initiative, which embraces bold ideas in helping people to see better.

The OCT modifications outlined in the study will help researchers who want to test therapiessuch as stem cells or gene therapy to treat retinal disease. They now have the tools to zoom in on the retina to evaluate whether the therapy is working.

Corresponding author Ramkumar Sabesan, a UW assistant research professor of ophthalmology, said the only wayto objectively measure the eye currently is to look at a wide retinal area. Sabesan said researchers currently can attach electrodes on the cornea but it captures a large area with around 1 million cells. Now they are talking about nanometers, or one billionth of a meter a small fraction of the size of a cell, providing orders of magnitude improvement.

Since photoreceptors are the primary cells affected in retinal generation and the target cells of many treatments, noninvasive visualization of their physiology at high resolution is invaluable, the researchers wrote.

Cone photoreceptors are the building blocks of sight, capturinglight and funneling information to the other retinal neurons. They are a key ingredient in how we process images and patterns of light falling on the retina.

Optical coherence tomography has been around since the 1990s. In this study, researchers used OCT with adaptive optics, line-scanning and phase-resolved acquisition to deliver the concept of Thomas Youngs interference to the human eye. With the ability to zoom in on the retina at high speeds, they found that cone photoreceptors deform at the scale of nanometers when they first capture light and begin the process of seeing.

As Sabesan explained: You can imagine a picture that looks visually and structurally normal. But when we interrogate the inner working of the retina at a cellular scale, we may detect a dysfunction sooner than what other modalities can do. A doctor then can prescribe medication to intervene early or follow the time-course of its repair via gene therapy or stem cell therapy in the future.

We will now have a way to see if these therapies are acting in the way they should, Sabesan said.

The study also involved researchers at Stanford University, University of California,Berkeley, and University of California, Riverside.

The study was funded by NIH grants U01EY025501, EY027941, EY029710, EY025501, and P30EY001730; Research to Prevent Blindness Career Development Award; Foundation Fighting Blindness; Murdock Charitable Trust; Burroughs Welcome Fund Careers at the Scientific Interfaces; and Unrestricted grant from the Research to Prevent Blindness.

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Seeing the eye like never before | Newsroom - UW Medicine Newsroom

DUBLIN, Sept. 14, 2020 /PRNewswire/ -- The "17th Annual Report and Survey on Biopharmaceutical Manufacturing Capacity and Production" report has been added to ResearchAndMarkets.com's offering.

The 2020 17th Annual Report and Survey of Biopharmaceutical Manufacturing Capacity and Production is the most recent study of biotherapeutic developers and contract manufacturing organizations' current and projected future capacity and production.

This report's data-rich analysis will help improve your decision-making in biomanufacturing operations, with in-depth analysis of capacity, production trends, benchmarks, and much more.

In-depth analysis and summary of the key survey findings, trends and implications for industry-wide biomanufacturing capacity and biotherapeutic production. Comparison of production by biotherapeutic developers and contract manufacturing organizations. Current and future potential industry bottlenecks.

Key Topics Covered:

CHAPTER 1: INTRODUCTION AND DISCUSSION1-1 Introduction: The Pharmaceutical and Biopharmaceutical Industries1-2 Current Status and Market Trends1-3 Market Potential1-4 Biopharmaceuticals and Biosimilars in the Pipeline1-5 Global Biopharmaceutical and Recombinant Protein/MAb Markets1-6 Biopharmaceutical Markets by Product Class1-7 Animal Derived Products and Biopharmaceuticals1-8 Future Trends in the Biopharmaceutical Industry1-9 Future Biopharmaceutical Market Trends

CHAPTER 2: FUTURE OF BIOPROCESSING: EXPERTS' PERSPECTIVE2-1 Cell and Gene Therapy - Future of Bioprocessing2-2 Continuous Processing - Present and Future Challenges2-3 China and Asia's position in Global Bioproduction2-4 Intersection of Biopharma and Small Pharma: Small Molecule Manufacturing Lessons for a New Industry2-5 Upstream and Downstream Biologics Manufacturing: Mapping the Future Challenges and Trends2-6 Suppliers' Contributions to Bioprocessing Advances2-7 Contract Manufacturing's Contributions to Bioprocessing Advances2-8 Worldwide Biopharmaceutical Manufacturing Capacity Analysis: Growth Continues Across the Board2-9 China's Advances in Global Biopharma and Bioprocessing: A 10-year Projection on Need for Quality Improvements

CHAPTER 3: EMERGING ISSUES IN BIOPHARMACEUTICAL MANUFACTURING3-1 Industry Trends in 20183-2 Budget Issues in 20183-3 Operational Changes3-4 New Bioprocessing Products Development Opportunities in 20183-5 Factors in Biomanufacturing Creating Improvements3-6 Cost-Cutting Actions & Development Timelines (2016 data)3-7 Average Cost per Gram Recombinant Protein3-8 Assay Development3-9 Perfusion Operations and Continuous Bioprocessing Operations Issues3-10 Perfusion Operations and Continuous Bioprocessing Trends3-11 Discussion3-12 Cell and Gene Therapy Platforms3-13 Selecting Bioreactors in New Facilities (2017 data)3-14 Discussion: Industry Trends and Issues

CHAPTER 4: CAPACITY UTILIZATION4-1 Capacity Utilization Trends4-2 Capacity Utilization: CMOs vs. Biotherapeutic Developers4-3 Capacity Utilization: U.S. vs. Western European Manufacturers4-4 Respondents' Current Total Production Capacity4-5 Discussion: Capacity Trends4-6 Range of Titers with mAb Production4-7 Discussion: Capacity and Industry Trends

CHAPTER 5: CURRENT AND FUTURE CAPACITY CONSTRAINTS5-1 Current Capacity Constraints5-2 Expected Capacity Constraints 210 Respondents' Expectations of Capacity Constraints by 20235-3 Factors Impacting Future Production Capacity5-4 Key Areas to Address to Avoid Future Capacity Constraints5-5 Discussion

CHAPTER 6: FUTURE CAPACITY EXPANSIONS6-1 Planned Future Capacity Expansions

CHAPTER 7: OUTSOURCING TRENDS IN BIOPHARMACEUTICAL MANUFACTURING7-1 Current Outsourcing by Production System7-2 Future Outsourcing7-3 Outsourced Activities in Biopharmaceutical Manufacturing7-4 Critical Outsourcing Issues7-5 CMOs' Problems with Their Clients7-6 Country Selections for International Outsourcing (Off-shoring) of Biomanufacturing7-7 Offshoring Trends7-8 5-Year Projection for Percentages of Biomanufacturing International Outsourcing/Off-shoring

CHAPTER 8: DISPOSABLES AND SINGLE-USE SYSTEMS IN BIOPHARMACEUTICAL MANUFACTURING8-1 Use of Disposables and Single-Use Systems8-2 Leachables and Extractables8-3 Reasons for Increasing Use of Disposables & Single-Use Systems 3268-4 Factors That May Restrict Use of Disposables8-5 Suppliers' Expectations for Standards Setting Bodies8-6 Need for Single-use Sensors, and Bioreactor Attributes8-7 Satisfaction with Single-use Device Vendors8-8 Single Use Operations and Trends8-9 Discussion of Single-use Bioprocessing

CHAPTER 9: DOWNSTREAM PURIFICATION9-1 Impact of Downstream Processing on Capacity9-2 Specic Purication Step Constraints9-3 Downstream Purication Issues9-4 mAb Purication Capacity Estimates9-5 New Downstream Processing Technologies9-6 Improvements to Downstream Operations9-7 Discussion

CHAPTER 10: QUALITY ISSUES, BATCH FAILURES, AND PAT IN BIOPHARMACEUTICAL MANUFACTURING10-1 Quality Initiative Implementation10-2 Hurdles to Implementing Process Analytical Technology10-3 Batch Failure Frequency in Biopharmaceutical Manufacturing10-4 Primary Cause of Batch Failures, Percentages of Failures10-5 Quality Problems in BioManufacturing Attributed to Vendors10-6 Discussion

CHAPTER 11: HIRING, EMPLOYMENT GROWTH, AND TRAINING IN BIOPHARMACEUTICAL MANUFACTURING11-1 Hiring Trends11-2 Hiring in 2023: 5-year Trends11-3 Hiring Challenges Today11-4 Training in Biopharmaceutical Manufacturing11-5 Intersection of Biopharma and Small Pharma11-6 Discussion

CHAPTER 12: New Methods: Continuous and Process Intensification, Cell and Gen12-1 PerfusionAreas of Involvement12-2 Trends in Fill-Finish and Related Bioprocessing Capacity12-3 Current Fill-Finish Trends12-4 Discussion

CHAPTER 13: SUPPLIERS TO BIOPHARMACEUTICAL MANUFACTURING AND LIFE SCIENCES13-1 Demographics13-2 Growth Rate of Sales by Suppliers13-4 Budget Issues and Problems Faced by Industry Suppliers13-5 Cost Cutting Actions by Vendors13-6 Problems Clients Have with Their Vendors13-7 Vendor Expansion Plans13-8 New Technology Areas in Development by Vendors13-9 Sales Staff Training 502 Days of Training Provided13-10 Biopharma Vendors' Financial Outlook for 201813-11 Discussion of Biopharma Suppliers

For more information about this report visit https://www.researchandmarkets.com/r/3bdm10

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

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Global Bio-pharmaceutical Manufacturing Capacity and Production Report 2020: Comparison of Production by Biotherapeutic Developers and Contract...

Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer

Experienced biotech executive to lead the next phase of growth

14 September 2020

LONDON, Silence Therapeutics plc, AIM:SLN and Nasdaq: SLN (Silence or the Company), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, today announces the appointment of Mark Rothera as President and Chief Executive Officer (CEO) and Board member, effective immediately. Iain Ross, who has been Executive Chairman since December 2019, has today assumed his previous position of Non-Executive Chairman.

Mr. Rothera brings more than 30 years of experience in the biopharmaceutical industry, with a strong record of commercial and operational leadership, including driving the successful build of multiple biotech companies, predominantly in the field of rare or specialty diseases. Prior to joining Silence, Mr. Rothera served as CEO of Orchard Therapeutics (Orchard), where he oversaw its transformation from a small U.K.-based, privately held company with two clinical-stage programmes into a leading gene therapy company with seven clinical-stage programmes and fully integrated capabilities. Under his leadership, Orchard completed an initial public offering of American Depositary Shares on the Nasdaq Global Market and during his tenure that company secured more than $600 million in financing and grew from a market capitalization of $250 million to more than $1.7 billion at its peak.

Prior to Orchard, Mr. Rothera served as Chief Commercial Officer of PTC Therapeutics (PTC), where he helped transition that company from a privately held R&D biotechnology company to a publicly traded, commercial-stage company with a global footprint, including the successful launch of two rare disease therapies. He also previously served as Global President of Aegerion Pharmaceuticals Inc. and Vice President and General Manager of commercial operations at Shire Human Genetic Therapies for Europe, Middle East and Africa. Mr. Rothera received an M.A. in Natural Sciences from Cambridge University and an M.B.A. from the European Institute for Business Administration (INSEAD).

Based out of Silences New York City office, Mr. Rothera will lead the continued global expansion of the Company. His appointment follows the completion of Silences Nasdaq listing on 8 September 2020 and aligns with the strategy of increasing the Companys presence in the United States.

Iain Ross, Chairman of Silence Therapeutics plc, said: "On behalf of the Silence Board and the entire Silence team, I welcome Mark to the Company. Following a thorough search, Marks appointment reflects his proven leadership skills and strong track record in growing successful biotechnology companies and building shareholder value. I believe he will now provide the leadership necessary to grow Silence into a leading international biotechnology company built upon our innovative siRNA technology platform, proprietary product pipeline and validating industry partnerships.

On a personal note, and on behalf of the Board, I would like to thank the management team and staff at Silence for their support, hard work and tremendous resilience during the current COVID-19 pandemic and over the past nine months whilst I have been Executive Chairman. The Company has made great strides during this period, and is now in a strong position, both operationally and financially, and ready for Mark to take the helm.

Mark Rothera, President and CEO of Silence Therapeutics plc, added: It is an honour to take the role of leading Silence at this time in the Companys history. I believe the Company is poised to capitalise on its important siRNA technology platform, pipeline and research capabilities built over 18 years, and position itself as a leader in the RNAi field. The Company has made great strides under Iains leadership and I look forward to working with the Board, the management team and Silence employees to build upon this momentum.

Director disclosures

The following information is being disclosed pursuantto Rule 17 and paragraph (g) of Schedule 2 of the AIM Rules for Companies.

Mark Rothera

Full name and age: Mark Andrew Rothera (aged 58)

Current Directorships or Partnerships:Genpharm

Previous Directorships or Partnerships in the last 5 years:Orchard Therapeutics plcPTC Therapeutics International LimitedAlliance for Regenerative Medicine

No further information in connection with his appointment is required to be disclosed under Schedule Two, paragraph (g) of the AIM Rules for Companies.

Enquiries:

About Silence TherapeuticsSilence Therapeutics is developing a new generation of medicines by harnessing the bodys natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet medical need. Silences proprietary technology can be used to engineer short interfering ribonucleic acids (siRNAs) that bind specifically to and silence, through the RNAi pathway, almost any gene in the human genome to which siRNA can be delivered. Silences wholly owned product candidates include SLN360 designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of Lipoprotein(a) and SLN124 to address beta-thalassemia and myelodysplastic syndrome. Silence is also developing SLN500 in partnership with Mallinckrodt Pharmaceuticals to reduce the expression of the C3 protein for the treatment of complement pathway-mediated diseases. Silence maintains ongoing research and collaborations with AstraZeneca, Mallinckrodt Pharmaceuticals and Takeda. For more information, please visit: https://www.silence-therapeutics.com/

The person who arranged for the release of this announcement on behalf of the Company was Rob Quinn, Chief Financial Officer.

Forward-Looking StatementsCertain statements made in this announcement are forward-looking statements, including with respect to the Companys clinical and commercial prospects. These forward-looking statements are not historical facts but rather are based on the Company's current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

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Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer - GlobeNewswire

Report Ocean announces the release of Cell and Gene Therapy Market research report. As per Report Ocean, the market is expected to grow at a healthy pace in the coming years. Leading market vendors are focusing on the development of their mergers & acquisitions with the main aim of providing a broad geographical presence to multiple industries. Most players are anticipated to adopt three key business strategies to cement their position in the market, i.e. expanding product portfolio, facilitating product differentiation, and participating in mergers and acquisitions.

Industry challenges together with the latest developments in the technological developments of the Cell and Gene Therapy Market have been elaborated on in the market intelligence report. It also provides a detailed picture of the trends of the changing industry structure and the challenges that are faced by various industry participants. The report elaborates on the major challenges that the participants of the said market could possibly across the globe.

Request Free Sample Report athttps://reportocean.com/industry-verticals/sample-request?report_id=mai52690

The outbreak of Covid-19 has brought in uncertainties and disruptions for the present and future of several businesses across the globe. The pandemic has claimed both lives and livelihoods, there leaving little or no hope till a vaccine for Covid-19 arrives. However, analysts at Report Ocean make a careful and meticulous assessment of the present situation and disruptions caused by the virus in the supply chain to draw estimates, projections and avenues of growth for the Cell and Gene Therapy Market.

The objectives of this study are to define, segment, and project the size of the Cell and Gene Therapy Market based on company, product type, end user and key regions.

Competitive Landscape:

The report also includes several valuable information on the Cell and Gene Therapy Market, derived from various industrial sources. The report studies the competitive environment of the Cell and Gene Therapy Market report is based on company profiles and their efforts on increasing product value and production.

Key parameters which define the competitive landscape of the Cell and Gene Therapy Market:

Revenue and Market Share by Player

Production and Share by Player

Average Price by Player

Base Distribution, Sales Area and Product Type by Player

Concentration Rate

Manufacturing Base

Mergers & Acquisitions, Expansion

Market Segmentation:

The major factors are also being considered while studying the various market segmentation. Some of the key factors are study of demand and supply of Cell and Gene Therapy Market, common interests and market share of the global Cell and Gene Therapy Market market across various geographies.

Geographical Analysis

Geographically, this report is segmented into several key regions, with sales, revenue, market share, and Cell and Gene Therapy Market growth rate in these regions, from 2015 to 2026, covering;

North America (U.S. and Canada)

Europe (UK, Germany, France, Russia, Italy and Rest of Europe)

Asia-Pacific (China, Japan, India, Malaysia, Singapore, Philippines, Indonesia, Thailand, Vietnam)

South America (Brazil, Argentina, Mexico, and Rest of South America)

The Middle East and Africa (Saudi Arabia, United Arab Emirates, Turkey, Egypt, South Africa, Nigeria)

Some of the Major Highlights of TOC covers:

Cell and Gene Therapy Market Production, Revenue (Value), Price Trend by Type

Production and Market Share by Type

Revenue and Market Share by Type

Price by Type

Cell and Gene Therapy Market Analysis by Application

Consumption and Market Share by Application

Cell and Gene Therapy Market Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Region

Production, Consumption, Export, Import by Country

Production, Revenue, Price and Gross Margin

Cell and Gene Therapy Market Manufacturing Analysis

Key Raw Materials Analysis

Market Concentration Rate of Raw Materials

Manufacturing Cost Analysis

Labor Cost Analysis

Manufacturing Cost Structure Analysis

Manufacturing Process Analysis of XYZ

Industrial Chain, Sourcing Strategy and Downstream Buyers

Cell and Gene Therapy Market Industrial Chain Analysis

Raw Materials Sources of Cell and Gene Therapy Market Major Players in 2019

Downstream Buyers

Market Dynamics

Market Drivers

Restraints

Opportunities

Increased Demand in Emerging Markets

Challenges

Porters Five Forces Analysis

Cell and Gene Therapy Market Forecast

Production, Revenue Forecast

Production, Consumption, Export and Import Forecast by Region

Production, Revenue and Price Forecast by Type

Consumption Forecast by Application

Note In order to provide more accurate market forecast, Cell and Gene Therapy Market report will be updated before delivery by considering the impact of COVID-19.

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COVID-19 Impact and Recovery Analysis - Cell and Gene Therapy Market 2020-2026 - The Market Chronicles

Global Exosome Therapeutic Market By Type (Natural Exosomes, Hybrid Exosomes), Source (Dendritic Cells, Mesenchymal Stem Cells, Blood, Milk, Body Fluids, Saliva, Urine Others), Therapy (Immunotherapy, Gene Therapy, Chemotherapy), Transporting Capacity (Bio Macromolecules, Small Molecules), Application (Oncology, Neurology, Metabolic Disorders, Cardiac Disorders, Blood Disorders, Inflammatory Disorders, Gynecology Disorders, Organ Transplantation, Others), Route of administration (Oral, Parenteral), End User (Hospitals, Diagnostic Centers, Research & Academic Institutes), Geography (North America, Europe, Asia-Pacific and Latin America)

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

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Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for an Analyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

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Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process.

The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

The major players covered in the report are evox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global. Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

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Exosome Therapeutic Market (Covid 19 Impact Analysis) Data Highlighting Major Vendors, Promising Regions, Anticipated Growth Forecast To 2027 - Good...

Population

During the first stage (first two rounds of interviews) five focus groups and 36 individual interviews were performed. In the second stage (round three), five focus groups and 21 individual interviews were performed.

A total of 136 individuals participated in the study: 85 (62.5%) women and 51 (37.5%) men; median age was 26 (range 1944) years for women and 29 (range, 1959) years for men; 77 (56.6%) participants had completed secondary school (Table1). Approximately 5% refused to participate in the study mostly citing personal reasons and lack of time.

The result sections are organized to highlight issues in contraception, ZIKV infection and PAC services, respectively.

Most participants knew at least one contraceptive method. The most frequently mentioned were pills, injections and implants, while less frequently mentioned methods were intrauterine devices (IUD), male condoms and surgical sterilisation. Both female and male participants knew about female condoms but lacked detailed information about them. Both men and women said that although they knew they could choose different contraceptive methods, they would like to receive more detailed information on each individual method, specifically regarding their adverse effects and efficacy.

They dont provide advice regarding family planning and they only ask What kind of family planning do you want? without informing the patient about the side effects of the different methods, for example the risks that an IUD can bring in the future, or the risks of the injection, so I end up choosing the method that a friend recommended. (Focus Group, women, Hospital).

Participants generally had a positive attitude towards the use of contraceptive methods. Women and their partners said they did not feel stigmatized for seeking information on methods at the health facilities. Few men acknowledged they did not allow their partners to use contraceptive methods, while others considered contraceptive use should be a shared decision. Despite the knowledge about contraceptive methods and the positive attitude, many participants specified that their children had not been planned. In fact, many women said that they had sought contraceptive methods only after giving birth.

Many participants shared doubts about the effectiveness and the adverse effects associated with contraceptive methods and had concerns on the lack of comfort when using them. Most participants believed that the effectiveness of each contraception method varied with each person. In fact, women thought that although physicians had information on the methods, they may not know how individual womens bodies would react to each method. Many participants provided anecdotes involving the experience of friends or family that supported their perception that any method, even surgical sterilisation, could fail.

This guy had sex with a lady, and she got pregnant, and he said it was not his baby . And then there was a fight at home. When he came to the hospital he got examined and he really was fertile even though he had been operated! Could you just imagine if he had killed that woman! (Focus Group, Women, Clinic).

Women most commonly accepted and used hormonal contraception; indeed, many preferred the three-month injection because it was freely distributed at the public hospital and was easily accessed. Participants main concerns regarding hormonal methods were their efficacy and their association with cancer, infertility, weight gain and skin blotches.

I am scared of implanon [implant], Im honest. No doctor will tell you the truth, they tell you the implanon will cover this or that, but they wont tell you that in the future it will give you cancer. (Focus Group, Women, Clinic).

Pills though had a burdensome daily regimen, were considered a very effective method. Participants had information on morning-after pills but explained that the use of this method was frequently associated with rape. For men, the most popular method were male condoms; however, they would use it for the prevention of sexual transmitted diseases (STI) rather than for contraception. Of note, only a few participants included Zika in the group of diseases that could be prevented through condom. Some male participants were aware of surgical sterilisation (vasectomy), although only very few men were considering having it.

Participants observed that opening hours at health centres were limited and that the distance between their homes and the health facilities was a significant access barrier. Therefore, participants often arrived at the hospital at 4am to secure an appointment. Most of the participants said that the admission process was disorganized and difficult and that waiting times were very long and thought the probable cause of delays was insufficient numbers of staff and lack of administrative organization.

Look, I came here on Sunday, at 9 am and they told me I had to come back at 1pm. I came back at 1pm and they told me they were not open because they were going to disinfect the place. So, I came back on Monday and told me to stay on a line of people, I tried to explain to the girl, so they told me to return in the afternoon. (Interview, Woman, Clinic).

Regarding infrastructure, some participants complained that the toilets were not clean; however, at the last round of survey, a few participants acknowledged there had been some improvements in cleanliness and infrastructure.

Participants considered that counselling services on reproductive health, especially on contraception were inconsistent and insufficient. Those who wanted to use contraception, received information provided at compulsory meetings only. Several participants suggested that health professionals should proactively approach people and give them more information on reproductive health and that counselling service should be extended to all even during the afternoons and evenings.

The service here (public health centre) is not the same as in a private clinic where you have to pay because here there are so many people. The doctors have to see so many people that they cannot have a special relationship with you (Focus Group, Women, Clinic).

Some women felt uncomfortable during the consultations due to lack of privacy. They would prefer female providers.

I dont know why, but with women we have more confidence, though some men are more sensitive sometimes, but for intimacy issues it is better to have a woman [as health personnel]. (Focus Group, Women, Hospital).

Often, single women sought counselling service and information in clinics outside their communities to avoid the embarrassment of being recognised by neighbours or healthcare staff. Moreover, it was reported that, sometimes, confidentiality was ignored by health staff, and this contributed to users mistrust in the services.

You know what Id change? Id make this office private for women who come here for the (DMPA) shot, because you have to pull down your trousers, and you really feel embarrassed, sometimes in presence of three, four, five other people there (Focus Group, Women, Clinic).

Participants stated that they could receive some contraception methods free of charge, including three-month injection, condoms and IUD. However, participants perceived that the condoms freely distributed at health facilities were small and of bad quality; therefore, when needed, they preferred buying at the pharmacies. Those condoms were considered to be inexpensive, of better quality and accessible in nearby vicinity. No improvement in services were perceived by the participants throughout the interview rounds.

Participants were aware that Zika was mainly transmitted by mosquitoes. Participants mentioned that pregnant women could be infected if they had sexual intercourse with an infected person and that this could be avoided using condoms.

I know that (Zika) is transmitted by the mosquito, that is the main thing, and that we have to pay attention if we have the basins, if we have plants and barrels, we have to keep them clean to avoid larvae and mosquitos at our houses (Focus Group, Women, Hospital).

However, from rounds one to three of survey, the number of participants who also knew of the possible sexual transmission of Zika, decreased (in correlation with distance in time with the outbreak).

Participants mentioned that Zika infection was associated with serious risks for pregnant women. Most mentioned that the baby could get sick, die in the womb, have problems in the head and brain; suffer from microcephaly. Fewer mentioned the Guillain-Barr syndrome and malformations. There was confusion among participants about the difference among Dengue, Chikungunya and Zika infections. For them, the three diseases had the same symptoms and consequences in men or in non-pregnant women. This perception didnt change in the different rounds of interviews.

Well, Ive heard that it affects people, pregnant women, and the baby. When someone is infected, they can infect the baby and cause complications (Focus Group, Women, Hospital).

We must be careful, if we are pregnant, we should be especially careful of not getting a bite, because the baby could be affected. (Focus Group, Women, Hospital).

Most frequently mentioned sources of information were television and radio, followed by Facebook and informative posters at health facilities. During round one of data collection, participants recalled having received information through mass or social media on how to avoid or control mosquitos at home and recalled having seen images of babies with microcephaly.

What I know is how to prevent the disease, but not how to take care of oneself once you get the disease; most of all I know about prevention, that includes a clean household and getting rid of all possible mosquito breeding places and that is what they mainly talk about in the news (Focus Group, Women, Hospital).

Health professionals were the most reliable source of information. However, at the last round of survey, at the end of epidemic, fewer participants received information or noticed any brochures or posters at the health facilities.

Information on Zika is only available when there is an outbreak, they give information to prevent the disease. Why is information available only during outbreaks? (Focus Group, Men, Hospital).

Participants mentioned that they knew of some preventive measures mostly related to the vector transmission; such as covering the water tank (called pila) and cleaning it; however, the information on the frequency of cleaning was not clearly communicated.

We should wash well the sink with chlorine, if you see a tire there with water you must empty it, if you have a leak, or containers full of water they have to be emptied, because that is where the mosquito comes from, wash all the ditches, everything has to be cleaned as much as possible (Focus Group, Women, Hospital).

They knew to use repellents, mosquito nets and coils, and cut tall grass and bushes. Less frequent practiced methods were covering buckets, cleaning gutters, or using a fan to repel mosquitos. Aside from the measures they knew of, participants explained they cleaned their house, and used larvicide and bleach in water tanks. However, many participants were against burning mosquito-repelling incense indoors because the smoke caused cough. Of note, by the third round of interviews, participants stressed that actions performed by public and technical teams in the prevention of Zika in neighbourhoods had ceased.

Upon suspicion of Zika, few participants sought care at a health centre and were referred to a hospital to confirm the diagnosis. However, not everyone attended a health centre; some were diagnosed by a relative who knew of the disease and its symptoms. Moreover, these patients acknowledge they were self-medicated with acetaminophen and liquids, because they knew how physicians treated other cases of Zika infection.

The process for women receiving PAC services at the Gynaecology and Obstetrics Emergency Room at the hospital is described in Fig.2.

Trajectory of women treated for post abortion care at the Gynaecology and Obstetrics Emergency Room and areas for improvement, Hospital, Tegucigalpa, Honduras, 2018

Women seek emergency obstetric care because either they are referred by a health centre or hospital to manage symptoms such as bleeding or pain; or because they are following up treatment with the drug misoprostol (A). Someone (partner, relative, etc.) may or may not accompany them to hospital (B). Those accompanying the women are not allowed inside the premises of the hospital. At the entrance of emergency gate, women are searched by security personnel and then escorted to a waiting area; those who are not ambulatory are assisted by hospital assistants in wheelchairs. In the waiting area, medical students interview the patients and refer them to specific services. Women needing PAC services are admitted to the Gynaecology and Obstetrics Emergency Room (C). Women in emergency are attended round the clock and those with suspected pregnancy are offered pregnancy test (rapid test).

Physicians register patients data and this registry is completed and collected daily by the statistics department. Misoprostol is prescribed and ultrasound is performed on all women. Patients under observation receive family planning counselling in the same room.

Women when referred, are warned that reaching hospital could take them up to two hours, or even more if transportation was unavailable. Participants complained that no other public hospitals offered PAC and highlighted the need for these services to be closer to their homes.

I decided to come today because I was bleeding so much, I fainted, had cold sweats, nausea and my body was shaking (Interview, Woman with MVA procedure, Hospital).

Women had to cover the transportation cost to the only hospital that offered PAC services in the city, and had to pay 200 Lempiras (approx. US$8) per treatment. However, it could reduce to 50 Lempiras (approx. US$2) if social services office certified that the patient could not afford the full fee. Moreover, the hospital also required the patients to bring a gallon of water to clean the medical equipment.

Some patients had to pay for their medication and most women paid for a rapid pregnancy test. It was mentioned that misoprostol, anti-inflammatory agents and analgesics were not freely available; instead, patients had to buy them out-of-pocket. Many times, these payments for specific treatments were out of reach of patients.

They asked me to buy a pregnancy test. Afterwards when they were about to perform the MVA they told me that I had to pay 200 Lempiras and purchase a container of water. (Interview, Woman, Hospital).

By the final round of this survey, following discussion of research team with health authorities, this requirement and the fee was eliminated. The hospital administration purchased a water filter system for emergency room and medical staff with research studys funds.

Participants complained that male partners and relatives were not allowed into the hospital and had to wait long hours without any protection from the weather; slept on the floor at night and received no updates on the health of their partners from physicians or nurses.

The cleaners just started to throw water on the floor, they didnt mind if I had my clothes there or if I was sitting there. I was sitting there, after two nights without any sleep, I fell asleep on my bags on the floor and when I woke up because someone told me to, they were throwing water at me (Interview, Man, Hospital).

Women seeking for emergency care remained unaccompanied in the waiting area and during the procedure.

Through emergency, I got her [wife] in and the guard treated me badly because I came with her and he told me that I couldnt go in and I told him that it was an emergency because she was my wife and she was really sick and had problems. (Focus Group, Men, Hospital).

A very common complaint during the first round was the lack of privacy for the women who sought assistance or during a procedure, including the lack of sufficient beds or rooms. Most participants complained that the admission process was disorganised, difficult and time-consuming, the public toilets were unclean, and medications were often lacking.

The reproductive health counselling was only given after MVA procedures were performed, with no privacy and in the presence of other patients. Over time, participants acknowledged some improvements specially ward cleanliness.

Many participants agreed they had been mistreated by physicians and nurses when seeking PAC or emergency care. Some of the women felt that they were laughed at, reprimanded and ignored by both physicians and nurses.

Today, I was undergoing the MVA, the doctor was doing the procedure with people standing by the door, there was a student asking one thing and another and doctors phone kept ringing. (Interview, Woman, Hospital).

The most common complaint was the staffs lack of empathy and indifference to their health situation.

My mom asked for information because when I was in the ward and there were many girls who had had an abortion and they told me you have to rest, you cant crouch, you cant travel. So, I told mom to get information. The first time I had an abortion, they didnt explain anything to me, they just told me go away and take care!, just that. So, my mom asked a nurse and she just said I had to rest, just that (Focus Group, Women, Hospital).

Accompanying male partners and female patients described instances of mistreatment by the security staff when being admitted to the hospital. The male partners mentioned that they did not receive information about the patients health and procedures during in-hospital stay, the remarked they feel mistreated and that produced distress and fear.

My partner was being accompanied by her sister. I could not get in touch with my partner, I could not even call her on the cell phone. Then, I could talk to her sister, she called me, she was crying and told me that my babys heart was not beating and the doctors had performed an ultrasound and had seen my baby was dead and that they going to do another ultrasound (Interview, Man, Hospital).

Some women complained of having to wait for long hours in pain, only to receive what they perceived as unsatisfactory care. Some women who were prescribed misoprostol felt that they had been discharged without adequate information as they felt staff did not sufficiently explain the procedures to them. Some patients reported that physicians did not treat and even ignored the symptoms they complained about, in particular, pain. After unsuccessful treatment in their own home, some participants had to return to hospital for treatment.

It is a horrible sensation, because one feels as if they are pulling the uterus and the doctors only tell us to cooperate but that is, it is impossible to stay relaxed, when one is going through something so painful. (Interview, Woman, Hospital).

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We know that dogs can be diagnosed with brain tumours however this is the first time we have reported on positive results in a preliminary brain cancer study for the treatment of dogs. Animal Life Sciences, Inc. (ALS), a pharmaceutical and nutritional development company announced that a comparative oncology trial being conducted using ALS101( a combination of two brain cancer drug candidates ALS licensed for use in animals) is showing promise in dogs suffering from malignant gliomas, including glioblastoma. Dogs suffer from these same types of aggressive tumours and treatment options, much like those for humans, are extremely limited. ALS have seen regression of tumour in a significant population of dogs treated in a formal Phase I clinical trial and while the tumour did not quite disappear, in some cases it shrank more than 95 percent and the animals lived longer.

News from Sweden now where a study has shown that one year after the diagnosis of low-grade malignant brain tumour just under three people in ten were in full-time employment. For this young patient group, returning to work is a key health factor however another year later, the proportion remained below half. For those of you unsure about the characterisation of a low grade, malignant tumour it is defined in this study as a low-grade glioma which is incurable and grows slowly but, thanks to modern treatments, its survival expectancy has successively increased. Given the patients' low age -- averaging 40 years when they fell ill -- their work capacity is seen as an especially important factor in quality of life. Being able to work again is, for many, a crucial aspect of returning to a normal life.

We have explained about orphan drug diagnosis in these updates before but to recap, in the US the Orphan Drug Act (ODA) provides for granting special status to a drug to treat a rare disease or condition. Benefits for a pharma company of having a drug given orphan designation include tax credits of 50% off the clinical drug testing cost awarded upon approval and eligibility for market exclusivity for 7 years post-approval.

Plus Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for its lead investigational drug, Rhenium NanoLiposomes (RNL) for the treatment of patients with recurrent glioblastoma. RNL is designed to safely, effectively, and conveniently deliver a very high dose of radiation - up to 25 times greater concentration than currently used external beam radiation therapy - directly into the brain tumour for maximum effect. Dr. Marc Hedrick, President and Chief Executive Officer of Plus Therapeutics said: We believe RNL has the potential to prolong survival for patients with malignant brain tumours and that of other difficult to treat radiosensitive tumours.

In the fight against cancer, scientists have long grappled with the ambiguous nature of stem cells. GBM tumours consist of stem cells which have the ability to self-renew making these tumours notoriously hard to treat with targeted radiation therapy and difficult to permanently remove through surgery. However, could gene therapy provide a potential breakthrough in brain cancer treatment and put the invincibility of tumour stem cells into question. This recent paper details a new technique of sensitizing stem cells to radiation therapy, thereby increasing the therapys efficacy.

You may find this overview of dendritic cell vaccines for brain tumours is helpful reading before moving onto this paperOnce, Twice, Three Times a Finding: Reproducibility of Dendritic Cell Vaccine Trials Targeting Cytomegalovirus in Glioblastoma. John Sampson, M.D., Ph.D. from Duke University a leader in cancer immunotherapy for brain tumours commented on the paper saying These study results not only advance our understanding of a virus role in cancer, but they also signal tremendous hope to patients and their families suffering from this devastating disease

An optimistic-sounding end to this weeks worldwide research update.

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Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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RESEARCH TRIANGLE PARK Dont call it AveXis anymore. Its now Novartis Gene Therapies.

Novartis has announced the renaming of its previously acquired AveXis gene therapy arm because of the growing importance of gene therapy to building a leading, focused medicines company with advanced therapy platforms.

AveXis, headquartered in Bannockburn, Illinois, was purchased in 2018 for $8.7 billion by the Swiss drugmakerNovartis.

The company announced early last year that it would put $60 million more into its then-new $55 million Durham County manufacturing facility, doubling the workforce here from 200 to 400. Now, according to a Novartis spokesperson, there are more than 400 people working at the Durham site, and more are being hired while the site is anticipating final FDA operational approval in 2021.

The company makes gene therapies for neurological genetic diseases. Its initial product is Zolgensma (onasemnogene abeparvovec-xioi), a gene replacement therapy to treat spinal muscular atrophy (SMA) Type 1, a deadly neuromuscular disease with limited treatment options.

AveXispurchased rightsto its gene therapy technology in 2015 for an undisclosed sum fromAsklepios BioPharmaceutical, a Chapel Hill gene therapy platform company commonly called AskBio.

AskBio said it received an upfront payment and stands to gain milestone payments and royalties based on AveXis successful development and commercialization of its treatment.

AskBios gene therapy platform is based on the work of Jude Samulski, Ph.D., a pioneering scientist in gene therapy who was recruited to the University of North Carolina School of Medicine from the University of Pittsburgh in 1993 with the help of about $250,000 in grant funding from the North Carolina Biotechnology Center. Samulski, renowned for his development of the harmless adeno-associated virus (AAV) as a vector, or delivery tool, for genes, directed UNCs Gene Therapy Center for many years.

Building on the success of Zolgensma, Novartis Gene Therapies said it will be responsible for the research, development, manufacturing and commercialization of the next wave of AAV-based innovative gene therapies. Novartis Gene Therapies will also provide manufacturing support for gene therapy work conducted by other Novartis units.

DavidLennon, Ph.D., previously president of AveXis, is now president of Novartis Gene Therapies and will continue reporting to Vas Narasimhan, CEO of Novartis.

Novartis sees tremendous potential in the future of gene therapy, and weve seen the impact gene therapy can have on so many lives, said Narasimhan. With the creation of Novartis Gene Therapies, we will continue to advance our gene therapy pipeline for rare genetic diseases, to accelerate the delivery of transformative innovation in areas of high unmet need, and to reimagine medicine for patients all around the world.

The most widely used gene therapy in the world, Zolgensma treats SMA, the leading genetic cause of infant death. If left untreated in its most common form, SMA leads to death or the need for permanent ventilation by the age of 2 in more than 90% of cases.

To date, more than 600 patients have been treated with Zolgensma, including in clinical trials, commercially and through managed access programs. In addition to Zolgensma being approved in the U.S., its approved in Japan, Europe and Brazil.

Novartis Gene Therapies is pursuing Zolgensma registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Australia, Argentina and South Korea in late 2020 or early 2021.

Novartis said the change to Novartis Gene Therapies is the natural evolution as the company scales up to deliver Zolgensma globally and expand its reach via a robust pipeline of AAV-based gene therapies for rare genetic diseases including investigational treatments for Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) and Friedreichs ataxia.

Novartis Gene Therapies also establishes a seamless global presence for Zolgensma and the gene therapies to come. Instead of alternating between the AveXis and Novartis umbrella brands by market, the company comes together under one banner as a unified entity.

Our patients and their families are the motivation for everything that we do, and under the banner of Novartis Gene Therapies, our dedicated team will continue to create a lifetime of possibilities to people suffering from rare genetic diseases, said Lennon.

Becoming Novartis Gene Therapies symbolizes the importance of our gene therapy advances for the future of Novartis and our industry leadership at large.

Novartis Gene Therapies comprises more than 2,000 employees across corporate, manufacturing and research facilities in the U.S. (Illinois, North Carolina, Colorado, California); Zurich, Switzerland; and Tokyo. With nearly 1 million square feet of gene therapy manufacturing capacity, Novartis Gene Therapies is the worlds largest gene therapy manufacturer.

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