Archive for the ‘Gene Therapy Research’ Category

Report Description

A recent market intelligence report that is published by Data Insights Partner onGene Therapy marketmakes an offering of in-depth analysis of segments and sub-segments in the regional and international Gene Therapy market. The research also emphasizes on the impact of restraints, drivers, and macro indicators on the regional and world Gene Therapy market over the short as well as long period of time. A detailed presentation of forecast, trends, and dollar values of international Gene Therapy market is offered. In accordance with the report, the global Gene Therapy market is projected to expand at a CAGR of 30% over the period of forecast.

Market Insight, Drivers, Restraints& Opportunity of the Market:

Gene therapy is a medical procedure which replaces defective genes or introduces new genes n order to prevent or cure genetic disorders. This procedure has become a bench mark in medical industry as there is no requirement of surgery or drugs or other procedure which has side effects on the individuals. Gene therapy was first commercialized in China in 2004 by China based SiBono Gene Tech (product Gendicine).

The global gene therapy market has been expanding due to the rigorous research conducted in the field of genetics. The rising awareness about the capability of cure of several rare genetic diseases by gene therapy is another important driver which leads the global gene therapy market during the forecast period. Gene therapy has capability cure several life threatening diseases such as cancer, cardiac diseases, AIDS, cystic fibrosis, age-related disorders, sickle cell anemia etc. In March 2019, the director of the National Health Institute (NIH), the U.S. announced that the recent clinical trials on the gene therapy for the treatment of sickle cell anemia showed promising result- therefore, increasing prevalence of aforementioned lie threatening diseases would likely to drive the growth of the global gene therapy market during the forecast period.

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On the other hand, treatment cost and stringent regulatory conditions etc. may hamper the growth of the global gene therapy market in the upcoming future. The results of Gendicine clinical trials were published in 2003 and the medicine got approval by the China State Food and Drug Administration in the same year. Although approved in China, Gendicines use is not very promising outside China. There are several concerns among the researchers about the quality of the clinical trials performed and safety and efficacy of the treatment. However, Gendicines equivalent Advexin (company Introgen Therapueitcs) is still waiting for the FDA approval.

Increasing investment to the gene therapy related research (around 10 Bn was invested in 2015 by private and public organizations), new product developments such as (Zolgensma in 2019), strategic alliance among the key players (such as collaboration between Axovant and Yposkesi) would bring the global gene therapy market an opportunity to propel during the forecast period. In May 2019, Avexis (a Novartis company) has got the FDA approval for Zolgensma for treatment of spinal muscular atrophy for the pediatric patients (less than 2 years of age).

Segment Covered:

This market intelligence report on the global gene therapy market encompasses market segments based on product, application, target user and geography. On the basis of product, the sub-markets is segmented into Yescarta, Kymriah, Strimvelis, Gendicine, Zolgensma and others (Advexin). Based on application, the global gene therapy market has been segregated into large B-Cell lymphoma, Car T Cell therapy, ADA-SCID (adenosine deaminase deficiency), muscular atrophy, head and neck squamus cell carcinoma, others (Crigler-Najjar syndrome). By target user, the global gene therapy market is also classified into adult and pediatric. By Geography, the global gene therapy market has been divided into North America (the U.S., Canada), Latin America (Brazil, Mexico, Argentina and other countries), Europe (Germany, France, the U.K., Spain, Italy, Russia, and other countries), Asia Pacific (India, Japan, China, Australia and New Zealand and other countries), Middle East and Africa (GCC, South Africa, Israel and Other countries).

Profiling of Market Players:

This business intelligence report offers profiling of reputed companies that are operating in the market. Companies such as Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others have been profiled into detail so as to offer a glimpse of the market leaders. Moreover, parameters such as gene therapy market related investment & spending and developments by major players of the market are tracked in this global report.

Report Highlights:

In-depth analysis of the micro and macro indicators, market trends, and forecasts of demand is offered by this business intelligence report. Furthermore, the report offers a vivid picture of the factors that are steering and restraining the growth of this market across all geographical segments. In addition to that, IGR-Growth Matrix analysis is also provided in the report so as to share insight of the investment areas that new or existing market players can take into consideration. Various analytical tools such as DRO analysis, Porters five forces analysis has been used in this report to present a clear picture of the market. The study focuses on the present market trends and provides market forecast from the year 2017-2027. Emerging trends that would shape the market demand in the years to come have been highlighted in this report. A competitive analysis in each of the geographical segments gives an insight into market share of the global players.

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Salient Features:

This study offers comprehensive yet detailed analysis of the Gene Therapy market, size of the market (US$ Mn), and Compound Annual Growth Rate (CAGR (%)) for the period of forecast: 2019 2027, taking into account 2017 as the base year

It explains upcoming revenue opportunities across various market segments and attractive matrix of investment proposition for the said market

This market intelligence report also offers pivotal insights about various market opportunities, restraints, drivers, launch of new products, competitive market strategies of leading market players, emerging market trends, and regional outlook

Profiling of key market players in the world Gene Therapy market is done by taking into account various parameters such as company strategies, distribution strategies, product portfolio, financial performance, key developments, geographical presence, and company overview

Leading market players covered this report comprise names such as. Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others

The data of this report would allow management authorities and marketers of companies alike to take informed decision when it comes to launch of products, government initiatives, marketing tactics and expansion, and technical up gradation

The world market for Gene Therapy market caters to the needs of various stakeholders pertaining to this industry, namely suppliers, manufacturers, investors, and distributors for Gene Therapy market. The research also caters to the rising needs of consulting and research firms, financial analysts, and new market entrants

Research methodologies that have been adopted for the purpose of this study have been clearly elaborated so as to facilitate better understanding of the reports

Reports have been made based on the guidelines as mandated by General Data Protection Regulation

Ample number of examples and case studies have been taken into consideration before coming to a conclusion

Reasons to buy:

vIdentify opportunities and plan strategies by having a strong understanding of the investment opportunities in the Gene Therapy market

vIdentification of key factors driving investment opportunities in the Gene Therapy market

vFacilitate decision-making based on strong historic and forecast data

vPosition yourself to gain the maximum advantage of the industrys growth potential

vDevelop strategies based on the latest regulatory events

vIdentify key partners and business development avenues

vRespond to your competitors business structure, strategy and prospects

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Gene Therapy Market Regulations and Competitive Landscape Outlook size 2027 - Skyline Gazette

The latest report on the Global Gene Therapy Technologies Market Report 2020-2026 is a systematic and insightful compilation of valuable evaluations of Gene Therapy Technologies market and relevant aspects. The report offers an in depth exploration of the market and its scope, trends, structure, production, profitability and maturity. The precise evaluation of market size, share, revenue, sales volume, demand, and rate of growth involved within the report drive investors, industry experts, researchers, also as novice and well-established market players to grasp the general Gene Therapy Technologies market structure.

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The Gene Therapy Technologies market report also delivers an in-depth analysis of the emerging industry trends along side the restraints, drivers, and opportunities within the Gene Therapy Technologies market to supply worthwhile insights also as a gift scenario for generating right decision. Moreover, the new report on the Gene Therapy Technologies industry covers the prominent vendors within the universal market alongside SWOT analysis, fiscal overview and major developments.

The global Gene Therapy Technologies Market report is considered as a detailed investigation of the respective market that will provide key solutions for establishment of profit-driven business strategies. It is helpful for offering details about futuristic Gene Therapy Technologies industry trends and in-depth assessment of the international industry. It permits you to determine remarkable challenges and risk factors alongside major opportunities available in the world Gene Therapy Technologies market. This report also exhibits the whole historical and current status of the Gene Therapy Technologies Market globally. Apart from this, the report on the Gene Therapy Technologies industry also represents the graphical representation of the upcoming Gene Therapy Technologies Market infrastructure and the Compound Annual Growth Rate (CAGR) in detail.

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The report on the Gene Therapy Technologies market is an exclusive and deep study which delivers a comprehensive overview of the industry contains the recent trends and future proportions of the Gene Therapy Technologies market in terms of product and services. Meanwhile, this report offers a professional research study on the Gene Therapy Technologies market so as to guage the remarkable vendors by calibrating all the relevant products or services to know the positioning of the key players within the Gene Therapy Technologies market globally.

Leading companies reviewed in the Gene Therapy Technologies report are:

Bluebird bioAdaptimmuneGlaxoSmithKlineMerckCelgeneShanghai Sunway BiotechBioCancellShenzhen SiBiono GeneTechSynerGene TherapeuticsOncoGenex PharmaceuticalsGenelux CorporationCell GenesysAdvantageneGenVecBioCancellCelgeneEpeius BiotechnologiesIntrogen TherapeuticsZiopharm OncologyShenzhen SiBiono GeneTechAltor Bioscience

The Gene Therapy Technologies Market report is segmented into following categories:

The product segment of the report offers product market information such as demand, supply and market value of the product.

The application of product in terms of USD value is represented in numerical and graphical format for all the major regional markets.The Gene Therapy Technologies market report is segmented into Type by following categories;Type IType II

The Gene Therapy Technologies market report is segmented into Application by following categories;Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovasular DiseasesOthers

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The global Gene Therapy Technologies marketing research report offers an in depth summary of the foremost desirable factors and informative details about the universal industry. Moreover, the study provides an in-depth summary and forecast of the worldwide Gene Therapy Technologies market on the idea of several segments. This report also delivers Gene Therapy Technologies market size and predicted estimations from the year 2020 to 2026 concerning various topological regions including Europe, North America, the center East and Africa, and South America.

The research study on the Gene Therapy Technologies Market is a valuable source of guidance for global customers as it will rapidly fulfil their requirement and speed up their business growth. It is an advantageous document for both existing industries manufactures including end-user industries, experts, managers, stakeholders and new entrants. We have designed this global Gene Therapy Technologies Market report in a deeply understandable format so that anyone can grasp each and every aspect related to the respective industry

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Global Gene Therapy Technologies Market Survey 2020-2026 Growth Prediction by Manufacturers Bluebird bio, Adaptimmune, GlaxoSmithKline, Merck - Galus...

Facts and Factors (FnF), A leading market research firm published the latest report on "Cell and Gene Therapy Consumables Market By Product Type (Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media) and By Application/ Therapeutics (Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027" which includes 180+ research pages for the forecast period. The Cell and Gene Therapy Consumables market report offers comprehensive research updates and information related to market growth, demand, and opportunities in the global Cell and Gene Therapy Consumables market.

The Cell and Gene Therapy Consumables market report encompasses the all-inclusive and overall analysis of the Cell and Gene Therapy Consumables market with all its related factors that might impact on the market growth. This report is anchored on the precise qualitative and quantitative assessment of the Cell and Gene Therapy Consumables market.

The goal of this Cell and Gene Therapy Consumables market report is to offer information and updates concerning the Cell and Gene Therapy Consumables market as well as see all the avenues for the development in the market. The report, to start with, comprises a market outline and provides market definition and a synopsis of the Cell and Gene Therapy Consumables market. The outline segment entails market dynamics consisting of market trends, opportunities, drivers, and restraints trailed by value chain analysis and pricing analysis.

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(The sample of this report is readily available on request).

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The overview or summary part of this report describes the definition of Cell and Gene Therapy Consumables as well as its uses, applications, product portfolios, pipeline analysis, and the other industry perspective. The Cell and Gene Therapy Consumables also discusses a couple of facets, for example, major key drivers, growth obstacles, and future predictions that have been found in the global market.

The Cell and Gene Therapy Consumables market research report also includes, a major key segment with the help of which discusses the market growth, regional aspects, sub-segment breakdown, etc., can be measure. The Cell and Gene Therapy Consumables market is also segmented on the basis of its type, uses, applications and regional and country level. The Cell and Gene Therapy Consumables research report covers an in-depth market segmentation that includes all market aspects containing in this intelligence report that could help to gain market share in the respective segments across regions.

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Some of Major Market Player ProfilesIncludedin this Report Are:

Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife

(To know the complete list of companies mentioned, Use This Link)

Furthermore, the report also categorizes the Cell and Gene Therapy Consumables market on the basis of types of products or services, application segments, end-user, regions, and others. Each of the segments growth is assessed along with their growth estimation in the forecast period. Also, the Cell and Gene Therapy Consumables market report on the Cell and Gene Therapy Consumables market provides a scrupulous study on sales volume, industry size, shares, demand & supply analysis, and value analysis of several firms together with segmental analysis, relating to important geographies.

The Cell and Gene Therapy Consumables market report includes major key drivers and its restraints to analyze the growth of the market. The report includes market size, share, trends, and growth analysis on the basis of different parameters. The report also offers the emerging trends observed in the market. The research study provides product picture and specifications, sales & revenue, market share and contact information of key market players of the Cell and Gene Therapy Consumables market. The report also enclosed the in-depth quantitative and qualitative analyses of the Cell and Gene Therapy Consumables market.

The report covers the historical and future analysis on the basis of applications, growth factors, and end-users. The research study provides an in-depth analysis based on size, share, developments, drivers and restraints.

The report analyzes the key opportunity, CAGR, and Y-o-Y growth rates to allow readers to understand all the qualitative and quantitative aspects of the Cell and Gene Therapy Consumables market. A competition analysis is imperative in the Cell and Gene Therapy Consumables market and the competition landscape serves this objective. A wide company overview, financials, recent developments, and long and short-term strategies adopted are par for the course.

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Available Customization Options:

The Cell and Gene Therapy Consumables market can be customized to the country level or any other market segment. Besides this, Report understands that you may have your own business need, hence we also provide fully customized solutions to clients.

Overview of Facts & Factors (FnF) Methodology

The qualitative analysis involved primary interviews, surveys, and vendor briefings. The data gathered as a result of these processes were validated through experts' opinions. The market dynamics have been determined after conducting a detailed study of the micro and macroeconomic indicators of the market.

Various parameters have been taken into account while estimating market size. The revenue generated by the leading industry participants in the sales of Keyword across the world has been calculated through primary and secondary research.

For this study, Facts & Factors Market Research has conducted all-encompassing primary research with key industry participants to collect first had data. Moreover, in-depth interviews with key opinion leaders also assisted in the validation of findings from secondary research and to understand key trends in the Cell and Gene Therapy Consumables. Primary research makes up the major source of data collection and validation.

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Facts & Factors is a leading market research organization offering industry expertise and scrupulous consulting services to clients for their business development. The reports and services offered by Facts and Factors are used by prestigious academic institutions, start-ups, and companies globally to measure and understand the changing international and regional business backgrounds. Our clients/customers conviction on our solutions and services has pushed us in delivering always the best. Our advanced research solutions have helped them in appropriate decision-making and guidance for strategies to expand their business.

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Cell and Gene Therapy Consumables Market 2020 Industry Analysis, Segmentation, Competitive Landscape, Regional Outlook and Forecast to 2027 - Global...

Human beings are a highlyadaptable bunch of bipeds, being able to swiftly change their lifestyles, clothes, hair, chewing gum brands, and political opinionsas the wind blows.But none of us are capable of intentionally altering our own genetic code as this superpowered species ofsquid has demonstrated!

In what could one day be used to affect gene-editing medicines and therapies to cure diseases in people, a new research paper publishedthis weekin the onlinejournalNucleic Acids Researchpresents evidence that a particular species of longfin inshore squid (Doryteuthis pealeii), commonly used as bait fish, isthe first-ever animal known to changemessenger RNAoutside of the cell nucleus.Most all creatures on this planet enact changes to their DNA from the cell nucleus via messenger RNA, but this crafty little oceanic creature takes a unique shortcut.

Scientists working out of the famous Woods HoleMarine Biological Laboratory inMassachusetts found that this extreme RNA editing process occurs withinthe squid on a fantasticscale, using over60,000 brain cells, and dwarfing the hundreds of similar sites registering in humans.

Joshua Rosenthal, a seniorscientist at Woods Hole,together withcolleagues from Tel Aviv University and the University of Colorado at Denver theorizethat thismethod of recodingallowsthe squid to better adapt to rapidly fluctuatingenvironmental conditions likewater temperature.This revolutionary RNA editing takes place inside the squids axon, the elongatedsectionof the brain cell that transmits electrical signals to neighboringneurons. Reformulatingtheir RNA outside the nucleus lets these squidmore effectively change protein function nearerto the segmentof the body that requires adaptation.

It works by this massive tweaking of its nervous system, Rosenthal explains. Which is a really novel way of going through life.

The implicationfor applications and exploration within today's gene-tweaking Crispr-centric technologies for fighting human ailments are enormous, sincethe squid does this type ofediting with messenger RNA instead ofDNA.Crispr medical research involvesirreversible changes inDNA, but due to the diminishing aspects ofunused messenger RNA, temporary errors includedby a certain genetherapy would be wiped away as opposed to remaining within someone's body permanently.

If you have some faulty information inside of your genomesay, you inherited a nucleotide base from your parents, and normally its a G [guanine] and its an A [adenine] in youthen you could potentially change it back by editing the RNA, Rosenthal notes, pointing toa pair ofbuilding blocks that compriseRNA. "RNA editing is a hell of a lot safer than DNA editing. If you make a mistake, the RNA just turns over and goes away."

So next time you order the calamari, remember that a member of your tasty squid's extended family could one day help in improving the health and vitality of all humanity. Now please pass the salt and melted butter!

More:
Crazy calamari! This superpowered squid can change its own genetic code - SYFY WIRE

There is always a well-known solution to every human problem neat, plausible, and wrong.From Prejudices (1920) by H L Mencken

There has never been a problem facing mankind more complex than understanding our own human nature. And no shortage of neat, plausible, and wrong answers purporting to plumb its depths.

Having treated many thousands of psychiatric patients in my career, and having worked on the American Psychiatric Associations efforts to classify psychiatric symptoms (published as the Diagnostic and Statistical Manual of Mental Disorders, or DSM-IV and DSM-5), I can affirm confidently that there are no neat answers in psychiatry. The best we can do is embrace an ecumenical four-dimensional model that includes all possible contributors to human functioning: the biological, the psychological, the social, and the spiritual. Reducing people to just one element their brain functioning, or their psychological tendencies, or their social context, or their struggle for meaning results in a flat, distorted image that leaves out more than it can capture.

The National Institute of Mental Health (NIMH) was established in 1949 by the federal government in the United States with the practical goal of providing an objective, thorough, nationwide analysis and reevaluation of the human and economic problems of mental health. Until 30 years ago, the NIMH appreciated the need for this well-rounded approach and maintained a balanced research budget that covered an extraordinarily wide range of topics and techniques.

But in 1990, the NIMH suddenly and radically switched course, embarking on what it tellingly named the Decade of the Brain. Ever since, the NIMH has increasingly narrowed its focus almost exclusively to brain biology leaving out everything else that makes us human, both in sickness and in health. Having largely lost interest in the plight of real people, the NIMH could now more accurately be renamed the National Institute of Brain Research.

Read: [How tech is helping brain-injured patients with decision-making]

This misplaced reductionism arose from the availability of spectacular research tools (eg, the Human Genome Project, functional magnetic resonance imaging, molecular biology, and machine learning) combined with the naive belief that brain biology could eventually explain all aspects of mental functioning. The results have been a grand intellectual adventure, but a colossal clinical flop. We have acquired a fantastic window into gene and brain functioning, but little to help clinical practice.

The more we learn about genetics and the brain, the more impossibly complicated both reveal themselves to be. We have picked no low-hanging fruit after three decades and $50 billion because there simply is no low-hanging fruit to pick. The human brain has around 86 billion neurons, each communicating with thousands of others via hundreds of chemical modulators, leading to trillions of potential connections. No wonder it reveals its secrets only very gradually and in a piecemeal fashion.

Genetics offers the same baffling complexity. For instance, variation in more than 100 genes contributes to vulnerability to schizophrenia, with each gene contributing just the tiniest bit, and interacting in the most impossibly complicated ways with other genes, and also with the physical and social environment. Even more discouraging, the same genes are often implicated in vulnerability to multiple mental disorders defeating any effort to establish specificity. The almost endless permutations will defeat any easy genetic answers, no matter how many decades and billions we invest.

The NIMH has boxed itself into a badly unbalanced research portfolio. Playing with cool brain and gene research toys trumps the much harder and less intellectually rewarding task of helping real people.

Contrast this current NIMH failure with a great success story from NIMHs distant past. One of the high points of my career was sitting on the NIMH granting committee that funded psychotherapy studies in the 1980s. We helped to support the US psychologist Marsha Linehans research that led her to develop dialectical behavior therapy; the US psychiatrist Aaron T Becks development of cognitive therapy; along with numerous other investigators and themes. Subsequent studies have established that psychotherapy is as effective as medications for mild-to-moderate depression, anxiety, and other psychiatric problems, and avoids the burden of medication side-effects and complications. Many millions of people around the world have already been helped by NIMH psychotherapy research.

In a rational world, the NIMH would continue to fund a robust psychotherapy research budget and promote its use as a public-health initiative to reduce the current massive overprescription of psychiatric medication in the US. Brief psychotherapy would be the first-line treatment of most psychiatric problems that require intervention. Drug treatments would be reserved for severe psychiatric problems and for those people who havent responded sufficiently to watchful waiting or psychotherapy.

Unfortunately, we dont live in a rational world. Drug companies spend hundreds of millions of dollars every year influencing politicians, marketing misleadingly to doctors, and pushing pharmaceutical treatments on the public. They successfully sold the fake marketing jingle that all emotional symptoms are due to a chemical imbalance in the brain and therefore all require a pill solution. The result: 20% of US citizens use psychotropic drugs, most of which are no more than expensive placebos, all of which can produce harmful side-effects.

Drug companies are commercial Goliath with enormous political and economic power. Psychotherapy is a tiny David with no marketing budget; no salespeople mobbing doctors offices; no TV ads; no internet pop-ups; no influence with politicians or insurance companies. No surprise then that the NIMHs neglect of psychotherapy research has been accompanied by its neglect in clinical practice. And the NIMHs embrace of biological reductionism provides an unintended and unwarranted legitimization of the drug-company promotion that there is a pill for every problem.

A balanced NIMH budget would go a long way toward correcting the two biggest mental-health catastrophes of today. Studies comparing psychotherapy versus medication for a wide variety of mild to moderate mental disorders would help to level the playing field for the two, and eventually reduce our massive overdependence on drug treatments for nonexistent chemical imbalances. Health service research is desperately needed to determine best practices to help people with severe mental illness avoid incarceration and homelessness, and also escape from them.

The NIMH is entitled to keep an eye on the future, but not at the expense of the desperate needs of the present. Brain research should remain an important part of a balanced NIMH agenda, not its sole preoccupation. After 30 years of running down a bio-reductionistic blind alley, it is long past time for the NIMH to consider a biopsychosocial reset, and to rebalance its badly uneven research portfolio.

This article was originally published at Aeonby Allen Frances and has been republished under Creative Commons.

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The distorted idea of 'cool' brain research is stifling psychotherapy - The Next Web

Glen Allen Mar 29, 2020 (Thomson StreetEvents) -- Edited Transcript of Precigen Inc earnings conference call or presentation Monday, March 2, 2020 at 10:30:00pm GMT

Precigen, Inc. - President & CEO

Precigen, Inc. - VP of IR

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

Good day, and welcome to the Precigen Conference Call. (Operator Instructions) Please note that this event is being recorded. I would now like to turn the conference over to Steven Harasym. Please go ahead, sir.

Steven Harasym, Precigen, Inc. - VP of IR [2]

Thank you, operator. Welcome to the Precigen Fourth Quarter and Full Year 2019 Business and Update Call. I'm Steven Harasym, Vice President of Investor Relations. And I'm pleased to be joined today by Dr. Helen Sabzevari, President and CEO of Precigen; and Tom Samuelson, Vice President of Finance.

During today's call, as seen on Slide 2, we will make various forward-looking statements. Investors are cautioned that our forward-looking statements are based on current expectations and are subject to risks and uncertainties that could cause actual results or outcomes to differ materially from those indicated by our forward-looking statements. Please read the safe harbor statement contained in this presentation as well as in Precigen most recent SEC filings for a more complete discussion of these risks and uncertainties. I would now like to turn the call over to Dr. Helen Sabzevari. Helen?

Helen Sabzevari, Precigen, Inc. - President & CEO [3]

Thank you, Steve. Please go to Slide 3. I'm extremely pleased to be here today as we have made great progress even since our last public presentation at JP Morgan Health Care Conference in early January. We enter 2020 with renewed optimism about our ability to deliver on our aggressive goals and add value to shareholders.

Precigen's mission is to improve patient care through innovative gene and cell therapy-based approaches. I will review our carefully curated portfolio of unique solutions to unmet needs in health in greater detail. Our approaches are novel and designed to treat conditions that are both difficult to treat and have limited treatment options for patients.

Before we give a quick recap of the divestments and transactions that enabled us to advance towards our goal of becoming a dedicated health care company, I thought it would be useful to provide a recap of how far we have come in such a short time, as shown in Slide 3. In Q4 of 2018, we started on the path to becoming a stand-alone health care company by reacquiring the rights to our oncology assets from our former partners. In just under a year, we advanced 2 programs from inception into the clinic and developed a pipeline of promising product candidates in immuno-oncology, autoimmunity and infectious diseases. We also took the necessary steps to expand on an already robust IP estate. Finally, we consolidated the majority of our operation in our Maryland headquarters and continue to bolster our scientific team here.

We believe the transactions that occurred over the last several months put us firmly on the path to being able to devote our resources to advance our health care assets and become a major player in the gene and cell therapy field. As you may recall, this included divesting the majority of Intrexon's non-health care assets, my appointment as a CEO, and name change from Intrexon to Precigen, and associated stock ticker change to PGEN. We also sold our position in AquaBounty for approximately $21.6 million; sold our 50% interest in EnviroFlight to our former partner, Darling Ingredients for $12.2 million; completed the sale of several assets, including our non-health subsidiaries, Oxitec, Okanagan Specialty Fruits and AgBio and ILH Holdings for a combined $53 million-plus certain contingent payment rights. These now divested non-health businesses accounted for $46 million in net cash operating expenses and capital expenditure in 2019 and raised $35 million in capital through the issuance of equity.

Through the financial transaction, we achieved our previously stated cash position goals, allowing the company to deliver on several value-creating milestones in 2020. Furthermore, we are happy to report that the going concern qualifier has been removed from our financial statement. Based on a combination of our cash position and reduced spending, we anticipate current capital on hand will allow us to operate well into 2021.

In light of our transition to a more health-focused company, we are evaluating all aspects of our operating structure and will provide updates in our next quarterly call. Concurrently, I want to acknowledge the contributions of General Bostick, COO and President of Intrexon Bioengineering; and Tom Reed, Founder and CFO of Intrexon, both of whom are no longer at the company. We thank them for their contribution and services.

Moving ahead, I want to share how we plan to manage Precigen going forward on the next slide. Slide 4, please. With our focus firmly on health care, we have aligned the entire company to operate in accordance with 4 key principles. First is adhering to strict fiscal responsibility. We will responsibly allocate capital to maximize value creation for stakeholders. Fiscal responsibility for us is not just about saving, maintaining cash and cutting costs. It is also about allocating resources to the right areas to create value. You will note that in 2019, we spent $43.7 million on our 2 most advanced health care subsidiaries, Precigen and ActoBio. Collectively, the health portfolio spent significantly lower than our non-health portfolio.

The second operating principle is actively managing our portfolio with a strict adherence to data-driven go and no-go decisions. Third is focusing on rapid execution, moving our valuable portfolio of assets quickly into the clinic and advancing them into registration-enabling studies in accordance with the appropriate scientific, clinical and manufacturing standards. And finally, forming a strategic partnership to advance or divest assets in our portfolio where appropriate, noting our fiduciary duty to operate in the best interest of shareholders.

Since January, we have aligned our portfolio, streamlined our operations and optimized our organizational structures to improve operational efficiency, especially at the corporate level. We will continue to take additional efficiency measures across the organization, and we'll update you on our progress. I strongly believe that the most important way we can deliver value to shareholders is to create value to patients through rapid development of novel therapeutics that are safe, effective and address unmet medical needs in a fiscally responsible manner.

Before moving on to a review of the anticipated 2020 milestones for Precigen, on the next slide, we want to discuss our strategy for the remaining non-health assets. Slide 5, please. Our subsidiary MBP Titan's technology platform has the potential to upgrade natural gas to higher value carbon output through our Methane Bioconversion Platform. As we prioritize our health portfolio, the Board of Directors and I are committed to significantly reducing our non-health spending, specifically for MBP.

To this end, we have already implemented efficiency measures at MVP to achieve this goal and have already significantly reduced their capital requirements compared to last year. We will continue to evaluate further efficiency measures that support its ongoing operations while establishing clear fiscal guardrails on spending.

Our goal is to partner or ultimately sell this business. Whatever we do will be in the long-term interest of our stakeholders and adherence to our mission. We will continue to update you on our decision when we have new information to share.

Another non-health business in our portfolio, Trans Ova, is the industry leader in advanced reproductive technologies for the cattle industry. We considered divestiture of this non-health business, but ultimately elected to retain it because we believe it has more value than the offers we received.

Nevertheless, we continue to strategically evaluate options for this business. In the meantime, however, we have implemented efficiency and cost reduction measures, such that we will not contribute any additional capital towards Trans Ova and expect Trans Ova to return to being a net contributor of capital to Precigen this year.

Moving to the next slide. You can see the breadth of our health care organization. Slide 6, please. The new Precigen enters 2020 with a promising portfolio of investigational gene and cell therapies. Under the new 1 Precigen umbrella is our transformative UltraCAR-T, AdenoVerse immunotherapy and ActoBiotics Therapeutic platforms, which share a focus on developing innovative therapies in immuno-oncology, infectious diseases and autoimmune disorders. We also are advancing an innovative approach from our subsidiary, Triple-Gene, in heart failure.

Slide 7, please. Moving to the next slide, we see the breadth and the value of our clinical portfolio, which includes our internal as well as partnered programs, including later-stage assets in Phase II and Phase III clinical trials. Two important things to remember about our portfolio are that: first, we are advancing unique programs that represent substantial therapeutic class innovation; and second, we expect most of these programs to have important data readouts in 2020.

Following the release of new data, we intend to assess and further prioritize our pipeline to optimize cash resources. Beyond this clinical pipeline, we also have a portfolio of novel preclinical assets that will be assessed and funded according to strict science and data development analysis. Our most significant role for preclinical assets for 2020 is to initiate a Phase I trial of PRGN-2009, our new off-the-shelf AdenoVerse immunotherapy in HPV-positive cancers.

Slide 8, please. Before reviewing our many exciting milestones for 2020, I want to take a few moments on the next slide to provide an overview of one of our most promising therapeutic platforms, UltraCAR-T. We believe it holds the promise to revolutionize the CAR-T landscape and provide benefits to patients and the health care system at large for the following reasons. First, unlike conventional CAR-T, UltraCAR-T cells do not require lentivirus and long ex vivo expansion in manufacturing facilities. Instead, UltraCAR-T uses nonviral, rapid, overnight manufacturing at hospital. This overnight manufacturing brings the potential for repeat dosing of UltraCAR-T to patients as well. This manufacturing approach, we believe, brings the convenience of allogeneic CAR-T administration to autologous CAR-T treatment without the potential risk associated with the allogeneic CAR-T therapy.

Second, unlike conventional CAR-T, UltraCAR-T cells express membrane-bound IL-15, which provides higher potential to expand and persist in vivo and to maintain a younger, less differentiated state.

Conventional CAR-T cells have limited potential for expansion after administration due to long manufacturing process that requires T cell activation and expansion outside the body.

Allogeneic CAR-T cells are even more limited in their persistence potential since they are foreign and risk rejection by patients' immune system and the occurrence of the graft-versus-host disease.

Furthermore, allogeneic CAR-T cells requires severe lymphodepletion of patients, which also limits antigen-spreading potential. We believe membrane-bound IL-15 provides our UltraCAR-T cells with a higher potential for expansion and persistence in patients after administration than conventional CAR-T.

Third, we have built our UltraCAR-T platform such that all modified T cells express a kill switch. This engineering provides us with an ability to selectively eliminate UltraCAR-T cells by administration of a kill switch activator in the event of toxicity, thus improving the safety profile.

With the potential to advance precision medicine and disrupt the current CAR-T landscape, our vision for the UltraCAR-T platform is to build and validate a library of UltraCAR vectors to provide personalized autologous CAR-T treatment for any cancer patients in a rapid and cost-conscious manner. During 2020, we expect to report numerous data sets and achieve new milestones.

Slide 9, please. Looking at our advanced assets,

3005 is the first UltraCAR-T in solid tumors, targeting ovarian cancer in Phase I clinical study. We currently are enrolling the second cohort for the IP arm. To date, we continue to have 100% manufacturing success and the preliminary findings regarding UltraCAR-T kinetics, which includes expansion and persistence in patients treated in the lowest dose cohort are very encouraging. We know that everyone is eager to see these early data points, and we share their excitement. We will continue to evaluate the appropriate time to provide additional details. Presently, we expect to provide an initial readout from the IP arm in the second half of 2020. We are also planning to present preclinical data for 3005 at an upcoming medical meeting later this year.

Slide 10, please. 3006 is the first UltraCAR-T therapy be evaluated in hematological cancers. We recently received orphan drug status from the FDA for this program. We are currently enrolling the second cohort for the non-lymphodepletion arm and the first cohort for the lymphodepletion arm. As with 3005, we continue to have 100% manufacturing success and the preliminary findings regarding UltraCAR-T kinetics from the trial are very encouraging. We expect to provide an initial data readout in the second half of 2020.

We are very excited about the Phase II trial for AG013 for oral mucositis in head and neck cancer with our partner Oragenics as we believe that it will further validate our ActoBiotics platform across multiple indications. Using our platform in partnership with Oragenics, we are targeting oral mucositis, a severe and painful side effect of chemoradiation therapy, especially in patients with head and neck cancer. There are currently no drugs approved to prevent this condition in the cancer patient population. As a result, the FDA has given this program Fast Track status, which validates the urgent need for a treatment. AG013 is formulated as an oral rinse and used as mouthwash to deliver the trefoil factor 1 gene, which prevents mucosal tissue damage and induces subsequent repair of the lining of the mouth. Based on the encouraging Phase I data, AG013 is currently in a Phase II trial. Enrollment in this study was completed in the fourth quarter of 2019, and we are looking forward to reporting interim data from the Phase II trial in the first half of 2020. We also expect to report interim data from an ongoing clinical trial of AG019 in type I diabetes patients. AG019 is a first-in-class disease-modifying antigen-specific immunotherapy for the prevention, delay or reversal of type 1 diabetes with encouraging results in preclinical studies. Currently, AG019 is in a Phase Ib/IIa trial. The Phase IIa portion of the trial is now enrolling, and interim data readout is expected in the third quarter of 2020.

Another exciting asset in our portfolio is INXN-4001, a novel gene therapy for heart failure patients, which is being developed by our majority-owned Triple-Gene subsidiary. We expect to complete the trial by the end of 2020. 2009 is an off-the-shelf AdenoVerse immunotherapy product candidate designed to activate the immune system to recognize and target HPV-positive solid tumors. This program is currently under development through a Cooperative Research and Development Agreement or CRADA with Dr. Jeffrey Schlom, a world renowned investigator in immuno-oncology at the NCI. We expect the NCI to start dosing patients in 2020.

I'll now turn the call over to Tom Samuelson to provide a financial update.

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Tom Samuelson, [4]

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Thank you, Helen, and good evening, everyone. There are 3 key points that I would like to address today. First, as Helen mentioned, we've made material progress in narrowing the company's focus to our core health care programs and reducing our other capital requirements. A critical component of this pivot was the divestiture of a number of our legacy bioengineering assets, including our shares in AquaBounty for $86.8 million plus certain contingent payment rights, and the sale of $35 million of our common stock. The business is sold in these recent transactions accounted for $46 million in 2019 segment adjusted EBITDA losses, capital that can be redeployed towards our health care assets in 2020. Please recall that segment EBITDA, which is more fully defined in our 10-K, is generally the sum of net cash operating expenses and capital expenditures. The proceeds from these transactions, combined with the company's cash and short-term investments on hand, provide sufficient capital to remove the going concern qualification from our 2019 financial statements. We've adjusted our 2019 and prior financial statements to reflect the effects of these businesses as discontinued operations.

Second, we reported fourth quarter and full year 2019 revenues of $17 million and $90.7 million and consolidated financial results from continuing operations, as we continued our shift from the business model focused on collaboration and licensing revenues to one wholly focused on our internal programs primarily in human health. Despite pivoting away from a collaboration model, we continue to own the rights to certain legacy milestones and royalties. Any of these, if successful, could result in additional sources of capital for us without requiring any further obligations on our part.

Fourth quarter and full year segment EBITDA losses including corporate costs, were $37.8 million and $144.4 million, respectively. These annual losses included only $30.2 million at Precigen and $13.7 million at ActoBio. Among our retained bioengineering entities, MBP Titan and Trans Ova Genetics accounted for $36.7 million and $6.3 million, respectively. As Helen mentioned, we are fully committed to reducing spend at these entities and have already implemented efficiencies that should substantially reduce their capital requirements going forward. I would further highlight that the aforementioned $46 million in segment EBITDA losses from transacted assets does not include an allocation of general corporate costs, which we do not allocate to particular segments. More details on segment information can be found in the financial discussion in our 10-K.

Third, the 2019 consolidated loss attributable to Precigen shareholders of $322.3 million or $2.09 per share includes $116.2 million or $0.75 per share associated with the discontinued operations. We further incurred a noncash impairment charge of $29.6 million for the write-down of goodwill associated with our Trans Ova Genetics subsidiary.

As discussed, we've initiated efforts in 2020 to improve the financial performance of Trans Ova Genetics going forward.

In concluding the financial component of our call, I will again reiterate that Precigen is wholly committed to deploying our precious capital towards our highest value health care assets in 2020 and beyond. We look forward to providing regular updates as our targeted value-generating milestones are achieved.

I would now like to turn the call back to Helen for concluding remarks.

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Helen Sabzevari, Precigen, Inc. - President & CEO [5]

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Thank you, Tom. In closing our call today, I want to confirm our optimism about Precigen's potential to transform the health care landscape with our innovative and focused portfolio. I think you will agree that there has never been a more exciting and promising time at Precigen. As Precigen's CEO, you have my commitment to manage our company in a financially prudent, fiscally disciplined and transparent manner with the paramount goal of achieving our mission to bring novel treatment options to patients. If we do this, all of our stakeholders will benefit.

With that, we'll now open the line for questions. Operator, please begin.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question will come from Jason Butler of JMP Securities.

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Jason Nicholas Butler, JMP Securities LLC, Research Division - MD and Senior Research Analyst [2]

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Helen, first one, just on the UltraCAR-T trials for both 3005 and 3006, can you frame for us how we should think about the readouts later this year in terms of patient numbers and maturity of data? And maybe if not specifically in terms of numbers, then in terms of how it could inform next steps or potentially progression to pivotal studies?

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Helen Sabzevari, Precigen, Inc. - President & CEO [3]

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Thank you, Jason. Actually, we are very excited for this upcoming interim data. First of all, in regards to the ovarian cancer, the IP arm and in regard to the AML that we have non-lymphodepletion arm to report. As you might have seen in our slides, our trials currently are 3 plus 3 plus 3, there are 3 doses. And as we finish these doses, we obviously will be reporting on the safety and dose, which are the paramount aspects of the Phase I. But at the same token, as we have always emphasized what is the most important thing for us is also show that our manufacturing in vivo directing patients to show the persistence and also the expansion of T cells. So we are looking forward to show some of the interim data by the second half of 2020. And it's quite exciting for us as we go through this journey, and we continue to then expand these patients to Phase Ib.

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Jason Nicholas Butler, JMP Securities LLC, Research Division - MD and Senior Research Analyst [4]

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Great. And then I had a question on AG013 and the interim results upcoming. How should we think about the magnitude of treatment effect here in terms of what would be clinically important to patients? And then in terms of the oral mucositis endpoint, can you just talk to us about subjectivity of the endpoint and how you're controlling for that? And any expectations for either reduction in analgesic use or potential for prolong radiation dose?

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Helen Sabzevari, Precigen, Inc. - President & CEO [5]

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Absolutely. Thank you. So our -- actually, the AG013 is a very exciting program for the platform of ActoBio. As you know, that ActoBio platform uses our elective delivery mechanisms for delivering different proteins, specifically to the mucosal linings. In the case of the oral mucositis, this is a devastating disease especially after the treatment of head and neck cancer patients with chemo and radiation. There are tremendous ulcers in the mouth, which is quite painful, and currently, there is really no treatment for this.

In the Phase I of the study that was done, we have designed the [elastics] in such a way that it delivers a gene that protects and also stimulates the lining of the mucosal lining of the mouth. And we had a very exciting results in the first Phase I showing reduction in the ulcers and actually the level of the pain. And now we have entered and actually finished enrollment of the patients in 2019 in a Phase II that it's done in a randomized fashion. So there is a placebo arm versus the treatment arm, which is quite important. And one aspect that is quite exciting based on the Phase I data, FDA has given us a Fast Track for this indication currently. And this is -- would be the first proof of this platform actually for ActoBio, which we also have exciting trial ongoing in the T1D, type 1 diabetes, which we are anticipating to actually report on in second half of 2020 using a similar actually platform, but delivering a proinsulin, and as well as IL-10, which suppresses the immune system, but actually, it helps the onset of -- it reduces onset of diabetes, and we had a very encouraging preclinical data, the Phase I data and now the Phase IIa will be reported in second half.

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Jason Nicholas Butler, JMP Securities LLC, Research Division - MD and Senior Research Analyst [6]

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Okay, great. That's helpful. And then just 1 last financial question, and sorry if I missed this in the prepared comments. But should we expect any further reorganization charges? And if so, how should we think about those in terms of cash versus noncash spend?

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Helen Sabzevari, Precigen, Inc. - President & CEO [7]

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So currently, I think we are not expecting any further reorganization charges. And as we mentioned by the end of January, all the assets will transfer to Third Security, and currently, we do not expect anything in that lab.

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Operator [8]

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(Operator Instructions) Our next question will come from Swayampakula Ramakanth from H.C. Wainwright.

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Swayampakula Ramakanth, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [9]

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This is RK from H.C. Wainwright. Helen, a couple of quick questions. For the non-oncology assets, AG019 and INXN-4001, what do you plan for these assets in the long term? The reason for that question is, as you can imagine, as these assets get into late-stage development, they could become cash-intensive because of the large clinical trials that you would need to conduct. Would these be assets that can become currency in terms of out licensing?

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Helen Sabzevari, Precigen, Inc. - President & CEO [10]

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Thank you, RK. Great question. So we agree. We have a portfolio of -- maybe I should just first give a few minutes on the reason that we have a portfolio that we have. The strength of our portfolio is that we are not a one-drug-product company. It becomes very important because when you are one-drug-product company, basically despite of what the science might be or might not be, you are committed somehow. Whereas the way we have arranged our portfolio is that it's in such a manner that we prioritize and make it go/no-go decision based on data, and we know the attrition of the portfolio. And therefore, it's very, very important that we keep a fiscal responsibility with very, very laser-focused decision-making to address our portfolio.

In regards to the assets that we have highlighted for this year, actually, this has been a principal factor behind it. We are looking at our data, we are evaluating the breadth of the data that comes in its totality. And then there will be decisions made that if these assets will go to the next levels of development or not. However, what is very important as the fourth pillar that I mentioned as part of our principle is a strategic partnership. And this is very, very important for us in our portfolio. And to your point, obviously, and I'm going to stress this over and over again. We will not take every asset forward ourselves. We will also look into strategic partnerships that will bring the most value for our shareholders. And I think this is a pillar for us that it would allow us to manage our portfolio accordingly and make the decisions with the right partners, with the right speed and with the right cash to make these things and move them forward. And definitely, as you have mentioned, this is one of our strategic responsibility and platforms that we have for these assets.

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Swayampakula Ramakanth, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [11]

Link:
Edited Transcript of XON earnings conference call or presentation 2-Mar-20 10:30pm GMT - Yahoo Finance

Growth Prospects of the Global Gene Therapy for Ovarian Cancer Market

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Gene Therapy for Ovarian Cancer Market top key players, size, Analysis, growth, research, Types, Regions and Forecast from 2019-2023 - Daily Science

Over 95% planned enrollment completed in Phase 3 oGVHD study; topline results anticipated by end of 2020

Conference Call and Webcast Today at 8:30 a.m. ET

MALVERN, Pa., March 27, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN), a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat rare and underserved ophthalmic diseases, today reported full year 2019 financial results along with a general business update.

We are extremely pleased with the progress we have made in enrolling patients in our Phase 3 clinical trial for OCU300, an orphan drug candidate for ocular graft versus host disease (oGVHD). Based on current enrollment, we anticipate topline results by the end of the year, commented Shankar Musunuri, PhD, MBA, Chairman, CEO and Co-Founder of Ocugen. The publication of preclinical data on OCU400 in Nature Gene Therapy earlier this month is a key achievement for this program. We continue to advance IND-enabling studies toward bringing this potential breakthrough modifier gene therapy platform to patients in a Phase 1/2a clinical trial for OCU400 in 2021. Similar to the situation with virtually all other biopharmaceutical companies, we are also assessing the potential impact of ongoing COVID-19 pandemic-related events on our programs and plans. We are grateful to healthcare professionals and others who are working hard to address and mitigate the challenges presented by the virus.

Business Highlights:

Full Year 2019 Financial Results:

Conference Call and Webcast Details

The Company has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the results and recent business highlights. Ocugen's senior management team will host the call, which will be open to all listeners. There will also be a question and answer session following the prepared remarks.

The call can be accessed by dialing (844) 873-7330 (U.S.) or (602) 563-8473 (international) and providing the conference ID 5373849. To access a live audio webcast of the call on the Investors section of the Ocugen website, please click here. A replay of the webcast will be archived on Ocugens website for approximately 45 days following the call.

About Ocugen, Inc. Ocugen, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing transformative therapies to treat the whole eye. Our Phase 3 small molecule drug candidate for ocular Graft Versus Host Disease (oGVHD), if approved, will be the first and only treatment for this orphan disease. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many. And our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

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

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OCUGEN, INC.CONSOLIDATED BALANCE SHEETS(UNAUDITED)

OCUGEN, INC.CONSOLIDATED STATEMENTS OF OPERATIONS(UNAUDITED)

Corporate Contact:Ocugen, Inc.Kelly Beck kelly.beck@ocugen.com +1 484-328-4698

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

Read more:
Ocugen Provides Business Update and Full Year 2019 Financial Results - Yahoo Finance

Facts and Factors (FnF), A leading market research firm published the latest report on "Gene Therapy Market By Type (Germ Line Gene Therapy and Somatic Gene Therapy), By Vector Type (Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome), and By Therapy Area (Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027" which includes 180+ research pages for the forecast period. The Gene Therapy market report offers comprehensive research updates and information related to market growth, demand, and opportunities in the global Gene Therapy market.

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Gene Therapy Market 2020 Upcoming Trends, Industry Share, Competitive Landscape with Top Key Players - Global Industry News 24

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DENVER (KDVR) -- Even with a stay-at-home order in place, there was no way Amber Freed was cancelling her 3-year-old twins birthday party.

The Denver mother set up an entire partys worth of decorations in the front yard, complete with a giant blow-up dinosaur, and dozens of balloons.

What I did was pull out every seasons decorations all at once.We had Thanksgiving, dinosaurs, Halloween, and everything for a birthday, Freed said, laughing.

She asked friends and neighbors to drive by in their car and wish her twinsMaxwell and Rileyahappy birthday, by honking three times.

I am going to remember this day and smile so big, said Amber Freed.

The drive-by style party brought a few hours of joy to the Freed family, during an otherwise time of darkness.

I definitely was up late last night, trying to piecemeal this party together, knowing that this might be Maxwells last healthy birthdayand also knowing that all the progress we made may be falling apart right now, said Freed, with tears in her eyes.

Maxwell suffers from a rare, genetic disorderso rare, in fact, it doesnt have a real name.For now, its referred to as SLC6A1 and is similar to Parkinsons disease.

In the next year, were expecting a debilitating form of epilepsy to begin, that is not treatable with drugs.His 4th birthdayhe may lose a lot of the skills he has fought so hard to gain, Freed said.

A drug, and possible cure, is already being tested on mice.

Gene therapy was set to start later this year, but the timeline has slowed down significantly in the face of the COVID-19 pandemic.

Only a few scientists can be in laboratory at a time, and a lot of students and PhDs were working on our projectsand universities have shut down, Freed explained.Because universities have shut down, there are just no resources, she added.

Its a devastating setback for her son.

Days and minutes count for us.We need to have him treated soon, or else he will have no quality of life.

Theyll still need an additional $3.5 million, minimum, to conduct a full clinical trailat a time where many Americans are now experiencing financial strain.

Im so deeply sad that we came this far, and everything was working out perfectly.I was fighting so hard to save my baby boy, and all of that is in complete limbo right now, Freed said.

Which is why Saturday was much more than just a party, in her eyes.

I cannot believe how many people showed up to support Maxwell and Riley.It made the day so special for us.And it just meant the entire world for my family.

Learn more on Milestones for Maxwell or donate to help the family fund research and a clinical trial.

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Drive-by birthday party for 3-year-old with rare disease - FOX 31 Denver

Fact.MR has adopted multi-disciplinary approach to shed light on the evolution of the global Gene Therapy market during the historical period of 2014 2019. The study presents a deep-dive assessment of the current growth dynamics, major avenues in the estimation year of 2020, and key prospects over the forecast period 2020 2026.

According to Fact.MR report the global Gene Therapy Market to hit US$ 5 Bn by 2026. Extensive rounds of primary and a comprehensive secondary research have been leveraged by the analysts at Fact.MR to arrive at various estimations and projections of the Gene Therapy market, both at global and regional levels. The analysts have used numerous industry-wide prominent business intelligence tools to consolidate facts, figures, and market data into revenue estimations and projections in theGene Therapy market.

After reading the Gene Therapy market report, readers get insight into:

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The Gene Therapy market report offers assessment of prevailing opportunities in various regions and evaluates their shares of revenue by the end of different years of the assessment period. Key regions covered comprise:

The evaluation of the competitive landscape in the Gene Therapy market covers the profile of the following top players:

Novartis AG, Gilead Sciences Inc., Spark Therapeutics Inc., Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., CELGENE CORPORATION, and Orchard Therapeutics Limited, among others.

To expand the understanding of opportunities in the global Gene Therapy market report looks at close quarters into the opportunities and new avenues in following key segments:

In addition to understanding the demand patterns of various applications, the report on the Gene Therapy market also enumerates trends expected to attract investments by other various associated industries.

On the basis of product types, the Gene Therapy market report offers insight into major adoption trends for the following segments:

The global Gene Therapy market report offers detailed assessments and quantitative evaluations that shed light on numerous key aspects that have shaped its evolution over the historical period. In coming years, some of the key aspects that will shape the growth prospects during the forecast period are objectively covered in the study.

Some important questions that the Gene Therapy market report tries to answer exhaustively are:

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Gene Therapy Market Foraying into Emerging Economies Insights 2020 to 2026 - The Cloud Tribune

The Global Gene Therapy Market is expected to grow from USD 1,636.49 Million in 2018 to USD 6,436.64 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 21.60%.The latest report on Gene Therapy Market added by Regal Intelligence, focus on market estimates and geographical spectrum of this industry. The report specifies information about Gene Therapy industry regarding a thorough and detailed assessment of this business.

Further, the Gene Therapy Market report details important challenges and factors that influence market growth. Further, a detailed comprehensive secondary research was done to collect information on the market segments and sub-segments. Further, primary research was performed to validate the assumptions and findings obtained from secondary research with industry professionals and experts.

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Competitive Landscape:

The report deeply explores the recent significant developments by the leading vendors and innovation profiles in the Global Gene Therapy Market including are Achieve Life Sciences, Inc., Adaptimmune, Bluebird bio, Inc., Gilead, Merck & Co., Inc, Abeona Therapeutics, Inc.,, AGTC, Audentes Therapeutics, Biogen, Editas Medicine, Novartis, Orchard Therapeutics, and Spark Therapeutics. On the basis of Type, the Global Gene Therapy Market is studied across Antigen Gene Therapy, Cancer Gene Therapy, Cytokine Gene Therapy, Suicide Gene Therapy, and Tumor Suppressor Gene Therapy.On the basis of Vector Type, the Global Gene Therapy Market is studied across Non-viral Vectors and Viral Vectors.On the basis of Application, the Global Gene Therapy Market is studied across Cardiovascular Diseases, Genetic Diseases, Infectious Diseases, Neurological Diseases, and Oncological Disorders.

In the primary research process, various sources from both the supply and demand sides were interviewed to obtain qualitative and quantitative information for this Gene Therapy Market report. The primary sources from the supply side include product manufacturers (and their competitors), opinion leaders, industry experts, research institutions, distributors, dealer and traders, as well as the raw materials suppliers, and producers etc.

The primary sources from the demand side include Gene Therapy industry experts such as business leaders, marketing and sales directors, technology and innovation directors, supply chain executive, End-User (product buyers), and related key executives from various key companies and organizations operating in the global Gene Therapy market.

Primary Types of the industry are

Primary Applications of the industry are

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This report is based on the synthesis, analysis, and interpretation of information collected on the Gene Therapy market from various sources. Our analysts have analysed the information & data and gained insights using a mix of primary and secondary research efforts with the primary objective to provide a holistic view of the Gene Therapy Industry.

The following market parameters were considered to estimate market value:

Market Overview

The report includes overviews market introduction, market drivers & influencing factors, restraints & challenges, and potential growth opportunities of Gene Therapy market. The report consists of market evaluation tools such as Porters five forces, PESTLE Analysis, and value chain analysis.

Key Questions Addressed in the Report:

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New Business Opportunities in Gene Therapy Market and Growth analysis - Skyline Gazette

The research study presented in this report offers complete and intelligent analysis of the competition, segmentation, dynamics, and geographical advancement of the Global Hemophilia Gene Therapy Market. The research study has been prepared with the use of in-depth qualitative and quantitative analyses of the global Hemophilia Gene Therapy market. We have also provided absolute dollar opportunity and other types of market analysis on the global Hemophilia Gene Therapy market.

It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the global Hemophilia Gene Therapy market. All findings and data on the global Hemophilia Gene Therapy market provided in the report are calculated, gathered, and verified using advanced and reliable primary and secondary research sources. The regional analysis offered in the report will help you to identify key opportunities of the global Hemophilia Gene Therapy market available in different regions and countries.

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The authors of the report have segmented the global Hemophilia Gene Therapy market as per product, application, and region. Segments of the global Hemophilia Gene Therapy market are analyzed on the basis of market share, production, consumption, revenue, CAGR, market size, and more factors. The analysts have profiled leading players of the global Hemophilia Gene Therapy market, keeping in view their recent developments, market share, sales, revenue, areas covered, product portfolios, and other aspects.

Competitive landscape

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Hemophilia Gene Therapy Market Size and Forecast

In terms of region, this research report covers almost all the major regions across the globe such as North America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. While Hemophilia Gene Therapy Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets. Hemophilia Gene Therapy Market in South, America region is also expected to grow in near future.

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The Hemophilia Gene Therapy Market report highlights is as follows:

This Hemophilia Gene Therapy market report provides complete market overview which offers the competitive market scenario among major players of the industry, proper understanding of the growth opportunities, and advanced business strategies used by the market in the current and forecast period.

This Hemophilia Gene Therapy Market report will help a business or an individual to take appropriate business decision and sound actions to be taken after understanding the growth restraining factors, market risks, market situation, market estimation of the competitors.

The expected Hemophilia Gene Therapy Market growth and development status can be understood in a better way through this five-year forecast information presented in this report

This Hemophilia Gene Therapy Market research report aids as a broad guideline which provides in-depth insights and detailed analysis of several trade verticals.

About Fact.MR

Fact.MR is a global market intelligence company providing business information reports and services. The companys exclusive blend of quantitative forecasting and trend analysis provides forward-looking insight for thousands of decision makers. Fact.MRs experienced team of analysts, researchers, and consultants use proprietary data sources and various tools and techniques to gather and analyze information.

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Hemophilia Gene Therapy Market to Generate Huge Revenue in Industry by 2026 - Monroe Scoop

- New company formed from combination of TranscripTx and ReCode Therapeutics

- Advancing mRNA-mediated protein replacement and tRNA NanoCorrector therapies for primary ciliary dyskinesia and cystic fibrosis

- Developing proprietary non-viral lipid nanoparticle delivery platform for organ-specific delivery of RNA therapies and gene editing components

MENLO PARK, Calif. & DALLAS--(BUSINESS WIRE)-- ReCode Therapeutics (ReCode) (the Company), a private biopharmaceutical company pioneering precision medicines for pulmonary diseases, today announced the close of an oversubscribed $80 million Series A financing round. OrbiMed Advisors LLC and Colt Ventures co-led the round, with participation from MPM Capital, Vida Ventures LLC, Hunt Technology Ventures, L.P. and Osage University Partners. ReCode will use the proceeds to continue the preclinical development of its lead programs in primary ciliary dyskinesia (PCD) and cystic fibrosis (CF). The Company expects to file an Investigational New Drug Application (IND) for both programs in 2021. In addition, the Company will advance its proprietary non-viral lipid nanoparticle (LNP) delivery platform for organ-specific delivery of RNA therapies and gene editing components.

Our preclinical studies demonstrate that our targeted RNA therapies have great potential for the treatment of life-threatening pulmonary diseases, commented David Lockhart, Ph.D., CEO and president, ReCode Therapeutics. We are pleased to close this financing round with world-class investors who believe in the bold vision of our new company. With these additional resources, were focused on advancing our preclinical programs into the clinic over the next two years.

Lockhart continued, Im especially grateful to ReCodes founders, professors Daniel Siegwart, Ph.D., and Philip Thomas, Ph.D., at the University of Texas Southwestern Medical Center, and Professor Emeritus Arthur Johnson from Texas A&M University whose foundational research played an instrumental role in cultivating both the LNP delivery platform and the CF therapeutic program. Finally, we are grateful to the Cystic Fibrosis Foundation, whose financial support has allowed us to play a pivotal role in understanding the pathogenesis of CF and led to the development of a novel therapeutic approach for correcting nonsense mutations for this severe disease.

Along with the financing, ReCode has appointed a new board of directors. The directors comprise representatives from the Company including Dr. David Lockhart and key investors including Dr. Peter Thompson, partner at OrbiMed, Ed Hurwitz, J.D., managing director at MPM Capital, Helen S. Kim, managing director at Vida Ventures, R.A. Session II, chief business officer of the gene therapy subsidiaries at BridgeBio, and ReCode founder and new vice president of R&D, Dr. Michael Torres.

We believe that ReCode has an exceptional opportunity to advance its targeted RNA therapies and LNP delivery platform to address the critical needs of patients living with devastating genetic respiratory diseases, said Dr. Peter Thompson, partner, OrbiMed. The board looks forward to supporting ReCodes evolution as it moves these compelling therapies into the clinic.

Chardan acted as sole placement agent on the Series A financing, and as M&A advisor to ReCode Therapeutics.

About ReCode Therapeutics

ReCode Therapeutics is a biopharmaceutical company developing precision medicines for genetic respiratory diseases with significant unmet medical need. ReCodes diverse pipeline includes lead programs for primary ciliary dyskinesia and nonsense mutations in cystic fibrosis. The Companys proprietary non-viral lipid nanoparticle platform enables the delivery of a variety of payloads, and precise organ targeting of nucleic acid and protein payloads via both systemic and local delivery. ReCode is leveraging its nanoparticle and nucleic acid technologies across multiple modalities, including mRNA-mediated protein replacement therapies, first-in-class tRNA NanoCorrectors for diseases caused by nonsense mutations, and gene editing. For more information, visit http://www.recoderx.com

About OrbiMed Advisors LLC

OrbiMed is a leading healthcare investment firm, with over $14 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, utilizing a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed maintains offices in New York City, San Francisco, Shanghai, Hong Kong, Mumbai and Herzliya. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and global team resources and support to help build world-class healthcare companies. To learn more, visit http://www.orbimed.com.

About Colt Ventures

Colt Ventures is a family office that was established in 2003 to invest the capital of Darren Blanton. Colt invests in a variety of private and public companies, and has deep domain expertise in biotechnology and energy. Dr. Sundeep Agrawal leads the firms investment activities with a focus on biotechnology/therapeutics. Colt is headquartered in Dallas with a presence in New York City. To learn more, visit http://www.coltventures.com.

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ReCode Therapeutics Raises $80 Million in Oversubscribed Series A Financing - BioSpace

The "Stem Cell and Gene Therapy Biological Testing Market, Size, Share, Outlook and Growth Opportunities 2019-2025" report has been added to ResearchAndMarkets.com's offering.

Stem Cell and Gene Therapy Biological Testing market outlook report presents key insights into global Stem Cell and Gene Therapy Biological Testing markets and companies with emphasis on market size, growth factors, R&D investments, market shares and 2019 market dynamics.

The report is structured to understand the key strategies of Stem Cell and Gene Therapy Biological Testing companies in terms of their investments, product portfolio, potential opportunities and future plans. The Stem Cell and Gene Therapy Biological Testing report provides 7-year outlook on market size across different Stem Cell and Gene Therapy Biological Testing types, applications, end-user industries and countries.

Scope of the report

Key Topics Covered:

1. Table of Contents

2. Global Stem Cell and Gene Therapy Biological Testing Industry Overview

2.1 Key Findings

2.2 Market Definition

2.3 Industry Overview

2.4 Report Guide and Research Methodology

3. Executive Summary

3.1 Key Trends Shaping the Future of Stem Cell and Gene Therapy Biological Testing Market to 2025

3.2 Key focus areas of Leading Manufacturers in the market

3.3 Potential Application Segments with strong growth prospects, 2019-2025

3.4 Key Emerging Markets vital for growth of Stem Cell and Gene Therapy Biological Testing Market

3.5 Prominent Types of Stem Cell and Gene Therapy Biological Testing set to Gain Market Shares, 2019-2025

4. Stem Cell and Gene Therapy Biological Testing Market Strategic Analysis Review

4.1 Porter's Five Forces Analysis of Global Stem Cell and Gene Therapy Biological Testing Market

4.2 Supply Chain Analysis of Stem Cell and Gene Therapy Biological Testing Industry

4.3 Pricing Analysis and Forecasts

4.4 SWOT Analysis

5. Global Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

5.1 Global Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

5.2 Global Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

5.3 Global Stem Cell and Gene Therapy Biological Testing Market Outlook by Region, 2019-2025

6. Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

6.1 Introduction

6.2 Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

6.3 Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

6.4 Asia Pacific Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

6.5 Leading Companies

7. Europe Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

7.1 Introduction

7.2 Europe Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

7.3 Europe Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

7.4 Europe Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

7.5 Leading Companies

8. North America Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

8.1 Introduction

8.2 North America Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

8.3 North America Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

8.4 North America Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

8.5 Leading Companies

9. Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook and Growth Opportunities

9.1 Introduction

9.2 Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook by Type, 2019-2025

9.3 Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook by Application, 2019-2025

9.4 Rest of World (RoW) Stem Cell and Gene Therapy Biological Testing Market Outlook by Country, 2019-2025

9.5 Leading Companies

10. Competitive Landscape

10.1 Leading Players

10.2 Market Shares of Top Companies in Revenue Terms, 2018

10.3 Company Benchmarking (Peer-to-Peer Comparison)

10.4 Financial Analysis

11. Business Profiles of Leading Stem Cell and Gene Therapy Biological Testing Companies

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12. Stem Cell and Gene Therapy Biological Testing Market Recent News and Deals Landscape

12.1 Mergers and Acquisitions

12.2 Stem Cell and Gene Therapy Biological Testing Market New Product Launches

12.3 Asset Transactions

12.4 Financial Announcements

13. Appendix

For more information about this report visit https://www.researchandmarkets.com/r/ol7uf2

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Contacts

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Global Stem Cell and Gene Therapy Biological Testing Market (2019 to 2025) - Potential Growth Opportunities and Areas of Focus -...

Three years after a brain cancer failure sent the company reeling, VBL Therapeutics is touting its first hint of positive Phase III results.

The Israeli biotech announced its lead drug, VB111, met an interim efficacy benchmark in a trial testing it against standard-of-care alone in recurrent, chemotherapy-resistant ovarian cancer. The patients in the treatment arm had a CA-125 response rate a measure of cancer antigen often used as a proxy in ovarian cancer studies and in detecting ovarian cancer at least 10% higher than those in the control, the independent review determined.

The response rate in the first 60 enrolled and evaluable patients was 53%. That indicated a treatment response rate of at least 58% an encouraging number, the company said, because of the CA-125 data from their earlier Phase II trial.

We are very pleased by the outcome of this interim analysis, which demonstrates the potential benefit of VB-111 over standard-of-care in a randomized-controlled study, VBL CEO Dror Harats said. The OVAL Phase 3 interim data are at least as good as the CA-125 response results observed in our VB-111 Phase 2 study.

In that earlier study, which indicated a dose-dependent response, patients who showed a CA-125 response ultimately had an overall survival rate of808 days, versus 351 days for those who did not. The primary endpoint for this study, which is set to be completed in 2022, is overall survival.

The data come two years after VBL announced the combo of VB-111 and Avastin had failed to beat out Avastin alone in a Phase III study of glioblastoma, a notoriously hard-to-treat indication. It was their first Phase III trial after a smattering of Phase II trials in different solid tumors had turned up mixed results.

VB-111 is what the company deems a gene therapy agent. It uses an adenovirus the viral vector traditionally used for gene therapy that carries a gene to cause explosive cell death in tumors, rupturing blood cells. They have anti-inflammatory and other cancer programs in development.

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VBL and its gene therapy cancer treatment are back with a peek at PhIII potential - Endpoints News

Basel, March 27, 2020 AveXis, a Novartis company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)has adopted a positive opinion recommending conditional marketing authorization of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. A rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, SMA results in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.2,3 Zolgensma is a one-time gene therapy designed to address the genetic root cause of the disease by replacing the function of the missing or nonworkingSMN1gene. Zolgensma is administered during a single intravenous (IV) infusion, delivering a new working copy of the SMN gene into a patients cells, halting disease progression. The positive opinion is an important step towards offering a new treatment option in Europe for babies and young children with SMA.

The European Commission (EC) reviews the CHMP recommendation and usually delivers its final decision in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway, Liechtenstein and the United Kingdom.

Todays positive CHMP opinion for Zolgensma marks a critical step closer to EC approval and to bringing the only gene therapy for SMA to Europe, helping to address the devastating impact the disease has on patients and their families, said Dave Lennon, president of AveXis. Zolgensma provides a transformational new way to treat this rare but debilitating disease delivering a potentially life-saving medicine with a one-time administered treatment. Given the urgency to treat SMA and the novel nature of gene therapy, we need to be equally innovative in advancing access, so we are offering governments and reimbursement bodies a Day One access program to enable rapid access to Zolgensma upon approval.

In the most severe forms of the disease, children who are not treated are unable to lift their heads, sit, stand, or even swallow, and typically do not survive beyond two years of age unless permanently ventilated, said Dr. Francesco Muntoni, Professor and Pediatric Neurologist at Great Ormond Street Hospital for Children, London. The results we have seen for Zolgensma to date from the STR1VE clinical trial show an impressive survival rate at the conclusion of the study, with the majority of patients being able to sit without support. And through follow-up on the START trial, an average of 4.5 years later, we can see the long-term potential this significant gene therapy may have for children with this rare disease.

The CHMP positive opinion is based on the completed Phase 3 STR1VE-US and Phase 1 START trials that evaluated the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients <6 months of age at dosing, who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. STR1VE-EU, a comparable Phase 3 study is ongoing. Zolgensma demonstrated prolonged event-free survival; rapid motor function improvement, often within one month of dosing; and, sustained milestone achievement, including the ability to sit without support, a milestone never achieved in untreated Type 1 patients.4

Additional supportive data included interim results from the ongoing SPR1NT trial, a Phase 3, open-label, single-arm study of a single, one-time IV infusion of Zolgensma in presymptomatic patients (<6 weeks at age of dosing) genetically defined by bi-allelic deletion of SMN1 with 2 or 3 copies of SMN2. These data demonstrate rapid, ageappropriate major milestone gain, reinforcing the critical importance of early intervention in SMA patients.4

The most commonly observed side effects after treatment were elevated liver enzymes and vomiting. Acute serious liver injury and elevated aminotransferases can occur. Patients with pre-existing liver impairment may be at higher risk. Prior to infusion, physicians should assess liver function of all patients by clinical examination and laboratory testing. And, they should administer systemic corticosteroid to all patients before and after treatment, and then continue to monitor liver function for at least 3 months after infusion.4

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We are delighted to know that EMA considers a new treatment effective to fight SMA and that it can benefit a part of our community. We rely on all relevant stakeholders, to work at their best to get it to patients without any delay. SMA Europe will continue working to ensure that all patients living with SMA in Europe have the possibility to access any treatment that can be beneficial for them in a timely and sustainable way, said Menca de Lemus, President of SMA Europe.

Zolgensma Day One access program

SMA is a significant burden to the healthcare system in Europe with cumulative estimated healthcare costs per child ranging between 2.5 to 4 million within the first 10 years alone.1Designed to work within existing pricing and reimbursement frameworks, yet recognizing the novel nature of a one-time gene therapy for a devastating and progressive disease, the Day One access program offers ministries of health and reimbursement bodies (in countries without pre-existing early access pathways) a variety of flexible options that can be implemented immediately at time of approval. The program is designed to ensure that the cost of patients treated before national pricing and reimbursement agreements are in place, are aligned with the value-based prices negotiated following clinical and economic assessments. The program is meant to ensure the continued integrity of the local pricing and reimbursement framework. AveXis is already in advanced discussions with multiple countries in Europe to agree on terms of the program. The following options can be customized for each country:

About Spinal Muscular AtrophySMA is the leading genetic cause of infant death.2,3 If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.5In Europe each year, approximately 550600 infants are born with SMA.6,7 SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.2 It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.8 This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities.9 More than 30% of patients with SMA Type 2 will die by age 25.10

Novartis will conduct a conference call with investors to discuss this news release on Monday, March 30, 2020 at 3 p.m. Central European Time and 9 a.m. Eastern Time. A simultaneous webcast of the call for investors and other interested parties may be accessed by visiting the Novartis website. A replay will be available after the live webcast by visiting https://www.novartis.com/investors/event-calendar.

About Zolgensma (onasemnogene abeparvovec)Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma represents the first approved therapeutic in the companys proprietary platform to treat rare, monogenic diseases using gene therapy.6 Approximately 400 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program in the U.S. AveXis is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada and Australia in late 2020 or early 2021.6

AveXis has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Genethon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

InMay 2019, the U.S. Food and Drug Administration approved Zolgensma for the treatment of pediatric patients less than two years of age with SMA with bi-allelic mutations in the SMN1 gene. 11 In the U.S. nearly all on-label patients have been approved by their payer for access to Zolgensma. On March 19, 2020, Zolgensma was approved by Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at diagnosis. Reimbursement with MHLW is expected by the end of 1H20, pending agreement Zolgensma will be available at that time.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as positive CHMP opinion, offering, recommending, step towards, step closer, potential, can, will, plan, may, could, would, expect, anticipate, pipeline, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for Zolgensma, or regarding potential future revenues from Zolgensma. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Zolgensma, will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that Zolgensma will be commercially successful in the future. In particular, our expectations regarding Zolgensma could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.About AveXisAveXis, a Novartis company, is the worlds leading gene therapy company, redefining the possibilities for patients and families affected by life-threatening genetic diseases through our innovative gene therapy platform. Founded in 2013 and headquartered in Bannockburn, IL, the goal of AveXis cutting-edge science is to address the underlying, genetic root cause of diseases. AveXis pioneered foundational research, establishing AAV9 as an ideal vector for gene transfer in diseases affecting the central nervous system, laying the groundwork to build a best-in-class, transformational gene therapy pipeline. AveXis received its first U.S. Food and Drug Administration approval in May 2019 for the treatment of spinal muscular atrophy (SMA). AveXis is also developing therapies for other genetic diseases, including Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) SOD1 and Friedreichs ataxia. For additional information, please visitwww.avexis.com.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 145 nationalities work at Novartis around the world. Find out more atwww.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisor follow @NovartisNews for the latest News & Media Updates at https://twitter.com/novartisnews.For Novartis multimedia content, please visit https://www.novartis.com/news/media-library.For questions about the site or required registration, please contact media.relations@novartis.com

References1. Estim. 10-year cumulative SMA costs for major EU markets based on available data including local healthcare resource utilizations studies, local databases and public information from previous SMA therapy economic assessments, as of February 21, 2020.2. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15(8):895-908.3. National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Accessed October 9, 20184. STR1VE-US, START and SPR1NT clinical data on file5. Finkel RS, et al. Neurology. 2014;83(9):810-817.6. Data on file.7. Verhaart IEC, et al. J Neurol. 2017;264(7):1465-1473.8. SolerBotija C, et al. Brain. 2002;125(7):1624-1634.9. Wang CH, et al. J Child Neurol. 2007;22(8):1027-1049.10. Darras BT, Finkel RS Spinal Muscular Atrophy. Chapter 25 - Natural History of Spinal Muscular Atrophy. October 2017.11. FDA approval: https://www.fda.gov/vaccines-blood-biologics/zolgensma

# # #

Novartis Media RelationsCentral media line: +41 61 324 2200E-mail: media.relations@novartis.com

Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

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AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy (SMA) - Yahoo Finance

Dosing Initiated in PROPEL Phase 1/2 Trial of PR001 for Treatment of Parkinsons Disease with GBA1 Mutations

Preparations Underway to Initiate PROVIDE Phase 1/2 Trial of PR001 for Treatment of Type 2 NeuronopathicGaucher Disease in Mid-2020

IND for PR006 for Treatment of Patients with Frontotemporal Dementia with GRN Mutation Active; Initiation of PROCLAIM Phase 1/2 Trial Planned for Mid-2020

Cash Runway into First Half of 2022

NEW YORK, March 26, 2020 (GLOBE NEWSWIRE) --Prevail TherapeuticsInc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reported financial results for the full year ended December 31, 2019, and reviewed recent business highlights.

2019 was an important year for Prevail as we continued to make important clinical and regulatory progress across our portfolio of novel AAV9-based gene therapy candidates for neurodegenerative diseases and brought the company public, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. Dosing has begun in our PROPEL clinical trial of PR001 for patients with Parkinsons disease with GBA1 mutations, or PD-GBA. Additionally, we are working to initiate three more Phase 1/2 clinical trials this year: the PROVIDE and PROGRESS trials of PR001 for the treatment of Type 2 and Type 3 neuronopathic Gaucher disease, or nGD, respectively, and the PROCLAIM trial of PR006 for the treatment of frontotemporal dementia patients with GRN mutation, or FTD-GRN potentially the first gene therapy for this condition to enter clinical trials. Our ability to advance multiple candidates towards the clinic over the last year reflects the dedication of our team, the potential reach of our platform, and the urgent need for novel potentially disease-modifying therapies for patients with these genetically-defined neurodegenerative diseases.

Recent Business Highlights and Updates:

Full Year 2019 Financial Results

About Prevail Therapeutics Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinsons disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with a GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinsons with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential impact of COVID-19 on Prevails ongoing and planned clinical trials, business and operations; the potential of Prevails gene therapies to modify the course of neurodegenerative diseases; the anticipated timing of Prevails Phase 1/2 clinical trials of PR001 in PD-GBA and in nGD and Prevails clinical trial of PR006; and the potential impacts of Orphan Drug and Rare Pediatric Disease Designations. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises.

These and other risks are described more fully in Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, the Companys Annual Report on Form 10-K for the fiscal year ended December 31, 2019, to be filed with the SEC on or about March 26, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Prevail Therapeutics Inc.Statements of Operations(in thousands, except share and per share data)

Prevail Therapeutics Inc.Balance Sheets(in thousands, except share and per share data)

Media Contact:Mary CarmichaelTen Bridge Communicationsmary@tenbridgecommunications.com

Investor Contact: investors@prevailtherapeutics.com

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Prevail Therapeutics Reports Full Year 2019 Financial Results and Business Highlights - BioSpace

Over 95% planned enrollment completed in Phase 3 oGVHD study; topline results anticipated by end of 2020

Conference Call and Webcast Today at 8:30 a.m. ET

MALVERN, Pa., March 27, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat rare and underserved ophthalmic diseases, today reported full year 2019 financial results along with a general business update.

We are extremely pleased with the progress we have made in enrolling patients in our Phase 3 clinical trial for OCU300, an orphan drug candidate for ocular graft versus host disease (oGVHD). Based on current enrollment, we anticipate topline results by the end of the year, commented Shankar Musunuri, PhD, MBA, Chairman, CEO and Co-Founder of Ocugen. The publication of preclinical data on OCU400 in Nature Gene Therapy earlier this month is a key achievement for this program. We continue to advance IND-enabling studies toward bringing this potential breakthrough modifier gene therapy platform to patients in a Phase 1/2a clinical trial for OCU400 in 2021. Similar to the situation with virtually all other biopharmaceutical companies, we are also assessing the potential impact of ongoing COVID-19 pandemic-related events on our programs and plans. We are grateful to healthcare professionals and others who are working hard to address and mitigate the challenges presented by the virus.

Business Highlights:

-- OCU300 (oGVHD) In December 2019, Ocugen announced 50% enrollment in its Phase 3 trial, and as of March 20, 2020, Ocugen has completed over 95% of the planned enrollment. Ocugen is anticipating topline results by the end of the year. -- OCU400 (AAV-NR2E3) On March 3, 2020, Nature Gene Therapy published preclinical data related to OCU400, supporting the use of nuclear hormone receptor gene NR2E3 as a genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases and potentially serve as a broad-spectrum therapy for retinitis pigmentosa. -- EB-5 Loan Agreement On March 26, 2020, Ocugen drew down an additional $0.5 million under its loan agreement with EB5 Life Sciences, L.P.

Full Year 2019 Financial Results:

-- Ocugens cash, cash equivalents and restricted cash totaled $7.6 million as of December 31, 2019, compared to $1.8 million as of December 31, 2018. -- Research and development expenses for the year ended December 31, 2019 were $8.1 million compared to $10.3 million for the year ended December 31, 2018. General and administrative expenses for the year ended December 31, 2019 were $6.1 million compared to $5.8 million for the year ended December 31, 2018. -- Ocugen reported a net loss of $20.2 million, or $1.46 loss per share, for the year ended December 31, 2019, compared to a net loss of $18.2 million, or $3.67 loss per share, for the year ended December 31, 2018. -- The Company had 52,625,228 shares of common stock outstanding as of December 31, 2019.

Conference Call and Webcast Details

The Company has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the results and recent business highlights. Ocugens senior management team will host the call, which will be open to all listeners. There will also be a question and answer session following the prepared remarks.

The call can be accessed by dialing (844) 873-7330 (U.S.) or (602) 563-8473 (international) and providing the conference ID 5373849. To access a live audio webcast of the call on the Investors section of the Ocugen website, please click here. A replay of the webcast will be archived on Ocugens website for approximately 45 days following the call.

About Ocugen, Inc. Ocugen, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing transformative therapies to treat the whole eye. Our Phase 3 small molecule drug candidate for ocular Graft Versus Host Disease (oGVHD), if approved, will be the first and only treatment for this orphan disease. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many. And our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

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

(tables to follow)

OCUGEN, INC.CONSOLIDATED BALANCE SHEETS(UNAUDITED)

December 31, December 31, 2019 2018 Assets Current assets Cash and cash equivalents $ 7,444,052 $ 1,628,136 Prepaid expenses and other current assets 1,322,167 313,499 Asset held for sale 7,000,000 Total current assets 15,766,219 1,941,635 Property and equipment, net 222,464 245,788 Restricted cash 151,016 150,477 Other assets 667,747 116,333 Total assets $ 16,807,446 $ 2,454,233 Liabilities and stockholders equity (deficit) Current liabilities Accounts payable $ 1,895,613 $ 3,277,525 Accrued expenses 2,270,045 1,402,750 Short-term debt, net 7,483,847 Derivative liabilities 1,741,222 Operating lease obligation 172,310 Other current liabilities 205,991 204,242 Total current liabilities 4,543,959 14,109,586 Non-current liabilities Operating lease obligation, less current portion 163,198 Long term debt, net 1,072,123 1,016,727 Other non-current liabilities 9,755 37,459 Total liabilities 5,789,035 15,163,772 Stockholders equity (deficit) Common stock 527,467 49,606 Treasury stock (47,864 ) Accumulated other comprehensive income 451 Additional paid-in capital 62,018,632 18,477,598 Accumulated deficit (51,479,824 ) (31,237,194 ) Total stockholders equity (deficit) 11,018,411 (12,709,539 ) Total liabilities and stockholders equity (deficit) $ 16,807,446 $ 2,454,233 - ---------- - - --------- -

OCUGEN, INC.CONSOLIDATED STATEMENTS OF OPERATIONS(UNAUDITED)

Year ended December 31, 2019 2018 Operating expenses: Research and development $ 8,085,522 $ 10,321,397 General and administrative 6,077,097 5,819,111 Total operating expenses 14,162,619 16,140,508 Loss from operations (14,162,619 ) (16,140,508 ) Other income (expense) Change in fair value of derivative liabilities (3,187,380 ) 1,664,689 Loss on debt conversion (341,136 ) Interest income 1,214 19,213 Interest expense (1,767,836 ) (3,750,630 ) Other income (expense) (784,873 ) (12,428 ) Total other income (expense) (6,080,011 ) (2,079,156 ) Net loss $ (20,242,630 ) $ (18,219,664 ) Net loss per share of common stockbasic and diluted $ (1.46 ) $ (3.67 ) Weighted average common shares outstandingbasic and diluted 13,893,819 4,960,552 ------------- - ------------- -

Corporate Contact: Ocugen, Inc. Kelly Beck kelly.beck@ocugen.com +1 484-328-4698 Media Contact: LaVoieHealthScience Emmie Twombly etwombly@lavoiehealthscience.com +1 857-389-6042

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Ocugen Provides Business Update and Full Year 2019 Financial Results - Associated Press

For biotechnology companies, responding to a sudden public health crisis, like the coronavirus, can be risky business. It can take more than a year to develop a vaccine or treatment. By that time, the threat may be gone, leaving little or no demand for a drug.

The current coronavirus pandemic appears to be different.

The race to beat the pathogen is on, with many biotechs now expecting a large market for coronavirus therapies in 2021 or beyond.But the starting gun didn't fire right away when the virus began spreading late last year.

"There were a lot of companies that kind of felt once bitten, twice shy," saidJose Trevejo, chief executive of SmartPharm Therapeutics in Kendall Square.

Companies were shy because drug makers have been bitten in the past when they've hustled to confront an outbreak, only to see the danger subside and their efforts go to waste. That's what happened during a previous coronavirus scare the SARS outbreak of 2003, which ended before any vaccine maker could earn a buck.

"There was also Ebola, and then a lot of people forget about the swine flu the H1N1," Trevejo said. "So, I think a lot of big pharma were a bit hesitant to plunge fully into coronavirus."

In recent weeks, the industry's view has changed. At LabCentral, the shared workspace where Trevejo's gene therapy startup operates, more than a dozen companies have turned their attention to the coronavirus, including SmartPharm.

And that's just one lab in Cambridge.

The number of companies pursuing coronavirus drugs is constantly growing, according to Yasmeen Rahimi, co-head of biotechnology research at Roth Capital Partners, an investment banking firm in Newport Beach, Calif.

"I track COVID-19 on a daily basis," she said. "Almost every day we get 10 or 12 companies that are coming."

Rahimi said the most notable may be Moderna, another Cambridge biotech. It's the first company to begin testing a potential coronavirus vaccine on humans in the U.S.

Moderna didn't respond to an interview request. But in a public document this week, the company said that "a commercially-available vaccine is not likely to be available for at least 12-18 months."

Moderna doesn't think the virus will peter out by then. Instead, it is "scaling up manufacturing capacity toward the production of millions of doses per month."

That may not be enough, according to Dr. Lee Wetzler, an infectious disease specialist at Boston Medical Center.

"Just like the flu vaccine, you're talking not just millions of doses," said Wetzler, who is also a professor at Boston University School of Medicine. "Tens tens of millions of doses and even more."

Then again, Wetzler added, it's hard to predict what this previously unknown virus will look like a year from now. It's possible that the pandemic will be under control, which would be good for the world but maybe not so good for companies, like Moderna and others, investing in drugs to stop it.

There's a cautionary tale in theonce-hot biotech Vical, which received federal money to chase vaccines for anthrax, SARS and Ebola. The fear and the funding always faded before Vical could cross the finish line, said John Carroll, editor of Endpoints News, a biotech publication.

"What Vical did was just flame out," he said. "They weren't able to come up with a vaccine and, after repeated failures, they just couldn't make it anymore."

The risk of failure is well understood by James Sietstra, chief business officer of Totient, one of the startups at LabCentral that have shifted to the coronavirus. But he maintains that working on the coronavirus won't be futile, even if the disease runs its course before Totient can bring a drug to market.

"As a business case for us, this is a great proof point for the power of our platform," he said.

Sietstra said the same method Totient will use to attack the coronavirus could also be used against other diseases including the company's original target, cancer.

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Vaccine Development Is Risky Business. Biotechs Are Tackling The Coronavirus, Anyway - WBUR

Patients urged to seek official medical advice to prevent surge in unethical treatments outside clinical pathways

Vancouver, Canada, March 25, 2020 -The International Society for Cell & Gene Therapy (ISCT), the global society of clinicians, researchers, regulators, technologists, and industry partners dedicated to the translation of cellular therapy into safe and effective therapies to improve patients lives, today announces it is discussing with scientific and industry organizations and regulators a range of actions against the proliferation of unproven and untested stem cellbased therapies with claims to treat COVID-19. It also urges patients and families to consult expert medical professionals for the most accurate available information on treatment options.

Organizations across the life science industry, including the cell and gene therapy (CGT) sector, are currently united in the key focus of finding evidence-based treatments and vaccines for the current COVID-19 pandemic. These therapies and vaccines could save vast numbers of lives globally, said Dr Daniel Weiss, MD, PhD, Chief Scientific Officer of ISCT. It is critical that these vital and urgent efforts are not hampered by unscrupulous companies profiting by offering false and unproven interventions. These false interventions can cause immense harm by eroding the confidence of the public, investors, and the wider communities in the medical and biopharma sectors at the very time it is needed most.

ISCT set up its Presidential Task Force in 2014 in its ongoing commitment to address the rise of commercially available unproven cell and gene therapy treatments. In October 2019, it formed a cell and gene therapy sector-wide coalition of organizations across the globe to combat the rise in the number of unproven commercial cell banking services. The PTF also publishes a biennial report detailing global CGT with market authorization which patients and physicians can utilize to make informed decisions on treatments. ISCT is currently in conversations with its consortia partners, as well as regulators and governments globally, regarding options to prevent organizations operating outside of current clinical guidance.

Prior to the current COVID-19 pandemic, ISCT identified that the unproven cell therapy market is currently worth up to USD 2.4 billion and involves approximately 60,000 patients annually, paying up to USD 40,000 per treatment. ISCT believes that during the current COVID-19 climate, the number of patients and the costs of these treatments will considerably increase.

The current climate of a quickly spreading COVID-19 infection and a lack of approved treatments creates a perfect storm that results in a plethora of unethical organizations offering unproven therapies. These organizations offering untested therapies outside of proper clinical pathways for COVID-19 create a real and immediate risk to life. These treatments often lack proof of efficacy, and potentially have a very high cost to desperate patients. Most importantly, these treatments produced with no evidence can be even more dangerous to the immediate health of patients and their communities, said Dr. Laertis Ikonomou, PhD, co-chair of the ISCT PTF on the Use of Unproven and/or Unethical Cell and Gene Therapy. ISCT will work with government agencies and regulatory bodies internationally to take action against those looking to profit from a pandemic that is affecting so many people across the globe.

About the International Society for Cell & Gene Therapy

Established in 1992, the International Society for Cell & Gene Therapy (ISCT) is a global society of clinicians, regulators, researchers, technologists and industry partners with a shared vision to translate cellular and gene therapy into safe and effective therapies to improve patients lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell and gene-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory and Commercialization. Through strong relationships with global regulatory agencies, academic institutions and industry partners, ISCT drives the advancement of research into standard of care.Comprised of over 1700 cell and gene therapy experts across five geographic regions and representation from over 60 countries, ISCT members are part of a global community of peers, thought leaders and organizations invested in cell and gene therapy translation. For more information about the society, key initiatives and upcoming meetings, please visit:www.isctglobal.org,@ISCTglobal.

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ISCT takes a stand against organizations marketing unproven COVID-19 cell and gene therapies - PharmiWeb.com

Global Gene Therapy for Inherited Genetic Disorders Market: Overview

Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

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Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Some of the bigplayerseyeing promising stakes in the gene therapy for inherited genetic disorders market areSpark Therapeutics Inc., Orchard Therapeutics, Novartis AG, bluebird bio Inc., and BioMarin Pharmaceutical.

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Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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Gene Therapy for Inherited Genetic Disorders Market Expected to Witness Significant Growth in the Forecast Years 2028 - Daily Science

REDWOOD CITY, Calif., March 23, 2020 (GLOBE NEWSWIRE) -- Codexis, Inc., a leading protein engineering company and developer of novel biotherapeutics, announces the signing of a strategic collaboration and license agreement with Takeda Pharmaceutical Company Limited (Takeda) for the research and development of novel gene therapies for certain disease indications, including the treatment of lysosomal storage disorders and blood factor deficiencies.

Under the terms of the agreement, Codexis will generate novel gene sequences encoding protein variants tailored to enhance efficacy as a result of increased activity, stability, and cellular uptake using its CodeEvolver protein engineering platform. Takeda will combine these improved transgenes with its gene therapy capabilities to generate novel candidates for the treatment of rare genetic disorders.

Our CodeEvolver platform technology enables the rapid engineering of novel genetic sequences that encode more efficacious proteins. The prospects of these improved sequences for the development of differentiated gene therapies for patients with rare diseases therefore holds great promise, stated John Nicols, Codexis President and CEO. Takedas expertise in developing novel treatments for patients with rare genetic disorders, and its commitment to developing the best possible gene therapies, makes them an ideal partner for our growing Novel Biotherapeutics business unit.

Gjalt Huisman, Codexis Senior Vice-President, and General Manager, Novel Biotherapeutics added, We are looking forward to working with Takeda to advance our pre-clinical assets for lysosomal storage disorders, and to broaden our biotherapeutics pipeline to now include blood factor disorders.

Terms of AgreementUnder the terms of the agreement, the parties will begin collaborative work on three initial programs. Codexis is responsible for the creation of novel enzyme sequences for advancement as gene therapies into pre-clinical development. Takeda is responsible for the pre-clinical and clinical development and commercialization of gene therapy products resulting from the collaboration programs. Under the terms of the agreement, in addition to the three initial programs, Takeda may initiate up to four additional programs for separate target indications. Subject to the terms of the agreement, Codexis is eligible to receive an upfront payment, reimbursement for research and development fees, development and commercial milestone payments, and low- to mid-single digit percentage royalties on sales of any commercial product developed through such initial programs and any other programs that Takeda may elect under the agreement. Back Bay Life Science Advisors served as strategic and financial advisors to Codexis.

About Codexis, Inc.Codexis is a leading protein engineering company that applies its proprietary CodeEvolver protein engineering technology to develop proteins for a variety of applications, including enzymes as biotherapeutics, as biocatalysts for the commercial manufacture of pharmaceuticals and fine chemicals, industrial enzymes, and for use in molecular diagnostics. For its Biotherapeutics pipeline, Codexis technology enables improvements in protein efficacy, through enhancement of activity, affinity, stability, as well as uptake by target cells. For more information, see http://www.codexis.com.

Forward-Looking StatementsTo the extent that statements contained in this press release are not descriptions of historical facts regarding Codexis, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including Codexis expectations regarding the prospects for the development and future commercialization by Takeda of novel gene therapies for specified target indications. You should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors that are, in some cases, beyond Codexis control and that could materially affect actual results. Factors that could materially affect actual results include, among others: Codexis dependence on its licensees and collaborators; Codexis dependence on a limited number of products and customers; and potential adverse effects to Codexis business if its customers products are not received well in the markets. Additional information about factors that could materially affect actual results can be found in Codexis Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 28, 2020, including under the caption Risk Factors and in Codexis other periodic reports filed with the SEC. Codexis expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

Investor Contact: LHA Investor RelationsJody Cain, 310-691-7100jcain@lhai.com

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Codexis Signs Strategic Collaboration and License Agreement with Takeda to Advance Novel Gene Therapies for Rare Genetic Disorders - GlobeNewswire

After nearly a year as president and COO at Paragon Biosciences, Jim Robinson has jumped to Urovant Sciences, where he will be president and CEO of the urology-focused biotech, succeeding founding CEO Keith Katkin. Robinson will continue as a member of Urovants board of directors. His previous stops include Alkermes as president and COO and a number of positions at Astellas and Schering-Plough Pharmaceuticals.

At the same time as an $80 million partnership with Astellas, CytomX has recruited Eli Lilly vet Carlos Campoy as SVP and CFO. Most recently, Campoy served as CFO of Alder BioPharmaceuticals, helping complete its $2 billion sale to Lundbeck. Prior to that, Campoy was VP of finance, international at Allergan.

Bristol Myers former top dealmaker Paul Biondi whos currently serving as an executive partner at Flagship Pioneering has joined the board of directors at Seres Therapeutics, succeeding Flagship CEO Noubar Afeyan whos stepping down.

UK-based Immune Regulation has wooed the former CEO of SteadyMed Therapeutics (acquired by United Therapeutics) Jonathan Rigby to the helm of their company as CEO. Rigby cofounded Zogenix and earlier in his career served at Merck and Bristol Myers Squibb.

Shanghai-based EpimAb Biotherapeutics has enlisted Xinyi David Gu as CFO. Gu makes the hop over from Millennium Management, and prior to that served at Jefferies as VP and global pharmaceutical equity research analyst. In addition, Gu has held a post at McKinsey & Company.

After bagging $26 million in funding and earning fast track designation for its lead drug candidate, gene therapy company Genprex has recruited Michael Redman as EVP and COO and Catherine Vaczy as EVP and chief strategy officer. Redman was the former CEO and president of Oncolix for more than a decade. Prior to Oncolix he served in posts at Bone Medical, Opexa Pharmaceuticals and Zonagen, among others. Vaczy was a co-founder of NeoStem (now Caladrius Biosciences) and served at ImClone Systems (acquired by Eli Lilly).

Menlo Therapeutics has tapped Andrew Saik as CFO and treasurer. Saik will be based at the companys headquarters at Bridgewater, New Jersey. Most recently, Saik served as CFO of PDS Biotechnology. Prior to that, he was CFO at Vertice Pharma and Auxilium Pharmaceuticals helping in the $2.6 billion sale of the company. His previous experience includes stints at Endo Health Solutions and Valeant Pharmaceuticals.

Lisa Rojkjaer has joined Viracta Therapeutics as CMO, effective May 1. Prior to her appointment at the precision oncology company, Rojkjaer was CMO at Oslo-based Nordic Nanovector and global clinical program head, oncology global development at Novartis. Among her responsibilities will be steering Viractas lead program toward regulatory approvals for Epstein-Barr virus (EBV) positive lymphomas.

After welcoming Shafique Virani last week as chief corporate development officer, AI biotech Recursion has added Michael Secora as CFO. Secora joins the company after a decade of service at Laurion Capital, where he served as managing director and head of capital markets and venture. Near the same time as Viranis appointment last week, former Celgene exec Robert Hershberg was appointed to the companys board of directors. Hershberg served as Celgenes CSO and EVP and head of business development and global alliance.

Belgian orthopedics-focused biotech Bone Therapeutics has a new CBO, naming Stefanos Theoharis to the post. With experience in the cell and gene therapy arena, Theoharis was formerly SVP at Cell Medica and CBO at apceth GmbH.

Pravin Dugel has been appointed EVP and chief strategy and business officer of IVERIC bio, which concentrates on treatment for retinal diseases. Dugel brings more than 25 years of experience in the field to the New York biopharma and has also been managing partner at Retinal Consultants of Arizona and the Retinal Research Institute.

I-Mab has selected Gigi Feng to be its VP, global head of corporate communications starting on April 20. She will oversee all communications at the China-based biopharma, which centers on novel or highly differentiated biologics to treat diseases with significant unmet medical needs. Feng previously had communications roles at Amgen and Sanofi.

Meenu Chhabra, the CEO and president of Proteostasis Therapeutics, has hopped aboard the board of directors at Vasommune Therapeutics a company working on a treatment for Covid-19/pathogen-induced ARDS.

NASH biotech 89bio has welcomed Steven Altschuler as chairman to its board of directors. Altschuler serves as managing director of healthcare ventures at Ziff Capital Partners.

Immuneering whose current pipeline focuses on diseases such as cancer cachexia and metastasis has brought on three new additions to its scientific advisory board. Daniel Ahn, director of the GI Oncology Translational Research Working Group; Mitesh Borad, director of the cancer cell, gene and virus therapy lab at the Mayo Clinic Center for Individualized Medicine; and Bijan Nejadnik, the CMO and head of research at SanBio.

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Jim Robinson takes over the reins at Urovant; Carlos Campoy moves to CytomX - Endpoints News

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How Innovation is Changing the Gene Therapy Market - Packaging News 24