StemCell Therapy

Global Cancer Gene Therapy Market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

The report titled Global Cancer Gene Therapy Market is one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Cancer Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Cancer Gene Therapy market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Cancer Gene Therapy market is carefully analyzed and researched about by the market analysts.

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The Essential Content Covered in the Global Cancer Gene Therapy Market Report:

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 Cancer 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. Cancer Gene Therapy Market in South, America region is also expected to grow in near future.

The key players covered in this studyAdaptimmuneBluebird bioCelgeneShanghai Sunway BiotechShenzhen SiBiono GeneTechSynerGene TherapeuticsAltor BioScienceAmgenArgenxBioCancellGlaxoSmithKlineMerckOncoGenex PharmaceuticalsTransgene

Market segment by Type, the product can be split intoOncolytic VirotherapyGene TransferGene-Induced Immunotherapy

Market segment by Application, split intoHospitalsDiagnostics CentersResearch Institutes

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

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Key questions answered in the report

*What will be the market size in terms of value and volume in the next five years?

*Which segment is currently leading the market?

*In which region will the market find its highest growth?

*Which players will take the lead in the market?

*What are the key drivers and restraints of the markets growth?

We provide detailed product mapping and analysis of various market scenarios. Our analysts are experts in providing in-depth analysis and breakdown of the business of key market leaders. We keep a close eye on recent developments and follow latest company news related to different players operating in the global Cancer Gene Therapy market. This helps us to deeply analyze companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study that will help you to stay on top of the competition.

Table of Contents

1 Cancer Gene Therapy Market Overview

1.1 Product Overview and Scope of Cancer Gene Therapy

1.2 Cancer Gene Therapy Segment by Type

1.2.1 Global Cancer Gene Therapy Production Growth Rate Comparison by Type 2020 VS 2026

1.3 Cancer Gene Therapy Segment by Application

1.3.1 Cancer Gene Therapy Consumption Comparison by Application: 2020 VS 2026

1.4 Global Cancer Gene Therapy Market by Region

1.4.1 Global Cancer Gene Therapy Market Size Estimates and Forecasts by Region: 2020 VS 2026

1.4.2 North America Estimates and Forecasts (2015-2026)

1.4.3 Europe Estimates and Forecasts (2015-2026)

1.4.4 China Estimates and Forecasts (2015-2026)

1.4.5 Japan Estimates and Forecasts (2015-2026)

1.5 Global Cancer Gene Therapy Growth Prospects

1.5.1 Global Cancer Gene Therapy Revenue Estimates and Forecasts (2015-2026)

1.5.2 Global Cancer Gene Therapy Production Capacity Estimates and Forecasts (2015-2026)

1.5.3 Global Cancer Gene Therapy Production Estimates and Forecasts (2015-2026)

2 Market Competition by Manufacturers

2.1 Global Cancer Gene Therapy Production Capacity Market Share by Manufacturers (2015-2020)

2.2 Global Cancer Gene Therapy Revenue Share by Manufacturers (2015-2020)

2.3 Market Share by Company Type (Tier 1, Tier 2 and Tier 3)

2.4 Global Cancer Gene Therapy Average Price by Manufacturers (2015-2020)

2.5 Manufacturers Cancer Gene Therapy Production Sites, Area Served, Product Types

2.6 Cancer Gene Therapy Market Competitive Situation and Trends

2.6.1 Cancer Gene Therapy Market Concentration Rate

2.6.2 Global Top 3 and Top 5 Players Market Share by Revenue

2.6.3 Mergers & Acquisitions, Expansion

3 Production Capacity by Region

4 Global Cancer Gene Therapy Consumption by Regions

5 Production, Revenue, Price Trend by Type

5.1 Global Cancer Gene Therapy Production Market Share by Type (2015-2020)

5.2 Global Cancer Gene Therapy Revenue Market Share by Type (2015-2020)

5.3 Global Cancer Gene Therapy Price by Type (2015-2020)

5.4 Global Cancer Gene Therapy Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End

6 Global Cancer Gene Therapy Market Analysis by Application

6.1 Global Cancer Gene Therapy Consumption Market Share by Application (2015-2020)

6.2 Global Cancer Gene Therapy Consumption Growth Rate by Application (2015-2020)

Read More..

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Cancer Gene Therapy Market Scope Analysis 2019-2025 - Science In Me

Located on the Tufts University Medford, MA campus, this new donor center will enable delivery of fresh blood, leukopaks and buffy coats for COVID-19, cell and gene therapy research within hours of collection

WESTBURY, NY - Apr 6, 2020 - BioIVT, a leading provider of research models and services for drug and diagnostic development, today announced the opening of its new blood donor center on the Tufts University campus in Medford, MA to support academic and pharmaceutical researchers involved in COVID-19, cell and gene therapy research.

BioIVT wants to play a leading role in supporting COVID-19 research efforts and blood donations are a vital resource for the research and development of new therapies, vaccines, and diagnostics. We have many years experience developing blood products, including blood-derived immune cells for cell and gene therapy research, and we want to make that expertise count, said BioIVT CEO Jeff Gatz. Researchers recognize and appreciate BioIVTs rapid response and commitment to high quality, fresh blood products and this new donor center will allow us to offer those attributes and services to additional US clients.

BioIVTs new Boston blood donor center is its seventh. The company has similar facilities located in California, Tennessee and Pennsylvania to serve US clients and in London, UK for EU-based clients.

While the initial focus at our Boston donor center will be on delivering fresh blood, leukopaks and buffy coats within hours of collection, we plan to add more capabilities and donors over time, said Jeff Widdoss, Vice President of Donor Center Operations at BioIVT.

Leukopaks, which contain concentrated white blood cells, are used to help identify promising new drug candidates, assess toxicity levels, and conduct stem cell and gene therapy research. They are particularly useful for researchers who need to obtain large numbers of leukocytes from a single donor.

BioIVT blood products can be supplied with specific clinical data, such as the donor age, ethnicity, gender, BMI and smoking status. Its leukopaks are also human leukocyte antigen (HLA), FC receptor and cytomegalovirus typed. HLA typing is used to match patients and donors for bone marrow or cord blood transplants. FC receptors play an important role in antibody-dependent immune responses.

COVID-19-related Precautions

Blood donor centers are considered essential businesses and will remain open during the COVID-19 quarantine. BioIVT is taking additional safety measures to protect both blood donors and its staff during this difficult time. It has instituted several social distancing measures, including increasing the space between chairs in the waiting room and between donor beds, and limiting the entrance of non-essential personnel. The screening rooms are disinfected between donors and all areas of the center continue to be cleaned at regular intervals.

As soon as each blood donor signs their informed consent form, their temperature is taken. If they have a fever, their appointment is postponed, and they are referred to their physician. Any donor who develops COVID-19 symptoms after donating blood is required to inform the center immediately.

All BioIVT blood collections are conducted under institutional review board (IRB) oversight and according to US Food and Drug Administration (FDA) regulations and American Association of Blood Banks (AABB) guidelines.

Those who would like to donate blood at BioIVTs new Boston-area donor center should call 1-833-GO-4-CURE or visit http://www.biospecialty.com to make an appointment.

Further information about the products available from BioIVTs new donor center can be found at https://info.bioivt.com/ma-donor-ctr-req.

About BioIVT

BioIVT is a leading global provider of research models and value-added research services for drug discovery and development. We specialize in control and disease-state biospecimens including human and animal tissues, cell products, blood and other biofluids. Our unmatched portfolio of clinical specimens directly supports precision medicine research and the effort to improve patient outcomes by coupling comprehensive clinical data with donor samples. Our PHASEZERO Research Services team works collaboratively with clients to provide target and biomarker validation, phenotypic assays to characterize novel therapeutics, clinical assay development and in vitro hepatic modeling solutions. And as the premier supplier of hepatic products, including hepatocytes and subcellular fractions, BioIVT enables scientists to better understand the pharmacokinetics and drug metabolism of newly-discovered compounds and their effects on disease processes. By combining our technical expertise, exceptional customer service, and unparalleled access to biological specimens, BioIVT serves the research community as a trusted partner in elevating science. For more information, please visit http://www.bioivt.com or follow the company on Twitter @BioIVT.

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BioIVT Opens New Blood Donor Center to Support Boston-area Research into COVID-19 Therapies, Vaccines and Diagnostics - Bio-IT World

While the world is working to slow the COVID-19 pandemic by social isolation and quarantine, numerous companies globally are working to develop a vaccine against the virus. The most likely timelinesat bestfor a viable vaccine against SARS-CoV-2, the coronavirus causing COVID-19, are 12 to 18 months. And thats if everything goes right.

As a recent article in Bloomberg Businessweek notes, For that to happen in the next year or so, an almost equally implausible set of circumstances has to occur: flawless scientific execution, breakneck trials and a military-style manufacturing mobilization unlike any the pharmaceutical industry has put in place before.

Typically, it takes 10 to 15 years of deliberate and careful work to develop a commercial vaccineand as both the common cold and HIV have demonstrated, sometimes its not possible even then. The infrastructure for the seasonal flu vaccines that come out every year has been in place for decades.

One of the companies that is gaining a lot of attention is Cambridge, Massachusetts-based Moderna. They have a head-start and are using a novel technology to develop their COVID-19 vaccine, mRNA-1273, which is already in clinical trials. mRNA-1273 is a mRNA vaccine that encodes for a prefusion stabilized form of the Spike (S) protein. Whats different about the vaccine is that it is almost like a form of gene therapyit codes for the genetic sequence for the spike protein, and when injected into the body, causes the patients own cells to produce the protein (not the virus), which triggers an immune reaction that will prep itself to battle the virus.

The technology is interesting, but unproven. There are no approved RNA therapies. And Moderna has never brought a product to the market before.

One thing thats of note is how early a start Moderna got on the vaccine. On January 11, 2020, Chinese researchers posted the genetic sequence of the coronavirus, well before most of the world was paying any attention. Moderna scientists had been working on a novel vaccine for a different coronavirus disease and jumped on this sequence and began working on a vaccine against it.

Bloomberg writes, By late February, when President Trump was still downplaying the risk of coronavirus, Modernas scientists had already delivered the first batch of candidate vaccines to researchers at the U.S. National institutes of Health. When the coronavirus was starting to explode in the U.S. in mid-March, the first healthy patient received a dose in a small, government-sponsored safety trial.

Moderna may be the leader, but they are not the only company employing this technology. Pfizer and a few others are as well. Pfizer is working with a German company, BioNTech, hoping to get their experimental RNA vaccine into human trials sometime this month.

Johnson & Johnson is using technology it employed for its experimental Zika and Ebola vaccine. Paris-based Sanofi is modifying technology it uses for its flu shots. There are more than two dozen companies working on coronavirus vaccines that are already in early-stage testing.

There is no precedent for the speed at which we are moving, said Clement Lewin, an associate vice president at Sanofi. I cant think of a parallel, in his thirty years of vaccine work.

The RNA vaccine technology, despite the apparent speed with which Moderna, Pfizer and BioNTech are moving, has some risks. Not as much is known about its efficacy. Early-stage data suggests it should be safe, but there are concerns about unwanted immune responses. And all vaccines run a risk of what is called disease enhancement, where the immune system responds in an unusual way, which can cause the illness to be worse.

It's possible that any vaccines just wont be effective, or the side effects will be such that they wont be considered safe for use.

Scale-up is also a major roadblock. As of April 7, there are 1,362,936 confirmed cases of COVID-19 around the globe. But there are another 8 billion possible people who would possibly benefit from a vaccine.

Under a scenario where Modernas vaccine turns out to be effective and safe, no one has experience in commercial production of RNA vaccines, let alone in the type of scale necessary. The company indicates it has the manufacturing capability to produce millions of doses of the vaccine per month at a factory that was already scaling up for a different vaccine. They are apparently also in discussions with possible partners. But millions is a very long way from producing vaccines for billions of peopleor even a few hundred million people.

If a vaccine were to become available in the next year, an optimistic timeline, the first candidates for the drug would be first-line respondersdoctors, nurses, paramedics, as well as infants, toddlers and pregnant women. Thats a figure that exceeds 26 million people in the U.S. alone.

The next round would be essential personnel and children with preexisting conditions, higher-risk patients and people over the age of 65. Once you add all those, just in the U.S., youre talking more than 100 million who would be considered high-priority for a vaccine.

Mark Feinberg, a former Merck & Co. executive in vaccines who now runs the International AIDS Vaccine Initiative, told Bloomberg that the real question wasnt just efficacy, but how quickly can you ramp up manufacturing to meet global need.

There arent a lot of drugs in the industry that are filled at these scales, period, said Stephen Hoge, president of Moderna. Even large pharma companies dont usually operate on this kind of scale. No one entity or one company will be able to do it by themselves.

With any luck, social distancing and quarantine will slow the spread of the virus. Perhaps the coming summer months will also decrease its spread, although whether there is a seasonality component to the disease is still unknown. This would give biopharma companies a little more room to develop and distribute vaccines or to determine which drugs are best-suited to preventing or treating the disease.

Meanwhile, biopharma companies worldwide are working desperately to develop solutions.

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The Hopes and Challenges of a COVID-19 Vaccine - BioSpace

The Food and Drug Administration has quietly closed out an investigation of data violations tied to Novartis' gene therapy Zolgensma, indicating it will not penalize the Swiss drugmaker for submitting manipulated testing results last year.

The violations, documented in an August 2019 inspection of Novartis' AveXis unit, were at the center of a scandal which last year engulfed the pharma and marred a landmark approval for its high-profile spinal muscular atrophy treatment.

In securing the FDA's blessing for the therapy, Novartis had knowingly filed an application containing altered data from preclinical tests in mice. At the time, the FDA warned it would consider civil or criminal penalties against the company, indicating its displeasure that Novartis was aware of the issue but proceeded to ask for approval anyway.

The statement was highly unusual and a signal, former FDA officials said, to other companies in the fast-growing gene therapy field. After seven months of review, however, the FDA has decided against leveling such sanctions, a spokesperson for the agency confirmed to BioPharma Dive.

Instead, the FDA has classified its inspection review as "Voluntary Action Indicated,"or VAI, which means violations were found during the inspection but didn't cross the threshold for regulatory action.

"FDA has completed its review of the information and records of the inspection, the evidence collected, and the firm's responses as well as the corrective actions to the inspectional observations, and the agency has classified the inspection as Voluntary Action Indicated," the FDA spokesperson said.

"Based on its review of the information available, FDA continues to find Zolgensmato be safe and effective for its intended use," added the spokesperson. Throughout its investigation, the FDA maintained that it found no evidence human data were changed.

FDA inspections, such as the one conducted of AveXis last August,result in what are known as Form 483s, technical documents that detail potential violations of the Food Drug and Cosmetic Act. Companies are allowed to respond, after which the FDA decides whether to proceed with a formal warning.

Novartis submitted its responseto the FDA's inspection in late August last year,pledging to retrain employees and improve quality control oversight. The company placed much of the blame for the manipulation on two senior AveXisexecutives, Brian and Allan Kaspar, who were fired in mid August. Through his lawyer, Brian Kasparhas publicly denied any wrongdoing.

Novartis also committed to informing the FDA within five business days of any credible allegations related to data integrity questions with any pending drug application in the future.

Novartis disclosed the FDA's Form 483 classification in a presentation Monday of new clinical data for Zolgensma, briefly noting the FDA's decision.

"We are pleased with this positive outcome and reiterate our firm commitment to data integrity and transparency in our engagements with regulators," a Novartis spokesperson said in a statement.

Zolgensma was only the second gene therapy for an inherited disease to win U.S. approval, showing in clinical studies it could keep alive infants who otherwise were likely to die from spinal muscular atrophy, or SMA. The neuromuscular disease robs newborns of a protein needed for muscle development, usually leading in its most severe form to death or permanent ventilation by age two.

Some 200 infants received Zolgensma in 2019, earning Novartis $361 million last year. The one-time therapy costs $2.1 million per patient, making it the most expensive drug in the world on a per-dose basis.

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No sanctions for Novartis as FDA ends review of gene therapy violations - BioPharma Dive

DUBLIN--(BUSINESS WIRE)--The "Innovations in Bioreactors, Gene Therapy, Vermifiltration, and Membranes for Desalination Processes" report has been added to ResearchAndMarkets.com's offering.

This edition of the Industrial Bioprocessing TOE features trends and innovations in fixed bed and disposable bioreactors used for processing viral vaccines and for the production of recombinant proteins used in gene therapy. The TOE also provides intelligence on the transformation of cellulose waste and other plant waste in to bio-char, levulinic acid, and formic acid that can be used for production of biofuels. The TOE features innovations based on the novel gasification systems that can be vital in converting biomass in to renewable energy. Additionally, the TOE provides intelligence on the use of innovative membrane technologies that can enhance water permeability and selectivity to generate more freshwater from desalination processes. The TOE also focuses on innovations that are based on the use of modular vermifiltration processes for wastewater treatment, and the generation of bioplastics and biofertilizers from plant and food waste.

The Industrial Bioprocessing TOE provides intelligence on technologies, processes and strategic insights of industries involving bioprocessing, including innovations in the development and production of chemicals, pharmaceuticals, nutraceuticals, alternative fuels, chemical feedstocks, food and beverages, and consumer products.

Key Topics Covered:

Advancements in Bioreactors, Gene Therapy, Vermifiltration, and Membranes for Desalination Processes

For more information about this report visit https://www.researchandmarkets.com/r/xrab0l

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2020 Innovations in Bioreactors, Gene Therapy, Vermifiltration & Membranes for Desalination Processes - ResearchAndMarkets.com - Business Wire

Novartis is on the final straight to approval of its spinal muscular atrophy (SMA) gene therapy Zolgensma in the EU, after getting a recommendation for approval from the CHMP.

The positive opinion gets Novartis marketing application for Zolgensma (onasemnogene abeparvovec) back on track after the company revealed last year that regulators in Europe and Japan had asked for more information on manufacturing of the gene therapy which delayed approval by some months.

At the time, Novartis AveXis unit which developed Zolgensma said the questions raised by the regulators werent related to a data manipulation scandal that emerged with the therapy last year, shortly after it was approved in the US. It was approved in Japan on 19 March.

The delay to its approval in Europe prompted Novartis to set up a managed access programme for some patients, which operated like a lottery and is expected to provide free access to the therapy for 100 patients, although it has come in for criticism as being an unfair way to determine access.

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Approximately 550 to 600 infants are born with SMA in Europe each year.

Now, the CHMP has backed conditional approval of Zolgensma for babies and young children with SMA, which could lead to full EMA approval within the next couple of months.

Novartis says it will offer a day one access programme to EU governments and reimbursement agencies to enable immediate access if the EMA gives it the nod.

The recommendation covers children with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1; or patients with 5q SMA with a bi-allelic mutation in SMN1 and up to three copies of the SMN2 gene.

Zolgensma is the worlds most expensive drug, with a price tag of more than $2.1 million per one-shot intravenous infusion, which Novartis argues is justified given the lifelong benefits the therapy can bring.

The company says SMA costs EU healthcare systems between 2.5 million and 4 million within the first 10 years of life alone.

The data scandal doesnt appear to have affected its uptake in the US sales in 2019 reached $361 million, more than half of that total from the last quarter of the year alone.

The quick uptake has come despite the established presence on the market of Biogens Spinraza (nusinersen), an antisense drug given by injection into the spine, which became the first treatment option for SMA when it was approved in 2016 and reached $2.1 billion in sales last year.

GlobalData recently predicted however that Zolgensma will generate global sales of $2.5 billion by 2025, overtaking Spinraza and leading the SMA market.

The CHMP recommendation is another shot in the arm for Novartis gene therapy, coming shortly after it reported additional safety and efficacy data with intrathecal delivery of the gene therapy i.e. delivery into the spinal column rather than intravenously.

Intrathecal administration is considered key to extending the use in therapy for children with milder forms of the disease who tend to exhibit symptoms later in childhood. Novartis also reported new data demonstrating long-term efficacy with Zolgensma, with patients showing benefits for up to five years after they were dosed with the therapy

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Novartis closes on EU approval for SMA gene therapy Zolgensma - - pharmaphorum

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

WALTHAM, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies and a mission to develop transformative medicines for devastating diseases, today announced it has closed an oversubscribed $60 million Series A financing. Seed venture investors F-Prime Capital and New Enterprise Associates (NEA) co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza and Partners Innovation Fund.

The proceeds will be used to advance the companys platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

The company is led by recently appointed Chief Executive Officer Rick Modi, who has a proven track record of building value at companies including AveXis, InterMune, MedImmune and Centocor. Joining the board of directors as part of the financing are Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, NEA; and Robert Weisskoff, Ph.D., Partner, F-Prime Capital. Industry veteran and gene therapy leader Sean Nolan will chair the board.

At Affinia Therapeutics, were setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors and gene therapies that have remarkable targeting properties, said Modi, CEO of Affinia. We are pleased to partner with such a distinguished syndicate to advance our platform and investigational product candidates toward the clinic for patients in need.

The companys technology was licensed from Lonza and Massachusetts Eye and Ear. It was developed at the Grousbeck Gene Therapy Center and further advanced under a sponsored research agreement with Lonza led by Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

In addition to Vandenberghe, joining Affinia Therapeutics as scientific co-founders are Botond Roska, M.D., Ph.D., Director, Institute of Molecular and Clinical Ophthalmology Basel; Aaron Tward, M.D., Ph.D., Assistant Professor, University of California, San Francisco; and Eric Zinn, Ph.D. student, Mass. Eye and Ear and Harvard University. Together, these scientists have authored more than 200 papers and filed more than 20 patents in the field of gene therapy.

Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies. By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine, said Vandenberghe, Director, Grousbeck Gene Therapy Center at Mass. Eye and Ear.

The Series A financing comes after Vandenberghe and his team successfully developed AAVSmartLibraries comprising thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vectors structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. Affinia Therapeutics has potentially the worlds largest library of patented functional AAV vectors.

Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines, said Mathers, General Partner at NEA. We are pleased to accelerate the impact of this exciting field.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need.www.affiniatx.com.

Link:
Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies - GlobeNewswire

FILE PHOTO: The company's logo is seen at a building of Swiss drugmaker Novartis in Rotkreuz, Switzerland, January 29, 2020. REUTERS/Arnd Wiegmann

ZURICH (Reuters) - Swiss drugmaker Novartis on Friday won a key European recommendation for its gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months.

The European Medicines Agencys (EMA) Committee for Human Medicines (CHMP) recommended conditional approval for Zolgensma for certain patients: those with Type 1 SMA, the severest form of the disease, or for SMA patients with up to three copies of the so-called SMN2 gene, an indicator of the diseases severity.

The EMAs conditional approval is meant to speed up access to medicines for unmet needs, based on less-complete data than normally expected.

Typically the European Commission approves medicines for use shortly after a CHMP recommendation, and Novartis is expecting a decision by June. The medicine, the worlds costliest one-time treatment at its U.S. list price, has already been approved in the United States and Japan.

Novartis is counting on the gene therapy becoming a billion-dollar-per-year seller. Zolgensma is the second treatment for SMA to be approved after Biogens Spinraza three years ago. Roche is expecting its oral drug risdiplam to win U.S. regulators blessing in May.

Reporting by John Miller; Editing by Michael Shields

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Novartis wins key European recommendation for gene therapy Zolgensma - Reuters

The Food and Drug Administration (FDA) has granted Fast Track designation to PR006 (Prevail Therapeutics), an experimental gene therapy, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN).

FTD-GRN is a progressive neurodegenerative disease caused by mutations in the GRN gene. Patients with a GRN mutation have reduced levels of progranulin, a protein critical for lysosomal function, neuronal survival, and normal microglial activities. PR006 delivers a healthy GRN gene using an AAV9 vector to increase progranulin levels in these patients.

The Food and Drug Administration (FDA) recently accepted the Investigational New Drug application for PR006, allowing the Company to proceed with a phase 1/2 trial.

FTD-GRN progresses rapidly and there are currently no therapeutic options available, said Asa Abeliovich, MD, PhD, Founder and CEO of Prevail. We believe PR006 has the potential to fill this unmet medical need and make a significant impact for patients.

The FDA previously granted Orphan Drug designation to PR006 for the treatment of patients with frontotemporal dementia.

For more information visit prevailtherapeutics.com.

This article originally appeared on MPR

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Gene Therapy for Frontotemporal Dementia With a GRN Mutation Gets Fast Track Status - Neurology Advisor

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDAs orphan status for remdesivir reserved to encourage the development of rare disease therapies Gilead CEO Daniel ODay had some explaining to do about the companys approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, ODay also reassured some analysts who had been fretting that Chinas quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

ODay emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall at least to the most heavily threatened groups.

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David Hallal bags another whopper round, looking to recruit more cell and gene therapy upstarts to ElevateBio BaseCamp - Endpoints News

Our first priority during this unprecedented time is the health of our employees, our patients and their communities, and the employees of our clinical sites, partners and vendors. In accordance with advice from health authorities, AVROBIO has moved quickly to virtual operations, except for essential laboratory work, which continues with additional COVID-19-related safety measures in place, said Geoff MacKay, AVROBIOs president and CEO. We continue to support patient identification efforts across our clinical trials in Canada, Australia and the United States. As the global healthcare community responds to the increase in COVID-19 cases, many hospitals, including our clinical sites, have temporarily paused elective medical procedures, which includes dosing of new patients in clinical trials of our investigative gene therapies. While were fully committed to moving our clinical programs forward, AVROBIO supports this temporary reallocation of resources to ensure hospitals can focus on meeting the needs of patients with COVID-19. We are closely monitoring this rapidly evolving situation and the potential impact on our clinical trial programs and business generally.

Program Updates AVR-RD-01 clinical trials in Fabry disease AVROBIO is conducting two clinical trials for its investigational gene therapy for Fabry disease (AVR-RD-01). Four patients have been dosed in the global Phase 2 trial (FAB-201), which is evaluating treatment-nave patients, and five patients in the fully enrolled Phase 1 (FACTs) investigator-led clinical trial of AVR-RD-01.

AVR-RD-04 Phase 1/2 clinical trial in cystinosis AVROBIOs investigational gene therapy for cystinosis (AVR-RD-04) is being studied in a Phase 1/2 investigational trial in collaboration with the University of California, San Diego (UCSD). The first patient was dosed in October 2019. The single-arm trial is expected to enroll up to six patients.

AVR-RD-02 Phase 1/2 clinical trial in Gaucher disease AVROBIO's investigational gene therapy for Gaucher disease (AVR-RD-02) is being studied in a Phase 1/2 clinical trial to evaluate the safety and efficacy in individuals with Gaucher disease type 1. The global trial is designed to enroll eight to 16 individuals with Gaucher disease type 1 including both those who are treatment-nave and those who are stable on enzyme replacement therapy.

Business Operations On March 10, AVROBIO created an internal, cross-functional COVID-19 Response Team to closely monitor the evolving situation and advise on the companys response. We have implemented a work-from-home policy for all employees excluding those providing essential services, such as our laboratory staff. For those employees, AVROBIO has implemented safety measures designed to comply with applicable federal, state and local guidelines in response to the COVID-19 pandemic. We may be required to take additional actions that impact our operations as required by applicable laws or regulations, or which we determine to be in the best interests of our employees. AVROBIO continues to evaluate the impact of the COVID-19 pandemic on its operations and will provide a further update in conjunction with its first quarter financial results announcement and Quarterly Report on Form 10-Q in May 2020. At this time, all preclinical programs and research activities remain on track, and we do not anticipate any material impact on our regulatory activities.

Financial Position In February 2020, the company raised gross proceeds of $100 million through a follow-on common stock offering. Based on the companys current operating plan, AVROBIO expects its cash and cash equivalents of $187.0 million as of Dec. 31, 2019, together with the net proceeds from the February 2020 follow-on common stock offering, will enable the company to fund its operating expenses and capital expenditure requirements into Q2 2022.

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

Forward-Looking Statements This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, the expected safety profile of our investigational gene therapies, the potential impact of the COVID-19 outbreak on our clinical trial programs and business generally, as well as our plans and expectations with respect to the timing and resumption of any development activities that may be temporarily paused as a result of the COVID-19 outbreak, and statements regarding our financial and cash position and expected cash runway. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

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

Cautionary Note on Future Updates The statements contained in this press release reflect our current views with respect to future events, which may change significantly as the global consequences of the COVID-19 pandemic rapidly develop. Accordingly, we do not undertake and specifically disclaim any obligation to update any forward-looking statements.

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AVROBIO Outlines Response to COVID-19 and Current Assessment of Business Impact - BioSpace

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A programme investigating the potential of gene therapy for the treatment of osteoarthritis in horses, dogs and cats, has begun at the Royal Veterinary College (RVC), led by Skeletal Regeneration expert, Dr Scott Roberts.

Dr Scott Roberts, who joined the RVC as Senior Lecturer in Translational Skeletal Research in the Department of Comparative Biomedical Sciences, has recently initiated the programme of investigative research.

The research is inspired by Dr Roberts interest in skeletal stem cell signalling and the effect of signal modulation on skeletal tissue regeneration. He initiated this work during his time at KU Leuven (Belgium) but has subsequently researched this topic as Principal Investigator at University College London and as Senior Principal Scientist at UCB Pharma. During his time at UCB, Dr Roberts identified musculoskeletal drug targets and led the subsequent drug discovery projects. His research has resulted in several patent applications on the use of cells and therapeutic antibodies to promote the regeneration of bone and cartilage.

The new gene therapy research programme in osteoarthritis will be undertaken in partnership with the LEP funded Vaccinology and Cell Therapy Hub at the RVC. This will not only provide an environment for further research on regenerative medicine but also the facilities to create reagents for clinical translation.

The Hub with its close connections to both scientists and veterinary clinicians is also well-placed to take the science from bench-to-bedside and facilitate clinical trials in veterinary patients. This will include horses at the Equine Referral Hospital, and dogs and cats at the Queen Mother Hospital for Animals at the RVC.

It is proposed that this treatment would transition into human clinical studies, exemplifying the RVCs commitment to the One Health approach which recognises and facilitates the synergy between animal and human health.

Dr Scott Roberts said:

This research has the potential to change the way that we approach degenerative joint disease and I am delighted to have access to the Vaccinology and Cell Therapy Hub while we undertake this work. We hope that this science will lead to a ground-breaking treatment for osteoarthritis in animals, and eventually humans. We are optimistic about the future of this research, particularly given its contribution to the RVCs One Health ethos. I look forward to taking advantage of the RVCs world-leading facilities, as well as the scientists and clinicians who will help us take this research forward.

Professor Jonathan Elliott, Vice Principal of Research and Innovation at the RVC, said:

We are delighted that Scott Roberts has joined the RVC and will pursue his translational research into novel therapies that stimulate repair of cartilage for osteoarthritis in horses, dogs and people. This appointment builds on the RVCs expertise in Skeletal Biology. Scotts links to the Pharma Industry are very important for his translational science to have impact. His work fits perfectly with the goals of our soon to open Vaccinology and Cell Therapy Hub, made possible by the funding from the Hertfordshire Local Enterprise Partnership.

The research Dr Roberts is undertaking is critical as cartilage has a very poor capacity to repair itself, with cartilage injuries often progressing to osteoarthritis. There is also currently no approved evidence-driven therapy for the treatment of this disease.

In addition to osteoarthritis, Dr Roberts research aims to create regenerative therapeutics for non-healing bone fractures. This is based on a comprehensive understanding of tissue development, as tissue repair is now regarded as a re-emergence of embryonic signalling cascades. Dr Roberts has used knowledge in this area to identify developmentally inspired methodologies to create laboratory grown tissue implants that have the capacity to drive bone fracture repair.

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Investigation Begins into Gene Therapy for Osteoarthritis - Everything Horse UK

TOKYO & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Modalis Therapeutics Corporation (Modalis) today announced that the company has entered into a license agreement with Editas Medicine, Inc., under which Modalis has obtained a license to certain intellectual property that is controlled by Editas Medicine. Modalis is utilizing its proprietary epigenetic gene modulation technology, CRISPR-GNDM (Guide Nucleotide Directed Modulation), to treat patients with serious genetic disorders. Additional details including financial terms of the agreement were not disclosed.

"Our goal is to create CRISPR based gene therapies for genetic disorders, most of which fall into the orphan disease category. There should be no disease that is ignored because of its small patient population, and our mission to develop disease modifying treatments for these diseases reflects our belief that Every Life Deserves Attention. We are proud to be the pioneer in CRISPR based gene modulation therapy, said Haru Morita, Chief Executive Officer of Modalis.

We are pleased to establish this license agreement with Modalis Therapeutics as their mission is aligned with our mission to make transformative medicines for people living with serious diseases of unmet clinical need. CRISPR technology has many uses and applications, and we are pleased to include Modalis in our expanding portfolio of licensees so the greatest number of patients may benefit in the future from transformative medicines, said Cynthia Collins, president and chief executive officer, Editas Medicine.

About Modalis

Modalis Therapeutics is developing precision genetic medicines through epigenetic gene modulation. Founded by Osamu Nureki and leading scientists in CRISPR gene editing from University of Tokyo, Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM technology which enables the locus specific modulation of gene expression or histone modification without the need for double-stranded DNA cleavage, gene editing or base editing. Modalis is focusing initially on genetic disorders caused by loss of gene regulation resulting in excess or insufficient protein production which includes more than 660 genes that are currently estimated to cause human disease due to haploinsufficiency. Headquartered in Tokyo with laboratories and facilities in Cambridge, Massachusetts, the company is backed by leading Japanese investors including Fast Track Initiative, SBI Investment, UTokyo-IPC, SMBC Venture Capital, and Mizuho Capital. For additional information, visit http://www.modalistx.com.

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Modalis Obtains Access to Foundational CRISPR IP - BioSpace

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

MALVERN, PA Ocugen, Inc. (NASDAQ: OCGN), a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat rare and underserved ophthalmic diseases, recently 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 inNature Gene Therapyearlier 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:

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Ocugen Provides Business Update and Full Year 2019 Financial Results - MyChesCo

Glen Allen Mar 30, 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]

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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

The comprehensive study on the Gene Therapy for Ovarian Cancer market provides crucial insights to the stakeholders who are vying to solidify their presence in the current and future market landscape. The various factors that are likely to shape the course of the Gene Therapy for Ovarian Cancer market over the next decade are thoroughly analyzed in the report. The study represents the market share in terms of US$ XX Mn/Bn and volume (XX units).

The report splits the global Gene Therapy for Ovarian Cancer market based on product type wherein a complete analysis of the various products including, product 1, product 2, product 3, and product 4 is provided. The market share of each product, the adoption rate, pricing analysis and more is included in the report along with accurate tables, graphs, and figures. The market is further segmented on the basis of end use and tracks the scope of each end use over the stipulated time frame. The various end uses covered in the report include end use 1, end use 2, end use 3, and end use 4.

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The key players covered in this studyTakara BioVBL TherapeuticsCELSIONTargovax

Market segment by Type, the product can be split intoIntravenousIntratumoralIntraperitoneal

Market segment by Application, split intoOvarian Cancer (unspecified)Recurrent Ovarian Epithelial CancerPlatinum-Resistant Ovarian Cancer

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

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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

Despite revolutionising healthcare, advanced therapies pose significant challenges to regulators and their approval pathways. Credit: Shutterstock.

Advanced therapies, including cell and gene therapies, have revolutionised healthcare. Created by altering a patient, or donors, cells using novel gene editing tools, these highly personalised therapies provide the possibility of long-term, life-improving treatment that could even be curative.

This approach to treating conditions, often those that were previously viewed as untreatable, has been gaining significant momentum; there are now three approved gene therapies on the market and more and more studies are being conducted in the area.

In the UK alone, there was a 45% increase in the number of advanced therapy trials from 85 in 2018 to 127 in 2019, according to Innovate UKs Cell and Gene Therapy Catapults clinical trials database. Non-profit Alliance for Regenerative Medicines 2019 clinical trials report highlighted although there was stagnation in the numbers of studies in Europe, there has been a global increase of 32% between 2014 and 2018.

However, the uniqueness of these therapies creates significant challenges for the pharma industry, particularly for regulators and their existing approval and reimbursement methods. The Alliance for Regenerative Medicine European public policy senior director Annie Hubert explains: Because advanced therapies are very different from traditional pharmaceuticals, both in the way they are manufactured and in the potential therapeutic effect for patients, traditional regulatory pathways are often ill-suited to the regulation of advanced therapies.

Often these therapies are developed for rare disorders with small patient populations meaning there is less safety and long-term efficacy data available upon approval than regulators are used to with randomised control trials. It is also often not seen as ethical to recruit placebo arms for the types of severe conditions these diseases treat.

As a result of their durable and sometimes curative effects, these therapies have a high upfront cost Novartiss gene therapy for spinal muscular atrophy Zolgensma is priced at $2.1m per patient, making it the most expensive drug in the world.

The Alliance for Regenerative Medicines 2019 Getting Ready: Recommendations for Timely access to advanced therapy medicinal products in Europe report also notes this cost is also linked to the incredibly complex, manufacturing process required for advanced therapies. Hubert notes the different manufacturing process also poses a challenge for regulators when applying traditional quality control and other standards to the production of advanced therapies.

How can regulators support continued R&D and allow for the commercialisation and reimbursement of these ground-breaking therapies?

Despite regulators creating new pathways for quicker assessment and approval of these challenging, but innovative medicines, there still remain significant barriers to development, approval and reimbursement of these therapies.

Since randomised, placebo-controlled trials with clinically validated endpoints are not usually appropriate for advanced therapies, there is a need for regulators to be adaptive to the different types of trials, data and endpoints to support the approval of advanced therapies.

In terms of innovative trial design, Hubert notes regulators can help to facilitate this by enhancing the acceptability of validated surrogate endpoints to estimate long-term outcomes, as well as improving the infrastructure for gathering real-world evidence [RWE] to supplement traditional clinical trial data.

RWE can help to mitigate some of the uncertainty around the long-term, durable therapeutic benefits of these advanced therapies. However, it can also be used to collect so-called natural history data of trial participator to be used as the comparator for trials, meaning there is no need for a placebo trial arm.

A primary recommendation of Alliance for Regenerative Medicines 2019 report is to consider to new payment structures that take into account the longer-term value these therapies have on patients and their families, as well as the overall cost-savings to the healthcare systems.

Increased predictability in reimbursement determinations post-approval would also likely promote growth in clinical development, adds Hubert.

The non-profit recommends a range of different models, including conditional reimbursement, paying based on outcomes and annuity-based payments.

All of these models would be supported by more and easier RWE collection, but Hubert picks out conditional approval schemes as benefitting the most; RWE can help to offset the perceived risk of providing and reimbursing products with limited follow-up data available, while still ensuring patients can access safe and effective therapies as efficiently as possible.

Conditional reimbursement schemes involve temporary reimbursement, which is contingent on the collection and review of further evidence; an example of an existing model is NHS Englands Cancer Drugs Fund, and Boris Johnsons Conservative Government has expressed a desire to expand this into an Innovative Drugs Fund to include disease areas beyond oncology.

Outcome-based reimbursement models are similar, but they are specifically linked to certain pre-determined benefits of the drug the UKs Cell and Gene Therapy say these can be clinical, economic or patient-reported experience measures, and the best way to support them is through the development of interconnected data registries. The Pistoia Alliances 2030 retrospective report also discussed the need to move away from a pay-per-pill approach towards payments based on outcomes when dealing with personalised medicines.

Finally, annuitypayments can help to spread the cost of therapy out over several years Hubert explains. This allows healthcare providers to plan their resource allocation for the therapy, as well as facilitates affordable access. The Alliances recommendations-focused report finds that these have been discussed, but not yet implemented.

The central vision of both the Cell and Gene Therapy Catapult and the Alliance for Regenerative Medicine is to better connect all the groups involved in advanced medicine, ranging from patients, drug developers to regulators.

Dialogue between industry, researchers, regulators, and other stakeholders is essential to ensure that safe and effective next-generation therapies are able to reach patients in a timely manner.

This becomes even more important as new, even more complex therapies are being developed; early dialogue would allow regulators to prepare their processes in advance, rather than trying to catch up after the fact.

In addition, the Alliances Getting Ready report also notes the need for regulators to communicate with one another to create international standards. This makes it easier for developers looking to launch a product in multiple countries, as well as reducing duplicative or contradictive regulatory requirements, which can be particularly burdensome for smalldevelopers with limited resources which dominate this field, Hubert concludes.

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Tips and tricks for regulators regarding advanced therapies - Pharmaceutical Technology

Press release content from Globe Newswire. The AP news staff was not involved in its creation.

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NEW YORK and CLEVELAND, March 27, 2020 (GLOBE NEWSWIRE) -- The COVID-19 pandemic has created extraordinary challenges across all aspects of healthcare. Many institutions and their brave staff of providers are now focused on caring for patients stricken by the virus. We at Abeona are thankful for their sacrifices and efforts, and stand behind these healthcare institutions whose resources are focused on the greater good. We are dedicated to being a good global citizen in doing our part by following guidance from local health officials in New York, Cleveland, and Madrid, and where remote employees reside.

Above all, we will take every reasonable measure to ensure the safety of our patients and employees, while sustaining our business operations during this uncertain time. Abeona is fully focused on getting through the pandemic by working closely with our clinical trial sites to ensure that patient safety remains paramount.

Clinical DevelopmentWe remain committed to advancing our clinical programs, but recognize delays are inevitable in these uncertain times, especially as healthcare resources are justly redirected to those who need them most. We are continually assessing the dynamic situation and implementing plans to minimize disruption. We are also constantly reviewing these plans and associated processes and policies to ensure our patients and employees are safe, and continuity in our scaled back operations remains.

While the full impact on our clinical programs cannot be quantified at this point, what we do know is that all current clinical trial sites remain active. We can say with certainty that some sites have paused screening and delays are expected as the situation evolves globally. What we wont know for the foreseeable future is the long-term impact. However, we remain dedicated to communicating frequently and openly with our stakeholders as more information becomes available, including updates on material changes to prior guidance as we continue to follow applicable government, regulatory and institutional guidelines.

Business OperationsLooking inward, the safety of our employees is a top priority. We have instituted additional protective measures since news of COVID-19 broke, and we frequently assess and improve our safety practices and policies. Operations at our Cleveland manufacturing facility have been significantly scaled back to ensure that employees and those around them have the best chance to remain safe, and to accommodate reduced clinical development activities. Only employees deemed essential by senior management to maintaining the manufacturing operation are entering the facility and under strict safety protocols to mitigate their risk. More traditional means of risk mitigation including a global work from home policy and suspended business travel are also in place.

As we try to rationalize the unprecedented developments sweeping the globe, please be assured that Abeona is doing all it can to protect our patients, employees, and the communities around us. This includes company operations that underpin our aspirations to bring new medicines to patients in need.

Abeona is about great Science and great People, and its important we continue to do everything we can to preserve both.

We will get through this but only if we work together.

About Abeona TherapeuticsAbeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Companys clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Companys portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). http://www.abeonatherapeutics.com

Investor Contact:Dan FerryLifeSci Advisors, LLC+1 (617) 535-7746 daniel@lifesciadvisors.com

Media Contact:Scott SantiamoDirector, Corporate CommunicationsAbeona Therapeutics+1 (718) 344-5843 ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics Stakeholder Letter in Response to the COVID-19 Pandemic - Associated Press

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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