StemCell Therapy

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

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

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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

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200330005703/en/

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

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

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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

Nomura analyst Christopher Marai maintained a Buy rating on Voyager Therapeutics (VYGR) on March 27 and set a price target of $16.00. The companys shares closed last Friday at $9.05, close to its 52-week low of $6.27.

According to TipRanks.com, Marai is a 2-star analyst with an average return of -0.6% and a 43.7% success rate. Marai covers the Healthcare sector, focusing on stocks such as Global Blood Therapeutics, Alexion Pharmaceuticals, and BioMarin Pharmaceutical.

Currently, the analyst consensus on Voyager Therapeutics is a Moderate Buy with an average price target of $19.67, implying a 129.5% upside from current levels. In a report issued on March 19, Benchmark Co. also initiated coverage with a Buy rating on the stock.

See todays analyst top recommended stocks >>

Based on Voyager Therapeutics latest earnings release for the quarter ending December 31, the company reported a quarterly revenue of $32.67 million and GAAP net loss of $12.57 million. In comparison, last year the company earned revenue of $2.01 million and had a GAAP net loss of $22.53 million.

TipRanks has tracked 36,000 company insiders and found that a few of them are better than others when it comes to timing their transactions. See which 3 stocks are most likely to make moves following their insider activities.

Voyager Therapeutics, Inc. operates as a clinical-stage gene therapy company, which develops treatments for patients suffering from central nervous system. Its pipeline of gene theraphy programs include VY-AADC, VY-SOD101, VY-HTT01, VY-FXN01, Tau Program, and VY-NAV01. The company was founded by Guangping Gao, Mark A. Kay, Krystof Bankiewicz and Phillip Zamore in June 2013 and is headquartered in Cambridge, MA.

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Nomura Sticks to Its Buy Rating for Voyager Therapeutics (VYGR) - Smarter Analyst

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (NASDAQ: BLUE) today provided an update in response to the global COVID-19 pandemic. The company has taken steps to ensure the safety of its patients and employees, while working to ensure the sustainability of its business operations as this unprecedented situation continues to evolve.

The COVID-19 pandemic has created new challenges for bluebird, the broader biotech community, and society as a whole. During this unprecedented time, we remain focused on caring for the patients who rely on us, said Nick Leschly, chief bluebird. In addition, we are prioritizing the safety and well-being of our employees, making a positive impact as a member of our local community, and continuing to execute on our business strategy. While we all face tremendous challenges and uncertainty at this time, I am confident in bluebirds ability to face these headwinds with ingenuity, empathy, and a relentless commitment to the people we serve.

bluebird bio continues to evaluate the impact of COVID-19 on the healthcare system and work with healthcare providers supporting its clinical studies to mitigate risk to patients while taking into account regulatory, institutional, and government guidance and policies. The company remains committed to maintaining its development plans but acknowledges the potential impact on clinical studies given the rapidly evolving global environment. Generally, the company expects the COVID-19 pandemic to shift the timing of enrollment and completion of clinical studies by at least three months and expects timing shifts to vary by clinical trial and by program.

Regulatory Activities

Commercial Launch of ZYNTEGLO

Operating PlanIn light of the impacts of the COVID-19 pandemic on the business and the anticipated changes to commercial, regulatory and development timelines, the company is currently re-evaluating its operating plan. The company will be adjusting priorities and overall expenses. An update will be provided by the companys Q1 2020 earnings release.

Ongoing Programs and Clinical StudiesBelow is the current status of bluebird bio sponsored clinical trials:

Our People and Businessbluebird bio is evaluating all business-critical actions to determine how best to mitigate risk while being as minimally disruptive as possible. Safety of bluebird bio employees remains a top priority for the company. As of March 10, the company transitioned to a global work from home policy. Business-critical laboratory, manufacturing and related support activities continue and have been subject to heightened precautions to ensure safety of employees and the continuation of highest priority activities. The company continues to assess company policies, business continuity plans and employee support.

Conference Call DetailsInvestors may listen to the call on March 26, 2020 at 8:30 am ET by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 2666529.

To access the live webcast of bluebird bios presentation, please visit the Events & Presentations page within the Investors & Media section of the bluebird bio website at http://investor.bluebirdbio.com. Replays of the webcast will be available on the bluebird bio website for 90 days following the event.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, Lenti-D and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the plans for development and commercialization for ZYNTEGLO and the companys product candidates, including plans for our ongoing clinical trials, expectations regarding timing for the first patient treated with ZYNTEGLO in the commercial context, our plans and timing expectations for regulatory submissions for ide-cel, LentiGlobin for -thalassemia, LentiGlobin for SCD, and Lenti-D for CALD, our plans for data disclosures in 2020, and our plans and expectations in light of and in response to the COVID-19 pandemic and its impacts on global healthcare systems and our business. Any forward-looking statements are based on managements current expectations of future events 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 risks that the COVID-19 pandemic may disrupt our business and/or the global healthcare system more severely than we have anticipated, which may have the effect of further delaying our ability to enroll and complete our ongoing clinical trials (which may include potential delays of the ide-cel clinical trials beyond April 13, 2020), further delaying our ability to treat the first patient in the commercial context and obtaining market access and reimbursement for our approved product, and further delaying our timelines for regulatory submissions for our product candidates; the risk that we will encounter further challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product, in the adoption of value-based payment models, or in obtaining sufficient coverage or reimbursement for our products; the risk that our collaborations, including the collaborations with Bristol-Myers Squibb, will not continue or will not be successful; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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bluebird bio Provides Assessment of Impact of COVID-19, Update on Business Operations and Clinical Program Development - Business Wire

- New company formed from combination of TranscripTx and ReCode Therapeutics

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

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

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

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

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

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

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

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

About ReCode Therapeutics

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

About OrbiMed Advisors LLC

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

About Colt Ventures

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20200326005123/en/

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

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

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

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

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

John Swartley

Permission granted by University of Pennsylvania

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

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

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

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

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

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

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

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

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

James Wilson

Permission granted by the University of Pennsylvania

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

BOSTON and LONDON, March 18, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that company founder and gene therapy pioneer Bobby Gaspar, M.D., Ph.D., has been named chief executive officer, effective immediately. Dr. Gaspar, previously president of research, chief scientific officer, and a member of the Orchard board of directors, succeeds Mark Rothera, who has served as the companys chief executive officer since 2017. As part of this transition process, Frank Thomas, Orchards chief operating officer and chief financial officer, will take on the role of president.

As a world-renowned scientist and physician, and accomplished strategic and organizational leader with more than 25 years of experience in medicine and biotechnology, Bobby Gaspar is uniquely qualified to lead Orchard into the future, said Jim Geraghty, chairman of the Orchard board of directors. In addition, Frank Thomas proven track record of success in leading operations, corporate finance and commercialization at a number of publicly traded life sciences companies will continue to be invaluable in his expanded role. On behalf of the entire Board of Directors, Id like to personally thank Mark for his many contributions to building Orchard into a leading gene therapy company over the last three years and wish him all the best in his future endeavors.

One of the companys principal scientific founders, Dr. Gaspar has served on Orchards board of directors and has driven its research, development and regulatory strategy since its inception. Over the course of his long career he has been a leading force in the development of hematopoietic stem cell (HSC) gene therapy bringing it from some of the first studies in patients to potential regulatory approvals. Dr. Gaspars unparalleled expertise, in addition to his deep relationships with key physicians and treatment centers around the world, will continue to be integral to efforts to identify and treat patients with metachromatic leukodystrophy (MLD) and other diseases through targeted disease education, early diagnosis and comprehensive newborn screening.

Dr. Gaspar commented: I am honored to become Orchards next CEO at a time of such opportunity for the company and for patients with severe genetic disorders. Through the consistent execution of our strategy, our talented team has advanced a leading portfolio of gene therapy candidates, expanding our R&D, manufacturing and commercial capabilities. We will now focus on driving continued innovation and growth, as well as strong commercial preparation and execution. I look forward to providing greater detail around our commercialization plan, pipeline prioritization and how we can realize the full potential of our HSC gene therapy platform, in the coming quarter.

Mr. Thomas commented: Im excited to be part of this next phase of Orchards evolution as a gene therapy leader as we look to refine our strategic priorities, ensure financial strength through improved operating efficiencies and prepare for a new cycle of growth, which includes our anticipated upcoming launch of OTL-200 in Europe. Im confident we will achieve long-term growth and value for our shareholders while turning groundbreaking innovation into potentially transformative therapies for patients suffering from devastating, often-fatal inherited diseases.

Mr. Rothera commented: It has been a great privilege to lead Orchard and this outstanding management team for the past three years. Orchard is poised to make a huge difference to the lives of patients worldwide living with devastating rare genetic conditions. Having worked closely with Bobby for the last several years, I know that he is tremendously talented, extremely passionate about the patient-centric mission, and fully prepared to lead Orchard as it enters its next phase as a company.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to permanently correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (twitter.com/orchard_tx and http://www.linkedin.com/company/orchard-therapeutics), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, the companys business strategy and goals, and the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the impact of the COVID-19 virus on Orchards clinical and commercial programs, the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 10-K for the year ended December 31, 2019, as filed with the U.S. Securities and Exchange Commission (SEC) on February 27, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee T. LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine C. HarrisonVP, Public Affairs & Stakeholder Engagement+1 202-415-0137media@orchard-tx.com

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Orchard Therapeutics Appoints Company Founder and Gene Therapy Pioneer Bobby Gaspar, MD, Ph.D., as New Chief Executive Officer - BioSpace

Abeona Therapeutics Inc.ABEO announced that it has treated the first patient in the pivotal phase III study VIITAL evaluating its lead pipeline candidate, EB-101, in patients with recessive dystrophic epidermolysis bullosa (RDEB). The rare connective tissue disorder, RDEB, is characterized by severe skin wounds and can lead to systemic complications.

The study is being conducted by investigators at Stanford University Medical Center and enrollment in it is ongoing.

Currently, there are no FDA-approved therapies for treating RDEB. A successful development of the gene-corrected cell therapy candidate, EB-101 will be a major boost for the clinical-stage pharma company.

Please note that the company has successfully completed a phase I/II study on EB-101 in RDEB patients. Data from the study showed that treatment with the candidate resulted in sustained and durable wound healing. It also had a favorable safety profile.

Shares of Abeona have lost 37.7% so far this year compared with the industrys decline of 12.7%.

Apart from EB-101, the company has two other clinical-stage pipeline candidates in its portfolio. The candidates ABO-102 and ABO-101 are adeno-associated virus (AAV)-based gene therapies, which are being developed for treating Sanfilippo syndrome type A and Sanfilippo syndrome type B, respectively.

The company is also planning to initiate a phase I/II study to evaluate pre-clinical AAV-based gene therapy candidate, ABO-202 in patients with CLN1 disease soon. An investigational new drug application to support the initiation of the study was approved by the FDA in May 2019.

Abeona Therapeutics Inc. Price

Abeona Therapeutics Inc. Price

Abeona Therapeutics Inc. price | Abeona Therapeutics Inc. Quote

Zacks Rank & Stocks to Consider

Abeona currently has Zacks Rank #3 (Hold) stock.

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Regenerons earnings estimates for 2020 have gone up from $28.31 to $29.18 and from $28.93 to $30.97 for 2021 over the past 30 days. Regenerons stock has returned 31% so far in 2020.

MeiraGTxs loss estimates for 2020 have narrowed from $2.41 to $2.06 and from $4.10 to $3.40 for 2021 over the past 30 days.

Veronas loss estimates for 2020 have narrowed from $3.95 to $2.65 and from $3.96 to $2.59 for 2021 over the past 30 days.

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Abeona Treats First Patient in Pivotal Gene Therapy Study - Yahoo Finance

Early last year, it was revealed that there were 289 cell and gene therapies held in the US pipeline, this figure has now increased to 362, according to PhRMA.

The updated figure only includes treatments that are currently going through Phase I-III trials, with this figure estimated to double once the preclinical pipeline is taken into account.

The jump from 289 to 362 clinical cell and gene therapy candidates represents a jump of 25% in just one year since the last assessment of the pipeline took place.

A knock-on effect of the burgeoning pipeline has been a significant capacity bottleneck experienced by contract development and manufacturing organizations to accommodate the work in the area.

One area that stayed the same compared to the previous years statistics is the concerted focus on oncology treatments. Again, around half of all clinical candidates (173) were focused on oncology, followed by genetic disorders being the next largest therapy area (34).

In total, a third of the drug candidates (132) are aimed at treating rare diseases, which is defined as a condition affecting fewer than 200,000 people in the US.

Thus far, nine cell and gene therapies have been brought to the market over the last few years, but PhRMA suggests that there are further challenges ahead once the current pipeline of products moves closer to approval.

In particular, delivery logistics and the manufacture of these products at scale, something companies are already struggling with, will need to be addressed through large capital investments.

Andrew Powaleny, director of public affairs at PhRMA, called for greater flexibility in reimbursement of such products.

He stated that current outdated federal rules and policies can create uncertainty for manufacturers and may limit the growth and expansion of innovative contracting arrangements.

Last year, Novartis gene therapy treatment, Zolgensma (onasemnogene abeparvovec),was approved in the US and given a price tag of $2.1m (1.8m) per patient.

This cost made it the most expensive treatment on the market, though the company moved to create an annualized payment plan that would see the treatment paid for over five years.

As more treatments reach the market these kinds of payment structure may become more common, with a spokesperson for Novartis telling us, at the time, that healthcare systems would have to rethink how to manage the cost of such treatments.

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Growing pipeline of cell and gene therapies - BioPharma-Reporter.com