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Archive for the ‘Gene therapy’ Category

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

Monday, March 30th, 2020

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.

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

Monday, March 30th, 2020

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

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

Monday, March 30th, 2020

- 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

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

Wednesday, March 18th, 2020

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

Wednesday, March 18th, 2020

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

Wednesday, March 18th, 2020

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.

Some better-ranked stocks from the biotech sector include Regeneron Pharmaceuticals, Inc. REGN, MeiraGTx Holdings PLC MGTX and Verona Pharma PLC VRNA, all sporting a Zacks Rank #1 (Strong Buy). You can seethe complete list of todays Zacks #1 Rank stocks here.

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

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

Wednesday, March 18th, 2020

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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Gene Therapies: Overcoming the Biggest Hurdles in… – Labiotech.eu

Wednesday, March 18th, 2020

By 2024, the gene therapy market is expected to reach an estimated worth of $13B (11.6B). With several therapies approved and hundreds of clinical trials underway, the sector is booming. However, its sudden and fast growth and the complexity of the therapy itself have resulted in a number of hurdles that need to be overcome.

Especially during manufacturing, researchers face challenges at different stages, including process development, safety testing, vector characterization, and when it comes to regulatory guidelines. One of the key issues is the highly compressed timeline researchers have to work with. Instead of the average eight to ten years that it takes to develop a drug, gene therapies are usually developed within three to five years.

Another challenge resulting from the rapid growth of the sector is the increasing demand for plasmids. As the key building blocks for the development of viral vectors, plasmids are needed for gene therapy development. Currently, the industry is struggling to meet the demand for plasmids, forcing companies to think outside the box. This bottleneck has resulted in the development of nonviral vector solutions, which we will see more of in the future.

But compressed timelines and viral vector bottlenecks are not the whole story. This infographic discusses the challenges at various stages of gene therapy manufacturing; what you can do to ace the manufacturing process; and what we can expect in the future.

We developed this infographic in collaboration with Merck. With decades of experience, Merck has already developed three gene therapy products through to commercialization; tested over 10,000 cell and gene therapy samples in one year; and has over 500 batches of different viruses to ensure a smooth manufacturing process.

Author: Larissa Warneck, Science Journalist at Labiotech.eu

Design: Elena Resko

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Gene Therapies: Overcoming the Biggest Hurdles in... - Labiotech.eu

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Cell And Gene Therapies Are Driving M&A Deal Activity – Law360

Wednesday, March 18th, 2020

Law360 (March 18, 2020, 4:34 PM EDT) -- There have been remarkable advances over the last several years in the development of cell and gene therapies, or CGTs. These therapies represent truly groundbreaking approaches to the treatment and prevention of diseases, many of which have proven resistant to traditional drugs or therapies.

Cell therapy generally refers to the transfer of live cells into a patient to treat a disease. The cells may originate from the patient (autologous therapy), where they are extracted, modified and reinfused into the patient, or from a donor (allogeneic therapy). Gene therapy involves a change in the genetic code of a patient by inserting or...

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Cell And Gene Therapies Are Driving M&A Deal Activity - Law360

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3 Mesothelioma Treatment Options on the Rise – Mesothelioma Guide

Wednesday, March 18th, 2020

People with mesothelioma dont have many treatment options.

A combination of surgery, chemotherapy and radiation is the standard of care. For years, chemotherapy was the only method with approval from the United States Food and Drug Administration.

In 2019, that changed. Now, in 2020 and looking forward, theres a lot of potential for another unorthodox treatment to emerge.

Last May, the FDA approved using the NovoTTF-100L System on a limited basis for pleural mesothelioma. Patients can use the treatment as a first-line option with chemotherapy and only if they arent candidates for surgery.

The NovoTTF-100L became the first mesothelioma treatment option in 15 years to receive FDA approval. In 2004, pemetrexed was approved for treating this cancer.

Hopefully, we wont have to wait another 15 years for the next approval. Here are three classes of cancer treatment that could break through soon and get approval from the FDA.

If youre interested in accessing any of these cancer treatment techniques, please email our registered nurse, Jenna Campagna. She can help you connect with a mesothelioma specialist and look into experimental treatments. Please email her at jenna@mesotheliomaguide.com.

Immunotherapy is at the forefront of many clinical studies involving mesothelioma. The treatment uses drugs to enhance the bodys immune system. The theory is that the body could better fight mesothelioma with just a little outside help.

There are a few variations of immunotherapy. The one used the most in experiments is checkpoint inhibitors. This type of immunotherapy drug targets the relationship between two proteins: PD-1 and PD-L1.

PD-1 is a protein on T-cells, which are the immune systems fighter cells against diseases like mesothelioma. PD-L1 is a protein in mesothelioma cells.

When the two interact, the T-cells cannot detect mesothelioma cells as dangerous to the body. Basically, the PD-1/PD-L1 connection is a mask for mesothelioma.

Checkpoint inhibitors block this connection, which allows the immune system to detect and attack infected cells. The two most common drugs are nivolumab (brand name Opdivo) and pembrolizumab (brand name Keytruda).

Dr. Patrick Forde and his team are testing immunotherapy with surgery and chemotherapy for pleural mesothelioma. Dr. Forde is a thoracic oncologist at Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

The clinical trial involves two stages of two immunotherapy drugs: nivolumab and ipilimumab. Patients receive three doses of nivolumab before surgery and one dose of ipilimumab. Then patients can receive further immunotherapy treatment after surgery.

Immunotherapys versatility is one of the treatments best qualities. It can be used before or after surgery, in conjunction with chemotherapy, and even paired with other emerging therapies.

Dr. Forde, who specializes in treating pleural mesothelioma, said immunotherapy can reduce the size of tumors and how widespread they are in the body. This regression can help surgeons remove the diseased cells during operation.

There are numerous forms of gene therapy. One type involves restructuring the genetic characteristics of infected cells. Another type one called suicide gene therapy uses genes to engage the immune system and attack the tumors.

Gene therapy usually isnt sufficient on its own. Instead, its used in conjunction with other mesothelioma treatment options.

The purpose of gene therapy is to repair or weaken infected cells. Repairing can reduce the number of dangerous mesothelioma cells in the body. Weakening can increase the effectiveness of other treatment methods, such as surgery, chemotherapy or immunotherapy.

A Phase 3 clinical trial involving gene therapy might be the most promising one available to pleural mesothelioma patients. Dr. Daniel Sterman is the director of the Multidisciplinary Pulmonary Oncology Program at New York University Langone Health. He is the lead investigator in the study, which combines gene therapy with chemotherapy.

The trial uses TR002, which is adenovirus-delivered Interferon Alpha-2B. The treatment is a gene therapy transported by a virus into the body. Interferon Alpha-2B is a gene therapy that causes tumors to produce interferon alpha, which is a cancer-fighting protein.

The increased production of this protein alerts the immune system of danger. The T-cells recognize that the cells are producing this protein and begin attacking them.

The Phase 2 results were astonishing in a positive way. The disease control rate meaning the cancer either was stagnant or reduced in size was 87.5% for enrolled patients. Additionally, the survival time for patients who received this gene therapy treatment nearly doubled compared to those who didnt.

Virotherapy involves using programmed viruses to either attack mesothelioma cells or kickstart the immune system. Virotherapy also is used as a vessel to transfer other forms of therapy into the body.

Its another outside-the-box method to help the body stop mesotheliomas progression. There are three types of virotherapy used in experimental mesothelioma treatment:

Oncolytic viruses are modified viruses that focus on cancer cells and ignore healthy ones. This form of virotherapy explicitly targets mesothelioma, and its the most common type of the treatment.

Viral vectors are transports for other forms of treatment. Viral vectors could deliver modified genes or an immune system enhancer.

Viral immunotherapy involves using viruses to activate the immune system. Since the viruses are considered intruders, the immune system senses danger and looks for these viruses. Doctors often program the virotherapy similar to the genetic structure of mesothelioma cells. Doing so allows the immune system to identify harmful cells based on these genetic characteristics.

A new clinical trial involving virotherapy and immunotherapy has potential. The study uses ONCOS-102, which is a modified virus that transports immune system stimulants (cytokines).

ONCOS-102 is a combination of viral immunotherapy and oncolytic viruses. The virus aspect breaks apart the mesothelioma cells, and the cytokines send danger signals to the immune systems T-cells. When the mesothelioma cells break apart, they release antigens that the T-cells recognize as cancerous.

This two-pronged process involving virotherapy and immunotherapy has helped patients. Those receiving ONCOS-102 plus chemotherapy had a progression-free survival of around nine months.

Progression-free survival means the patient didnt die and the disease didnt spread further in the body. By comparison, patients who only received chemotherapy had a progression-free survival of around seven months.

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3 Mesothelioma Treatment Options on the Rise - Mesothelioma Guide

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Gene therapy giving Flames executive Snow hope in ALS fight – NHL.com

Wednesday, March 18th, 2020

He swipes once. Twice. Three times.

The lidocaine goes in, to freeze the skin. Then the needle.

Slowly, precisely, Rasquinha removes spinal fluid through the lumbar puncture, commonly known as a spinal tap. He then gives him the injection that Snow and his wife, Kelsie, believe is saving his life.

They believe because they want to, because they have to, and because -- against every single odd, against every single thing you've ever heard regarding amyotrophic lateral sclerosis, or ALS -- it just might be working.

Snow, a 38-year-old assistant general manager for the Calgary Flames, was diagnosed with ALS on June 17. It was not a surprise. His family has been ravaged by the familial form of the disease, with his father, two uncles and a cousin having died from ALS caused by the SOD1 genetic mutation.

ALS affects motor neurons, the cells that control muscle movement. As ALS progresses, the motor neurons die, the muscles become weaker, and eventually movement slows or becomes impossible.

It's what Snow saw happen in his right hand, how he began to suspect that the disease had come for him too. But almost immediately after his diagnosis, which usually carries a life expectancy of between six and 18 months, Snow enrolled in a phase 3 clinical trial at Sunnybrook Health Sciences Centre in Toronto for those with the SOD1 mutation, which affects 2 percent of ALS patients.

In this, he was lucky, both that it was available and that he qualified.

He has made the trip to Toronto with Kelsie every four weeks since then, though initially there was a two-in-three chance that every trip brought him an injection of tofersen, a drug that doctors and researchers hoped could slow the progression of the disease.

For the first six months of the trial, two-thirds of the study participants are given the actual drug. One-third are given a placebo.

He does not know for sure whether he was in the group given the medication or the placebo. The Snows believe he was one of the lucky ones, one of those given tofersen. They believe because they, remarkably, have not seen a progression of the disease since he entered the trial. He still does not have use of his right hand. He has use of everything else, all the things they feared might slip away from him in the weeks and months after they confirmed the diagnosis.

The injection takes two minutes, two minutes of silent meditation for Snow. He thinks about his mom, Linda, who committed suicide in 2012. "Because," as he says, "she'd be really happy and really sad if she were here. One of the things I got from her was a real joy for life. She always was happy that I was that way. So, I think about her and how I get to move on because of this." He thinks about his dad, Bob, who died of ALS in 2018, and his uncles and his cousin.

He wishes they had had the opportunity for these two minutes. For that two-in-three chance.

"I move through those thoughts fairly quickly," he says. "I usually say a prayer or two. For them. Probably in part for myself."

****

The Snows have already been at Sunnybrook for hours at this point, Feb. 20 marking their 11th visit to this hospital, his 10th lumbar puncture. At 9:15 a.m., the driver picks them up at their hotel for the 20-minute ride. When they walk in the doors at the hospital, a massive campus that looks like a small airport, Snow is on edge.

They find their way down to Room UG21, where Snow will undergo a battery of tests, all of them designed to determine whether the progress of the disease has slowed, whether it has stopped, whether it has -- God forbid -- picked up again.

They start with a detailed neurological assessment.

"Have you experienced any changes to your health since you were last here?"

"No."

"Have you noticed any difference in your speech?"

"No."

They test knowledge next, the year, the season, the province, the city. A series of words that never changes from visit to visit: apple, penny, table. He counts backward from 100 by seven.

These are the hardest weeks, when the anxiety sweeps up and the visit looms closer and the fear that "no change" might have turned into "some change" pierces the bubble they have formed around themselves.

"My bad hand, I don't have any expectations for it being better," Snow says. "It's more the opposite, that I'm always kind of concerned about and testing the good parts. The other hand, in particular. Living without one hand is not difficult. Living without two hands is difficult.

"It's testing my good hand, and then I overtire it. And then I convince myself something is wrong."

This is when Kelsie can sense his perpetual optimism faltering.

"None of the physical aspects of any of that, the lidocaine, the needle itself, I couldn't care less about those things," Snow says. "It's just the emotional anguish that you can put yourself through, with wondering, am I a little bit different? And if I'm a little bit different, what does that mean?

"Because that's totally unknown. But then you worry, that could be a slippery slope."

He does a breathing test, to determine lung capacity, and one to test muscle strength. That is the one that gives him the most anxiety, because they are testing his hand and his strength, and that is where the disease started to eat at him.

"This is our 10th time doing most of these tests," Snow says.

"And there has not been any changes," research coordinator Jahan Mookshah says.

"Those are our favorite words," Kelsie says.

Kelsie bends down and ties her husband's shoes. The testing is over, for the moment.

****

There is no blood test for ALS. It's only diagnosed by process of elimination, a factor that often can be problematic, as the disease progresses while the patient is still waiting for answers. Snow was told June 10 by an EMG technician in Calgary that it was likely ALS, a diagnosis that was confirmed a week later by Dr. Michael Benatar at the University of Miami.

It was the last answer they wanted in the world. It was a death sentence.

They crumbled, seeing an unknown present, a future robbed.

"I don't know how we did it," Snow said. "We did our days, and then we cried."

Back in Calgary, fellow assistant general manager Craig Conroy got the news on the phone from Snow, while looking at Snow's kids, Cohen and Willa, now 8 and 5, who were staying at his house while their parents were in Miami. As Conroy said, "That just breaks your heart."

But there was some good news.

Snow had the best-case scenario: a family history and a mutation of the SOD1 gene. He had pressed the issue with doctors, over and over again, after starting to feel the weakness in his right hand while lifting weights during the Stanley Cup Playoffs in April, even as he wanted to believe it was a pinched ulnar nerve, desperate for any answer but this. For Snow, that meant it was only a matter of months between the onset of symptoms and the diagnosis, as opposed to 1 1/2 to 2 years, which is the average.

Which was why when the Snows talked to Benatar on June 17, it was like being flattened and lifted up in the same moment. They confirmed Snow had ALS. They also learned there was a trial, with openings for those with a fast-progressing form of the disease, like him. There was something they could do. There was a place they could go. There was a medicine he could take, produced by Biogen, a company whose corporate headquarters are on Binney Street in Cambridge, Massachusetts, the exact street where Snow lived when he moved there to work at The Boston Globe, another career and another lifetime ago.

There was -- maybe, possibly -- hope. An impossibility in this world.

"It's just like someone believably telling you you're going to die, then telling you you might not die," Kelsie said. "You can't describe that in a more dramatic way than that. You feel like you got your life back. You've got a chance. Just a chance, right?"

Most of the time ALS is sporadic -- that is, not inherited. But in 5 to 10 percent of cases, there is a familial link, an altered gene that has folded. When Kelsie researched trials in the immediate aftermath of the diagnosis, she realized that the studies that were furthest along happened to be for SOD1, Snow's mutation, the second-leading cause of familial ALS.

They would head to Toronto every month, their kids left with a rotating selection of friends, off to get the injection that might -- or might not -- include the liquid hope that his future rested on.

They had six months in which he could have been receiving either the drug or the placebo, until last month, when they were finally assured he would be getting the medication from this point on.

They told their kids that they would have to have a summer to last a lifetime.

"Because we felt like it was maybe going to have to," Kelsie said.

They jammed in everything, all the bike rides and ice cream and pictures, the trip to Merrymeeting Lake in New Hampshire, Snow's family cabin. They jammed happiness in every moment, the tears reserved for private times, for when they couldn't hold it in any longer, wiped away when the kids approached. She wondered if he would make it to February and, if he did, whether he would want to hang around once he got there. She told him to just stay the way he was. They put one foot in front of the other and, still, they don't know exactly how.

By October, they had started to feel differently. Hopeful.

Snow got back on the ice, courtesy of a Flames equipment manager, who sewed his glove into a fist, enabling him to grip a hockey stick. He shot a puck. It rang off the crossbar. Kelsie took a video. It was evident to her that the disease had not progressed, not robbed him of any more strength.

That moment, that month, changed them.

"I felt like that was probably the first moment where I could really breathe," she said.

They went public on Dec. 18, and it ricocheted around the hockey community. Kelsie wrote a letter detailing their diagnosis and their hope, something she has continued to chronicle on her blog, kelsiesnowwrites.com, and recently, in a story that ran in Sports Illustrated.

It has become a piece of her every day, every thought, something that still fells her at a moment's notice. When she is asked if there's been a day that she hasn't thought about ALS since the diagnosis, tears start to flow down her face.

"These three letters are a part of my life forever," Kelsie says. "I sure wish they weren't. But I know that a lot of good things will come to us as a result of this. And I've seen a lot of good in a lot of people because of this.

"There's so much beauty in grief, and there's so much beauty in sadness and tragedy. Because you get to see the best in people. And that's not a small thing. But I wish I didn't have a byline in Sports Illustrated. I don't want a byline in Sports Illustrated. I don't want to be telling this story. But if this is what I'm here for, then I'm OK with that. It's not small. I know that. I know that being there for somebody you love is significant. It's enough for me."

She swipes at the tears, running her fingers underneath her eyes. She sniffles.

"This is because I know you, too," Kelsie says, starting to laugh amidst the tears. "That's the problem."

****

It has been 15 years since I met Kelsie and Chris, back when she was still Kelsie Smith and he was still a baseball writer. The summer of 2005 was one of those summers that's only possible when you are young and there is no responsibility, no ties, no worry. When a White Russian just before the bar closes seems like a good idea, and a shut-off car in a parking lot is the right place to bare a soul, for a friend to reveal she just might be falling in love.

As Kelsie texted me recently, "Honestly, best summer of my life."

They were too young, realistically, for it to work. She was 21. He was 23. They met in a bar, the White Horse Tavern, down the street from the apartment that Snow would buy in Allston, Massachusetts, and that I would later buy from him and live in for the next decade.

He was the Boston Red Sox beat writer at the Globe, she was an intern in the Globe's sports department out of the University of Kansas, and I would be hired into the sports staff that summer as a general assignment reporter.

They got engaged the next summer, and married in December 2007, on a frigid, 9-degree day in St. Paul, Minnesota, where they moved after he was hired as the director of hockey operations for the Minnesota Wild, an unorthodox move that would launch him on a career in hockey and give me a chance to succeed him on the Red Sox beat at the Globe.

She covered the Minnesota Twins for the St. Paul Pioneer Press. We spent spring trainings together in Fort Myers, Florida.

It's hard to think about that day in 2007, so many years ago now, when they promised a lifetime to each other. Because, as Kelsie said, "That I have extra months is not lost on me. I know that I've been already given a gift. It's just that I want what we all thought we were getting when we got married. That we are going to grow old together."

Kelsie has always trusted in Snow, something she has never been shy about expressing. She's always believed he would do what he said, that he would succeed despite all the odds: That he would make the unheard-of jump from baseball reporter to NHL front office member; that he would figure it out after the Wild let him go; that they could find their way on one income (first hers, then his) in a new city, in a new country with a new baby.

And he did. They did.

"It was always like, Chris will figure this out," she said. "And that's probably an unfair amount of pressure that I put on him, but I just believed in him. I've always believed in him that much."

She still does, even in a battle that, up to this point, has been unwinnable.

****

At 1 p.m., after a pizza lunch, it's time to head back to UG21 for the lumbar puncture. Snow laughs about the diet he has been instructed to stick to -- high protein, high fat, high carb -- because losing weight is a marker of the disease. It's one thing he doesn't mind.

This session, too, starts with some tests. Of reflexes. Hands. Ankles. Feet. Jaw.

"This might hurt," Rasquinha says. "Sorry."

Rasquinha flips his hands over, examines them, tells Snow to relax. This is not Snow's forte.

Snow acknowledges yet again that he cannot do anything with his right hand. That went in June, and the atrophy has set in up to his elbow. Kelsie -- or, if he's at work with the Flames, one of his colleagues -- makes sure to cut his food for him, if needed; they tie his skates and his shoes. "Tight, but not too tight," as she puts it.

Testing done, it's time for the lumbar puncture. Snow is now on the open-label extension of the trial. After this visit, he is assured that the fluid sent into his spine will be tofersen. This is a comfort, even as they believe he has been receiving the medication all along.

"All right," Snow says, "let's rock and roll."

The risks are read out, the warnings given, as they always are. The Snows know this nearly by heart. Rasquinha snaps on sterile green gloves and a baby blue face mask. The lidocaine goes in. "Mosquito bite," Rasquinha calls it. "A little burn. Sorry."

Lorne Zinman enters the room. He is part of the reason for their hope, a man who oozes sunshine as he talks, despite having devoted his life to a disease that to this point has claimed every one of its victims.

"This is exciting," says Zinman, the director of the ALS/Neuromuscular Clinic at Sunnybrook and an associate professor of neurology at the University of Toronto. "This is gene therapy for ALS. I've been talking about this for, like, two decades. The fact that it's a reality and we could be helping people like Chris, it's just the happiest thing. I always say -- apology to my children -- the happiest day of my life will be when we finally have something for this disease, because I've seen too many people go through it."

It is easy for Zinman to be joyful these days. He, like all those who work with ALS, has spent years and years in the darkness, and he somehow just might see a little light emerging, a speck that grows bigger with every day that Snow, and potentially others, are stable.

"It's been decades and decades of, really, failure after failure in ALS," Zinman said. "When I give lectures, I put up a slide and I call it the graveyard of failed trials. And it's just failure after failure. The big turning point came when we found the first gene for ALS in the early '90s."

Researchers put the gene into mice, and the mice started to show signs of disease. They thought a cure would come soon after. They were wrong.

Things started to turn when advances in gene therapy led the focus to shift. They knew SOD1 made a protein, and that the protein misfolded, leading to the killing of motor neurons that led to ALS. That became their target.

On Feb. 3, Kelsie posted a video to Twitter showing Snow lifting his right hand at his wrist. It was something he hadn't been able to do since the disease took hold. They don't know what it means, and they try not to read too much into it, but the idea of stability, let alone improvement, sometimes seems like a dream they do not want to end.

And that is what makes Zinman so excited.

"We don't usually see that in ALS trials," he said. "The objective is to slow things down, not to make things improved. It's really exciting when you hear something like that, where I'm actually able to do something that I couldn't do before. You always have to take it with a grain of salt it's just really exciting when you hear something like that."

He envisions a day when a patient like Snow or one of his family members could find out he has the SOD1 mutation, that they could start him on the drugs in the pre-symptomatic phase, as a preventative, similar to the way that doctors have been able to use PrEP to keep HIV from taking hold, or the way that doctors use vaccines to prevent diseases.

"We're diagnosing ALS about five to six times a week, and three to four of our patients die every week," Zinman said. "I've been doing this for 15 years now. I see the faces of these patients, not just them, their family members and what they went through, so to be able to offer someone something like this -- research is hope."

When Zinman and I exited the room, I later learned, Snow grew emotional. He cried. It was joy and relief and release. He had finished another session at Sunnybrook. There had been no change. He soon would be assured of getting more of the medicine that has given him more, where before, he expected there would be less and less.

Because these two minutes, every four weeks, might just be saving him.

"The first few times he was dosed I had full-on panic attacks," Kelsie said. "It was all about the fact that I didn't know what [he was getting]. They'd come in and they'd put this little Ziploc baggie on the desk in there and it had this syringe in it. I remember noticing right away that it was 15 milliliters of this clear fluid. And that was every ounce of hope I had in my life."

****

The appointment over for the month, Snow walks down the corridor, a drab, shabby, beige hallway. It is hardly inspiring, with its fluorescent lights and well-trod flooring. But Snow is nearly bouncing. This is the best he feels all month, his hopes confirmed, the medication, he assumes, flowing through his body.

"Every time I walk this hallway, it's a good feeling," he says. "Because you picture yourself being wheeled."

But he can walk. He can breathe. He can do his job, almost the same way he always has, with a few simple modifications. He wears a suit without a tie these days, because he cannot tie one and, really, who needs it? He has not yet transitioned to slip-on shoes, perhaps out of stubbornness.

Link:
Gene therapy giving Flames executive Snow hope in ALS fight - NHL.com

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Exploring Avenues in Personalized Medical Care Defines Advances in Red Biotechnology Market: Valuation to Touch Massive US$512 Bn By 2027, Finds…

Wednesday, March 18th, 2020

Research for novel cancer therapeutics pool in massive investments in red biotechnology market

Player leverage AI to extract potential of red biotechnology in preserving health and controlling diseases

ALBANY, New York, March 18, 2020 /PRNewswire/ -- A wide assortment of applications of genetic engineering technology, vaccine research, and biologics have helped expand the potential of the red biotechnology market, which was worth US$314.2 billion in 2018. Growing application of biotechnology in medicine has unlocked promising prospects particularly in chronic and rare diseases treatment.

Transparency Market Research

Future Outlook

Emerging applications of gene therapy, pharmacogenomics, and genetic testing in the preservation of health, notably in oncology, are shaping the future growth trajectories in the red biotechnology market. By 2027-end, the revenues are projected to reach US$512 bn, clocking CAGR of ~6% from 2019 to 2027. In developing countries, many new collaborations are likely to be forged, defining future initiatives on cloud-based technology and AI.

Analysts' Viewpoint

"Staying abreast with standardized regulations and regulatory norms will enable biotechnology and pharmaceutical companies to chart new growth avenues in the red biotechnology market," note the analysts. Marked prevalence of cancer world over expands scope for market stakeholders, they further opine.

Key Takeaways of Red Biotechnology Market Study

Explore 176 pages of top-notch research, incisive insights, and detailed country-level projections. Gain business intelligence on Red Biotechnology Market (Application: Biopharmaceutical Production, Gene Therapy, Pharmacogenomics, and Genetic Testing; End User: Biopharmaceutical Industry, CMOs & CROs, Research Institutes, and Others) - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2019 - 2027 at https://www.transparencymarketresearch.com/report-toc/2063

Red Biotechnology Market: Key Driving Factors and Promising Avenues

A few striking trends in investments in healthcare sector shape the evolution of the red biotechnology market. Growing number of clinical trials in cancer research and incessant efforts of biotechnology players to find therapies for rare diseases are boosting the pace of new drug approvals. A few statistics support research and developments in aforementioned realms.

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Red Biotechnology Market: Regional Outlook

Among the various regional markets, North America leads the pack, and is expected to retain its dominance throughout the assessment period. Spate of investments in clinical trials for chronic and rare diseases and massive investments in developing cancer drug pipeline have helped the North America red biotechnology market to reach dominant position. Stakeholders are harnessing artificial intelligence will continue new prospects in the regional market. Prominently, relentless efforts of biopharmaceutical players in the U.S. test new drugs and vaccines form crucial trend in the growth of this regional market.

On the other hand, a growing numbers of players have shifted their attention to Asian economies to tap into the vast latent potential in personalized healthcare. The region will see new strategic collaborations among healthcare companies and biopharmaceutical players.

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Red Biotechnology Market Competition Landscape

Growing demand for biologics and biosimilars in developing economies has been crucial to the expansion of red biotechnology prospects, globally. Most notably, Asia Pacific has been the key focus for players to gain competitive edge over their peers and rivals.

Biopharmaceutical players have begun inking new partnership deals and distribution agreements in the region, particularly in on oncology therapeutics and development. Focus on novel therapeutics and disease pathways for bettering human health has been one of the key winning imperatives for companies in the red biotechnology market. To consolidate their positions, top players are aiming to expedite product approvals for rare and chronic diseases, and have increased their stakes in advanced stages of clinical trials.

A handful of big pharmaceutical and biotech players, and multinational healthcare companies jointly contribute half of the global revenues. These include Pfizer Inc., Gilead Sciences Inc., Amgen Inc., and F. Hoffmann-La Roche.

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The red biotechnology market is segmented on the basis of application, end user, and region.

Explore Transparency Market Research's award-winning coverage of the global Healthcare industry:

Bioinformatics Market- The global bioinformatics market is projected to grow at a significant CAGR during the forecast period and is likely to touch a valuation of US$9.1 bn by 2018.

Gene Therapy Market -Expanding at a stellar, double-digits CAGR (Compound Annual Growth Rate) of 40% over the forecast period of 2018 to 2026, the global gene therapy market is a dizzying trajectory, marking out a rosy landscape for players operating in the playfield. As per a Transparency Market Research report, based on extensive primary and secondary research, states that over the period states, the market would accrue a worth of USD 5164.03 million a steep and impressive increase from the USD 17 million worth noted in 2017.

Biosimilars Market- Advancements in oncology settings are improving patient quality of life. Due to effective biosimilars, healthcare providers are able to receive better outcomes in several cancer patients who can opt for convenient treatments such as a painless injections rather than receiving a lengthy chemotherapy session. Patent expiry of biologic drugs for the treatment of cancer are creating a scope for incremental opportunities. As such, oncology indication segment of the biosimilars market is estimated to reach a value of~US$ 21.1 Bnby the end of2027.

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Exploring Avenues in Personalized Medical Care Defines Advances in Red Biotechnology Market: Valuation to Touch Massive US$512 Bn By 2027, Finds...

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Correcting Barth Syndrome With Gene Therapy – Gilmore Health News

Wednesday, March 18th, 2020

Barth syndrome is an X-linked metabolic disorder, affecting only males. It has widespread systemic effects presenting with cardiomyopathy, neutropenia, muscle weakness, stunted growth, exercise intolerance and abnormal skeletal structures. In many cases, it results in stillbirth. It is strongly related to mutations in the tafazzin gene, also known as TAZ. Currently only symptomatic treatment exists, and no definite cure has been developed for Barth syndrome.

Read Also: CRISPR Used for the First Time to Treat a Blind Patient

Researchers at Boston Childrens Hospital have proposed gene therapy as a potential treatment method to inhibit processes that lead to heart failure. The researchers conducted the study on mouse models with Barth syndrome.

A research to better understand Barth syndrome was conducted in 2014 by William Pu, MD and colleagues at Boston Childrens Hospital. Together, they created heart on chip models of Barth syndrome by using cardiac myocytes derived from patients with TAZ mutation. This led the researchers to discover the correlation between Barth syndrome and dysfunction. When the defective mutated TAZ myocytes was replaced by healthy TAZ gene myocytes, the cardiac dysfunction was spontaneously corrected.

Pu and colleagues realized that in order to fully understand the effects of Barth syndrome on the system, an animal body was crucial. Attempts at creating a whole body model had previously been done, but had not been successful.

The Beatson Institute for Cancer Research in the U.K has recently been successful in creating mouse models of Barth syndrome. Two categories of these mouse models were created, in the first category TAZ gene was deleted throughout the whole system whereas in the second category of mouse models ha TAZ gene deleted only from the cardiac myocytes.

The mouse models with whole body TAZ deletion died before birth mostly due to hypotonic weak musculature. However, some of the mice survived and developed cardiomyopathy, similar to the dilated cardiomyopathy in humans. The hearts left ventricle had thinner walls and dilated substantially which decreased the systolic pressure resulting in decreased cardiac output.

In those mice with deleted TAZ in heart muscle cells, all subjects survived but had cardiomyopathy issues and reduced cardiac output. Under electron microscope, the heart muscles were found to have abnormal structures and poor organization.

Read Also:UC Berkeley Researchers Restore Vision in Mice Through Gene Insertion

Using gene therapy, the researchers replaced the TAZ gene by administering a gentically engineered virus subcutaneously or intravenously. Whole body TAZ deletion mice survived to an average life span of healthy mice. It successfully prevented cardiac dysfunction in all mice models.

Only when more than 70 percent cardiac myocytes had taken up the modified TAZ gene, significant improvement was seen.

The problem is that neutralizing antibodies to the virus develop after the first dose, said Pu. Getting enough of the muscle cells corrected in humans may be a challenge.

Post introduction of TAZ gene corrected cells, the major problem was seen in sustaining the levels of modified gene cells. In comparison to cardiac myocytes, the number of corrected gene cells in skeletal muscles declined progressively.

https://www.sciencedaily.com/releases/2020/03/200309165231.htm

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Correcting Barth Syndrome With Gene Therapy - Gilmore Health News

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Engineering specialist selected to deliver expansion of CGT Catapult – EPM Magazine

Wednesday, March 18th, 2020

Engineering and construction specialists Merit has been chosen to deliver the third phase expansion of the Cell and Gene Therapy Catapult (CGT Catapult) manufacturing centre in Stevenage, further increasing the capacity of the facility.

The company will use its offsite manufacturing technology and expertise in building controlled environments to deliver seven new quality control laboratories.

Merit completed construction of the expansion phase of the manufacturing centre in October last year using its off-site PAM (pre-assembled module) approach.

For these new laboratories, only 10% of the labour content will be carried out on site. The company will engineer pre-assembled analytical QC laboratory space at its offsite manufacturing centre reducing the build time by 33%, aiming to deliver cost savings to the project while also avoiding disruption to research and manufacturing at the Stevenage facility.

Tony Wells, managing director, Merit, said: We are delighted to have the opportunity to continue to work with the team at CGT Catapult and are proud to have an integral role in the development of such a pioneering off-site initiative. Our proprietary Instant Building technology combines advanced offsite manufacturing with the technical expertise to deliver complex solutions in significantly shorter build schedules. We have a detailed knowledge of the project after successfully completing the expansion phase last year and look forward to working closely with CGT Catapult to once again deliver a high-quality facility.

Backed by over 75 million of funding, including investment from the UK Governments Industrial Strategy Challenge Fund; the department of Business Energy and Industrial Strategy, from Innovate UK, the UKs innovation agency, the European Regional Development Fund and from Hertfordshire Local Enterprise Partnership, the centre is providing the infrastructure and expertise to enable companies to develop their manufacturing capabilities and systems for large scale, commercial cell and gene therapy supply. Companies currently collaborating at the centre are Adaptimmune, Autolus, Freeline Therapeutics and TCRTherapeutics.

Keith Thompson, CEO, Cell and Gene Therapy Catapult, said: It is a pleasure to work with Merit again to expand our laboratory space to provide additional analytical capabilities at the Cell and Gene Therapy Catapult manufacturing centre. Merit provided diligent support and ensured minimal disruption during the building of our expansion phase at the centre, and this is crucial to maintaining the facility operational for us and our collaborating companies manufacturing therapies, in an operational GMP compliant state. We look forward to working with Merit again on what is another significant project for CGT Catapult.

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Engineering specialist selected to deliver expansion of CGT Catapult - EPM Magazine

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Gene Therapy and Antisense Drugs Market Growth Trends, Key Players, Competitive Strategies and Forecasts to 2025 – 3rd Watch News

Wednesday, March 18th, 2020

Global Gene Therapy and Antisense Drugs Market: Snapshot

Since an escalating number of patients are being diagnosed with cancer every day, the global demand for gene therapy and antisense drugs is bound to multiply. The growing incidence of health conditions such as Parkinsons disease and high cholesterol is also boosting the demand for gene therapy and antisense drugs. Several new drugs and therapies have been making their debut in the global gene therapy and antisense drugs market of recent.

For instance, in June 2017, it was announced that a group of scientists at the University of Queensland have developed a new technique that is capable of permanently silencing severe allergies triggered by shellfish, peanuts, and venom. The treatment was found to be successful in animal trials. The technique holds considerable potential for treating asthma completely. During the same month, results of another research study for treating multiple myeloma, a type of blood cancer, were announced at a conference held by the American Society of Clinical Oncology. The new CAR-T therapy consists of filtering the blood of the patients in order to eliminate T cells which are then genetically altered and given back to the patients.

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However, the difficulty pertaining to the delivery of antisense technology to the brain can present key challenges to the expansion of the global market for gene therapy and antisense drugs. Moreover, the unavoidable toxic aftereffects associated with the technology can also inhibit the growth of the gene therapy and antisense drugs market worldwide. However, the present attempts at developing safe and efficient antisense drugs by several market participants including Ionis Pharmaceuticals, which undertook testing of the technology on transgenic mice, can promote the further expansion of the market.

Global Gene Therapy and Antisense Drugs Market: Overview

Antisense gene therapy is emerging as one of the most beneficial therapeutics for various diseases such as tumors, cancer etc. These newer therapies are based on increased knowledge of molecular events that lead to disordered cellular growth. The therapy involves using a gene silencing technique rather than a gene repairing technique for silencing the genes effect.

The research report is a valuable tool for comprehending the progression of the global gene therapy and antisense drugs market between 2017 and 2025.

Global Gene Therapy and Antisense Drugs Market: Treatment Insights

Antisense drugs attach to the mRNA of a target protein, which inhibits the protein production process. Antisense therapeutics can obstruct the expression of oncogenes and other cancer-related genes that express growth factors. Antisense gene therapy involves the utilization of various therapeutic strategies which requires a clear knowledge of the molecular anatomy of cancer related genes. Antisense gene therapy is used to treat various diseases such as hemorrhagic fever, cancer, cystic fibrosis, renal diseases, HIV/AIDS, spinal muscular atrophy, and cardiovascular diseases.

Global Gene Therapy and Antisense Drugs Market: Market Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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Gene Therapy and Antisense Drugs Market Growth Trends, Key Players, Competitive Strategies and Forecasts to 2025 - 3rd Watch News

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Anchiano Therapeutics Reports Year-End 2019 Financial Results – GlobeNewswire

Wednesday, March 18th, 2020

CAMBRIDGE, Mass., March 18, 2020 (GLOBE NEWSWIRE) -- Anchiano Therapeutics Ltd. (Nasdaq: ANCN) (Anchiano), a biopharmaceutical company focused on discovery and development of novel therapies to treat cancer, today reported financial results forthe year ended December 31, 2019.

Key Developments

Year Ended December 31, 2019 Financial Results:

On December 31, 2019, Anchiano had total cash and cash equivalents of approximately $17.6 million. Financial resources are expected to suffice through the end of 2020.

Research and development expenses for the year ended December 31, 2019 were approximately $13.3 million, compared to approximately $7.5 million for the same period in 2018. This increase was mainly due to an increase in clinical trial expenses, manufacturing expenses and manpower expenses, as well as additional startup and initial ongoing expenses in connection with the Collaboration Agreement with ADT.

General and administrative expenses for the year ended December 31, 2019 were approximately $6.2 million, compared to expenses of approximately $5.5 million for the same period in 2018. The increase was mainly due to increases in professional fees, insurance and manpower expenses, offset by a decrease in share-based payment.

Financing expenses, net, in the year ended December 31, 2019 were approximately $4.2 million, compared to approximately $457 thousand for the same period in 2018. This change was mainly due to a revaluation of investor warrants at fair value during a period where these could not be classified within shareholders equity.

Restructuring expenses in the year ended December 31, 2019 were approximately $3.4 million, and were comprised principally of contract termination costs and employee severance and associated termination costs related to the reduction of workforce resulting from Anchianos decision to discontinue its Phase 2 Codex as described above.

Net loss for the year ended December 31, 2019 was approximately $27.1 million compared to approximately $13.8 million for the same period in 2018.

About Anchiano

Anchiano is a biopharmaceutical company dedicated to the discovery, development, and commercialization of novel targeted therapies to treat cancer in areas of significant clinical need, with its headquarters in Cambridge, MA. Anchiano is developing small-molecule pan-RAS inhibitors and inhibitors of PDE10 and the -catenin pathway. For more information on Anchiano, please visit http://www.anchiano.com.

Forward-Looking Statements

This press release contains forward-looking statements that are subject to risks and uncertainties. Words such as believes, intends, expects, projects, anticipates and future or similar expressions are intended to identify forward-looking statements. These forward-looking statements are subject to the inherent uncertainties in predicting future results and conditions, many of which are beyond the control of Anchiano, including, without limitation, the risk factors and other matters set forth in its filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2019. Anchiano undertakes no 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.

Company Contact:Frank Haluska, M.D., Ph.D.President and Chief Executive Officerinfo@anchiano.com

Investor Contact:Ashley R. RobinsonManaging DirectorLifeSci Advisors, LLC617-430-7577arr@lifesciadvisors.com

RESULTS OF OPERATIONS (unaudited)

U.S. dollars in thousands

STATEMENTS OF FINANCIAL POSITION (unaudited)

U.S. dollars in thousands

CASH FLOWS (unaudited)

U.S. dollars in thousands

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Anchiano Therapeutics Reports Year-End 2019 Financial Results - GlobeNewswire

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Gene Therapy Market size Witness Growth Acceleration during 2027 – Packaging News 24

Wednesday, March 18th, 2020

Report Description

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

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

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

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

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

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

Segment Covered:

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

Profiling of Market Players:

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

Report Highlights:

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

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Salient Features:

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

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

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

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

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

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

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

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

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

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

Reasons to buy:

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

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

vFacilitate decision-making based on strong historic and forecast data

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

vDevelop strategies based on the latest regulatory events

vIdentify key partners and business development avenues

vRespond to your competitors business structure, strategy and prospects

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Gene Therapy Market size Witness Growth Acceleration during 2027 - Packaging News 24

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Global Cancer Gene Therapy Market, Trends, Analysis, Opportunities, Share and Forecast 2018-2025 – Galus Australis

Wednesday, March 18th, 2020

Global Cancer Gene Therapy Report available at Digits n Markets contains an overview of the Global Cancer Gene Therapy which covers market size, opportunities, trends, growth rate, and competition landscape. The Global Cancer Gene Therapy is segmented Source, Product Type Applications and regions. With forecast to 2027.

Digits n Markets has recently published a comprehensive market research report on the Global Cancer Gene Therapy that includes evaluation of market size and various segments. The competitive environment is analyzed along with study of winning strategies adopted by key players.

The report is a detailed study on the accounting Global Cancer Gene Therapy with details regarding an in-depth evaluation of the industry vertical. The study is performed taking into consideration a twofold aspect of consumption and production. Speaking of the product category, the report provides detailed product remuneration, manufacturing of the product and the gross margins of the firms manufacturing the products. With regards to the consumption, the study reveals the product consumption value and the product consumption volume along with the status of import as well as the export of the products.

The Global Cancer Gene Therapy Market Anticipated to exhibit a CAGR 35.1% during Forecast Period. 2018-2025

Avail a free sample in PDF format along with a quick look at vital report briefs:https://digitsnmarkets.com/sample/6650-global-cancer-gene-therapy-market

Key Questions Answered by the Report:

Numerous micro and macro-economic factors impacting the growth of the market are analyzed and the data is represented in a way to aid the clients to enhance their strategic decision making. Key players operating in the Global Cancer Gene Therapy are:

Table of Content

Chapter 1: IntroductionChapter 2: Executive SummaryChapter 3: Market OverviewChapter 4: Cancer Gene Therapy Market, By TypeChapter 5: Cancer Gene Therapy Market, By ApplicationChapter 6: Cancer Gene Therapy Market, By RegionChapter 7: Competition Landscape

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Global Cancer Gene Therapy Market, Trends, Analysis, Opportunities, Share and Forecast 2018-2025 - Galus Australis

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Gene therapy used in clinical trial for person with haemophilia – The Irish Times

Friday, March 6th, 2020

Gene therapy has been used to treat a person with haemophilia for the first time in Ireland, a patient group has announced.

The Irish Haemophilia Society (IHS) confirmed on Thursday morning that the person received gene therapy as part of a clinical trial. IHS chief executive Brian OMahony said the treatment is a momentous occasion for the haemophilia community in Ireland.

The general term haemophilia describes a group of inherited blood disorders in which there is a life-long defect in the clotting mechanism of the blood.

Since the 1970s, haemophilia has been treated by the administration of intravenous infusions of the missing clotting factor. However, work done by companies and academic institutions has given new hope that an effective treatment could be based on gene therapy, the IHS said.

The clinical trial uses a viral vector to deliver gene therapy to the persons liver intravenously. In the past, viruses such as HIV and Hepatitis C decimated the haemophilia population in Ireland through contaminated blood. It is ironic that a virus could now be the delivery system which offers the best hope of a practical cure for severe haemophilia, Mr OMahony said.

It is hoped that the effect of the gene therapy infusion will last for many years and possibly for a lifetime.

The principal investigator on the trial in Ireland is Dr Niamh OConnell of the National Coagulation Centre in St. Jamess Hospital. She said the gene therapy was ground breaking.

The opportunity to participate in clinical trials is part of the commitment of the National Haemophilia Service to personalise treatment and to improve the quality of life and outcomes for people with haemophilia.

The study, which is being run by drug manufacturer UniQure, involves three Irish patients among a total of 60 around the world. There will be an intensive period of monitoring of effectiveness at first, followed by a longer term evaluation over five years. Only one treatment is administered to trial patients.

The particular gene therapy is focused on patients who are missing clotting factor IX, the second most-common type of haemophilia. Earlier results show that the level of clotting factor increased from 1 per cent - generally seen as severe haemophilia - to between 33 and 51 per cent in a small number of individuals treated, levels seen in mild cases or even amongst the non-haemophiliac population.

Professor Martina Hennessy of the Wellcome HRB Clinicial Research facility in St Jamess, where the gene therapy was infused, said that access to high quality research is an integral part of good healthcare because it raises standards and pushes the boundaries of what can be achieved.

Delivering gene therapy requires specialised training and equipment, we have been preparing with Dr OConnell and her team for over a year to undertake this exciting work, in partnership with the Irish Haemophilia Society. Other trials are planned, we hope this expertise leads other Irish patient groups also being able to access potentially life changing treatments in the future, she said.

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Gene therapy used in clinical trial for person with haemophilia - The Irish Times

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Serial child rapist sentenced to up to 126 years in jail; new gene therapy from UPMC could cure diabetes: Tod – PennLive

Friday, March 6th, 2020

Listen to the latest episode at this link, or on your favorite app including Alexa, Apple, Google, Spotify and Stitcher. Episodes are available every morning on PennLive. Subscribe/Follow and rate the podcast via your favorite app.

A serial child rapist is going behind bars for life after being sentenced to up to 126 years in jail. Meanwhile, a couple is being charged with arson and insurance fraud after allegedly setting fire to their newspaper business. In Pittsburgh, a UPMC researchers new gene therapy could cure diabetes. Also, with Easter on the horizon, Cadbury is on the hunt for a different kind of bunny -- and one Pennsylvania llama is throwing its hat in the race.

Those are the stories we are covering in the latest episode of Today in Pa, a daily weekday podcast from PennLive.com and hosted by Julia Hatmaker. Today in Pa is dedicated to sharing the most important and interesting stories in the state.

Todays episode refers to the following articles:

Special thanks to Apple Podcast listener wildbill95 for leaving Today in Pa. a review. Heres what they wrote:

Great start to the day! Julia provides the most-needed details on the days news, and makes a great way to begin the morning. Just as important, Julia gives us the lede to stories we now know to look for the in-depth article on the website.

Thanks, Julia, for giving us a boost on a ho-hum morning!"

If you enjoy Today in Pa, consider leaving us a review on Apple Podcasts or on Amazon. Reviews help others find the show and, besides, we like to know what you think of the program.

Thanks for visiting PennLive. Quality local journalism has never been more important. We need your support. Not a subscriber yet? Please consider supporting our work.

Go here to see the original:
Serial child rapist sentenced to up to 126 years in jail; new gene therapy from UPMC could cure diabetes: Tod - PennLive

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