Category Archives: Gene therapy

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the Companys plans for the buildout of its new Research and Development (R&D) and Chemistry, Manufacturing and Controls (CMC) operation which will also serve as the Companys new headquarters in Cranbury, New Jersey. This new 103,720 ft2 facility will support clinical development of Rockets growing pipeline of lentivirus (LV) and adeno-associated virus (AAV) gene therapies from discovery through pivotal trials, with space for potential future expansion and commercialization. This buildout comes on the heels of the Companys recent successful capital raise of approximately $300 million that provides a cash runway into the second half of 2023.

Investing in R&D and manufacturing innovation, talent, and capacity through this new world-class facility will allow us to deliver on our mission of bringing five curative gene therapies to rare disease patients by 2025, said Gaurav Shah, M.D., President and Chief Executive Officer of Rocket. With data on five clinical programs expected this year, including two that are in registration-enabling trials, these new capabilities will enable us to work with continued urgency and purpose towards bringing transformational therapies to patients.

We are collaborating with some of the best scientists and innovators worldwide. This new facility is instrumental in bolstering Rockets gene therapy capabilities to rapidly advance multiple platforms and programs efficiently and effectively. Producing clinical drug product will enable greater control of supply, cost, quality, and timing to pave a smoother path toward commercialization, added Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development.

Approximately one-half of the facility is being scaled for AAV Current Good Manufacturing Practice (cGMP) production. The other half features state-of-the-art R&D labs to support the expanding pipeline and Quality Control (QC) laboratories to support CMC development for process and analytics.

Rocket recently reported positive interim Phase 1 results for its first AAV-based gene therapy, RP-A501 for the treatment of Danon Disease. The first cGMP production at this facility will be initiated in 2021 and will be used in a planned Phase 2 registrational study evaluating RP-A501, following the completion of the current Phase 1 trial.

Approximately $300 million secured in public equity offering

On December 14, 2020, Rocket closed an upsized underwritten public offering of 6,035,714 shares of its common stock, inclusive of greenshoe, at the public offering price of $56.00 per share. The offering was ~7.3x oversubscribed based on the initial deal size of $175 million. Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the U.S. and Europe, accelerate the buildout of in-house manufacturing capabilities, and for general corporate purposes. This capital raise extends Rockets cash runway to the second half of 2023.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the preclinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, filed November 8, 2019 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Buildout of R&D and Manufacturing Facility to Support Development of Innovative Gene Therapy Pipeline - Business Wire

PARIS--(BUSINESS WIRE)--Regulatory News:

Lysogene (Paris:LYS) (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announces that it has received MHRA and Research Ethics Committee approvals to initiate the gene therapy clinical trial in the United Kingdom with LYS-GM101 for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease. LYS-GM101 builds on Lysogenes extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.

This is the first regulatory authorization to initiate this global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The company expects to dose the first patient in the first half of 2021.

This trial builds on Lysogenes considerable expertise for AAV gene therapy and clinical development. Since the most damaging effects of GM1 gangliosidosis occur in the brain and spinal cord, LYS-GM101 will be administered into a cerebrospinal fluid-filled space at the back of the head called the cisterna magna so that enough therapy reaches those tissues. I expect the study to provide us with data demonstrating the transformative potential of LYS-GM101 said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene. LYS-GM101 is our second gene therapy program entering the clinical phase after LYS-SAF302 for MPS IIIA, followed by a discovery stage pipeline, including gene therapy candidates for Fragile X syndrome, Gaucher and Parkinson's disease.

LYS-GM101 (adeno-associated viral vector serotype rh.10 expressing beta-galactosidase) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016.

Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors in infantile and juvenile GM1 gangliosidosis (NCT04310163).

About Lysogene

Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

About Manchester University NHS Foundation Trust

Manchester University NHS Foundation Trust is one of the largest NHS trusts in England and a leading provider of specialist healthcare services. Its nine hospitals are home to hundreds of world class clinicians and academic staff committed to finding patients the best care and treatments. More information is available at http://www.mft.nhs.uk.

Forward Looking Statement

This press release may contain certain forward-looking statements, especially on the Companys progress of its phase 2-3 clinical trial and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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Lysogene Receives MHRA and Research Ethics Committee Approvals to Initiate the Gene Therapy Clinical Trial in the UK with LYS-GM101 for the Treatment...

Few government agencies were as front and center in the public eye last year as the Food and Drug Administration, which came under immense pressure to authorize unproven drugs as the world grappled with the pandemic and conducted high-stakes reviews of two coronavirus vaccines.

As COVID-19 cases continue to soar in the U.S., most of those pressures haven't gone away. But the inauguration next week of Joseph Biden as president will almost certainly bring new management to the agency, a change that can often result in policy shifts both subtle and far-reaching.

There will also be new faces at the FDA's parent agency, the Department of Health Human Services, which has battled FDA leadership at several points during the Trump administration. Even now, with days left to Trump's term in office, HHS officials are reportedly attempting to ram through several new policies that impact the FDA.

The pace and frequency of drug approvals, meanwhile, has quickened in recent years, prompting some to question whether reviewers are showing enough caution. Depending on who's appointed commissioner, the Biden administration could pull the reins somewhat.

"I think after so many years of faster, faster, faster, we're going to see a little bit of a pendulum shift," Remy Brim, co-lead of the health and life sciences practice at lobbying firm BGR Group, said Dec. 14 during the virtual Biopharma Congress sponsored by Prevision Policy.

"FDA, especially under Democratic administrations, really feels that a product is not safe and effective until it's shown it's safe and effective," Brim said. "It doesn't necessarily take the stance that a six -month or a one-year delay is detrimental."

Here are five issues before the FDA that will define its first year under a Biden administration:

The FDA commissioners under President Donald Trump, Scott Gottlieb and Stephen Hahn, have been relatively steady leaders. Even though Hahn yielded to pressure from Trump to grant some controversial coronavirus drug authorizations, he became a fierce defender of the agency's review process for vaccines developed by Moderna and partners Pfizer and BioNTech.

While Hahn offers continuity amid the pandemic, the Biden administration is almost certain to appoint a new commissioner. The appearance of David Kessler, an FDA commissioner under Presidents George H.W. Bush and Bill Clinton, on the Biden transition team raised eyebrows. Two other Obama administration-era FDA officials, Luciano Borio and Lisa Barclay, are also members of the team.

Kessler, however, is reportedly out of the running for a FDA commissioner appointment, according to BioCentury.

Other names that have emerged include Gottlieb and two former commissioners, Margaret Hamburg and Mark McClellan. From within the agency, Amy Abernethy, the principal deputy commissioner whose name surfaced the last time there was a vacancy, has also been mentioned, as has a past holder of Abernethy's post, Joshua Sharfstein, now at Johns Hopkins University.

In a Nov. 16 note to clients, Cowen analyst Rick Weissenstein wrote Sharfstein's continued involvement in agency issues since leaving a decade ago makes him the best nomination for the sector. "We see a pick like Sharfstein as the best possible outcome as it would mean continuity at the FDA and maintain a positive environment for drug developers," he wrote.

Authorizing two coronavirus vaccines in two weeks last December was a monumental task, but the job isn't done. Three more vaccines could soon have late-stage study results supportive of authorizations, too, while testing continues for new experimental antivirals and antibodies that aim to help infected patients stay alive and out of the hospital.

If the latest vaccines succeed in the clinic, the FDA would likely convene an advisory committee meeting for each one as it did with the Pfizer and BioNTech and Moderna vaccines, a time-consuming task that is nonetheless considered important for building public trust.

Meanwhile, drugmakers that developed coronavirus treatments now cleared for emergency use, including Regeneron and Eli Lilly, may seek to convert their authorizations into full approvals.

The job of reviewing all of them will be made more complicated by the companies' efforts to expand the populations in which they can be used. Both the Regeneron and Lilly antibody drugs were authorized after results in comparatively small numbers of patients. Data from larger groups could change the conclusions of the drugs' effectiveness and pose thorny questions for reviewers.

The massive trials to push coronavirus vaccines through from design to emergency authorization in less than a year, and a separate one to quickly evaluate treatments of hospitalized patients, have proven that big studies do not necessarily need to be multi-year affairs. The RECOVERY trial in the U.K., in particular, showed how a simple trial asking only one or two urgent public health questions can yield answers within weeks or a few months. The U.S. government's COVE trial, which tested Moderna's vaccine, did something similar.

The public health emergency of 2020 no doubt inspired agency reviewers to be more flexible about clinical trial design and the speed at which they were conducted. While normal times won't bring the same pressure, the experience could have lessons for regulators' oversight of trials in the future.

Particularly in cancer and rare diseases, the FDA has shown that it's willing to work with drug developers to design trials that can determine whether or not treatments work in short order. RECOVERY and COVE point toward a way to do the same for broader public health questions, and the agency may need to consider how the study of new drugs for major conditions like diabetes or cardiovascular disease could be sped up.

The FDA's surprise rejection of BioMarin Pharmaceutical's hemophilia gene therapy Roctavian, along with scrutiny of manufacturing processes for other gene therapy developers, has led some to argue the FDA has raised standards. But Wilson Bryan, head of the agency's gene therapy review center, has argued the tough oversight is tied to "an increase in activity in the field."

Roctavian's rejection was unexpected, as two previous gene therapies, Roche's Luxturna and Novartis' Zolgensma, had largely non-controversial approvals. (The clearance for the latter, however, was marred by a post-approval data manipulation scandal.)

The FDA's concern, according to BioMarin, was that early data from later-stage testing showed a somewhat weaker treatment effect than a smaller, early study, raising questions about the durability of Roctavian's benefit. And in hemophilia, patients have many existing drug options available to them, which may have changed the agency's risk-benefit calculation.

"What does unmet need mean, when you have a gene therapy versus other products on the market?" Brim said at the Biopharma Congress session. "Once a patient takes [a gene therapy], they can't untake it."

More than 1,000 Investigational New Drug applications for gene therapies have already been submitted to the FDA, according to Bryan, meaning the demands on the agency will only grow with time.

The laws authorizing the user fees that partially fund FDA drug reviews known in shorthand as PDUFA sunset every five years, requiring Congress to pass a new one to sustain the program. The current edition is set to expire Sept. 30, 2022, so work is already underway to set the agenda for the seventh iteration of the law.

In the past, reauthorization of the PDUFA program has forced the agency to set firmer deadlines for drug approval decisions and has created expedited approval pathways such as the Breakthrough Therapy designation. The laws can also become a vehicle for Congress to achieve changes within the agency, which has been under the microscope during the coronavirus pandemic.

"How FDA handles itself on COVID is going to play into what type of policy riders we're going to see," Brim said.

A chief FDA request has been for more resources to beef up the staffing in its gene and cell therapy division. The agency, meanwhile, may be called upon to expand its work on new models of clinical trials, such as adaptive studies, that can shorten the amount of time experimental drugs spend in development.

Drugmakers could also seek to streamline the manufacturing paperwork involved in expedited review pathways to further shorten timelines.

Excerpt from:
5 questions facing the FDA in 2021 - BioPharma Dive

Washington: The research is published in Science Advances.

The gene, called TSC2, codes for tuberin, a protein that acts to inhibit cell growth and proliferation. When mutations occur in TSC2, resulting in a lack of tuberin in cells, the cells enlarge and multiply, leading to the formation of tumors.

To restore the function of TSC2 and tuberin in a mouse model of tuberous sclerosis complex, researchers developed a form of gene therapy using an adeno-associated virus vector carrying the DNA that codes for a condensed form of tuberin (which fits within the vector's carrying capacity) and functions like the normal full-length tuberin protein.

Mice with tuberous sclerosis complex had a shortened life span of about 58 days on average, and they showed signs of brain abnormalities consistent with those that are often seen in patients with the disease.

When the mice were injected intravenously with the gene therapy treatment, however, their average survival was extended to 462 days, and their brains showed reduced signs of damage.

"Current treatments for tuberous sclerosis complex include surgery and/or lifelong treatment with drugs that cause immune suppression and potentially compromise early brain development. Therefore, there is a clear need to identify other therapeutic approaches for this disease," said co-lead author Shilpa Prabhakar, an investigator in the MGH departments of Neurology and Radiology.

"Adeno-associated virus vectors have been used widely in clinical trials for many hereditary diseases with little to no toxicity, long-term action in nondividing cells, and improvement in symptoms," adds Prabhakar.

She notes that benefits can be seen after a single injection, and some forms of the viral vector can efficiently enter the brain and peripheral organs after intravenous injection.

The US Food and Drug Administration has approved a limited number of gene therapy products for use in humans, and the results from this study suggest that clinical trials are warranted to test the strategy's potential in patients with tuberous sclerosis complex.

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Gene Therapy Strategy Found Effective in Mouse Model of Hereditary Disease TSC - DTNEXT

BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy for the treatment of early-onset metachromatic leukodystrophy (MLD). In late 2020, the FDA cleared the companys Investigational New Drug (IND) application for OTL-200, and the therapy also recently was approved in the European Union (EU) under the brand name, LibmeldyTM.

Receipt of RMAT designation for OTL-200 underscores both the severe nature of MLD and the transformative potential of the therapy for young patients suffering from this devastating, fatal neurodegenerative condition, said Bobby Gaspar, M.D., Ph.D., chief executive officer,Orchard Therapeutics. Alongside our open IND, RMAT designation provides an opportunity for enhanced interactions with the FDA to determine the optimal path to submit a Biologics License Application (BLA) for OTL-200 in the U.S.

Established under the 21st Century Cures Act, the RMAT designation program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition.The FDA granted Orchard RMAT designation for OTL-200 based on data submitted on 39 patients, including 9 patients from theU.S., who have received OTL-200 as part of clinical studies and compassionate use programs conducted at theSan Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)inMilan, Italy. This data set includes post-treatment follow-up data of up to eight years in the earliest treated patients in these programs.

We look forward to continued engagement with the FDA in the coming months to discuss the comprehensive data set we have already collected in the OTL-200 clinical development program and agree on the potential next steps on the regulatory path to approval for this innovative gene therapy, said Anne Dupraz, chief regulatory officer at Orchard.

About Libmeldy / OTL-200

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the European Medicines Agency (EMA) website.

Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein, and Norway. OTL-200 is an investigational therapy in the U.S.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

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 andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, 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, Orchards business strategy and goals, the therapeutic potential of Libmeldy (OTL-200), the likelihood that data from clinical trials will support further clinical development and regulatory approval of OTL-200, and the outcome of planned FDA interactions regarding the potential approval pathway for OTL-200. 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, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of OTL-200, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that OTL-200 or any one or more of Orchards product candidates 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 Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; 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 or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize OTL-200, if approved, or Libmeldy in the EU; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. 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 quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. 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 LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

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Orchard Therapeutics Announces OTL-200 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation by FDA for the Treatment of Metachromatic...

Sana Biotechnology is following the Moderna playbook to a tee: Promise a lot based on very early science, be vague, nab a major VC raise, then gun for an IPO.

While Moderna has, in some respects, come up good after its monster $600 million-plus IPO a few years back and it now looking to literally save the world and the economy with its mRNA vaccine, Sana is looking for a $150 million IPO for its range of preclinical stem cell and gene control platforms.

That $150 million could, if history repeats itself, swell to be much more than that: It raised a gigantic $700 million last summer from a whos who of VCs including Arch Venture Partners, Flagship Pioneering, the Canada Pension Plan Investment Board, Baillie Gifford, F-Prime Capital, the Alaska Permanent Fund, the Public Sector Pension Investment Board, Bezos Expeditions, GV, Omega Funds, Altitude Life Science Ventures and multiple unnamed institutional investors.

Many other early-stage biotech have also lowballed their IPO target only to see it eventually grow to 50% or 100% more than that.

RELATED: In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience': report

Last year, Sana licensed technology from Harvard University to further its efforts to develop off-the-shelf cell therapies, its main pipeline focus. The goal is to genetically modify and differentiate stem cells to create cell therapies that are cloaked from the immune system.

Using this, Sana said at the time it plans to harness these resources to create off-the-shelf cell therapies capable of treating a range of diseases. To achieve that goal, Sana will need to find ways to stop the immune system from rejecting the cells as foreign.

The biotech, run by a bunch of former Juno execs, is focused on a series of disease areas including oncology, diabetes, central nervous system disorders, cardiovascular diseases and genetic disorders.

All of its candidates are, however, in preclinical development, with IND submissions for clinical work not expected until 2022 and 2023, according to its Securities and Exchange Commission filing.

Our vision is to build the pre-eminent company focused on engineered cells to create medicines for patients, it said in its filing. Our mission is to do so at a scale that allows broad accessibility for patients so that we can democratize access to curative therapies.

To achieve this, we have strategically focused on the key limitations for generating engineered cell therapies, whether the cell modulation occurs in vivo or ex vivo. We also continue to aggregate the people and technologies that will allow us to research, develop, manufacture, and ultimately commercialize differentiated products across a range of diseases.

It plans to list as "SANA" on the Nasdaq.

More:
Secretive Sana, still a year away from the clinic, files for an IPO after a mammoth raise - FierceBiotech

Gene therapy pioneer Jim Wilson and the University of Pennsylvania are teaming up with Regeneron to help deliver its COVID-19 antibody cocktail using adeno-associated virus (AAV) tech in the hope of curbing infection via a nasal spray.

The antibody cocktail, made up of casirivimab and imdevimab, was given a speedy authorization by the FDA less than two weeks ago as a treatment for certain COVID-19 patients. But, keeping up with the fast pace of SARS-CoV-02 R&D, Regeneron is not resting on its laurels and now wants to find a quicker way of delivering its therapy while also working on it as a prophylactic.

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

RELATED: Regeneron, following in Lilly's footsteps, wins FDA emergency nod for COVID-19 antibody cocktail

These antibodies are currently injected into patients, but Regeneron and Penn will use Wilsons gene therapy know-how to attempt a nasal spray formulation using AAV vectors. The belief is that this could prevent infection with the virus using a technology typically used in high-tech gene therapies.

The group plans to study the safety and effectiveness of using AAV vectors to introduce the sequence of the cocktails virus-neutralizing antibodies directly to nasal epithelial cells and see whether it can help protect against the disease.

The first step is to finish preclinical trials; if successful, an IND will be sent off to the FDA for human trials.

Wilsons team said it was hopeful that introducing the therapy via single dose of AAV will be able to produce similar protection Regeneron has seen for its cocktail, but for potentially a longer duration.

Regeneron scientists specifically selected casirivimab and imdevimab to block infectivity of SARS-CoV-2, the virus that causes COVID-19, and we have been encouraged by the promising clinical data thus far, said Christos Kyratsous, Ph.D., vice president of research, infectious diseases and viral vector technologies at Regeneron.

In the quest to use cutting-edge science to help end this disruptive and often very devastating disease, we are excited to explore alternate delivery mechanisms such as AAV that may extend the potential benefits of this investigational therapy to even more people around the world.

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Wilson, Penn ink Regeneron pact to use gene therapy tech to deliver COVID-19 antibodies - FierceBiotech

SALT LAKE CITY (Ivanhoe Newswire)

Gene replacement therapy: Its a game-changer when it comes to treating life-threatening illnesses. It can replace disease-causing genes with healthy genes, knock out a gene thats not working right, or add a new gene to the body to help fight disease. To date, the FDA has approved four types of gene therapy including one that was given the OK just in time to save one little boys life.

No doubt about it, Cinch Wight is going to be a cowboy just like his dad.

He loves the dog and the horses and the cows, shared Cinchs dad, Alex Wight.

But it has been a wild ride for this young bronco. A mandatory newborn screening test at birth revealed Cinch had spinal muscular atrophy or SMA.

Cinchs mom, Amber Wight recalled, That was the first time Id ever even heard the term and what it was. And so, it was very scary.

A neuromuscular disorder that can paralyze a baby in the first few weeks of life.

My first thought was, hes never going to be able to ride broncs or anything like that, expressed Alex.

But just one day after Cinch was born, the FDA approved a new gene therapy.

We were pretty excited to get a phone call from the department of health, you know, and have this baby here who we can use this treatment on after its approval, explained Russell Butterfield, MD,pediatric neurologist at University of Utah Health and Intermountain Primary Childrens Hospital.

A critical gene in little Cinch was missing. Pediatric neurologist Russell Butterfield used an infusion to deliver a virus carrying a new copy of the gene into Cinchs nerve cells.

Its like a delivery truck to deliver genes to where you want them to go. What that does do, is it stops the disease right where it is, elaborated Dr. Butterfield.

Just a few years ago, most children born with SMA didnt make it to their second birthday. Now?

The hardest is holding a baby in one hand and holding that drug in the other and really feeling the weight of that. And understanding that how different this childs life will be with his new medicine, expressed Dr. Butterfield.

It took a lot of courage for this family to get this far. Thats why Alex wrote a book for his son. A true story about how real cowboys never give up.

I wanted to let him know that no matter how hard it gets, as long as he keeps going, hell be all right, shared Alex.

Doctors dont know if the one-time infusion will last a lifetime or will have to be repeated and there could be a possible risk of inflammation to the liver that doctors will closely monitor. The gene replacement therapy costs 2.1 million dollars. Insurance paid for most of it, but Alex hopes sales from his childrens book will help pay the rest. You can find the book, A Cowboy and His Horse, at https://www.amazon.com/COWBOY-HIS-HORSE-ALEX-WIGHT/dp/B08CWG46ZX.

Contributors to this news report include Cyndy McGrath, Executive Producer; Marsha Lewis, Field Producer; Rusty Reed, Videographer; Roque Correa, Editor.

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A little cowboy saved by groundbreaking gene replacement therapy - Wink News

Researchers based at the Neuroscience Institute at the University of Sheffield in the UK have identified a new genetic risk factor for Motor Neurone Disease (MND) in so-called 'junk DNA'.

The newly discovered genetic changes are present in up to 1% of MND patients.

The research, published in the journal Cell Reports, focused on genetic mutations in non-coding DNA, often known as junk DNA because it does not directly encode protein sequences. Non-coding DNA makes up more than 99% of the human genome, but currently is relatively unexplored.This research also includes new methods for studying mutations in non-coding DNA which could be applied to other diseases.

The authors of the study reported that they determined an existing neuroprotective drug developed at the University of California San Diego (UCSD) called SynCav1 could help MND patients carrying the newly discovered genetic mutation.

An experimental gene therapy for the treatment of neurological disorders such as MND and Alzheimers disease, SynCav1 has been licensed to CavoGene LifeSciences.

MND or Amyotrophic Lateral Sclerosis (ALS), as it is also known, affects motor neurons in the brain and spinal cord that form the connection between the nervous system and muscles to enable movement of the body. The progressive disease affects a patient's ability to walk, talk, use their arms and hands, eat and breathe.

Around 5,000 people in the UK and 30,000 people in the US are currently living with MND, with numbers expected to rise.

High-income countries currently have the highest rates of motor neuron diseases worldwide, and the burden is increasing with the ageing population, shows an analysis of the Global Burden of Diseases, Injuries, and Risk Factors Study (GBD) 2016.

Dr Jonathan Cooper-Knock, lead author of the study and NIHR clinical lecturer in Neurology at the Neuroscience Institute at the University of Sheffield, said: "Until now scientists have never systematically examined non-coding or junk DNA in relation to the development of MND.

"Not only have we identified a mutation in junk DNA which puts people at risk of developing a certain form of the MND, but we have also found that by targeting the mutated gene with the established neuroprotective drug called SynCav1, it might be possible to halt or potentially prevent the disease progressing in those patients.

"This is a significant breakthrough in terms of genetic risk factors driving personalized medicine for MND patients."

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An experimental gene therapy may be effective for MND patients with a newly discovered genetic mutation - BioPharma-Reporter.com

In the oncology world, theres no better hunting ground for cancer R&D execs than Genentech. The biotech franchise at Roche has worked on some of the leading drugs in the field, proven themselves with blockbuster returns, and carries weight for whatever it says and does.

The exodus of R&D talent out of the South San Francisco hub is a testament to their success.

Now one of their top research execs has been raided by a top China biotech player to satisfy not just their need for an oncology R&D chief as they build up their muscle in discovery and drug development, but also add a spotter for new cancer drug deals.

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Regeneron teams with gene therapy pioneer James Wilson, adapting its Covid-19 antibody cocktail to an AAV-based nasal spray - Endpoints News