Seven abstracts accepted show the potential of HSC gene therapy to transform the treatment of multiple severe rare disorders

First peer-reviewed presentation from companys work in transduction enhancers showcases promise to improve manufacturing efficiency of lentiviral-based gene therapy

BOSTON and LONDON, April 28, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of seven abstracts at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place virtually from May 11-14. Accepted abstracts include clinical data from several of its hematopoietic stem cell (HSC) gene therapy programs, including: OTL-203, being investigated for the treatment of mucopolysaccharidosis type I (MPS-I), and OTL-101, being investigated for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID), as well as the companys efforts to improve efficiency of HSC gene therapy manufacturing though the development of proprietary transduction enhancers.

Our significant presence at ASGCT this year showcases not only the depth and breadth of our HSC gene therapy pipeline and the strength of our clinical data but also how we are using innovation to improve the way our HSC gene therapies will be manufactured in the future, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. These data demonstrate the transformative potential of HSC gene therapy and, along with our partners, we look forward to sharing results with the broader scientific and medical communities.

The presentations are listed below, and the full preliminary program is available online on the ASGCT website.

Oral Presentation Details:

Development of an optimized lentiviral transduction process for ex vivo CD34+ hematopoietic stem cell gene therapy drug product manufactureAbstract Number: 1Presenting Author: Pervinder Sagoo, Ph.D., Orchard TherapeuticsDate/Time: Tuesday, May 11 at 5:30 p.m. EDT

Autologous ex vivo lentiviral gene therapy for the treatment of ADA-SCIDAbstract Number: 40Presenting Author: Claire Booth, Ph.D., UCL GOSH Institute of Child HealthDate/Time: Tuesday, May 11 at 6:30 p.m. EDT

Immune reconstitution in transfusion dependent beta-thalassemia patients treated with hematopoietic stem cell gene therapyAbstract Number: 79Presenting Author: Samantha Scaramuzza, Ph.D., San Raffaele Telethon Institute for Gene TherapyDate/Time: Wednesday, May 12 at 5:45 p.m. EDT

Hematopoietic reconstitution and lineage commitment in HSC gene therapy patients are influenced by the disease backgroundAbstract Number: 82Presenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene TherapyDate/Time: Wednesday, May 12 at 6:30 p.m. EDT

Liquid-biopsy-integration-site-sequencing allows safety studies and longitudinal monitoring of vector integration sites in LV and AAV-based in-vivo GT applicationsAbstract Number: 250Presenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene TherapyDate/Time: Friday, May 14 at 12:15 p.m. EDT

Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome): An interim analysis with a median follow up of 19 monthsAbstract Number: 219Presenting Author: Bernhard Gentner, M.D., San Raffaele Telethon Institute for Gene TherapyDate/Time: Friday, May 14 at 1:15 p.m. EDT

Poster Presentation Details:

Dissecting bone remodeling mechanisms and hematopoietic stem cell gene therapy impact in mucopolysaccharidosis type I Hurler bone defects Abstract Number: 609Presenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene TherapyDate/Time: Tuesday, May 11 at 8:00 a.m. EDT

About Orchard TherapeuticsOrchard 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, and follow us on Twitter and LinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (, the investor relations website (, 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 StatementsThis 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, 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, these risks and uncertainties include, without limitation: 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, will be insufficient to support regulatory submissions or marketing approval in the US or EU, as applicable, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product 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 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 its product candidates, 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 Annual Report on Form 10-K for the year endedDecember 31, 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.


InvestorsRenee LeckDirector, Investor Relations+1

MediaBenjamin NavonDirector, Corporate Communications+1

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Orchard Therapeutics Announces Multiple Presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy - GlobeNewswire

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