Orchard Therapeutics Outlines Comprehensive Presence at 2021 WORLDSymposium™
Nine abstracts accepted demonstrating potential of HSC gene therapy to treat multiple neurodegenerative disorders
New clinical data from all eight patients treated with OTL-203 for Mucopolysaccharidosis type I (MPS I)
Biomarker data from first three patients treated with OTL-201 for Mucopolysaccharidosis type
Multiple abstracts highlighting clinical and real-world data for OTL-200 and Metachromatic Leukodystrophy (MLD)
Company to host virtual investor webinar to review symposium data on
“Together with our clinical partners, we’re proud of our presence at the upcoming WORLDSymposium, which for the first time features clinical data on cognitive function and growth in all eight MPS-I patients treated with gene therapy,” said
The presentations are listed below and the full preliminary program is available online on the WORLDSymposium website. The ePosters will open at
Orchard is planning to host a virtual investor webinar on
Platform Oral Presentation Details:
Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial
Presenting Author: Jane Kinsella, on behalf of the Clinical Trials Team at Royal Manchester Children’s Hospital, part of Manchester University NHS Foundation Trust – 2021 Young Investigator Award Recipient Date/Time: Tuesday, February 9, 2021, 11:12 a.m. ET
Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome)
Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early juvenile metachromatic leukodystrophy
ePoster Presentation Details:
Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200
Abstract Number: 25
Initial signs and symptoms of metachromatic leukodystrophy: A caregiver perspective
Abstract Number: 64
Demographic and clinical characteristics of patients with metachromatic leukodystrophy in the
Abstract Number: 110
Quality of life of patients with metachromatic leukodystrophy and their caregivers in the US,
Abstract Number: 186
Health-related quality of life in metachromatic leukodystrophy based on a societal utility study in the
Abstract Number: 187
Newborn screening for metachromatic leukodystrophy in
Abstract Number: 269
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. This includes Royal Manchester Children’s Hospital (RMCH), one of the largest children’s hospitals in the UK with an international reputation as a centre of excellence for research and innovation. More information is available at www.mft.nhs.uk.
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
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 EMA website.
Libmeldy is not approved outside of the
Libmeldy was developed in partnership with the
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 and LinkedIn), 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 Orchard’s investor relations website and may include additional social media channels. The contents of Orchard’s 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.
This press release contains certain forward-looking statements about Orchard’s 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, Orchard’s business strategy and goals, and the therapeutic potential of Orchard’s 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 Orchard’s 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 Orchard’s 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 Orchard’s 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 Orchard’s 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 Orchard’s regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchard’s product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchard’s ability to commercialize its product candidates, if approved, or Libmeldy in the EU; the risk that the market opportunity for Libmeldy, or any of Orchard’s product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchard’s 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 Orchard’s quarterly report on Form 10-Q for the quarter ended September 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchard’s 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.
Director, Investor Relations
Vice President, Corporate Affairs
Source: Orchard Therapeutics (Europe) Limited