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Orchard Therapeutics plc (ORTX) is a pioneering biotechnology company dedicated to transforming the lives of children with life-threatening rare diseases through advanced gene therapies. The company focuses on the development, manufacturing, and commercialization of ground-breaking hematopoietic stem cell (HSC) gene therapies, which hold the potential to provide one-time treatments that address the underlying causes of severe genetic disorders.
Founded in 2015, Orchard Therapeutics collaborates with prestigious research centers globally to harness the life-saving potential of gene therapy. The company’s leadership team and partners bring over a decade of experience in advancing therapies for orphan diseases.
Orchard’s flagship product, Libmeldy® (atidarsagene autotemcel), is designed for the treatment of early-onset metachromatic leukodystrophy (MLD), a rare metabolic disorder. Libmeldy has received approval from the European Commission and the UK Medicines and Healthcare products Regulatory Agency and is under Priority Review by the U.S. FDA with a decision expected by March 18, 2024. The therapy aims to halt or slow disease progression by restoring enzymatic function.
The company is also advancing OTL-203 for mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome. These programs utilize the same HSC gene therapy platform as Libmeldy.
In a strategic move, Kyowa Kirin recently acquired Orchard Therapeutics, enhancing its portfolio with Orchard's clinically differentiated gene therapy platform. This acquisition is expected to drive the development of new treatments and support ongoing and future launches, including the continued growth of Libmeldy in Europe and potential new approvals in the U.S.
Orchard Therapeutics continues its mission to deliver curative gene therapies for severe diseases by translating promising early-stage research into approved treatments available to patients worldwide. The team remains committed to leveraging its extensive experience and cutting-edge technology to address unmet medical needs in rare and underserved conditions.
Orchard Therapeutics (Nasdaq: ORTX) announced a strategic shift, focusing on hematopoietic stem cell gene therapies for severe neurometabolic diseases and early-stage research. The company aims to extend its cash runway into 2024 by reducing its workforce by 30% and discontinuing investment in primary immunodeficiency programs. Therapeutic candidates OTL-200 for MLD and OTL-203 for MPS-IH are highlighted for continued development, with a BLA filing for OTL-200 expected by early 2023. Recent commercial momentum for Libmeldy in Europe is gaining traction, enhancing the company's value proposition.
On March 24, 2022, Orchard Therapeutics (Nasdaq: ORTX) announced a conference call on March 30, 2022, at 8:00 a.m. ET to discuss business updates and its fourth quarter and fiscal year 2021 financial results. The call will be accessible via phone and live webcast. The company focuses on innovative gene therapies using hematopoietic stem cell (HSC) modifications to treat severe diseases. With a strong pipeline following the acquisition of GSK’s rare disease gene therapy portfolio, Orchard aims to deliver new curative treatments to patients.
Orchard Therapeutics (Nasdaq: ORTX) announced its upcoming presentations at two investor conferences. The management team will participate in the Cowen 42nd Annual Healthcare Conference on March 8, 2022, at 2:10 p.m. ET and the Oppenheimer 32nd Annual Healthcare Conference on March 15, 2022, at 11:20 a.m. ET. Both events will be virtual. Live webcasts can be accessed via the company’s website, with replays available afterward. Orchard Therapeutics focuses on innovative gene therapies for serious diseases, leveraging hematopoietic stem cell technology to offer potential cures.
Orchard Therapeutics (Nasdaq: ORTX) announced five presentations at the 18th Annual WORLD Symposium from February 7-11, 2022. The presentations include clinical updates on investigational gene therapies OTL-203 for MPS-I and OTL-201 for MPS-IIIA, alongside data on Libmeldy® (OTL-200) focused on patient identification and market access. Key presentations will feature updates on gene therapy for Hurler syndrome and cost-effectiveness analyses for metachromatic leukodystrophy treatment in Europe. Libmeldy is the first approved therapy for early-onset MLD in the EU.
Orchard Therapeutics (Nasdaq: ORTX) announced an agreement with NHS England for the reimbursement of Libmeldy® (atidarsagene autotemcel) for children with metachromatic leukodystrophy (MLD) in England and Wales. This follows the positive evaluation by NICE, recognizing the significant clinical impact and economic value of Libmeldy. Additionally, two children have been treated with Libmeldy in Germany and France, while a third patient from the Middle East has been referred for treatment in Italy. These developments mark crucial milestones in the European launch of Libmeldy, highlighting its potential to address a severe genetic disorder.
Virica Biotech announced a collaboration with Oxford Biomedica to enhance the yield and production of lentiviral vector gene therapies using Virica's Viral Sensitizers (VSEs™). This partnership aims to meet the increasing demand for effective and commercially viable cell and gene therapies. Ella Korets-Smith, Chief Business Officer, highlighted the significance of their technology in supporting industry advancements. Oxford Biomedica's LentiVector® platform is integral to the production of breakthrough therapies and has established partnerships with major pharmaceutical companies.
Orchard Therapeutics (ORTX) announced a publication in The Lancet, showcasing long-term benefits of Libmeldy® (atidarsagene autotemcel) for treating early-onset metachromatic leukodystrophy (MLD). The treatment led to a 90% overall survival rate in a study of 29 pediatric patients over a median follow-up of 3.2 years. Notable improvements in motor skills and cognitive function were recorded, particularly in pre-symptomatic patients. Libmeldy is the only approved gene therapy for MLD in Europe, with plans for commercialization and future regulatory approvals.
Orchard Therapeutics announced significant progress in 2021 with the launch of Libmeldy in Europe, receiving a major therapeutic benefit rating in Germany. The company is preparing for a U.S. BLA submission for OTL-200 and an EU MAA submission for OTL-103 in 2022. Continued focus on health technology assessments is expected to enhance market access. The company ended 2021 with approximately $220 million in cash, anticipated to support operations through the first half of 2023, excluding potential additional funding from future partnerships.
Orchard Therapeutics (Nasdaq: ORTX) will present at the 40th Annual J.P. Morgan Healthcare Conference on January 13, 2022, at 9:00 a.m. ET. A live webcast will be available on their website under 'News & Events'. Orchard focuses on gene therapy, particularly hematopoietic stem cell gene therapy, aiming to treat severe diseases with limited options. The company acquired a rare disease gene therapy portfolio from GSK in 2018, enhancing its pipeline of innovative treatments. More information can be found at www.orchard-tx.com.
Orchard Therapeutics (Nasdaq: ORTX) announced high survival rates and promising early clinical outcomes for OTL-203, a gene therapy for Hurler syndrome (MPS-IH). With a 100% overall survival rate over a median follow-up of two years, all eight treated patients achieved supraphysiologic blood IDUA activity. The therapy demonstrated stable cognitive and motor function development, significant metabolic correction, and normalization of urinary glycosaminoglycans. A global Phase 3 trial is planned for 2022 to further evaluate the therapy's impact.
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