Welcome to our dedicated page for Orchard Therapeutics Plc news (Ticker: ORTX), a resource for investors and traders seeking the latest updates and insights on Orchard Therapeutics Plc stock.
Orchard Therapeutics Plc (ORTX) delivers groundbreaking gene therapies for rare inherited disorders, focusing on transformative treatments for pediatric patients. This page provides centralized access to all official company announcements, regulatory filings, and clinical development updates.
Investors and researchers will find timely information on clinical trial progress, regulatory milestones, and strategic partnerships shaping the future of gene-based medicine. The curated news collection spans therapy approvals, research collaborations with leading institutions, and manufacturing advancements critical to orphan disease treatment accessibility.
Regular updates ensure stakeholders maintain current awareness of ORTX's innovations in hematopoietic stem cell therapies and neurometabolic disorder treatments. Bookmark this page for efficient tracking of material developments affecting the company's position in the advanced therapies sector.
Orchard Therapeutics (Nasdaq: ORTX) announced four oral presentations and 11 posters focused on gene therapy and newborn screening for severe neurometabolic diseases at the upcoming 19th Annual WORLD Symposium from February 22-26, 2023, in Orlando, Florida. Highlights include clinical results from an extensive analysis of patients treated with OTL-200 (Libmeldy) for metachromatic leukodystrophy (MLD) and efforts to enhance newborn screening programs. With over 10 years of follow-up data, the updated analysis of OTL-200 aims to support future regulatory submissions in the U.S. The event will also feature expert discussions on newborn screening's critical role in timely diagnosis.
Indapta Therapeutics has announced the appointment of Moya Daniels as Senior Vice President of Regulatory, Quality and Clinical Operations. With 25 years of experience in cell and gene therapies, Daniels previously worked at Aruvant Therapeutics and Orchard Therapeutics. Indapta is advancing its G-NK cell therapy for treating multiple myeloma and lymphoma, aiming to enter clinical trials this year. The G-NK therapy is designed to enhance the cytotoxicity of monoclonal antibody treatments, utilizing a proprietary manufacturing process that ensures higher efficacy and persistence compared to conventional NK cells.
Orchard Therapeutics reported Libmeldy revenue of $5.8M in Q4 2022 and $18.8M for the year. The company ended 2022 with approximately $144M in cash and investments, significantly reducing its burn rate to support operations until Q2 2024.
Key achievements include FDA clearance for OTL-203 IND application and scheduled Type B meeting for OTL-200 prior to BLA submission. Upcoming milestones target expansion of commercial operations and further clinical advancements in 2023.
Orchard Therapeutics (Nasdaq: ORTX) has received FDA approval for its Investigational New Drug (IND) application for OTL-203, a gene therapy aimed at treating the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). A global registrational trial is expected to begin in the second half of 2023 to evaluate the safety and efficacy of OTL-203 against standard care treatments. The trial will involve 40 patients and aims to demonstrate the superiority of OTL-203 in addressing severe symptoms of MPS-IH after two years. Preliminary data showed promise in cognitive development and growth outcomes for previous patients treated.
Orchard Therapeutics (Nasdaq: ORTX) announced early clinical findings from its proof-of-concept study of OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA). Following a median follow-up of 1.5 years, all patients showed sustained engraftment and supraphysiological levels of SGSH enzyme. Four out of five patients demonstrated improvement in cognitive skills, aligning with healthy developmental benchmarks. Treatment was generally well-tolerated, with manageable side effects noted. The ongoing trial aims to further assess safety and neurocognitive outcomes over three years.
Orchard Therapeutics (Nasdaq: ORTX) announced the acceptance of its marketing authorization application (MAA) for Libmeldy® by Swissmedic, aimed at treating early-onset metachromatic leukodystrophy (MLD). This follows prior EU approval in December 2020. The firm anticipates receiving an assessment report by mid-2023 as part of its efforts to expand commercial reach in Europe. MLD is a rare, life-threatening condition affecting about 1 in 100,000 live births, leading to severe neurological issues. Libmeldy, the first approved therapy for early-onset MLD, has known adverse reactions such as anti-ARSA antibodies.
Orchard Therapeutics reported Q3 2022 financial results, achieving $4.8M in Libmeldy sales and $13.0M year-to-date. The company confirmed the first case of metachromatic leukodystrophy from a newborn screening study, aiding in universal screening adoption. A Type B meeting with the FDA is planned before the BLA submission for OTL-200. The firm ended Q3 with $146.6M in cash, extending its runway into Q2 2024. R&D expenses decreased by 13% year-over-year, while the net loss was $47.6M, up from $36.4M in Q3 2021.
Orchard Therapeutics (Nasdaq: ORTX) announced participation in two investor conferences. Management will present at the Barclays Gene Editing & Gene Therapy Summit on November 14, 2022, at 1:40 p.m. EST, discussing reimbursement and patient access for genomic disorders. Additionally, they'll present at the Stifel 2022 Healthcare Conference on November 15, 2022, at 2:25 p.m. EST in New York.
A live webcast of the Stifel presentation will be accessible on the company's website.
Orchard Therapeutics (Nasdaq: ORTX) will host a conference call on November 14, 2022, at 8:00 a.m. EST to discuss business updates and Q3 2022 financial results. Interested parties can access the live webcast via the company's website. Orchard is focused on gene therapy for severe diseases, utilizing hematopoietic stem cell (HSC) therapies. The company aims to address serious medical conditions where treatment options are limited. Its pipeline includes various clinical and commercial gene therapies.
Orchard Therapeutics (Nasdaq: ORTX) announced early clinical findings from its proof-of-concept study of OTL-201 for treating MPS-IIIA, to be presented at the 64th ASH Annual Meeting on December 12, 2022. The oral presentation will include neurocognitive data and follow-up results after 1.5 years. The study involves five patients aged 6 to 24 months, focusing on safety and neurocognition. Orchard will host a virtual KOL investor webcast at 5:00 p.m. EST on the same day to discuss the findings further.
