Orchard Therapeutics Plc - ORTX STOCK NEWS

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.