2023年9月18日 · Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of metachromatic leukodystrophy (MLD) in patients characterized by...

2023年8月3日 · BLA Submission Completed for OTL-200 in the U.S. The company has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for OTL-200, in children with early …

BOSTON and LONDON, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast...

BOSTON and LONDON, July 15, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company has entered into two worldwide royalty-bearing license agreements with GlaxoSmithKline plc (GSK) (LSE/NYSE: GSK) for use of their proprietary lentiviral stable cell line technology (LV-SCLT ...

2021年6月29日 · Based on the feedback from this meeting and previous interactions, the company is now preparing for a BLA filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using...

BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted...

2021年1月14日 · --Orchard Therapeutics, a global gene therapy leader, today announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation to OTL-200, an...

OTL-105 is an investigational HSC gene therapy designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks. OTL-105 inserts one or more functional copies of the SERPING1 gene into patients own HSCs ex vivo which are then transplanted back into the patient for potential durable C1-INH production.

2019年4月25日 · OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

BOSTON and LONDON, Jan. 05, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has cleared its...