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The first patient has been dosed in an open-label, six-subject Phase 1/2 clinical trial evaluating a cryopreserved formulation of Orchard Therapeutics' (NASDAQ: ORTX ) lentiviral gene therapy OTL-103 in patients with Wiskott-Aldrich syndrome (WAS), a disorder characterized by abnorma...
Program on Track for MAA and BLA Regulatory Submissions in 2021 Recent Lancet Hematology Publication Describes Encouraging Safety and Efficacy Data from Interim Analysis of OTL-103 Fresh Formulation Registrational Trial BOSTON and LONDON, April 25, 2019 (GLOBE NEWSWIRE) --...
Mustang Bio ( MBIO ) surged higher the prior week, after it highlighted substantially positive data in a phase 1/2 study using its gene therapy to treat patients with X-linked severe combined immunodeficiency ((XSCID)). It was shown that treatment with MB-107 was able to restore the immu...
BOSTON and LONDON, April 15, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced ...
Complete results from a registrational Phase 2 clinical trial evaluating Orchard Therapeutics' ( ORTX -4.8% ) OTL-200 in patients with a rare inherited disorder called metachromatic leukodystrophy (MLD) showed a treatment benefit. The data were presented at the Annual Meeting of the E...
Compared to Natural History, Late Infantile Patients Demonstrated Gross Motor Function Scores Higher by 65 and 72 Percentage Points at 2- and 3-Years Post Gene Therapy; Early Juvenile Patients Demonstrated Scores Higher by 40 Percentage Points at Both Time Points No Effective Treatmen...
Orchard Therapeutics PLC (ORTX) Q4 2018 Results Conference Call March 21, 2019 9:00 AM ET Company Participants Renee Leck - Director, Investor Relations Mark Rothera - President, Chief Executive Officer Bobby Gaspar - Co-Founder and Chief Scientific Officer Andrea Spezzi - Co-F...
Orchard Therapeutics (NASDAQ: ORTX ): FY GAAP EPS of -$10.22. More news on: Orchard Therapeutics plc, Earnings news and commentary, Healthcare stocks news, Read more ...
Three Lead Programs for MLD, ADA-SCID and WAS on Track for Regulatory Filings Over the Next Three Years, with MLD and ADA-SCID Expected in 2020 Registrational Data Showing 100% Overall Survival and 100% Event-Free Survival at Two Years in ADA-SCID Patients Recently Presented at ASBMT;...
BOSTON AND LONDON, March 18, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced ...
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TOKYO, LONDON and BOSTON, May 03, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced that Bobby Gaspar, M.D., Ph.D., co-founder and chief executive officer, ...
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Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient’s own hematopoietic stem cells HURCULES study to enroll 40 patients at clinical sites across the U.S....