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Today, we take a look at Orchard Therapeutics, a developmental concern from across the pond that has discernible potential catalysts on the horizon. With a couple of drug candidates in late-stage development, this is an intriguing name in the gene therapy space. Orchard will be ad...
Orchard Therapeutics (ORTX) has reported initial data from an ongoing proof-of-concept trial evaluating OTL-201, an autologous hematopoietic stem cell gene therapy candidate, for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA, or Sanfilippo syndrome type A), an inhe...
BOSTON and LONDON, Dec. 08, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, yesterday reported initial data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-201, an investigational ex vivo ...
The largest pharmaceutical companies in the world are restructuring. Biopharma represents a far more valuable future than growth-dilutive consumer health revenues. GSK cleared room for next generation oncology cellular medicines by selling their prior portfolio to Novartis. ...
BOSTON and LONDON, Nov. 24, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that its chief executive officer, Bobby Gaspar, M.D., Ph.D. will participate in a pre-recorded fireside chat as part of the Piper Sandler 32nd Annual Virtu...
The FDA has signed-off Orchard Therapeutics' ([[ORTX]] +3.2%) Investigational New Drug application for OTL-200, an autologous, hematopoietic stem cell, lentiviral vector-based gene therapy in development for the treatment of metachromatic leukodystrophy ((MLD)). The company also has applied f...
BOSTON and LONDON, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for OTL-200, an autologous, he...
Developing drugs to treat or cure rare diseases can be a lucrative line of business, as Orchard Therapeutics (NASDAQ: ORTX) and Alexion Pharmaceuticals (NASDAQ: ALXN) know. While it might seem highly limiting to target conditions that only affect a handful of patients worldw...
First look at preclinical data in f rontotemporal dementia with progranulin mutations ( GRN- FTD) and new amyotrophic lateral sclerosis (ALS) program NOD2 mutation revealed as Crohn’s disease (CD) genetic target,...
BOSTON and LONDON, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company is scheduled to present at the Barclays Gene Editing & Gene Therapy Summit on November 16, 2020 at 11:15 AM ET. A live webcast o...
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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....