The US Food and Drug Administration (FDA) is proposing the creation of two lists of drugs or drug categories that are difficult to compound under sections 503A and 503B of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as well as criteria for inclusion on the lists.
US approves first gene therapy for children with rare genetic disease
TOKYO and LONDON and BOSTON, March 18, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients...
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 ...
BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on...
Kyowa Kirin successfully completes acquisition of Orchard Therapeutics, a global gene therapy leader for rare diseases
BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has...
Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH
BOSTON and LONDON, Oct. 26, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced a range of interim clinical outcomes, in addition to the...
Eight presentations highlight the differentiated profile and broad applicability of the company™s HSC gene therapy platform Data from proof-of-concept study of OTL-203 in MPS-IH demonstrate...