TOKYO, LONDON and BOSTON — Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, for the treatment of children...
treatment News
BOSTON and LONDON — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I...
BOSTON and LONDON — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, announced various business accomplishments along with its financial results for the quarter ended March 31, 2023. “Following multiple productive interactions this year with the FDA on our clinical and CMC packages, I am delighted to announce the...
Boston and London — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, announced that the company will make a virtual presentation at the Guggenheim Genomic Medicines and Rare Disease Day on Tuesday, April 4, 2023 at 1:35pm ET. A live webcast of the presentation will be available under “News...
TARRYTOWN, N.Y. — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Commission (EC) has approved Ordspono™ (odronextamab) to treat adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), after two or more lines of systemic therapy. This marks the first...
SAN DIEGO, Calif. — Organovo Holdings, Inc. (Nasdaq: ONVO), a clinical stage biotechnology company focused on developing FXR314 in inflammatory bowel disease (IBD), including ulcerative colitis, based on demonstration of clinical promise in three-dimensional (3D) human tissues, today announced the presentation of preclinical data related to the company’s FXR314 development...
SHANGHAI, China and ROSELAND, New Jersey – Oricell Therapeutics (Oricell), a clinical-stage biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for OriCAR-017 for patients with relapsed/refractory multiple myeloma (R/R MM). OriCAR-017 is a chimeric antigen receptor (CAR) T...
Espoo, Finland – Orion presented last week at the 2024 ASCO GU Cancers Symposium a poster with additional data from the ongoing Phase II CYPIDES trial evaluating the safety and efficacy of ODM-208 (or MK-5684), an investigational, oral CYP11A1 inhibitor, in heavily pretreated patients with metastatic castration-resistant prostate cancer (mCRPC)...
ELK GROVE VILLAGE, Ill. — Orsini Specialty Pharmacy has been selected by AstraZeneca and Ionis as the exclusive specialty pharmacy partner for WAINUA™ (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN). WAINUA is a subcutaneous injection that can be self-administered,...
ELK GROVE VILLAGE, Ill. – Orsini Specialty Pharmacy announced today that it is now dispensing tiopronin, the first available generic version, in the US, of THIOLA® (tiopronin). Tiopronin is a reducing and complexing thiol indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation...