WebJun 4, 2024 · The US biotech company bluebird bio has obtained EU conditional market approval for a gene therapy that could remove the need for blood transfusions for people with the blood disorder beta-thalassemia. Called Zynteglo, the gene therapy is aimed at treating people with beta-thalassemia who are over 12 years of age and have any but … WebNov 5, 2024 · Response of patients with transfusion-dependent β-thalassemia (TDT) to betibeglogene autotemcel (beti-cel; LentiGlobin for β-thalassemia) gene therapy based on HBB genotype and disease genetic modifiers ... All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information ...
bluebird bio Announces FDA Approval of ZYNTEGLO
WebAfter a rough takeoff into 2024, beleaguered gene therapy specialist bluebird bio has alighted on a spot of good news, courtesy of prominent U.S. drug cost watchdog ICER. WebWith a dedicated focus on severe genetic diseases, bluebird has industry-leading programs for sickle cell disease, beta-thalassemia and cerebral … electric scooters valencia
New CRISPR, gene therapy results strengthen potential for treatment …
WebSep 8, 2024 · First gene therapy for β-thalassemia approved Bluebird Bio’s gene therapy eliminates the need for blood transfusions in patients with β-thalassemia. Charlotte … WebAugust 17, 2024 – bluebird bio, Inc. announced the FDA has approved ZYNTEGLO ® (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and … WebJun 12, 2024 · Bluebird is much further along, having treated 60 patients with beta thalassemia and 37 with sickle cell disease across six different studies. Updated results from three of those studies showed 23 of 27 evaluable patients with beta thalassemia were transfusion independent for at least a year following treatment. electric scooters with gps