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Zynteglo
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. It was developed by bluebird bio, Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015. The most common adverse reactions include reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nosebleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus (itch). It was approved for medical use in the European Union in May 2019, Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. and in the United States in August 2022. Medical uses Betibeglogene autotemcel is indicated for the treatment of people twelve years and older with T ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Bluebird Bio
bluebird bio, Inc., based in Somerville, Massachusetts, is a biotechnology company that develops gene therapies for severe genetic disorders. The company is owned by investment funds managed by The Carlyle Group and SK Capital Partners. One of the company's approved drugs is betibeglogene autotemcel (Zynteglo), which treats transfusion-dependent beta thalassemia (TDT), a rare genetic blood disorder, and has been approved for use by the European Medicines Agency. The company has been criticized for the $1.8 million cost of the drug, which is the second most expensive drug in the world. The company is developing LentiGlobin gene therapy for the treatment of sickle cell disease and cerebral adrenoleukodystrophy. It is also developing T cell product candidates to treat acute myeloid leukemia, Merkel-cell carcinoma, diffuse large B-cell lymphoma, and MAGEA4 solid tumors. History The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Lebou ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |