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Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [1] [5] [2] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.
EMA/129698/2012: Concept paper on extrapolation of efficacy and safety in medicine development. [10] FDA-2015-D-1376: Leveraging existing clinical data for extrapolation to pediatric uses of medical devices. Guidance for Industry and Food and Drug Administration Staff. [11] ICH E5 (R1): Ethnic factors in the acceptability of foreign clinical ...
The therapy known as Casgevy [11] works through editing a dysfunctional protein that interferes with creation of adult hemoglobin. This gene is known as the BCL11A, and when people have Beta thalassemia, their bodies do not make enough adult hemoglobin. Casgevy uses precise gene editing of stem cells, and reduces the activity of BCL11A.
Elivaldogene autotemcel was designated an orphan drug by the European Medicines Agency (EMA) in 2012. [23] Elivaldogene autotemcel was granted orphan drug, rare pediatric disease, and breakthrough therapy designations by the US Food and Drug Administration (FDA). [24] In September 2022, elivaldogene autotemcel was granted accelerated approval. [25]
The Committee for Advanced Therapies (CAT) was established in accordance with Regulation (EC) No 1394/2007 on advanced-therapy medicinal products (ATMPs) such as gene therapy, somatic cell therapy and tissue engineered products. It assesses the quality, safety and efficacy of ATMPs, and follows scientific developments in the field. [24]
In April the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis [238] [239] and the European Commission approved it in June. [240] This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene ...
"Gene Therapy Arrives". Scientific American This page was last edited on 10 November 2024, at 06:28 (UTC). Text is available under the Creative Commons Attribution ...
Idecabtagene vicleucel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. [ 4 ] [ 7 ] Each dose is customized using a patient's own T-cells, which are a type of white blood cell, that are collected and genetically modified to include a new gene that facilitates ...