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Based on preliminary trial data, the FDA had designated Zolgensma a breakthrough therapy, one of three special designations that helped it race from human trials to regulatory approval in five ...
Jesse Gelsinger (June 18, 1981 – September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency , an X-linked genetic disease of the liver , the symptoms of which include an inability to metabolize ammonia – a byproduct of ...
Clinical trials of gene therapy for sickle cell disease were started in 2014. [227] [228] In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the ...
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
Between 1989 and December 2018, more than 2,900 clinical trials of gene therapies were conducted, with over half of them reaching the phase I stage. Over the years, several gene therapy-based drugs have been developed and made available to the public, marking important milestones in the treatment of genetic disorders. Examples include Zolgensma ...
The journal content is aimed at academic and industrial scientists with technical focus on gene and cell therapy, preclinical and clinical research. The journal publishes articles concerning basic research and clinical applications of gene and cell therapy of diseases including new scientific methods, gene delivery systems, gene therapy ...
The treatment was developed at San Raffaele Telethon Institute for Gene Therapy and developed by GlaxoSmithKline (GSK) through a 2010 collaboration with Fondazione Telethon and Ospedale San Raffaele. GSK, working with the biotechnology company MolMed S.p.A., developed a manufacturing process that was previously only suitable for clinical trials ...
The use of gene therapy in treating neurological disorders such as epilepsy has presented itself as an increasingly viable area of ongoing research with the primary targets being somatostatin, galanin, neuropeptide y, potassium channels, optogenetics and chemogenetics for epilepsy. As the field of gene therapy continues to grow and show ...