Bypassing broken genes Researchers at Penn State have developed a new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other rare diseases. The…..
Trend Chart On Innovative Bioindustries - June 2020, 2nd FEATURE STORY ● Novartis snags EU nod for SMA gene therapy After a manufacturing-related delay, Novartis has won conditional approval in the EU for Zolgensma®, the one-time gene therapy to treat patients with a clinical diagnosis of SMA type 1 and…..
Trend Chart on Innovative Biotherapies - January 15th, 2020 FEATURE STORY ● The National Gene Vector Biorepository. Eleven years of providing resources to the gene therapy community GENE THERAPY ● Recommendations for the development of cell-based anti-viral vector neutralizing antibody assays DISRUPTIVE TECHNOLOGIES ● A small molecule-controlled Cas9 repressible system…..
BioPharmAnalyses and OctopusyX BioConsulting are proud to announce the launch of their up-dated and enriched 2019 edition of their report entitled “Landscape… in Gene Therapy Companies”. This report provides you with an in-depth analysis of more than 230 companies involved in this expanding field. (UPDATED SEP 2019) In…..
FEATURE STORY ● Bluebird bio and Novo Nordisk enter into research agreement to develop in vivo genome editing candidates CLINICAL TRIALS - DATAS ● Risdiplam Spinal Muscular Atrophy data demonstrating continued benefit ● ProQR announces positive top-line results from the phase 1/2 study of Sepofarsen in LCA10 patients ● Ionis'…..
Gene Therapy is a rapidly and obviously evolving field that is now definitively recognized as a well-acknowledged major medical and scientific area. BioPharmAnalyses is proud to announce the launch of its new global commercial report "Landscape in Gene Therapy Companies". This is the first such report that provides you with an actually updated information and an accurate international mapping…..
« La thérapie génique va trouver sa place dans l’arsenal thérapeutique » – Entretien avec Serge Braun, directeur scientifique de l’AFM-Telethon
Vingt-cinq ans après les premiers essais de thérapie génique chez l’homme, Serge Braun, directeur scientifique de l’AFM-Telethon (photo), fait le point sur la situation actuelle de ces nouveaux traitements qui intéressent maintenant un nombre croissant de big pharma et de biotech. BioPharmAnalyses : Quelles sont les technologies les plus utilisées…..