Trend Chart on Orphans - February 15th, 2020 FEATURE STORY ● The potential of gene therapy for mucopolysaccharidosis type I GENE THERAPY ● Repair of retinal degeneration following ex vivo Minicircle DNA gene therapy and transplantation of corrected photoreceptor progenitors RARE DISEASES ● Genetics and other omics in pediatric pulmonary…..
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…..
FEATURE STORY ● The Alliance for Regenerative Medicine outlines recommendations to increase the number of European-based ATMP clinical trials GENE THERAPY ● Long-term correction of copper metabolism in WD mice with AAV8 vector delivering truncated ATP7b ● Enhancing the therapeutic potentiel of sulfamidase for the treatment of MPSIIIA DISRUPTIVE TECHNOLOGIES…..
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'…..
FEATURE STORY ● The landscape of early clinical gene therapies outside oncology BASIC SCIENCE ● Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL CLINICAL TRIALS/DATA ● ATL1102 for DMD commencement of European regulatory interactions ● UniQure stays a step ahead in hemophilia B FINANCIAL DATA ● Passage Bio Closes $110…..