FEATURE STORY ● A safe and potent anti-CD19 CAR T cell therapy GENE THERAPY ●Chimeric antigen receptor T cell persistence and memory cell formation DISRUPTIVE TECHNOLOGIES ●Hemophilia A : Restoration of FVIII expression by targeted gene insertion in the FVIII CLINICAL TRIALS-DATA ● Avexis data reinforce effectiveness of Zolgnesma® in treating…..
FEATURE STORY ● Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy GENE THERAPY ● Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model ● Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 RARE DISEASES ● Characterization and treatment of congenital thrombotic thrombocytopenic purpura ● Targeting angiogenesis in…..
FEATURE STORY ● Luxturna®: FDA documents reveal the value of a costly gene therapy GENE THERAPY ● The application of Adeno-Associated Viral vector gene therapy to the treatment of Fragile X Syndrome ● Adeno-associated virus vector as a platform for gene therapy delivery ● Integrative analysis of pooled CRISPR genetic screens…..
The International Society for Cell and Gene Therapy (ISCT), the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, has published its first annual report detailing global cell and gene therapies (CGT) with market authorization. This report is intended to provide…..
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…..