New prospects for liquid biopsies ? Dr Muneesh Tewari, professor of internal medicine at the U-M Medical School. © Joseph XU. U-M College of Engineering Advancing technology is allowing scientists increasingly to search for tiny signs of cancer and other health issues in samples of patients' blood and urine. These "liquid biopsies"…..
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…..
A new gene and cell therapy production facility in Maryland Precigen's new manufacturing facility has officially opened its doors in Germantown, Maryland. The wholly-owned subsidiary of Intrexon commenced the build-out of the nearly 5,000 square foot manufacturing facility in 2018 to support gene therapy manufacturing. The good manufacturing practices (GMP) facility…..
Waylivra™ (volanesorsen), first therapy for familial chylomicronemia syndrome The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending conditional marketing authorization for Waylivra™ (volanesorsen) for the treatment of familial chylomicronemia syndrome (FCS). This antisense oligonucleotide developped by Ionis Pharmaceuticals and its affiliate Akcea…..
First European Market Approval for Nanobiotix The French nanomedicine company Nanobiotix has received its first European market approval (CE mark) enabling commercialisation of its lead product Hensify® (NBTXR3) in the 27 EU countries for the treatment of locally advanced soft tissue sarcoma. “We could not be more proud to receive market approval…..
Merck chooses France for its first M Lab™ collaboration center in Europe Molsheim is now home to Merck Life Science's first M Lab™ collaboration center in Europe. This new center supports pharmaceutical and biotechnology customers from pre-clinical through full-scale production in non-GMP environment. Molsheim site provides biopharmaceutical manufacturers with a shared,…..
Illustration of lymphocytes attacking cancer cells.©La Jolla Institute How to counteract T cell exhaustion in CAR T cancer therapies? In CAR T cell therapies, a patients' own T cells are removed and genetically engineered to better recognize cancer cells. The cells are then returned to the patient's body, where…..
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…..
1eres Journées Polepharma de microbiomique Le cluster de production pharmaceutique Polepharma organise ses premières journées de microbiomique les 9 et 10 avril prochains à l'UFR Santé de l'université de Rouen. Le programme a été co-construit entre académiques, start-up, PME et grands industriels de la pharma présents au sein du premier…..
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…..