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,…..
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…..
Imabiotech, a French CRO based in Lille (France) and in Boston (USA), is expanding its range of services. This company is offering innovative imaging services and software to the pharmaceutical research sector. These services are based on histology combined to label free molecular imaging (Quantitative Mass Spectrometry Imaging, QMSI) . This technology…..
RNA science is now translated into new therapies… Last August, the FDA approved the first RNAi therapy, Onpattro®… RNA drugs allow precised silencing or activation of genes… RNA drugs can address a wide range of common and rare diseases… More than 60 rare or common diseases are currently targeted…. BioPharmAnalyses…..
The European Medicine Agency (EMA) is launching a six-month public consultation on its draft ‘ Regulatory Science to 2025’ strategy for a six-month public consultation. This sixty-page document proposes the plan for advancing the Agency’s engagement with regulatory science over the next five to ten years, covering both human and veterinary medicines. The…..
A poorly designed and improperly executed trial can « kill » a product An interview with Régine Rouzier, president of the CRO Cap Research What is the central issue to successfully perform a phase I trial? The success of a phase I study depends on the speed in execution and…..