close

LATEST POSTS from Anne-Lise Berthier

Trend Chart on Innovative Biotherapies 26th April

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

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

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

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…..

Trend Chart on Orphans – February 23rd, 2019

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

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…..