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…..

Imabiotech boosts its strategic partnerships

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…..

EMA’s Regulatory Science to 2025 Strategy

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…..


  BioPharmAnalyses is happy to announce the release of the first issue of its « BRIEFING ON CART-T CELLS TRIALS » CAR-T cells therapies are at the forefront of innovation in oncology “BRIEFING ON CART-T CELLS TRIALS” provides you with an accurate overview to explore the latest trials in this hot and rapidly evolging…..

Chiffres clés de la recherche clinique en France

A l’occasion de la 7ème journée de la recherche clinique, l’AFCROs, qui regroupe 75 sociétés-membres représentant environ 70% de l’activité dans le domaine de la recherche clinique et épidémiologique, a présenté les chiffres clés de la recherche clinique en France. Cet outil réalisé par les groupes de travail de l’association vise…..