Myotubular Trust is holding a 2021 call for research grants. We will require completed applications by 1700 hours GMT Friday 29th October 2021. We anticipate making awards in April 2022.
Un message du président de la SFTCG
Review written by Alessandro Aiuti, Giuliana Ferrari & Adrien Thrasher
New Horizons in the Management of ADA-SCID is a free educational resource for healthcare professionals managing patients with this rare disease.
Transparence : et la science ? Nos concitoyens et concitoyennes ont droit à l'intégrité scientifique Chapeau : La confiance du public en la science a pu être ébranlée lors de la pandémie actuelle, mais le mal est ancien et profond : les grandes déclarations ne suffisent plus. Seule la loi peut désormais garantir à nos compatriotes le respect de règles d'intégrité scientifique indiscutables.
Le prix Nobel de Chimie 2020 a été attribué à Emmanuelle Charpentier et Jennifer Doudna pour leur travaux sur CRIPSR/CAS9
CDTI invests together with Inveready Biotech III and EASO Ventures in VIVEbiotech, a biotechnology startup that develops and produces viral vectors to be applied in gene therapy
MeiraGTx is very excited to let you know they released positive data for their AAV-RPGR program to treat X-Linked retinitis pigmentosa!
To further encourage the development of treatments for rare diseases, EMA will waive all fees for scientific advice for academia developing orphan medicines. The academic sector plays an important role in the development of innovative medicines. Their scientific research is often at the source of novel methodologies and innovative medicines with a potential to benefit patients with rare diseases.
San Sebastián, Spain, June 4, 2020 - VIVEbiotech, S.L., a gene transfer technologies Contract Development and Manufacturing Organization (CDMO) developing and GMP manufacturing lentiviral vectors, today announces a collaboration with Xyphos Biosciences for the development its CAR-T™ cell therapy for people with hematological cancer.
the gene therapy for the treatment of ADA-SCID was originally developed by Ospedale San Raffaele (OSR) and Fondazione Telethon (Telethon), through their joint San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and was taken forward by GSK through a strategic collaboration formed in 2010
New gene therapy success in a rare disease of the immune system: Wiskott-Aldrich syndrome