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.
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.
The Japan Society of Gene and Cell Therapy (JSGCT) has announced that Professor Shigetaka Asano, the first president of JSGCT, passed away on 12th August 2020. We extend our thoughts and best wishes to all at JSGCT.
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.
Non-viral genetic therapy strategies for the treatment of ataxia telangiectasia
James Grey received the ESGCT-sponsored prize for best poster presentation at the British Society for Gene and Cell Therapy (BSGCT) Annual Conference, which took place from 19-21 June 2019. Read about his winning presentation here!
Gene therapy for thrombotic thrombocytopaenic purpura
Robyn Bell received the ESGCT-sponsored prize for best oral presentation at the British Society for Gene and Cell Therapy (BSGCT) Annual Conference, which took place from 19-21 June 2019. Read about her winning presentation here!
CRISPR-Cas9 genome editing - molecular scissors a bit too sharp?
Grégoire Cullot received the ESGCT-sponsored prize for best oral presentation at the Société Française de Thérapie Cellulaire et Génique (SFTCG) Thematic Day on Viral Vectors, which took place on 17 May 2019 in Paris. Read about his winning presentation here!
Dgkk as a new therapeutic target in the Fmr1-KO murine model of Fragile X
Karima Habbas received the ESGCT-sponsored prize for best poster at the Société Française de Thérapie Cellulaire et Génique (SFTCG) Thematic Day on Viral Vectors, which took place on 17 May 2019 in Paris. Read about her winning presentation here!