News
21.12.20
Orchard Therapeutics Receives EC Approval for the Treatment of Early-Onset MLD
First gene therapy to receive full EU marketing authorization for eligible MLD patients.
03.11.20
Potential adverse reaction to gene therapy in a patient treated with Strimvelis for the treatment of ADA-SCID
Fondazione Telethon has recently learned that a patient suffering from a rare immunodeficiency of genetic origin, ADA-SCID, and treated in 2016 with gene therapy has developed leukemia.
16.10.20
Orchard Therapeutics Receives Positive CHMP Opinion for the Treatment of Early-Onset MLD
First therapy recommended for EU approval for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants.
07.09.20
Osteopetrosis: new opportunity of treatment thanks to a licensing agreement
SiSaf announces a research collaboration and licensing agreement with the University of L’Aquila for the treatment of the debilitating bone disease, Autosomal Dominant Osteopetrosis Type 2.
10.07.20
Discovery of a novel drug candidate to develop effective treatments for brain disorders
Researchers at IIT-Istituto Italiano di Tecnologia discovered a novel chemical compound, which has the potential to became a new drug for the treatment of core symptoms of brain disorders like Down syndrome and autism.
01.07.20
Study of genetic condition leads to discovery of mechanism that promotes cance development
At TIGEM scientist have described how the study of a rare genetic condition has provided the key to understanding a mechanism that leads to the formation of cysts and tumours in certain organs, in particular the kidneys.
04.06.20
Groundbreaking Gene Therapies for Hereditary Diseases
Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Kröner Fresenius Prize for Medical Research 2020 endowed with 2.5 million euros.
20.04.20
Sofinnova Partners Announces Third Investment from its Italian Fund
This investment brings the total financing by Sofinnova Partners to €25 million over the past six months.
11.04.19
Gene Therapy Demonstrates Efficacy in Treating Wiskott-Aldrich Syndrome
A clinical study confirms the efficacy of gene therapy as a treatment for Wiskott–Aldrich Syndrome (WAS), a serious rare genetic disease affecting blood cells.
