Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)

First gene therapy to receive full EU marketing authorization for eligible MLD patients. One-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele.

From Telethon Foundation


Awarded Telethon Farmindustria Prize to three young researchers

Farmindustria and Fondazione Telethon have awarded three prizes of 10,000 euros to young researchers who work in the neonatology field.

From Telethon Foundation


Fondazione Roche rewards a young researcher of Tigem in Naples

Funded the research project by Paolo Grumati dedicated to the study of a neurodegenerative disease that causes generalized sensory loss.

From Telethon Foundation


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.

From Telethon Foundation


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.

From Telethon Foundation


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.

From Telethon Foundation


Tigem and SR-Tiget involved in UPGRADE project, funded by the European Union

Fondazione Telethon announces the launch of the UPGRADE project, funded by the European Union with 15 million euros, aiming at developing more precise and effective gene therapies approaches.

From Telethon Foundation


Restore Health – Therapies that cure

The European Commission (EC) announced that the FET Flagship RESTORE, which was initiated by the Berlin-Brandenburg Center for Regenerative Therapies (BCRT), has been selected to enter the next phase of the FET Flagship competition.

From Telethon Foundation


Editas Medicine Announces Scientific Multi-Year Collaboration with Fondazione Telethon and Ospedale San Raffaele

Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, and Fondazione Telethon and Ospedale San Raffaele, have entered into a scientific collaboration to research and develop genome edited hematopoietic stem cell (HSC) and T cell therapies.

From Telethon Foundation

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