GlaxoSmithKline, Fondazione Telethon and Fondazione San Raffaele have entered into a strategic alliance to research and develop novel treatments for rare genetic disorders.

The collaboration will build upon research carried out at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), a partnership between Fondazione Telethon and Fondazione San Raffaele, using gene therapy carried out on stem cells derived from a patients’ bone marrow.

Under the agreement, GlaxoSmithKline will have an exclusive licence to develop and commercialise an investigational gene therapy for ADA Severe Combined Immune Deficiency.

GlaxoSmithKline will also co-develop six further applications of ex vivo stem cell therapy for the treatment of a range of rare disorders with its new partners, with the help of a new gene transfer technology developed by HSR-TIGET scientists,.

The companies will develop treatments for disorders including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, beta-thalassemia, mucopolysaccharoidosis type I, globoid leukodystrophy and chronic granulomatous disorder.

Clinical trials for metachromatic leukodystrophy and Wiskott-Aldrich syndrome treatments have been initiated at HSR-TIGET, and are currently enrolling patients.

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Fondazione Telethon will receive an upfront payment of €10m ($13m) from GlaxoSmithKline and is eligible to receive further payments depending on successful completion of a number of predetermined development milestones.