Collaboration with the pharmaceutical and biotech industry is a key accelerator in the development of therapies for rare genetic diseases. By partnering with companies, Fondazione Telethon gains access to additional financial resources, technologies, and expertise that complement the work carried out within our research institutes.
Industrial agreements are guided by a set of principles that safeguard our mission:
- maintaining ownership of intellectual property and know-how (licensed to partners as appropriate)
- ensuring scientific independence for our researchers
- and securing royalties and returns that are reinvested into research.
Partners are also required to commit to developing the therapies covered by the agreement.
In 2024, Telethon managed 32 active industrial agreements (19 at SR-TIGET and 10 at TIGEM), spanning licensing, research collaborations, material transfer, and confidentiality agreements. Current collaborations include:
- Orchard Therapeutics - Licensing of the gene therapy for Metachromatic Leukodystrophy (approved both in EU and US) and development of the program for Mucopolysaccharidosis type I-H.
- Akouos - Licensing of the technology developed at TIGEM for large-gene transfer, applied to innovative gene therapy programs for hearing loss caused by mutations in the OTOF gene.
- CanFite - Collaboration for the clinical development of a potential treatment for Lowe syndrome, based on preclinical data generated at TIGEM.
Through these and other partnerships, Fondazione Telethon ensures that discoveries made in the laboratory can be translated into accessible therapies for patients, while maintaining the integrity of its scientific mission.
