The interest of public and private investors in Telethon-funded discoveries has led to the creation of several biotech start-ups, each focused on advancing promising therapeutic technologies. Fondazione Telethon promotes and governs this process through its Business Development Office, which identifies opportunities for spin-offs, supports researchers in the transition toward entrepreneurship, and nurtures the start-up in its early years.
Fondazione Telethon is currently a founding partner in five start-ups:
- GeneSpire – Developing liver-directed gene therapies with lentiviral vectors designed for long-term correction after a single treatment. Founded in 2020 at SR-TIGET by Luigi Naldini and Alessio Cantore, together with Fondazione Telethon and Ospedale San Raffaele, with the support of Sofinnova Partners.
- AAVantgarde Bio – Focused on next-generation gene therapies for inherited retinal diseases, with an innovative platform capable of transferring large genes. Co-founded by Alberto Auricchio (TIGEM) and Fondazione Telethon, supported by Sofinnova Partners.
- Epsilen Bio (now part of Chroma Medicine) – Developing epigenetic editing technologies to silence disease-causing genes without permanently altering DNA. Founded in 2019 at SR-TIGET by Angelo Lombardo and Luigi Naldini, together with Fondazione Telethon and Ospedale San Raffaele. Supported by the Sofinnova Telethon Fund.
- Innovavector – A CDMO (Contract Development and Manufacturing Organization) specialized in the design and production of AAV vectors for gene therapy. Founded in 2022 as a spin-off of TIGEM’s Vector Core group, building on over a decade of expertise in viral vector manufacturing.
- Negedia – Developing next-generation molecular diagnostics for rare genetic diseases and cancer through advanced DNA sequencing. Founded in 2022 as a spin-off of TIGEM, leveraging over thirty years of experience in rare genetic disease research.
Other start-ups have also originated from Fondazione Telethon-funded research, where the Foundation is not a shareholder but remains co-owner of the originating patents. These include:
- IAMA Therapeutics - Developing innovative therapies for central nervous system disorders, including epilepsy and autism. Founded in 2021 by Laura Cancedda and Marco De Vivo at the Italian Institute of Technology (IIT) Genoa.
- Sibylla Biotech - Uses artificial intelligence and computational simulations to identify molecules that interfere with protein folding, targeting proteins once considered “undruggable.” Founded in 2017 by researchers from the Italian National Institute for Nuclear Physics (INFN) and the Universities of Trento and Perugia.
- Genenta Science - Specializing in stem cell-based gene therapies for cancer, with a proprietary lentiviral platform (Temferon). Founded in 2014 by Luigi Naldini, Bernardo Tavazzi, and Pierluigi Paracchi, as a spin-off of SR-TIGET.
- TR1X - Developing therapies for autoimmune and inflammatory diseases, based on Tr1 regulatory T cells. Founded in 2021 by Maria Grazia Roncarolo at SR-TIGET (Ospedale San Raffaele, Milan).
By promoting entrepreneurship, Fondazione Telethon ensures that innovative ideas born in its research ecosystem can attract the expertise and investments needed to become therapies, creating tangible opportunities for patients.
Genespire
Aims to develop gene editing technologies and gene therapy for the treatment of inherited diseases, in particular primary immunodeficiencies, haemophilia and metabolic diseases. GeneSpire's technology is based on advanced lentiviral vector platforms, designed to be curative after a single treatment, enabling stable and long-term expression of therapeutic genes. It is a spin-off of Milan-based SR-TIGET and was founded in 2020 by Fondazione Telethon and Ospedale San Raffaele, with the support of Sofinnova Partners. GeneSpire's research and development activities are based at SR-Tiget, and led by gene therapy experts Luigi Naldini and Alessio Cantore.
Aavantgarde Bio
The company was founded with a specific focus on the development of gene therapies for inherited retinal diseases, and aims to make next-generation gene therapies available. AAVantgarde is optimising gene transfer with adeno-associated vectors (AAV) via a technology platform to transfer large genes, overcoming the current limitation of AAV-based gene therapies. AAVantgarde was co-founded with Alberto Auricchio and grew out of research activities carried out at TIGEM, and supported by Sofinnova Partners.
Epsilen Bio
Aims to develop therapies based on the stable and long-lasting switching off of genes involved in pathological processes by means of epigenetic silencing, which does not irreversibly change the DNA. It is a spin-off of SR-TIGET and was founded in 2019 by Fondazione Telethon and Ospedale San Raffaele, and gene therapy experts Angelo Lombardo and Luigi Naldini. Initiated through seed funding from the Sofinnova Telethon Fund, in 2021 it was acquired by the US-based Chroma Medicine, which, through Epsilen Bio, continues to enjoy a strategic partnership with SR-Tiget, carrying out research and development activities at the Institute.
Innovavector
Designs and tests techniques for the production of AAV vectors for gene therapy to safely and efficiently transport genetic material into target cells. As a CDMO (contract development and manufacturing organisation) it absorbs the expertise of the Vector Core group already present in TIGEM, which has consolidated its experience in the production of research-grade AVs and AAVs.
Negedia
Aims to perfect the application of next-generation DNA sequencing systems in the molecular diagnosis of rare genetic diseases and tumors, in order to develop genuine diagnostic services. Negedia (formerly Next Generation Diagnostic Srl) is a laboratory born from over thirty years of experience of TIGEM in the study of rare genetic diseases. Negedia's laboratory was inaugurated in 2022 and was certified in the same year ISO:9001 for research and diagnostics.
The ability to influence and govern these start-ups varies from case to case and evolves as the companies grow and attract new financiers. In addition to participated start-ups, there are also start-ups arising from Telethon-funded research in which the Foundation is not a partner, although in some cases it is co-owner of the patents from which they originated: Sibylla
