Amongst our strategies to develop therapies and accelerate the path to access to cures is collaboration with public and private investment funds. Investors' interest in our scientific discoveries has given rise to several start-ups, which attract and focus funds and expertise on a promising technology or therapeutic approach.
Our Business Development Office identifies the most suitable research projects for the creation of a spin-off and, if an investor is interested, accompanies the project and the researchers towards the creation of the start-up. It creates the legal structure of the alliance with the start-up and accompanies it in the first years of its life to foster its growth and monitor the alliance. We participate in five start-ups as a founding partner and sit on four boards of directors.
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 Biotech, IAMA Therapeutics, Genenta Science, Tr1X Bio.
