RARE Daily

Genespire Launched to Develop Gene Therapies

April 20, 2020

Venture firm Sofinnova Partners said it has invested in Genespire, an Italian biotech founded in March by gene therapy pioneer Luigi Naldini, Alessio Cantore, The Telethon Foundation, and San Raffaele Hospital.

Genespire will focus on developing gene therapies for patients affected by genetic diseases, primarily imunodeficiencies and inherited metabolic disorders. The company is a spin-out of SR-Tiget, a leading cell and gene therapy research institute.

The financing is the third investment from Sofinnova’s Telethon Fund, the largest fund in Italy making early-stage investments in Italian companies targeting cures for rare and genetic diseases.

The fund has invested $27 million (€25 million) over the past six months. Besides Genespire, the Sofinnova Telethon Fund has invested in Epsilen Bio, which is developing a technology to turn off genes linked to specific pathologies without irreversibly modifying the DNA, and PinCell, a preclinical-stage biotech focused on therapies for rare dermatological diseases.

The Sofinnova Telethon Fund, managed by Sofinnova Partners, received commitments totaling $117 million (€108 million), to invest exclusively in Italy, seeking to identify, create and develop some of the best companies in the field of rare and genetic diseases.

Francesca Pasinelli, CEO of The Telethon Foundation, said the biggest winners of the partnership would be the patients who suffer from rare and genetic diseases.

“This Fund has always been about them,” he said, “and additionally is dedicated to elevating Italian science, supported by Italian investors and focused on Italy’s best entrepreneurs and companies.”

Italy has been at the forefront of rare disease care and research. The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) was created in 1995 as a joint venture between the Telethon Foundation and IRCCS Ospedale San Raffaele, with the mission to perform cutting-edge research in gene and cell therapy and to translate its results into therapeutic advances, focusing on genetic diseases. It has been a fertile ground for innovative therapies, including Strimvelis, which was the first ex-vivo gene therapy to win approval in Europe for ADA-SCID immunodeficiency.

The Fondazione Telethon is a non-profit organization created in 1990 to advance scientific research in genetic diseases. Through a television marathon, along with other initiatives and a network of partners and volunteers, Telethon has raised more than $575 million (€ 528 million) over 28 years to finance scientific research on rare genetic diseases, with the ultimate objective of making the treatments developed available to everyone who needs them.

Photo: Francesca Pasinelli, CEO of The Telethon Foundation

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