Genespire raises €46.6 million (~$52 million) in a Series B round to advance its first pediatric in-vivo gene therapy into the clinic

One of the largest private Italian Biotech company financings to date

Financing co-led by Sofinnova Partners, XGEN Venture and CDP Venture Capital forming a strong syndicate with Indaco SGR

Funds dedicated to advancing off-the-shelf pediatric gene therapy for Methylmalonic Acidemia (MMA) into the clinic, and to bolstering the Company’s gene therapy pipeline

Genespire, a biotechnology company developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases, today announced the closing of a €46.6 million (~$52 million) Series B financing, co-led by Sofinnova Partners, XGEN Venture and CDP Venture Capital through its Large Venture Fund forming a strong syndicate with Indaco SGR.

The financing will enable the development of GENE202, the company’s lead candidate, up to a Phase I/II clinical trial for the treatment of MMA, a devastating genetic disorder impairing the metabolism of certain amino acids and fats.

MMA has onset in early infancy, is associated with significant mortality and morbidity, and is characterized by severe clinical manifestations including muscular weakness, seizures, developmental delays and organ damage. Currently, there are no disease-modifying treatments for MMA. GENE202 is a pioneering off-the-shelf gene therapy which harnesses the company’s Immune Shielded Lentiviral Vector (ISLV) platform. ISLVs are designed to be used intravenously, enabling the patient's liver to produce the therapy throughout its lifetime. This unique mode of action makes Genespire’s approach optimally suited to address pediatric patients affected by genetic diseases who currently face the most pressing unmet medical needs, while also benefitting adult patients. The ISLV technology was developed by Genespire’s scientific co-founders and gene therapy leaders, Pr. Luigi Naldini and Dr. Alessio Cantore, at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget).

This Series B financing round will also strengthen the Company’s pipeline by fostering discovery and preclinical work on candidate products targeting several additional genetic diseases.

Karen Aiach-Pignet, Genespire’s CEO said, “Our highly innovative ISLV platform gives Genespire the unique ability to bring groundbreaking treatments that have the potential to transform the lives of children suffering from genetic disease. The support from our new and existing investors reflects the enthusiasm and confidence in our platform’s potential, with further validation achieved from the promising data generated to date. We extend a heartfelt gratitude to our previous CEO, Julia Berretta, for her instrumental role in this financing round, and we look forward to working closely with CDP Venture Capital, XGEN Venture and Indaco SGR, alongside our founding investor, Sofinnova Partners, as well as the SR-Tiget team, as we drive our first therapy in MMA towards the clinic.”

“Since our initial investment, we’ve been deeply convinced by Genespire’s world-class team and its groundbreaking lentiviral technology.” said Lucia Faccio, Partner at Sofinnova Partners. “We remain firmly committed to supporting the company’s growth in collaboration with our colleagues from XGEN, CDP Venture Capital and Indaco, to pave the way for single treatment approaches for several genetic disorders in children.”

“Gene therapy is starting to deliver results with several recent clinical successes and product approvals and we believe that Genespire’s approach offers huge opportunities. We look forward to working with fellow investors and the Management team to help realize this potential.” commented Paolo Fundarò, Founder and Managing Partner from XGEN Venture.

“Health, particularly Biotechnology Research dedicated to therapeutic needs, is one of the strategic macro-areas prioritized in our 2024-2028 Business Plan,” says Agostino Scornajenchi, CEO and General Manager of CDP Venture Capital, “Genespire’s research started in the renowned laboratories of the San Raffaele Institute, and is being advanced by an ambitious team that, through advanced gene therapy technologies and gene therapies, aims to offer curative treatments for diseases that currently lack effective therapeutic options, such as MMA. We look forward to supporting the company to set a new international gold standard.”

In connection with the Series B round, Marco Dieci, Senior Advisor at CDP Venture Capital and Chief Executive Officer of Simis S.r.l., will join Genespire’s Board of Directors as the representative for CDP Venture Capital, alongside Paolo Fundarò, Founder and Managing Partner from XGEN Venture.