This is one of the largest private financing rounds for Italian biotech companies. The funds will be used to bring the pediatric off-the-shelf gene therapy for methylmalonic acidemia (MMA) to clinical trials and to strengthen the company’s gene therapy pipeline.
Italian biotech company Genespire has raised €46.6 million (about $52 million) in a Series B funding round. This infusion of capital will enable the development—up to phase 1-2 clinical trials—of Gene202, its off-the-shelf pediatric gene therapy aimed at combating a devastating genetic disease called Methylmalonic Acidemia (MMA). The funding round was co-led by Sofinnova Partners, XGEN Venture, and CDP Venture Capital through its Large Ventures Fund, alongside Indaco as part of a strong pool of investors, as noted in the company’s announcement of the funding closure.
MMA is a disorder that affects the metabolism of certain amino acids and fats. It manifests in early childhood, has a high mortality rate, and is characterized by severe symptoms such as muscle weakness, seizures, developmental delays, and organ damage. Currently, there are no treatments that modify the course of the disease. The Gene202 therapy uses the ISLV (Immune Shielded Lentiviral Vector) platform, which are lentiviral vectors designed for intravenous use. This technology was developed by Genespire’s co-founders, gene therapy pioneers Luigi Naldini and Alessio Cantore at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget). The strategy is to enable the patient’s liver to produce the therapy for a lifetime. This method makes Genespire’s approach particularly suitable for pediatric patients with genetic diseases, addressing urgent unmet medical needs while also offering benefits for adult patients, according to the company.
This funding round will strengthen the company’s pipeline, supporting the discovery and preclinical development of products to treat various genetic diseases, the statement continues.
“Our ISLV platform,” stated Genespire CEO Karen Aiach-Pignet, “gives Genespire the unique ability to offer innovative treatments that have the potential to transform the lives of children suffering from genetic diseases. The support of our new and existing investors reflects the enthusiasm and confidence in the potential of our platform, further reassured by the promising data generated so far. We extend heartfelt thanks to our former CEO Julia Berretta for her pivotal 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 and the SR-Tiget team, in bringing our first MMA therapy to the clinic.”
“Since our initial investment, we have strongly believed in the internationally recognized Genespire team and their groundbreaking lentiviral technology,” emphasized Lucia Faccio, partner at Sofinnova Partners. “We remain fully committed to supporting the company’s growth in collaboration with our colleagues from XGEN, CDP Venture Capital, and Indaco, to pave the way for transformative therapeutic approaches for various pediatric genetic diseases.” Gene therapy “is proving its effectiveness with several recent clinical successes and product approvals, and we believe Genespire’s approach has tremendous potential,” added Paolo Fundarò, founder and managing partner of XGEN Venture.
Agostino Scornajenchi, CEO and General Manager of CDP Venture Capital, noted that Genespire’s research “began in the renowned labs of the San Raffaele Institute and is driven by an ambitious team aiming, through advanced gene therapy technologies, to offer curative treatments for diseases currently lacking effective therapeutic options, such as MMA. We look forward to supporting the company in establishing a new international gold standard.”
As part of the Series B round, Marco Dieci, Senior Advisor at CDP Venture Capital and CEO of Simis Srl, will join the Board of Directors at Genespire as a representative of CDP Venture Capital, along with Paolo Fundarò, founder and Managing Partner of XGEN Venture.
Source: Sanità33