Genespire Secures $52 Million to Advance Gene Therapy for Methylmalonic Acidemia
Key Insights
Genespire (search) has raised $52 million in a Series B financing round to advance its gene therapy program for Methylmalonic Acidemia (search) (MMA (search)).
The funding will support the development of GENE202, an off-the-shelf gene therapy, through a Phase I/II clinical trial.
Genespire (search)'s Immune Shielded Lentiviral Vector (ISLV) platform delivers the therapy directly to the liver for lifelong production.
Genespire (search), a biotechnology company focused on developing gene therapies for pediatric genetic diseases, has announced the closing of a €46.6 million (~$52 million) Series B financing. The funding will primarily support the advancement of GENE202, the company's lead candidate, into a Phase I/II clinical trial for the treatment of Methylmalonic Acidemia (search) (MMA (search)). The financing was co-led by Sofinnova Partners (search), XGEN Venture (search), and CDP Venture Capital (search), with participation from Indaco SGR (search).
GENE202: A Novel Approach to MMA Treatment
MMA (search) is a rare, inherited metabolic disorder characterized by the body's inability to properly process certain amino acids and fats. The condition typically manifests in early infancy and can lead to severe clinical complications, including muscular weakness, seizures, developmental delays, and organ damage. Currently, there are no disease-modifying treatments available for MMA, highlighting a significant unmet medical need.
GENE202 is designed as an off-the-shelf gene therapy that utilizes Genespire (search)'s proprietary Immune Shielded Lentiviral Vector (ISLV) platform. This platform is designed for intravenous administration, enabling the patient's liver to produce the therapeutic protein throughout their lifetime. The ISLV technology, developed by Genespire's scientific co-founders Pr. Luigi Naldini and Dr. Alessio Cantore at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), aims to overcome the limitations of traditional gene therapy approaches by reducing the risk of immune rejection.
The ISLV platform utilizes the baculovirus-derived GP64 envelope protein to target the therapeutic RNA encoding functional methylmalonyl-CoA mutase (search) (MUT (search)) to hepatocytes and liver sinusoidal endothelial cells (LSEC).
Addressing Unmet Needs in Pediatric Genetic Diseases
"Our highly innovative ISLV platform gives Genespire (search) the unique ability to bring groundbreaking treatments that have the potential to transform the lives of children suffering from genetic disease," said Karen Aiach-Pignet, Genespire’s CEO. The company's focus on pediatric patients with inherited metabolic diseases reflects a commitment to addressing critical unmet needs in this population.
Expanding the Pipeline
In addition to advancing GENE202, the Series B financing will also support the expansion of Genespire (search)'s pipeline through discovery and preclinical work on candidate products targeting several additional genetic diseases. This strategic investment underscores the company's long-term vision of developing a portfolio of innovative gene therapies for a range of pediatric disorders.
Investor Confidence
The Series B round saw Marco Dieci, Senior Advisor at CDP Venture Capital (search) and Chief Executive Officer of Simis S.r.l., join Genespire (search)’s Board of Directors as the representative for CDP Venture Capital, alongside Paolo Fundarò, Founder and Managing Partner from XGEN Venture (search).
"Since our initial investment, we’ve been deeply convinced by Genespire (search)’s world-class team and its groundbreaking lentiviral technology," said Lucia Faccio, Partner at Sofinnova Partners (search). "We remain firmly committed to supporting the company’s growth in collaboration with our colleagues from XGEN, CDP Venture Capital (search) and Indaco, to pave the way for single treatment approaches for several genetic disorders in children."