Italian biotech Genspire is set to launch a Phase I/II clinical trial of its lead gene therapy, GENE202, as a treatment for a group of rare, inherited diseases – methylmalonic acidemia.
The company will be able to fund the trial after it received €46.6m ($52.1m) in a Series B financing round. The financing will also help to fund discovery and preclinical work for other gene therapy candidates.
Investors included European investment firms such as Sofinnova Partners, XGEN Venture and CDP Venture Capital. Genspire is a spinout from Milan-based San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), and has previously raised €16m in Series A financing in 2020.
Methylmalonic acidaemia is a group of rare genetic disorders where an individual is unable to metabolise proteins and fats properly. This results in high levels of acid in the blood (acidaemia) and body tissues. Methylmalonic acidaemia often manifests within the first few months of life and its severity can vary from mild disease to life-threatening.
According to the US National Organization for Rare Disorders (NORD), methylmalonic acidaemia usually affects 1 in 50,000 to 1 in 100,000 live births. The disease is usually managed through dietary modifications to limit certain proteins and fats.
GENE202 is an off-the-shelf, one time gene therapy developed using the company’s Immune Shielded Lentiviral Vector (ISLV) platform. ISLV-based therapies are designed to allow a patient's liver to produce the therapy throughout its lifetime.
Gene therapies are a growing area of interest, with multiple gene therapies gaining approval from the US Food and Drug Administration (FDA) in recent years. In recent months, multiple companies have entered the gene therapy market by acquiring or merging with specialist companies and pipelines.
Last year, Alexion AstraZeneca Rare Disease signed a definitive agreement, acquiring and licensing Pfizer’s early-stage gene therapy portfolio for rare diseases. The deal included a $1bn payment along with net sale royalties from the acquired therapies. Despite the recent interest, the uptake of gene therapies is limited due to their high price tags.
Furthermore, not all gene therapies make it to market, especially for rare diseases, as their high cost and limited commercial market can deter companies from investing. In some cases, academic institutes and non-profit organisations have ‘rescued’ these gene therapy programmes.
Another therapy in development for methylmalonic acidaemia is Moderna’s mRNA therapy, mRNA-3705. The therapy is being investigated in the Phase I/II Landmark study (NCT04899310) as a treatment for a specific type of methylmalonic acidaemia, one due to CoA mutase (MUT) deficiency. In June 2024, mRNA-3705 was included in a Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot programme by the US Food and Drug Administration (FDA). The START programme is aimed at expediting the development of novel drugs and biological products for rare diseases.
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