Genspire secures US$52M to advance paediatric gene therapy

ITALY – Genspire, an Italian biotech company, is preparing to initiate a Phase I/II clinical trial for its flagship gene therapy, GENE202, aimed at treating methylmalonic acidemia (MMA), a group of rare, inherited metabolic disorders.

The company secured funding for the trial after raising €46.6 million (US$52.1 million) in a Series B financing round.

This funding will support the GENE202 trial and advance the discovery and preclinical development of other gene therapy candidates.

According to Pharmaceutical Technology, prominent European investors, including Sofinnova Partners, XGEN Venture, and CDP Venture Capital, participated in the Series B round.

Genspire, which originated as a spinout from the Milan-based San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), had previously raised €16 million(US$ 17.9 million) in Series A funding back in 2020.

GENE202 is a one-time, off-the-shelf gene therapy developed using Genspire’s Immune Shielded Lentiviral Vector (ISLV) platform.

ISLV-based therapies are designed to continually enable the patient’s liver to produce the therapeutic protein throughout their lifetime.

Gene therapy has become an exciting area of research, with several therapies receiving approval from the US Food and Drug Administration (FDA) in recent years.

In addition to Genspire’s efforts, the field of gene therapy is attracting significant attention, with multiple companies entering the market through acquisitions and partnerships.

 For instance, in 2023, Alexion AstraZeneca Rare Disease signed a deal to acquire and license Pfizer’s early-stage gene therapy portfolio, which focuses on rare diseases.

This agreement included a $1 billion payment in addition to potential royalties from future sales. However, despite growing interest, the uptake of gene therapies remains limited due to their high cost.

Another promising therapy in development for methylmalonic acidemia is Moderna’s mRNA-based therapy, mRNA-3705.

This treatment is currently being investigated in a Phase I/II trial, known as the Landmark study (NCT04899310), specifically targeting a form of the disorder caused by CoA mutase (MUT) deficiency.

In June 2024, mRNA-3705 was selected for the US FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program, which aims to expedite the development of novel treatments for rare diseases.

Methylmalonic acidemia is a rare genetic condition in which individuals cannot properly metabolize specific proteins and fats.

 This results in an abnormal accumulation of acid in the blood (acidemia) and body tissues, which can be risky for overall health.

 The disorder typically manifests within the first few months of life, with severity ranging from mild symptoms to life-threatening complications.

According to the US National Organization for Rare Disorders (NORD), the condition affects approximately 1 in 50,000 to 1 in 100,000 live births.

Managing the disease often involves strict dietary modifications to limit the intake of specific proteins and fats.

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