Clinical Trials Arena January 21, 2026
GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
Rare disease biotech Genespire is looking to take its single-dose gene therapy – GENE202 – to the clinic in 2026, CEO and partner at Sofinnova Partners, Lucia Faccio, tells Clinical Trials Arena.
Genespire is currently preparing investigational new drug (IND)-enabling studies on GENE202 in methylmalonic acidaemia – a rare genetic disease characterised by a buildup of amino acids and fats in the bloodstream.
If all goes to plan, the Italian biotech will initiate a clinical trial by the end of 2026, while also performing observational studies to better characterise this rare disease and the unmet patient needs.
This news comes as GENE202 was granted orphan...
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