Genespire plans to start clinical testing of its single‑dose gene therapy candidate, GENE202, for methylmalonic acidaemia by the end of 2026 after completing IND‑enabling studies. GENE202 uses an immune‑shielded lentiviral vector to integrate a functional MUT gene into liver cells and has received orphan designation from both the FDA and the European Commission. The company raised $52 million in a September 2024 Series B to advance the programme and will run observational studies to better understand patient needs. If successful, the single‑dose approach could reduce transplant and chronic immunosuppression requirements and improve patients’ quality of life.
Genespire Aims To Begin Clinical Testing Of Single‑Dose GENE202 For Methylmalonic Acidaemia In 2026
Genespire is looking to take its single-dose gene therapy
Italian rare‑disease biotech Genespire plans to move its single‑dose gene therapy candidate, GENE202, into clinical testing by the end of 2026, Lucia Faccio, CEO of Genespire and partner at Sofinnova Partners, told Clinical Trials Arena. The company is completing investigational new drug (IND)‑enabling studies and preparing observational research to better define patient needs and disease characteristics.
Why GENE202 Matters
GENE202 targets methylmalonic acidaemia (MMA), a rare inherited disorder most commonly caused by mutations in the methylmalonyl‑CoA mutase (MMUT) gene. MMUT encodes a mitochondrial enzyme required for the breakdown of certain amino acids and fats; when it is defective, methylmalonic acid accumulates in blood and tissues, causing severe damage to the liver, brain and kidneys. MMA is estimated to affect roughly 1 in 50,000 to 1 in 100,000 live births, and today there are no approved therapies for the condition.
Approach And Potential Advantages
Genespire’s candidate is a hepatocyte‑directed, single‑administration gene therapy designed to drive durable expression of a functional MUT gene in liver cells. GENE202 is delivered using an immune‑shielded lentiviral vector (ISLV) that integrates into the recipient’s DNA. According to Faccio, integration can "contribute to the stable expression of the MUT gene in target cells," which may avoid the repeat dosing required by mRNA or some adeno‑associated virus (AAV) approaches.
If GENE202 proves safe and effective in clinical trials, it could reduce the need for organ transplants and chronic immunosuppression, potentially improving long‑term outcomes and quality of life for patients with MMA.
Competitive Landscape And Development Plans
GENE202 recently received orphan drug designation from both the US Food and Drug Administration and the European Commission, an achievement the company announced on 20 January. Moderna’s lipid nanoparticle mRNA therapy, mRNA‑3705, is currently the only programme in the clinic for MMA and is being evaluated in a Phase I/II study (NCT04899310) as a repeat‑dosing treatment.
Genespire closed a $52 million Series B round in September 2024 to support progression of GENE202 into the clinic. While preparing for human testing, the company plans to run observational studies to refine patient characterisation and will engage clinicians and patient communities to build confidence in its therapeutic approach.
Lucia Faccio: "There have been ups and downs in the field, but we see therapies gaining approval and commercial traction. Our aim is to show that a single‑dose, durable gene therapy can address unmet needs for patients with MMA."
Outlook
Genespire expects to initiate a first‑in‑human trial by the end of 2026, pending successful completion of IND‑enabling studies and regulatory clearance. Broader adoption of gene therapies will depend on continued innovation in manufacturing, pricing and reimbursement, areas the industry is actively addressing as the sector matures.
Note: This article is based on reporting by Clinical Trials Arena and information announced by Genespire and regulatory authorities.
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