Rare disease biotech Genespire is looking to take its single-dose gene therapy – GENE202 – to the clinic in 2026, CEO and Sofinnova Partner, 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.
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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 designation from both the US Food and Drug Administration (FDA) and the European Commission (EC), which was announced by the company on 20 January.
Methylmalonic acidaemia most commonly arises from a faulty copy of the methylmalonyl-CoA mutase (MUT) gene, which encodes a mitochondrial enzyme responsible for the breakdown of proteins and fats.
This means that patients with this genetic condition have an unusually high level of clear methylmalonic acid in the blood, resulting in severe organ damage in the liver, brain and kidneys.
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By GlobalDataGENE202’s designation comes at a time when there are no approved therapies available for patients with methylmalonic acidaemia, which is estimated to impact between 1 in 50,000 and 1 in 100,000 livebirths.
Now, companies across the pharmaceutical industry are developing therapies to treat methylmalonic acidaemia. Moderna’s lipid nanoparticle-powered mRNA therapy, mRNA-3705, is currently the only drug in the clinic – where it is being evaluated as a bi- or tri-weekly treatment for the rare disease in a Phase I/II study (NCT04899310).
Taking a single-dose approach to treatment
While a majority of the therapies currently in development are life-long treatments, Genespire is taking a different approach with GENE202 by creating a single-dose, hepatocyte-directed gene therapy that aims to encourage lifelong expression of the functioning MUT gene in a recipient’s liver cells.
GENE202 is powered by an immune-shielded lentiviral vector (ISLV), which can be directly inserted into a recipient’s DNA, which Faccio noted can “contribute to the stable expression of the MUT gene in target cells,” potentially overcoming the need for the repeat dosing necessary with mRNA or adeno-associated virus (AAV) alternatives.
If the drug is proven effective in clinical trials, it could mitigate the need for organ transplants and the chronic use of immunosuppressants, potentially improving a patient’s overall quality of life (QoL).
While exploring GENE202 in the clinic, Faccio stated that Genespire will place a keen focus on building awareness and confidence in the therapeutic approach.
In September 2024, Genespire raised $52m in series B fundraising to help advance GENE202 into the clinic.
The future of gene therapies
As a partner under Sofinnova’s Telethon Fund, Faccio acknowledges that gene therapies have faced several challenges from a commercial, pricing and reimbursement standpoint.
“There have been ups and downs in the field, but there are also several therapies that have gained approval and are continuing to grow in commercial value,” she said.
Currently, a strong proportion of marketed or pipeline gene therapies are indicated for rare diseases, though Faccio believes the modality is also set to become increasingly prevalent in common indications.
As an example of this shift, Faccio named AstraZeneca’s $1bn acquisition of in vivo cell therapy developer, EsoBioTec, which is currently developing a range of lentiviral therapies for the treatment of certain cancers.
“I believe we will soon see what we observed 20 years ago with biologics and monoclonal antibodies (mAbs),” Faccio stated, noting that these drugs are “halfway” to becoming widely accessible and common across a broad selection of disease areas.
However, to achieve this, Faccio caveated that the industry will need to see innovation around manufacturing, pricing and reimbursement.
According to GlobalData, parent company of Clinical Trials Arena, the cell and gene therapy (CGT) sector is shifting amid a more selective and strategy-driven funding landscape.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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