US-based gene therapy startup, GEMMABio has raised $34m in seed funding to help advance its portfolio of gene therapy programmes.
GEMMABio was founded by gene therapy scientist Jim Wilson who led an influential research group at the University of Pennsylvania. The group developed methods for delivering genes to cells, which was a major step for gene therapies. Wilson departed the University of Pennsylvania earlier this year and founded GEMMABio as well as contract research organisation (CRO) Franklin Biolabs, that specialises in viral vectors for gene therapies.
The $34m seed round was led by Double Point Ventures, Bioluminescence Ventures and Earlybird Venture Capital, with additional backing from Savanne Life Sciences.
Building on a $100m boost secured by GEMMABio in October, the company said it will manufacture and test six gene therapies in Brazil. The therapies are targeting a range of rare diseases, including a fatal disorder called GM1 gangliosidosis that destroys neurons in the brain and spinal cord.
GEMMABio secured the funds via a partnership with Fiocruz – the main supplier of immunobiologics, biopharmaceuticals and diagnostics to the Brazilian Ministry of Health – to sponsor research that will bring gene therapy treatments to the people of Brazil under Brazil’s publicly funded health care system, the Sistema Único de Saúde (SUS).
Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Last year was a record year for cell and gene therapy approvals, with seven treatments approved by US Food and Drug Administration (FDA).
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By GlobalData
Gene therapies make up some of the world’s most expensive drugs, mainly because of their curative potential and single administration requirement. Their high price tags are also partly attributed to high manufacturing costs, the use of advanced technologies and relatively small target populations.
Orchard Therapeutics’ gene therapy Lenmeldy (atidarsagene autotemcel) is the most expensive drug on the market with a price tag of $4.25m, according to GlobalData analysis. The gene therapy is developed for children with metachromatic leukodystrophy (MLD). GlobalData predicts that the therapy will generate $154m in sales in 2030.
GlobalData is the parent company of Pharmaceutical Technology.
Despite the successes in the last few years, many companies fall short at the earlier stages of therapeutic development due to manufacturing challenges and a failure at scaling up. In the announcement accompanying the funding, Wilson said: “Their [funding partners’] involvement comes at a pivotal moment in the industry, and together, we are committed to expanding and speeding access to affordable gene therapies for the patients who need them most.”
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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