GEMMABio secures $100M partnership with health ministry to bring rare disease gene therapies to Brazil

GEMMABio, one of two new companies spun out of the now-folded Gene Therapy Program at the University of Pennsylvania, has announced its first major step in improving global access to rare disease gene therapies.

The Philadelphia-based biotech, founded and run by Jim Wilson, M.D., Ph.D., will receive up to $100 million from the Oswaldo Cruz Foundation (Fiocruz) for clinical research and manufacturing, the firm said in an Oct. 8 release. Fiocruz is a public health research institution that is part of Brazil’s Ministry of Health.

"By combining our resources and knowledge, this collaboration represents a major advancement in making gene therapies accessible to more patients quickly and at a fraction of the typical cost," Wilson, who is president and CEO of GEMMABio, said in the release. "We are optimistic that this deal will serve as a global example for expanding access to advanced medical therapies as well as pursuing non-traditional ways to capitalize biotech companies."

A primary piece of the deal is technology transfer, with GEMMABio’s manufacturing capabilities to be replicated in Brazil so that the company and Fiocruz can collaborate on research and production in the country, according to the release. In return for its funding, Fiocruz will be able to license related treatments for the Brazilian public health system and can use the tech with other programs and sponsors.

The release also answered the burning question of which rare diseases GEMMABio will focus on first. In Brazil, there will be six programs comprising GM1 gangliosidosis (GM1), Krabbe disease, metachromatic leukodystrophy (MLD) and three unnamed programs, GEMMABio said in the release.

GM1 leads to the progressive destruction of nerve cells in the spinal cord and brain due to a mutated gene that prevents patients from breaking down large sugar molecules. In Krabbe disease, patients have a mutation in the gene for an enzyme called galactosylceramidase; this causes the protective myelin coating of nerves to fray, exposing the nerves to damage. MLD also degrades myelin through a buildup of sulfatides due to a mutated gene. All three diseases commonly affect children.

Fiocruz is the main supplier of immunobiologics, biopharmaceuticals and diagnostics for the Brazilian Ministry of Health. The ultimate goal of the partnership is to deliver affordable rare disease gene therapies to Brazilian patients through the country’s publicly funded healthcare system, the Sistema Único de Saúde, the partners said in the release.

"Brazil has the largest public health system in the world, offering health services and products to more than 200 million people,” Fiocruz Vice President of Production and Innovation in Health Marco Krieger said in the release. “A technology that was previously restricted to those with higher incomes, will now be accessible to the entire Brazilian population."

In August, Wilson elected to shut down the Gene Therapy Program he led at Penn for more than 30 years to form GEMMABio and another company, contract research organization Franklin Biolabs, which recently launched its genetic medicine services. Combined, the two companies hope to bring treatments for rare diseases to patients around the world.