Fiocruz and GEMMABio announce partnership for the development of gene therapies

Fiocruz and GEMMA Biotherapeutics (GEMMABio) announced last Tuesday (10/8) an agreement to sponsor research focused on gene therapy treatments for the Brazilian population within the scope of the Unified Health System (SUS). The ultimate goal of the international partnership with the company founded by gene therapy expert Jim Wilson is to obtain approval for gene therapy programs that will enable the country to increase the distribution of treatments for rare diseases at a more affordable cost.

With the support of the Ministry of Health (MS), the Foundation will invest up to US$100 million in GEMMABio over four years. The funds will be used to develop clinical research and production to expand the distribution of treatments for rare diseases in Brazil at a more affordable cost for the SUS.

"This partnership with GEMMABio reaffirms Fiocruz's commitment to innovation in health and its mission to ensure that scientific advances reach all Brazilians," said the Foundation's president, Mario Moreira. "By expanding access to gene therapies through the SUS, we are not only democratizing cutting-edge technologies, but also strengthening our ability to treat rare diseases in an affordable and effective manner. It is an important step to establish Brazil as a global reference in public health and innovation," he added.

"By combining our resources and expertise, this collaboration represents a major breakthrough in making gene therapies accessible to more patients faster and at a fraction of the usual cost," said Jim Wilson, president and CEO of GEMMABio. "We are optimistic that this agreement will serve as a global example for expanding access to advanced medical therapies, as well as for seeking non-traditional ways to capitalize biotechnology companies."

The partnership, which will be implemented through the Immunobiological Technology Institute (Bio-Manguinhos/Fiocruz), involves the transfer of technology used in GEMMABio's manufacturing processes to be replicated in Brazil. The agreement also provides for clinical development activities in Brazil for six main programs. Three of these programs focus on developing gene therapy treatments for GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy.

GM1 gangliosidosis is an inherited disorder that progressively destroys nerve cells in the brain and spinal cord. Krabbe disease is a rare and usually fatal disorder of the nervous system. Metachromatic leukodystrophy is a rare inherited disease that causes fatty substances to accumulate in cells, particularly in the brain, spinal cord and peripheral nerves.

The disease targets for the other three programs have not yet been disclosed. In exchange for the financial support, Fiocruz will be able to license the related treatments to supply the SUS. "Brazil has the largest public health system in the world, offering health services and products to over 200 million people. We are very proud to bring this innovation to the Brazilian system. A technology that was previously restricted to those with higher incomes will now be accessible to the entire Brazilian population free of charge," said Marco Krieger, vice president of Production and Innovation in Health at Fiocruz.

GEMMABio

GEMMABio is a therapeutics company focused on speeding the research of and global access to life-changing advanced therapies for those living with rare diseases. The company will provide research and product development functions to bring gene therapy discoveries from the bench to the bedside faster and affordably. GEMMABio is led by gene therapy industry pioneer Jim Wilson and his team of experts, who previously conducted their work in academia.