Jim Wilson’s new gene therapy company Gemma is getting up to $100M from Brazil

Research scientist Jim Wilson’s new gene therapy venture, Gemma Biotherapeutics, will receive up to $100 million from Brazil’s Ministry of Health for research designed to bring treatments for rare diseases to market in the South American country, the company recently spun out of the University of Pennsylvania announced Tuesday.

The agreement covers clinical development activities for six genetic disorders affecting the central nervous system. The ultimate goal is for the Brazilian government, which operates the world’s largest national health system, to manufacture and distribute the treatments for its population. Gemma would collect a royalty from each treatment.

Gemma is one of two companies the University of Pennsylvania board of trustees voted in July to launch out of its pioneering Gene Therapy Program, which Wilson had led for decades.

Wilson now leads Gemma as CEO and is chair of the second company, Franklin Biolabs, which will perform research under contract for other companies. Combined, the companies employ 240 people.

One of Wilson’s goals in the creation of two new companies — at a time of decreased investment in biotech — was to diversify funding streams for gene therapy treatments beyond traditional Wall Street investors to include philanthropists, foundations, and governments, as is the case with the Brazilian partnership.

“I can’t tell you how excited I am about achieving this milestone. My hope is, it reinforces our message about innovation, not only in science, but innovation in business,” Wilson said in an interview.

As of Oct. 1, he no longer works for Penn.

Gemma identified three of the six diseases it is tackling in Brazil. They are GM1 gangliosidosis, an inherited disease that destroys nerve cells in the brain and spine; Krabbe disease, a disorder that leads to the loss of the protective coating on nerves; and metachromatic leukodystrophy, another disorder that destroys the protective sheath on nerves.

Those three potential treatments are licensed from Passage Bio Inc., a Philadelphia company that relies on research from Wilson’s lab at Penn into neurodegenerative diseases. Wilson did not disclose details on the other three therapies.

The funding from the Brazil Ministry of Health’s Oswaldo Cruz Foundation is expected to cover the costs of bringing the six treatments to market over the next three to five years, Wilson said.

“We feel very comfortable in Brazil and confident in the stakeholders that we’re working with,” Wilson said, noting that Passage Bio and another company he helped found have had positive experiences there during clinical trials.

If Brazil’s equivalent of the U.S. Food and Drug Administration approves the treatments, the Oswaldo Cruz Foundation, known as Fiocruz, will be responsible for manufacturing and distributing them.

Marco Krieger, vice president of production and innovation in health at Fiocruz, said in a news release that Brazil has the largest public health system in the world, serving more than 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,” he said.