UPenn gene therapy pioneer’s biotech gets $34 million in funding

A company founded by a University of Pennsylvania gene therapy pioneer announced Monday it has received $34 million in seed funding.

GEMMA Biotherapeutics, founded by Jim Wilson earlier this year, focuses on developing gene therapies for rare diseases. GEMMABio has made global access to gene therapy — which is often expensive — a priority, and in October, the company announced it will receive up to $100 million from Brazil’s Ministry of Health for research that can lead to treatments for rare diseases in the South American country.

The $34 million in seed funding is designed to help GEMMABio expand its operations and gene therapy programs, and was co-led by multiple venture capital firms, including Double Point Ventures, Bioluminescence Ventures, and Earlybird Venture Capital.

“We are delighted by the enthusiastic support from our engaged and high-quality partners, who share our vision for the future of gene therapy,” Wilson said in a statement. “Their 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.”

For decades, Wilson was a titan of the field at Penn, which notes that dozens of companies have been using Wilson’s viruses to develop new gene therapies.

His team’s work also helped establish eight biotech companies and nearly 100 patents.

He became widely known as the lead scientist in a gene therapy trial that led to the 1999 death of 18-year-old Jesse Gelsinger, the first person to die as a result of gene therapy. The tragedy led to a government investigation, and Wilson was barred from conducting clinical trials for five years.

He continued to dedicate his career to using gene therapy to fight rare disease, and focused on experimenting with different viruses to deliver corrected genes to patients without triggering deadly side effects.

His work proved successful, and in 2019, the U.S. Food and Drug Administration approved the first commercial gene therapy using a virus from Wilson, which treated spinal muscular atrophy.

In July, Penn decided to spin out the Gene Therapy Program led by Wilson into two for-profit companies: GEMMABio, where Wilson would serve as CEO, and Franklin Biolabs, a contract research organization that offers services to other companies in the industry, including research and manufacturing vectors for gene therapy. Wilson, who left Penn as a result of the transaction, is also chairperson of Franklin Biolabs.

As a result of the transfer agreement, both companies have to keep their corporate headquarters in the Philadelphia area.