A gene therapy pioneer thinks he has a solution to the field’s struggle to commercialize breakthrough cures: Go global.
Jim Wilson’s Philadelphia-based company GemmaBio on Tuesday announced up to $100 million in funding to manufacture and test six gene therapies in Brazil. The company’s plan, funded by Brazil’s Ministry of Health, looks to produce the potential medicines at a fraction of the millions of dollars that they often cost.
Drugmakers big and small have found that the price tag for gene therapies made them a tough sell, or impractical to develop. In an interview, Wilson told Endpoints News that he’ll soon board flights to Korea, Saudi Arabia and other countries in hopes of making similar partnerships with organizations there.
“What I’d like to be remembered for, and this is the chapter that we’re working on now that we have not yet achieved, is to be an advocate for global access of genetic medicines,” Wilson said.
Wilson, initially known for a tragedy that halted the gene therapy field 25 years ago, managed a comeback by leading an influential group at the University of Pennsylvania. His academic unit developed methods for delivering genes to cells, a major step in breakthrough therapies, like Novartis’ therapy Zolgensma for spinal muscular atrophy.
That progress, however, couldn’t overcome another trend: Companies began to abandon rare disease medicines that were deemed commercially unviable. Endpoints reported last year that dozens of companies had shelved dozens of treatments for rare conditions, often citing the cost of development and commercial uncertainty.
Those failures led Wilson to conclude that the existing model for those rare diseases and their tiny groups of patients had reached its limit.
“We had sort of run our course of what we can do in an academic setting,” Wilson said.
In late July, the University of Pennsylvania board of trustees approved a spinout of Wilson’s research group that became GemmaBio. The company is initially working on eight therapies, three of them licensed from Passage Bio, a company Wilson co-founded in 2018. Under a licensing deal reached in August, GemmaBio paid Passage Bio $10 million upfront, and the agreement calls for additional milestone payments.
Many drugmakers have turned away from gene therapies aimed at rare conditions because of the limited size of the markets. Wilson believes that GemmaBio’s global approach will maximize the number of patients treated. To reach them, the company is zeroing in on countries that look to benefit the most from its potential medicines.
One of the diseases it’s targeting — a fatal disorder called GM1 gangliosidosis that destroys neurons in the brain and spinal cord — is 10 times as common in Brazil compared to the rest of the world.
“You have to go to where the prevalence is higher to conduct the clinical trials,” Wilson said.
For GemmaBio, Brazil made sense in additional ways. The company is partnering with Fiocruz, a division of Brazil’s Ministry of Health, that has built up the country’s biomanufacturing sector to lessen costs and be closer to patients.
During the pandemic, Fiocruz partnered with AstraZeneca to locally produce the company’s Covid-19 vaccine. Fiocruz has also worked with a nonprofit to domestically manufacture CAR-T therapies.
Fiocruz’s up to $100 million in funding to GemmaBio will go toward clinical trials and manufacturing, with an eye toward securing approval for the six therapies in the region and potentially other countries.
“Brazil could be a foothold to gain broader access throughout Latin America,” Wilson said.
In exchange for Fiocruz’s financial support, the organization has the option to license related treatments to supply the Brazilian public health system.
Often, drugmakers first worry about FDA clearance, which quickens approvals in other countries. But Wilson said he’s encouraged by an FDA pilot program that looks to develop uniform standards across countries.
“While we’re focusing this announcement on Brazil, we’re thinking globally,” Wilson said.
GemmaBio’s mission aligns with calls to make gene therapies accessible.
“Researchers, drug developers and drug manufacturers are squandering the chance to make gene therapies viable even in wealthier countries, let alone transformative for the world,” co-authors Evelyn Mwesigwa Harlow and Jennifer Adair wrote earlier this year in Nature. “To seize the opportunity, they must take into account the populations most in need, as well as the global market for treatments — both when developing and when valuing the drugs.”
It was once unthinkable that Wilson could be at the forefront of bringing gene therapies to the world. In 1999, he led a first-of-its-kind experiment in which 18-year-old Jesse Gelsinger died because of a gene therapy that triggered an immune system reaction.
The death was a major setback for the field and for Wilson, whose gene therapy center was disbanded, and he was barred from working on clinical trials for five years. In a comeback over the intervening years, Wilson’s lab discovered a family of viruses that could be engineered to shuttle gene therapies to cells, leading to a resurgence of the field.
GemmaBio’s mission is being cheered on by rare disease advocates, who have watched drugmakers leave the space en masse.
“You don’t do that unless you really care,” Christine Waggoner, the co-founder of the Cure GM1 Foundation, which jumpstarted one of the therapies that was paused by Passage Bio and licensed by GemmaBio.
GemmaBio still faces a major challenge. Companies like Taysha Gene Therapies have tried to standardize gene therapy development only for their plans to crumble. A cash crunch at Taysha led the company to scrap most of its work, and it has been slow to offload paused programs that were initially funded by families.
Wilson said GemmaBio distinguishes itself through both a global strategy and a focus on diseases with a smoother path for regulatory approval.
“Many of the diseases that they selected were complicated,” Wilson said of other efforts. “Clinical development wasn’t clear. Endpoints weren’t clear. Natural history wasn’t clear. Potential for accelerated approval wasn’t clear.”
Beyond just disease selection, GemmaBio could benefit from the FDA’s increasing openness to accelerated approval for gene therapies. And the agency is developing a so-called platform policy that could allow drug developers to simultaneously target multiple diseases without redoing critical work.
Alongside GemmaBio, Wilson founded Franklin Biolabs, a contract research organization that specializes in viral vectors for gene therapies. It has received an undisclosed amount in funding from Savanne Life Sciences, the investment arm of Bioculture Group, a breeder of non-human primates for research.
Plans call for soon disclosing how much GemmaBio received in seed funding, and from whom, according to Wilson. Besides venture capital, he’s hoping for a long-term extension of a federal voucher program that’s designed to incentivize the development of rare disease drugs.
And the $100 million from Fiocruz is the first, he hopes, of more government partnerships.
“It sends a message there is a different way for all of us to succeed in our business,” Wilson said.