Jim Wilson raises $100M for global gene therapy revival

A pioneering gene therapy company, GemmaBio, led by Jim Wilson, has announced a significant funding initiative aimed at making gene therapies more accessible globally. The Philadelphia-based company revealed on Tuesday that it has secured up to $100 million in funding to produce and test six gene therapies in Brazil. This funding, provided by Brazil’s Ministry of Health, aims to manufacture these potentially life-saving medicines at a fraction of the cost typically associated with gene therapies, which often reach millions of dollars.

The high cost of gene therapies has been a significant barrier for drugmakers, making it challenging to develop and commercialize these treatments. In a recent interview, Wilson expressed his ambition to establish similar partnerships in other countries, including Korea and Saudi Arabia, to further this global initiative.

Wilson has a storied history in the field of gene therapy. He initially gained notoriety 25 years ago due to a tragic incident that halted progress in the field. However, he made a remarkable comeback by leading a significant research group at the University of Pennsylvania. His team made substantial advancements in delivering genes to cells, a crucial development for therapies like Novartis’ Zolgensma, used to treat spinal muscular atrophy.

Despite these advancements, many companies have abandoned the development of rare disease treatments due to the high costs and commercial uncertainties involved. This trend led Wilson to realize that the traditional model for developing these therapies had reached its limit. Consequently, the University of Pennsylvania approved the spinout of Wilson’s research group, resulting in the formation of GemmaBio.

Initially, GemmaBio is focusing on eight therapies, three of which are licensed from Passage Bio, a company Wilson co-founded in 2018. GemmaBio paid $10 million upfront for these licenses and has agreed to additional milestone payments. Wilson believes GemmaBio’s global approach will maximize patient reach, especially in countries that stand to benefit the most from these treatments.

One of the diseases GemmaBio is targeting is GM1 gangliosidosis, a fatal disorder that is ten times more prevalent in Brazil than in other parts of the world. This higher prevalence makes Brazil an ideal location for conducting clinical trials. The partnership with Fiocruz, a division of Brazil’s Ministry of Health, is crucial. Fiocruz has significant experience in biomanufacturing, having partnered with AstraZeneca to produce the Covid-19 vaccine locally and worked on manufacturing CAR-T therapies domestically.

Fiocruz’s $100 million funding will support clinical trials and manufacturing efforts, with the goal of securing approval for the six therapies within Brazil and potentially other countries. Wilson sees Brazil as a potential gateway to broader access throughout Latin America.

In return for its financial support, Fiocruz has the option to license the therapies for the Brazilian public health system. Unlike many drugmakers who prioritize FDA clearance to expedite approvals in other countries, Wilson is encouraged by an FDA pilot program aiming to establish uniform standards across nations.

GemmaBio’s mission is to make gene therapies accessible globally, a sentiment echoed by researchers and advocates who believe that drug developers must consider the global market and the populations most in need when developing these treatments.

Wilson’s journey in gene therapy has seen significant highs and lows, from the tragic death of Jesse Gelsinger in 1999 to leading the resurgence of the field with new viral vectors designed for gene therapy. GemmaBio’s efforts are being supported by rare disease advocates who have seen many companies exit the space.

GemmaBio aims to overcome the challenges that have hindered previous gene therapy efforts by focusing on diseases with clearer regulatory paths and benefiting from the FDA’s openness to accelerated approvals. Additionally, Wilson has founded Franklin Biolabs, a contract research organization specializing in viral vectors for gene therapies, which has received funding from Savanne Life Sciences.

As GemmaBio continues its mission, Wilson hopes to secure more government partnerships and further funding, emphasizing that there are innovative ways for the industry to succeed.