As noted in a recent article published in Inside Precision Medicine, in 1992 gene therapy was often discussed but seldom practiced.  When Jim Wilson MD, PhD attended Penn State, gene therapy was considered a scientific dream.  Simply stated, one infusion of a viral vector transporting a normal gene to cure one genetic disease did not seem feasible.

Yet even then, Wilson was troubled by a question that had no precedent: how do you price a one-time cure?

Wilson was fascinated with the idea of pricing a therapy which would be given to a patient once but would last a lifetime. Wilson’s determination formed the trajectory of his career.

Now, Wilson is launching Rare Therapeutics, a spin-out from one of his earlier companies. He is rewriting how gene therapies are created and how they reach patients.  The primary goal of his new company, RareTx, is to enhance the affordability and the quality of gene therapy products.

At the time Wilson began working at Penn State, adenovirus was the preferred vector. It was used to carry DNA segments into a host cell. Yet it was often rejected by a patient’s immune system.

The Penn Lab eventually discovered a family of AAVs including AAV9 which is currently one of the most widely used vectors.  The discovery prompted a licensing arrangement with GlaxoSmithKline (GSK) that provided resources but proved to be temporary due to GSK’s varied corporate priorities.

Wilson was instrumental in arranging sub-licensing to a new startup, but as part of that arrangement he had to agree to personally participate in management of the new company.

Wilson agreed and in 2009 REGENEXBIO was formed and became one of the few pure plays in the industry. Pure play describes a company that specializes in only one product rather than a diversified conglomerate.

Wilson’s company eventually grew to be unique as a biotech inside a university.  Most labs focus on discovery while Wilson’ lab focused on taking ideas from proof of concept to regulatory submission.  During its best season, there were over 400 people employed by the group which eventually saw funds and investments begin to dry up.  The programs evolved into spin-out companies. With the help of Wilson’s lab, eight companies were started, including REGENXBIO.

In 2020, when the flow of investor participation fell considerably, Wilson and two partners co-founded ICLM, a non-profit focusing on developing gene therapies, but it was difficult to rely on philanthropy.  Wilson decided that the only solution to solvency was starting his own company.

The Plan: Wilson et al

The leadership at Penn supported the plan as they shared Wilson’s imperative to translate science into medicine.  Wilson founded GEMMABIO in 2024 that included a portfolio of rare-disease programs falling into two categories:

• One category of licensed programs originated from Penn, while others were licensed from companies Wilson had started.
• One group of companies focused on large, rare diseases that are attractive as pharmaceutical partnerships while others focused on various orphan diseases.

In 2025, Wilson moved a step forward with his strategy.

Stay tuned for Part 2.