Joseph La Barge On The Challenges Of Advancing Gene Therapy, Finding The Right Support

March 28, 2024

By Irene Yeh 

March 28, 2024 | Joseph La Barge, CEO of Apertura Gene Therapy, began his career raising capital and building biotech companies. At Spark Therapeutics he served as both Chief Legal and Chief Business Officer overseeing business development, patient advocacy, quality assurance, legal affairs, compliance, finance, public policy, and global pricing and reimbursement. In 2019, La Barge led the negotiation of Spark’s acquisition by Roche and stayed until 2022. Today he leads Apertura Gene Therapy, helping his team find effective treatments for patients through gene expression and capsid engineering. In the newest episode of Trends from the Trenches, host Stan Gloss speaks with La Barge about his unique perspective on growing biotech businesses. La Barge delves into the challenges he and his team face in gene therapy advancement and how a company—especially one just starting out—can pave the path to successful research. 

Advancing Research Against Adversity 

Gene therapy uses the capsid of a virus to package and deliver a genetic payload to a specific organ or tissue to treat a disease. At Apertura, there have been developments in effective treatments, including developments in capsid engineering and a novel AAV virus that targets the human transferrin receptor, which is highly expressed on the blood-brain barrier, a semi-permeable barrier that blocks out external matter. 

Researchers are still working on accessing hard-to-reach tissues, says La Barge, including the kidneys and muscles, and crossing the blood-brain barrier. They are also working to fine tune gene expression. “There are a lot of opportunities to improve the ways in which the genetic payloads that we're delivering can be expressed,” says La Barge. “That is, trying to tune expression so that it is higher or that it is lower or that it hits a kind of a Goldilocks zone where you're not giving too much and you're not getting too little.” 

Finding The Right Partner 

La Barge emphasizes the importance of finding the right partners to support one’s studies. Having raised capital for companies himself, La Barge explains that a committed partner who can secure funding prevents researchers from “worrying day-to-day, week-to-week” over having enough finances to advance research. Additionally, partners should share the same goals and be as passionate as the team carrying out the studies. 

“Having a company—an investor—like Deerfield, who was committed to the therapeutic space [and] who's committed to the modality and has capital to deploy, can allow people like me and the people that I hire on my team to focus on advancing the science and developing programs that can ultimately get to the clinic,” says La Barge about Apertura’s partnership with Deerfield. “They are committed to the gene therapy space. They have a large amount of capital that they are able to tap and deploy, which means you have a stable investor behind you.” 

However, it is important to keep in mind that even the most committed partners and largest capitals are not finite. Furthermore, patients are still waiting for solutions and discoveries that will provide the treatment they need and deserve. Researchers should not let perfection be the enemy of the good, warns La Barge. At some point, it is more important to deem something good enough to keep moving forward. 

To hear La Barge discuss more on gene therapy advancements, bringing novel science to the market, and more, be sure to listen to this month’s episode of Trends from the Trenches here