Waukesha-based professional society looks to support rare disease treatments
The Waukesha-headquartered American Society of Gene & Cell Therapy is working with another nonprofit to connect investors with rare disease treatments that have stalled in development because they’re not considered commercially viable.
“Because of our position and our mission, there has to be something that we can do to catalyze the field around this and find some solutions,” ASGCT CEO David Barrett told Wisconsin Health News in a recent interview.
ASGCT represents about 6,500 researchers, scientists, physicians and other professionals working in the field of gene and cell therapy, which involves the use of genetic material or the transfer of cells to treat and prevent disease.
ASGCT will work with the Massachusetts-based Orphan Therapeutics Accelerator to develop a database of under-development therapies for matching with investors, expanding their potential to secure funding. CGTxchange will roll out this year.
While other associations have headquarters in Washington, D.C. and Chicago, ASGCT came to southeast Wisconsin more than 20 years ago to be managed through a consulting group in the area. It decided to stay, and the majority of its employees live within commutable distance of its office.
Barrett spoke about how CGTxchange will work, the state of the cell and gene therapy industry and what’s next for ASGCT.
Edited excerpts from the interview are below.
WHN: How do you plan to connect sponsors with these therapies?
Barrett: There are groups that very much want to put programs into the clearinghouse. This is seen as a potential benefit to those groups that are looking for additional alternative investors and for ways that they can facilitate the advancement of a clinical program in a way that might work for other businesses, but not those that have deprioritized or are looking at shelving these. Collecting those individuals and assembling that data is absolutely something that we have the network, the expertise and the ability to perform. It’s not an easy thing to do. But it is something that we’re well suited to do and that we’re looking forward to taking on right now.
On the marketplace side, that’s an area where we’re encouraged by what we learned in our efforts up to the point of making this announcement. There is a constellation of individuals and organizations that are invested and want to become more active in this space. We are expecting to be able to identify non-traditional investors and sponsors, or traditional sponsors that just might not be engaged in this space. We think about nonprofits. We think about patient advocacy organizations. There are nonprofit biotechs. There are academic institutions. There are other charitable foundations that could be interested in this sort of work. There’s a lot of opportunity out there.
WHN: What’s your assessment of the cell and gene therapy field?
Barrett: The Alliance for Regenerative Medicine just released their state of the cell and gene therapy industry … This is an environment where we continue to see scientific advancement and refinement of the underlying technology. We continue to see an investment space that is more discerning, but still very active. We’re also seeing a commercial sector that has learned how to respond to some of the initial challenges, including things like manufacturing bottlenecks, high costs, coverage and reimbursement issues on the back end, and ensuring access for patients.
Over the course of the last several years, by being able to refine this and understand the industry much better, the commercial space is beginning to rebound. There’s a lot of optimism for cell and gene therapy in 2026, making this a really exciting time.
The Food and Drug Administration has started to make big statements in the cell and gene therapy space about their support for making safe and efficacious cell and gene therapies available … We’re seeing a lot of effort by the administration right now to allow for regulatory flexibility, particularly for cell and gene therapies for ultra-rare disorders.
WHN: What’s next for ASGCT?
Barrett: There are a number of clinical programs that we expect over the course of this year and next year to have (Prescription Drug User Fee Act) dates with FDA and hopefully become approved gene therapies.
We are interested in helping to support the clinical implementation of cell and gene therapy. Now that there are a couple dozen cell and gene therapies that are approved in the United States, there’s a growing need to be able to provide these in hospitals and even for some in outpatient settings. Building those clinical support networks is something that ASGCT is going to be focused on.
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