New Initiative Goals to Revive Stalled Uncommon Illness Therapies

Cell and gene therapies (CGTs) have shown extraordinary promise, exemplified by baby KJ, who was the first person in the world to receive a personalized, in vivo CRISPR gene-editing therapy for a fatal genetic disorder. Yet despite scientific breakthroughs, companies have withdrawn several CGTs from the market or discontinued them during clinical development.

For many in the rare disease community, including patients, families, clinicians, and advocates, this trend has been deeply discouraging. David Barrett, the chief executive officer of the American Society of Gene & Cell Therapy (ASGCT), said, “It was not because of a lack of scientific or clinical success, but because of the challenges with their commercial modeling.”

Such factors include concerns about the high cost of development, manufacturing, and making treatments accessible to patients, the loss of policy-based market incentives, and regulatory requirements. These challenges rendered many CGTs as not commercially viable by traditional for-profit biopharma companies and investors, despite the rare disease therapies’ clear benefits to patients in trials.

This spurred a series of conversations with stakeholders and external groups to better understand the underlying barriers and explore potential solutions. Among those groups was the Orphan Therapeutics Accelerator (OTXL), a nonprofit biotechnology organization dedicated to completing development and expanding access to stalled rare, or “orphan,” disease treatments.

Craig Martin, chief executive officer of OTXL, observed a noticeable pullback in investment and long-term commitment to certain rare disease programs—even among companies with longstanding histories in the space. A central question emerged: How could the field identify new clinical sponsors beyond the traditional venture-backed biotech model or large pharmaceutical companies? Many CGT programs, the leaders concluded, required a fundamentally different pathway to secure continued development support.

In response, OTXL developed AI-based tools to evaluate programs at scale, conduct predictive modeling, assess resource requirements, and estimate timelines for advancement. “But as we got into it, we realized, well, these are tools that we’re using for [OXTL], but they are also tools that could be very useful at a broader scale,” said Martin. This aligned with ASGCT’s mission to revive deprioritized CGTs and so the two organizations decided to combine forces to build out their vision.

In January 2026, ASGCT and OTXL announced a partnership to establish CGTxchange, a jointly owned entity that will serve as a clearinghouse and marketplace for deprioritized CGTs. While the platform is currently in development, Martin described the clearinghouse as “a Zillow for shelved gene and cell therapies.” As for the types of CGTs, Bennett remarked, “We would imagine that the therapies that we were looking to fill the clearinghouse with would be across all developmental stages, [though] I envision many of them would be in clinical stages.” Martin added that the team is aiming for near-term impact at scale by focusing on clinical stage assets.

Then, the marketplace serves as a space for potential partners and funders to peruse listed programs as well as provide them with access to other resources, such as a clinical development network willing to work at reduced or deferred costs. This approach expands the potential to secure funding and sponsors.

“This new effort by ASCGT and [OXTL] is really exciting,” said Phillip (“PJ”) Brooks, the acting director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences, who was not involved in the initiative.

The success of baby KJ brought more attention to the CGT field, Brooks remarked. “It was a wonderful thing we didn’t anticipate. It gave the whole field a shot in the arm.” Now, with this initiative, Brooks expressed hope that the collaboration will attract new types of sponsors capable of advancing deprioritized therapies and ultimately delivering them to patients.

Harold Trent Spencer, the director of the Gene and Cell Therapy Program at Emory University, was also not involved in the partnership but understood the challenges firsthand. Over the years, his team developed several CGTs, primarily targeting pediatric conditions, including a treatment for hemophilia A.

The therapy used genetically engineered hematopoietic stem cells, but the financial model to develop and deliver it at an affordable price point just wasn’t there. Despite it all, Spencer remarked, “There are opportunities abound right now. I’m glad somebody’s thinking about [this challenge]…to try and move the needle on some of these programs.”

Spencer also remarked that part of the opportunity lies in the scale. Manufacturing demands for many orphan diseases are far smaller than for more common conditions. “A lot of these diseases don’t require large production runs,” he explained. For example, he remarked that instead of 60 liters of product, it may be possible to make six liters to treat 10 people a year. That smaller scale, he suggested, creates opportunities for innovative development and manufacturing models.

Brooks added that as the field searches for new financial and operational models, the science itself is evolving beyond the traditional paradigm of one drug for one disease. Emerging technologies, including adeno-associated virus vectors and ex vivo gene therapies, are increasingly being developed as adaptable platforms, potentially streamlining development and lowering costs.

Echoing the optimism, Bennett emphasized the shared goal across the field: ensuring that promising therapies do not languish. “I don’t think that anybody really wants to see [the therapies] stall,” said Bennett. “We want to see them succeed. We want to see patients have access eventually to safe and effective cell and gene therapies. And it’s bringing forward a lot of individuals and organizations that are interested in cooperating.” ASGCT and OTXL envision a mid-year launch and rollout in 2026.