A Second Chance for Cures: AI Marketplace to Rescue Shelved Gene Therapies

MILWAUKEE and CAMBRIDGE, Mass. – January 07, 2026 – A groundbreaking partnership announced today aims to create a lifeline for hundreds of potentially life-saving therapies for rare diseases that have been abandoned by the biopharmaceutical industry. The American Society of Gene & Cell Therapy (ASGCT) and the non-profit Orphan Therapeutics Accelerator (OTXL) are joining forces to launch CGTxchange, a first-of-its-kind marketplace designed to rescue and advance stalled cell and gene therapies (CGTs).

The new venture directly confronts a growing crisis in drug development: promising treatments, many of which have already shown clear benefits to patients in clinical trials, are being "deprioritized" and left on corporate shelves. The reason is not a lack of scientific merit, but a harsh economic reality. This initiative seeks to build a new bridge over the commercial "valley of death" for therapies targeting ultra-rare conditions, offering new hope to patient communities long overlooked by traditional market forces.

The Growing Graveyard of Promising Therapies

In recent years, a confluence of policy changes and economic pressures has led to a difficult reality within the biotech sector. The high cost of development combined with the small patient populations for ultra-rare diseases—conditions often affecting only a few thousand people worldwide—creates a challenging business case. While the science behind a gene therapy may be revolutionary, the projected return on investment often fails to meet the high thresholds set by traditional venture capital and large pharmaceutical companies.

"At ASGCT, we have observed that a host of cell and gene therapies are being set aside not because they lack clinical merit, but rather due to the challenges of commercializing these therapies once approved," said David Barrett, CEO of ASGCT.

This has created a paradox where innovation outpaces commercial viability, leaving a graveyard of shelved assets. Industry analyses suggest hundreds of such programs exist, representing immense sunk costs in research and, more importantly, lost opportunities for patients. With over 7,000 identified rare diseases and effective treatments available for only about 5%, the need for alternative development pathways is more urgent than ever. CGTxchange is being positioned as a direct response to this market failure, aiming to ensure that a therapy’s potential to save lives, not just its profit potential, dictates its future.

How AI Becomes the Matchmaker

At the heart of the CGTxchange initiative is a sophisticated, AI-enabled platform developed by OTXL. This technology will serve as the engine for the clearinghouse, systematically identifying, analyzing, and matchmaking stalled therapies with new potential sponsors. The platform is designed to transform a chaotic and opaque problem into a structured, data-driven solution.

Here’s how it will work: Information on deprioritized CGT assets will be uploaded into a secure, searchable database. The AI will then analyze the complex data—spanning preclinical results, clinical trial data, manufacturing processes, and intellectual property—to generate comprehensive profiles and risk-based assessments for each therapy. This automates a due diligence process that would otherwise be prohibitively time-consuming and resource-intensive.

The platform's most critical function is its ability to act as an intelligent matchmaker. By understanding the specific needs of a therapy and the unique capabilities, risk tolerance, and mission of potential partners—be they academic institutions, other non-profits, or mission-aligned investment funds—the AI can facilitate connections that would likely never occur otherwise.

"Typical venture capital and biopharma expectations for returns are set well above what most CGTs for ultra-rare diseases can meet in light of recent policy and market shifts," explained Craig Martin, CEO of OTXL. "Yet many of these shelved therapies can still offer meaningful returns to the right partners, as well as tremendous benefits to patients. This partnership allows us to surface high-quality, clinical-stage CGT programs to investors and organizations whose missions, timelines and objectives align with what these therapies can deliver."

Forging a New, Sustainable Path to Patients

Beyond the technology, CGTxchange represents a fundamental disruption to the standard drug development model. By operating outside the constraints of traditional for-profit structures, the venture can champion therapies based on clinical need and scientific promise. This new ecosystem relies on a different definition of value and a more flexible approach to funding and regulation.

The funding model will draw from a diverse pool of sources, including philanthropy, government grants, and impact investors who are motivated by more than just financial returns. ASGCT will leverage its vast global network of scientists, clinicians, donors, and industry leaders to build support and find sponsors. OTXL, in turn, will utilize alternative financing structures, such as success-based agreements, to lower the upfront cost of acquiring and advancing these programs. The goal is to create a self-sustaining model where any proceeds from successful therapies are reinvested to rescue the next wave of promising treatments.

This new pathway is also supported by existing regulatory frameworks designed to encourage rare disease research. Rescued therapies are prime candidates for programs like the FDA’s Orphan Drug Designation, which provides powerful incentives including seven years of market exclusivity and fee waivers. Furthermore, therapies that have already demonstrated patient benefit can leverage accelerated approval pathways, which allow for earlier market access based on surrogate endpoints while confirmatory studies are completed. This regulatory flexibility is critical for making development feasible for diseases with very small patient populations.

By combining ASGCT's deep scientific and community expertise with OTXL's innovative platform and non-profit business model, the partnership is creating an entirely new infrastructure for hope. ASGCT and OTXL plan to begin development of the CGTxchange platform in early 2026, with a targeted launch and rollout by the middle of the year, signaling a swift move to turn this vision into a reality for patients in need.