American Society of Gene & Cell Therapy and Orphan Therapeutics Accelerator Partner to Advance and Commercialize Promising Rare Disease Treatments
PR Newswire
MILWAUKEE and CAMBRIDGE, Mass., Jan. 7, 2026
First-of-its-kind venture to facilitate and catalyze development of deprioritized cell and gene therapies, supported by AI-enabled platform
MILWAUKEE and CAMBRIDGE, Mass., Jan. 7, 2026 /PRNewswire/ -- The American Society of Gene & Cell Therapy (ASGCT), the leading professional organization for the advancement of cell and gene therapies (CGTs), and the Orphan Therapeutics Accelerator (OTXL), a non-profit biotech focused on completing development and enabling access to stalled rare disease treatments, today announced a partnership to establish CGTxchange, a jointly owned entity that will serve as a clearinghouse and marketplace for deprioritized CGTs.
The new joint venture will address a growing and urgent challenge facing the CGT field: policy and economic shifts in recent years have led biopharma sponsors to halt development of hundreds of CGTs no longer considered commercially viable by traditional industry standards, including many that delivered clear benefits to patients in trials.
CGTxchange leverages the combined capabilities of the organizations in an unprecedented way to engage a broad array of potential partners and funders to efficiently identify, evaluate, finance, develop, and provide sustainable, reliable, and scalable access to potentially transformative CGTs for ultra-rare diseases.
"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. "In partnership with OTXL, we are creating an entirely new way to ensure these CGTs find their way to organizations – whether companies, non-profits or academic institutions – that are ready to take on the challenges of development and commercialization so patients may ultimately benefit."
Using OTXL's AI-based platform, CGT assets and related information can be input into a secure, searchable database and analyzed, generating listings and profiles of shelved therapies and providing risk-based assessments. From there, listed assets can be matched with prospective investors or partners in the marketplace, thereby expanding the potential to secure funding and new clinical sponsors.
"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," said 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."
Within the partnership, ASGCT will convene and engage its extensive network of CGT leaders, experts, donors and investors, while helping establish CGTxchange as the leading solution for currently 'pre-viable' CGT programs. OTXL will develop the platform for the clearinghouse and marketplace, contributing resources from its AI-based infrastructure and partner network to continue to build, adapt and scale the platform for CGT community adoption and use.
ASGCT and OTXL will initiate development of CGTxchange in early 2026, with a targeted mid-year launch and rollout.
About ASGCT
The American Society of Gene & Cell Therapy (ASGCT) is the premier professional organization for scientists, physicians, and patient advocates working to advance the understanding, development, and application of genetic and cellular therapies. With members in more than 60 countries worldwide, ASGCT is committed to the alleviation of human disease through its extensive educational offerings, active engagement in public policy, and a wide array of support resources.
About the Orphan Therapeutics Accelerator
The Orphan Therapeutics Accelerator (OTXL) is a non-profit biotech focused on obtaining, completing development, and enabling commercial access to promising clinical-stage therapies for ultra-rare conditions that have been shelved for financial or strategic reasons. OTXL utilizes alternative development and financing models to advance programs efficiently and sustainably, with proceeds reinvested to support additional rare disease therapies.
For further information, contact:
Sarah Kikkert, director of communications
American Society of Gene & Cell Therapy
skikkert@asgct.org
(262) 649-4914
SOURCE American Society of Gene & Cell Therapy and Orphan Therapeutics Accelerator
