Hundreds of cell and gene therapies (CGTs) that showed clinical promise have been shelved in recent years — not because they failed patients, but because they failed to meet traditional commercial return expectations. A new joint venture announced January 7, 2026, is attempting to create an alternative pathway for those programs.
The American Society of Gene and Cell Therapy (ASGCT) and the Orphan Therapeutics Accelerator (OTXL) announced the launch of CGTxchange, a jointly owned clearinghouse and marketplace designed to identify, evaluate, and reconnect deprioritized clinical-stage CGTs with investors and development partners willing to take them on.
The backdrop is a familiar tension in rare disease drug development. Policy and economic shifts — including changes to how orphan drugs are treated under Medicare price negotiation rules — have led biopharma companies to pull back from programs that still carry meaningful clinical data but limited commercial upside. For ultra-rare diseases in particular, the math rarely works under conventional venture or biopharma return models, even when the underlying science holds up. The result is a growing pool of clinical-stage assets that are neither failures nor successes — they simply ran out of institutional will to move forward.
The challenge is compounded by how fragmented the rare disease development landscape already is. Programs are often paused mid-development, with data scattered across sponsors who have moved on, making it difficult for potential new partners to even identify what is available, let alone evaluate it efficiently.
How CGTxchange works
The platform is built around OTXL's AI-enabled infrastructure. Detailed asset information is uploaded into a secure data room, where it is analyzed to generate standardized profile pages and risk scores across areas including preclinical and clinical evidence, development pathway, regulatory readiness, and market attractiveness. A first layer of information is broadly accessible to qualified investors and partners; more sensitive data and analyses are shared selectively by asset listers in response to individual requests.
The intent is to reduce the due diligence burden on both sides of a potential deal and improve the odds of matching an asset with a partner whose mission, timeline, and return expectations are actually compatible with what a given therapy can realistically deliver. That pool of potential partners extends beyond traditional biopharma — the model is explicitly designed to accommodate nonprofits and academic institutions as well.
"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," Craig Martin, CEO and cofounder of OTXL, said in the press release. "Yet many of these shelved therapies can still offer meaningful returns to the right partners, as well as tremendous benefits to patients."
For a program to be considered for inclusion, it must be a clinical-stage asset for a lower-prevalence rare disease, available for acquisition, licensing, or financing, and supported by robust safety and clinical data. Regulatory readiness, manufacturing complexity, and intellectual property position are also factored into the evaluation.
ASGCT brings the network side of the equation. The society has members in more than 60 countries and will work to engage its community of CGT leaders, donors, and investors to surface viable assets and build awareness around the platform. Professional societies occupy an unusual position in this kind of effort — they carry scientific credibility without the commercial pressures that shape how biopharma companies prioritize their pipelines, which makes them better positioned to advocate for programs that fall outside conventional development logic.
"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, nonprofits, or academic institutions — that are ready to take on the challenges of development and commercialization so patients may ultimately benefit."
Whether the model works in practice will depend on whether the marketplace can attract both quality assets and credible partners at sufficient scale. CGTxchange development began in early 2026, with a mid-year launch targeted. For drug developers working in the rare disease space, it represents a potential new channel — both for offloading programs that no longer fit a pipeline and for sourcing clinical-stage assets that might otherwise go dark entirely.
