Nonprofit Orphan Therapeutics Accelerator Announces Partnerships

Rare Daily Staff

The nonprofit Orphan Therapeutics Accelerator, which is focused on completing development and commercializing therapies for ultra-rare conditions, announced a series of collaborative partnerships that it said establish the foundation for a new development and commercialization pathway for these treatments.

The Orphan Therapeutics Accelerator was established in 2024 to overcome significant challenges facing ultra-rare disease drug development and commercialization in recent years, driven by shifting financial, market, and policy dynamics that have caused many promising experimental therapies to be abandoned or de-prioritized.

The nonprofit works to obtain rights to shelved therapeutic programs, advancing them efficiently and at low cost through its network of partners. It will provide commercial access through several potential pathways, including directly from academic or non-profit institutions, via spinouts under an affiliated LLC, or out-licensed to mission-aligned biotech partners.

“Many of us involved in this venture have been working in rare diseases for decades,” said Craig Martin, founder and CEO of the Orphan Therapeutics Accelerator. “We’ve seen the ebbs and flows and know that this recent stretch isn’t merely a ‘market correction,’ it’s a sea change that we need to radically adjust our thinking to confront.”

The partnerships include ones with rare disease-focused global biotech and pharmaceutical companies, a contract development and manufacturing organization, contract research organization, and AI-based platform providers.

Chiesi and BIAL have committed funding and will become the first founding members. They will help guide and support selection and late-stage development of a set of initial programs expected to be announced in the coming months. Funding from the two companies will be used to complete evaluations and secure and initiate work on a set of two to three lead programs.

Landmark Bio will help lower development and manufacturing costs, and Uncommon Cures will collaborate with Orphan Therapeutics Accelerator to streamline clinical trial processes. DVLP Medicines is providing an end-to-end drug development platform through an early access program, utilizing autonomous AI agents to perform complex analysis and generate plans, reports, and other tools for expert use. Vibe Bio will provide AI-based solutions for due diligence and analysis of potential therapeutic assets, enabling highly efficient clinical development, asset management, and other vital functions.

The Orphan Therapeutics Accelerator said these organizations will participate in Orphan ClinDevNet, a connected ecosystem of its partners that it will rely on for clinical- and commercial-stage services.

“The recent market dislocation and capital rotation out of biotech have prevented many rare disease research programs from moving forward,” said Giacomo Chiesi, head of Global Rare Diseases at Chiesi. “We believe rare diseases are a long-term societal responsibility and, with this collaboration, we commit to build partnerships across stakeholders to meet these newly emerging challenges of ultra-rare diseases.”