The Critical Path Institute (C-Path) announced on March 28 the launch of its One to Millions program, a global public-private initiative aimed at accelerating the development and access of advanced therapies for very rare diseases. The program seeks to address regulatory and reimbursement challenges that currently limit patient access to personalized treatments.
This initiative is significant because recent advances in technologies such as genome editing, gene therapy, and RNA-based treatments have enabled targeted interventions for small patient groups. However, existing frameworks were designed for broader populations and are not well-suited for these new approaches. The One to Millions program aims to close this gap by providing scalable pathways for developing and approving these therapies.
Klaus Romero, M.D., MS, FCP, Chief Executive Officer of C-Path said: “Words cannot express the importance of this moment in transforming lives and realizing an innovative vision long awaited.” Romero explained that the partnership leverages a centralized data platform ready for regulatory use along with integrated preclinical, translational, clinical outcomes and actionable evidence frameworks needed to optimize safety assessments. “There is simply no other program like it,” he said.
Julia Vitarello, founder of Mila’s Miracle Foundation and co-founder of N=1 Collaborative added: “This is a very exciting time in genetics. Today we have science that can help many children with severe life-changing rare diseases but our access system was not designed for thousands of genetic diseases each affecting small populations.” She continued: “We are excited to work with regulators so we can move from approving one drug per disease at a time toward building processes that work across many diseases… This shift could be game-changing for millions of patients but will only succeed if we ensure continuous iterative learning through systematic data collection and sharing.”
Janet Woodcock, M.D., former director at CDER and acting FDA commissioner commented: “New technologies offer potential correction of root causes behind devastating monogenic diseases. But progress can stall—and regulatory requirements remain overly conservative—when information isn’t available collectively.” Woodcock stressed the need to share knowledge quickly so patients benefit from advances.
The collaborative effort includes organizations such as n-Lorem Foundation—which has developed over 25 personalized ASO drugs—and Mila’s Miracle Foundation. Sarah Glass, Ph.D., Chief Operating Officer at n-Lorem said: “With encouraging clinical benefits observed in our patients… there is significant momentum towards solving some challenges facing the ultra-rare disease community… We look forward to joining One to Millions.” Timothy Yu, M.D., Ph.D., also highlighted how this framework allows developers to leverage modularity without restarting regulatory processes each time.
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