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Transforming Personalized Medicine Globally

The Critical Path Institute® (C-Path) made a significant announcement on March 27, 2026, introducing its latest initiative, "One to Millions". This ambitious public-private partnership seeks to scale advanced therapies, ultimately striving to improve patient access to innovative treatments worldwide. The global initiative aims to bridge the existing gap between the rapid advancements in personalized medicine and the regulatory frameworks that currently govern the approval and reimbursement processes.

Over recent years, breakthroughs in therapeutic technologies, such as antisense oligonucleotides, genome editing, gene therapies, and RNA-based treatments, have enabled the design of precise interventions tailored to very small patient populations—or sometimes even to individual patients. However, regulatory and reimbursement systems have lagged behind the swift pace of these innovations, resulting in delays and disparities in access to life-saving therapies.

To address this critical bottleneck, the "One to Millions" initiative will leverage the Plausible Mechanism Framework established by the U.S. Food and Drug Administration (FDA) and the Rare Disease Evidence Principles. This framework will be supported by C-Path's centralized data platform, which meets regulatory-grade quality standards, fostering the evidence collection necessary for regulatory decision-making and creating scalable development pathways.

Klaus Romero, CEO of Critical Path Institute, expressed the groundbreaking nature of this initiative: "Words cannot fully capture how momentous this is for changing patient lives and realizing a long-awaited innovative vision. The 'One to Millions' initiative, designed to scale personalized therapies for a broader population, is a partnership only C-Path could initiate. It encompasses a regulatory-compliant centralized data platform, a unique pre-competitive research environment that captures the entire ecosystem, integration of preclinical and clinical data, and practical evidence frameworks. There has never been an initiative quite like this."

The platform's modernized approach ensures consistency and reliability within advanced therapeutic technologies. Standardization of manufacturing and quality control processes directly enhances efficiency and reduces the high costs typically associated with small-scale drug development. By creating new therapies utilizing existing frameworks, developers can build on prior knowledge, enabling regulators to focus solely on novel elements instead of reassessing the entire foundation anew.

Julia Vitarello, founder of Mila's Miracle Foundation and co-founder of the N=1 Collaborative, emphasized the urgent need for a coordinated approach to rectify flaws in the current system. "We are at an incredibly exciting moment in genetics. Today, we possess scientific knowledge capable of helping a vast number of children suffering from severe, rare diseases that profoundly impact their lives. However, our access system is not designed for the thousands of genetic disorders, each affecting small populations. We look forward to collaborating with regulatory agencies to shift from approving one drug for one disease to processes that can operate across multiple diseases. This transformation could be groundbreaking for millions of patients, but it will only succeed if we ensure continuous learning from these therapies through systematic data collection and sharing to develop safer and more effective drugs."

The integration of data collection post-approval into development processes serves as a pivotal element of this initiative. Incorporating long-term registries directly into the operational framework ensures that the data used in regulatory decision-making also supports payer assessments regarding sustainability, safety, and efficacy. Integrating information from across the ecosystem helps to prevent redundancy and accelerates knowledge expansion. In this context, Janet Woodcock, a long-time director at CDER and former acting commissioner of the FDA, remarked, "New technologies allow potential corrections to the root causes of debilitating monogenic diseases. However, progress may be stalled when there is a lack of data for joint analysis and knowledge expansion. Regulatory requirements remain excessively conservative without data to support this. We should aim for rapid knowledge flow and flexible development in this new field through shared experiences. Our patients deserve this."

The compilation of existing preclinical, translational, and clinical data will streamline toxicology studies and dose selection. Amassing solid data maximizes the usefulness of alternative methods, reducing the need for animal testing while simultaneously fostering a continuous model of knowledge development and confirmation of findings.

"This represents a crucial new pillar in the field of interventional genetics, delivering a long-missing element in terms of approval and reimbursement while closing the loop initiated by the FDA's guidelines on personalized antisense therapies from 2021," stated Timothy Yu from Boston Children's Hospital and co-founder of the N=1 Collaborative. "By systematically using the modularity of these frameworks, data can be applied across different therapies targeting various genetic variants without needing to restart the regulatory process from scratch for each mutation. This indicates a future of 'plug-and-play' genetics; however, such a system cannot emerge in isolation. Developing therapies that truly add value will require a collective expansion of knowledge through broad data sharing, where each breakthrough supports the next."

Sarah Glass, operations director at the n-Lorem Foundation, shared insights on the success of their solid and scalable process for delivering personalized ASO therapies to patients with ultra-rare diseases. "So far, we have identified and developed over 25 such therapies, enabling treatment for more than 45 patients. Many of these new drugs can be used to help additional individuals, and we are committed to reaching them. The clinical benefits we observe suggest substantial potential to address some challenges faced by the ultra-rare disease patient community, increasing the availability of these therapies and developing commercial solutions for broader accessibility. We are thrilled to join the 'One to Millions' initiative and look forward to contributing insights gained through our efforts."

Participants in this initiative include the n-Lorem Foundation, Mila's Miracle Foundation, and the N=1 Collaborative, with more organizations joining as founding members. For further information, visit c-path.org/programs/one-to-millions or contact [email protected]. Additionally, viewers can attend the C-Path webinar titled "Transforming Drug Development in Precision Medicine - A Practical Path Beginning with Evidence Gathering" and register for the upcoming online meeting on Wednesday, April 8, at 12:00 PM ET, titled "Scalable Approaches to Benefit-Risk Assessment from the Patient Perspective: Defining Patient-Centered Drug Development in Precision Medicine."

About Critical Path Institute

Founded in 2005 as a public-private partnership in response to the Critical Path Initiative by the U.S. FDA, the Critical Path Institute (C-Path) operates with the mission to collaborate towards the advancement of better treatment methods for people worldwide. C-Path is recognized globally as a pioneer in projects that speed up drug development processes. The organization has formed numerous international consortia, programs, and initiatives currently involving over 1,600 scientists and representatives from regulatory agencies, academia, patient organizations, disease foundations, pharmaceutical, and biotechnology companies. Headquartered in Tucson, Arizona, C-Path Europe is based in Amsterdam, the Netherlands, with local offices operating globally. More information is available at c-path.org. In the United States, Critical Path Institute receives support from the FDA and is funded 56% by FDA/HHS, totaling $23,740,424, and 44% from non-government sources totaling $18,881,611. The content provided is authored and does not represent the official views of the FDA/HHS or the U.S. government or indicate endorsement thereof.