Global initiative seeks to fast-track personalized therapies for rare diseases

Written by Emma Hall (Editor)
Data processing and interpretation Drug development News Regulated bioanalysis

A new international effort aims to revamp how highly individualized treatments are developed, approved and delivered to patients with rare diseases.

The Critical Path Institute (C-Path; AZ, USA) has launched “One to Millions”, a global public-private collaboration designed to accelerate the development of advanced therapies for rare and ultra-rare diseases. Announced on 26 March 2026, the initiative brings together regulators, researchers and patient groups to address barriers slowing access to innovative treatments, aiming to modernize outdated systems and enable scalable solutions for very small patient populations.

Breakthroughs in technologies like antisense oligonucleotides, gene therapies and RNA-based therapies are facilitating increasingly tailored treatments for small groups and even individuals. However, regulatory and reimbursement systems still rely on traditional models built for large populations, often delaying access for those in urgent need.

One to Millions will expand on existing regulatory frameworks, such as the US FDA’s (MD, USA) Plausible Mechanism Framework and Rare Disease Evidence Principles, by using C-Path’s centralized data platform to generate robust evidence for decision-making. The initiative promotes standardized development methods, shared data and a collaborative environment spanning preclinical research through to patient outcomes. This approach could reduce duplication, lower costs and allow regulators to focus on novel treatment components rather than reassessing entire systems.

“Words cannot fully express how pivotal this moment is for the transformation of lives and the long-awaited materialization of an innovative vision,” explained Klaus Romero, C-Path’s CEO. “Built to make individualized therapies scalable for even more people, One to Millions is a partnership that only C-Path could convene. It features a centralized, regulatory-ready data platform; a unique precompetitive environment across the entire ecosystem; integrated preclinical, translational, clinical and patient-level outcomes; actionable evidentiary frameworks to optimize the evaluation of efficacy and safety; and the ability to generate the regulatory-grade tools needed to establish a continuous learn-and-confirm process. There is simply no other initiative like it.”

“This is a very exciting time in genetics. Today, we have the science to help a massive number of children with severe, life-altering rare diseases, but our system of access wasn’t designed for thousands of genetic diseases, each affecting small populations,” commented Julia Vitarello, Founder of Mila’s Miracle Foundation (CO, USA).

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Consolidating preclinical, translational and clinical data sources can enhance toxicology and dose selection processes, accelerate approvals, improve safety monitoring and support payer decisions related to durability and effectiveness. Collecting this comprehensive data can also reduce excessive dependence on animal testing and encourage a continuous cycle of learning and validation. If successful, it may establish a “learn-and-adapt” model where each new therapy strengthens the system for the next.

“By effectively doubling down on modularity, this framework enables developers to leverage data across therapies targeting different genetic variants without restarting the regulatory process for each mutation. It points toward a future of ‘plug-and-play’ genetics, but such a system cannot be built in isolation. Advancing cures that are truly greater than the sum of their parts will require shared learning through robust data sharing, where each breakthrough informs the next,” commented Timothy Yu, Co-Founder of the N=1 Collaborative (MA, USA).

Click here to view the press release