BioCentury Article: Critical Path Institute Aims to Bring Scale to Individualized Medicines
FDA’s plausible mechanism framework aims to accelerate and expand drug development for patients...
One to Millions
Overview
Founded in 2026, One to Millions advances therapies from individualized breakthroughs to scalable global solutions.
The Problem
Science has moved faster than the systems designed to evaluate it.
Breakthrough modalities like antisense oligonucleotides, gene editing, RNA therapies, and cell-based therapies now make it possible to design treatments for a single patient. Yet the regulatory and reimbursement systems governing development were built for population-scale medicines.
The result: a growing gap between what science can achieve and what the system can deliver.
The Solution
One to Millions is a new initiative led by Critical Path Institute to modernize how advanced therapies are developed, evaluated, and reimbursed.
Anchored in FDA’s evolving frameworks, and aligned with emerging global regulatory science approaches, One to Millions is built on a centralized, regulatory-grade data platform that integrates:
- Preclinical, clinical, and real-world evidence
- Manufacturing standards and quality systems
- Quantitative regulatory tools and biomarkers
- Longitudinal patient registries
- Early and sustained payer engagement
The Impact
A new regulatory framework that enables therapies to scale predictably to move from one patient, to many, to millions. This initiative is designed to create a scientifically grounded and economically sustainable pathway, trusted by regulators, while preserving the rigor and credibility essential to therapeutic innovation.
FAQs
What is One to Millions?
One to Millions is a global initiative designed to make highly personalized medicines, like therapies tailored to a single patient, scalable and accessible to many more people. Today, science allows researchers to create treatments for individuals based on their unique genetic makeup, but there’s no practical system to develop and deliver these therapies at scale. One to Millions aims to change that by building a collaborative framework where data, tools, and expertise are shared across organizations. This allows each individual treatment to contribute to a larger body of knowledge, so future patients can benefit faster and more efficiently, turning one-off breakthroughs into a repeatable, scalable model. Read more about One to Millions from this informative BioCentury article, Critical Path Institute Aims to Bring Scale to Individualized Medicines, here.
Why aren’t individualized therapies already widely available?
While technologies like ASOs and gene editing can be tailored to individual patients, the development and regulatory systems are still built for large populations and traditional clinical trials. This creates a fundamental mismatch that prevents scalable adoption.
What is One to Millions aiming to achieve?
The initiative aims to create a scalable framework for developing and evaluating individualized therapies by integrating data, advancing analytics, and enabling collaboration across stakeholders.
How is this different from current approaches?
Current approaches are largely one-off and siloed. One to Millions creates a continuous learning system, where each therapy contributes to a growing body of evidence that informs future development.
What is meant by a “process-based” regulatory model?
Instead of evaluating each therapy from scratch, regulators can assess therapies within a validated framework. This allows for more efficient review, focusing on what is novel rather than revalidating known components.
How will data sharing work?
Data is shared within a secure, precompetitive environment managed by C-Path. Companies contribute data, and C-Path develops tools based on aggregated insights. Companies benefit from these tools without accessing each other’s data.
Why start with ASOs and gene editing?
These technologies are well-suited for individualized applications and have sufficient scientific and clinical grounding to serve as initial use cases for building scalable frameworks.
How does this benefit patients directly?
It creates a pathway where patients no longer depend on isolated, resource-intensive efforts. Instead, each patient’s experience contributes to a system that accelerates future therapies.
What does this mean for pharmaceutical companies?
It enables a viable business model by reducing uncertainty, shortening development timelines, and allowing companies to build on shared knowledge rather than starting from scratch.
Is artificial intelligence central to this initiative?
AI is an important tool for analyzing complex datasets, but it is part of a broader toolkit that includes multiple analytical and scientific approaches.
What does success look like in the near term?
- Establishing a collaborative framework
- Launching initial data integration efforts
- Engaging founding industry partners
- Beginning development of shared tools and standards
What is the long-term vision?
A healthcare ecosystem where individualized therapies can be developed and delivered routinely, supported by a scalable, data-driven, and globally aligned framework.
Are there any resources available to learn more?
Yes, you can view C-Path’s initial webinar on this initiative, here on our YouTube channel.
Partner with Us
Interested in partnering with One to Millions?
Contact us to learn how organizations can contribute to shaping the future of advanced therapy development. The era of individualized medicine has arrived and C-Path is building the system needed to deliver it at scale.
