Synthèse
The second track of our rare disease research grants program will support biotechnologists and early-stage biotechs working to accelerate drug development for rare diseases. Today, it takes one to two years to move from a confirmed genetic diagnosis to a treatment available to patients, with much of this time spent waiting in queues for certified manufacturing slots, running safety studies sequentially instead of in parallel, and hand-assembling the thousands of pages of chemistry and regulatory
The second track of our rare disease research grants program will support biotechnologists and early-stage biotechs working to accelerate drug development for rare diseases. Today, it takes one to two years to move from a confirmed genetic diagnosis to a treatment available to patients, with much of this time spent waiting in queues for certified manufacturing slots, running safety studies sequentially instead of in parallel, and hand-assembling the thousands of pages of chemistry and regulatory documentation required for in-patient testing. We think it is possible to radically compress phases of this process with Claude—in particular, by making it easier to complete documentation (for example, drafting and reviewing the regulatory dossier), but also by speeding up therapeutic strategy selection (for example, by analyzing whether a target is druggable across a suite of modalities, such as small molecules, antibodies, genetic medicines, and so on) and looking for shared mechanisms across individual genetic therapies, which could allow them to be approved under a single “basket trial” instead of requiring a separate IND for each patient. Although many aspects of drug development are difficult to expedite because of manufacturing constraints or safety testing, we believe much can be done to move more quickly. By granting API credits and Claude Science access to the many biotechnologists and startups working in this space, we hope to encourage the experiments necessary to explore and identify such solutions.
We also hope grantees will amplify and emulate the efforts of our other partners working in rare disease therapeutics.
Source originale
nejm.org
Track one: Scaling our basic science partnerships ↗
mondo.monarchinitiative.org · 19 juil.