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The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

17 Apr 2025

Abstract excerpt

Developing customized gene-targeting therapies for the millions of individuals affected by ultra-rare diseases globally requires breaking new ground in therapeutic and regulatory innovation. To address this need, the N=1 Collaborative (N1C) was established to unite academia, industry, patients, and regulators, building an open, shared ecosystem for personalized medicines. Initially focusing on antisense...

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PMID: 40277082
DOI: 10.1093/nar/gkaf346

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