• Definition of CMC framework for therapeutic oligonucleotides.
• Phase-appropriate overview of the CMC framework, spanning from IND-enabling studies to commercialization.
• Practical roadmap for navigating scientific and operational complexities through the oligonucleotide therapeutic lifecycle

The field of oligonucleotide therapeutics has gained significant momentum, underscored by the regulatory approval of multiple drugs that validate its clinical potential. However, to further enhance therapeutic efficacy, improve cellular uptake, increase stability, and enable delivery to previously inaccessible tissues, the development and integration of novel chemistries and molecular constructs are essential.

These innovations, while promising, introduce new challenges from a Chemistry, Manufacturing, and Controls (CMC) perspective. This talk will highlight key stages, technical hurdles, and strategic considerations that can help early-stage researchers and innovators anticipate and avoid common pitfalls, factors that can critically impact the success and progression of novel oligonucleotide therapies.

The presentation will provide a phase-appropriate overview of the CMC framework, spanning from IND-enabling studies to commercialization. Emphasis will be placed on how CMC requirements evolve across development stages, offering a practical roadmap for navigating scientific and operational complexities through the oligonucleotide therapeutic lifecycle.