Genetic Therapies Bootcamp: Foundations, Frontiers, and Clinical Engagement OnDemand-03-10 15-35 318-323 Brooks Session 3 Overview of Academic-Government-Industry Relationships

03-10 15-35 318-323 Brooks Session 3 Overview of Academic-Government-Industry Relationships

Video Transcription

Video Summary

Dr. P.J. Brooks, Acting Director of NIH's Office of Rare Disease and Innovation, discussed various government-academic-industry collaborations to advance genetic therapies for rare diseases. He highlighted direct NIH funding mechanisms such as cooperative agreements, prize competitions like the Targeted Genome Editor Delivery Challenge, and other transaction awards. He emphasized the importance of developing genome editing as a therapeutic platform, illustrated by projects targeting multiple diseases and regulatory efforts to streamline clinical trial pathways. Brooks showcased indirect support through the Rare Disease Clinical Research Network (RDCRN), which facilitates natural history studies and clinical trials, and public-private partnerships such as the Bespoke Gene Therapy Consortium (BGTC) that optimize AAV gene therapy development for ultra-rare diseases. He also described NIH small business grants and programs like URGENT and TRND that provide research support and contract resources to accelerate therapy development. Throughout, Brooks highlighted collaborative, platform-based, and regulatory strategies designed to improve efficiency and inclusivity for rare disease treatment innovation.

Keywords

NIH funding mechanisms

genome editing therapies

Rare Disease Clinical Research Network

Bespoke Gene Therapy Consortium

AAV gene therapy

rare disease treatment innovation