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Regulatory Pathway: Where We Are Today

Monday, October 16 at 12:30 pm US EDT



This session will start with an overview of the contents of an N=1 IND application process from preIND meeting through IND submission and maintenance. It will include a brief discussion of the differences between an N=1 IND and both research INDs and expanded access INDs. The session will transition to a discussion tackling some of the most confusing things about ultra rare trials: How do we get from having a drug in hand - to an approved IND? This session covers the essentials of regulatory affairs and communicating with the FDA, as well as streamlined nonclinical precedents.


Ashley Kuniholm graduated from Boston University with a BS in Human Physiology. She was an IRB administrator at Boston Children’s Hospital for seven years before transitioning to the Institutional Centers for Clinical and Translational Research: Clinical Research Operations Center as a clinical research project manager. Ashley now serves as the Regulatory Affairs Managers, assisting investigators across the Hospital with their FDA-regulated research and providing regulatory education to the BCH Research Community.


An ex-FDA reviewer, Lauren Black is now a Distinguished Scientist at Charles River - with more than 30 years in drug development, focusing on accelerating Phase 1 trials for unmet medical needs. Dr. Black served as a pharmacologist at FDA CDER and CBER; and represented FDA externally on oligos, relevant tox species, and streamlined preclinical research. Her impacts are recognized by the SOT/BTSS Career Service Award and the President’s Award at Charles River. Lauren earned her BS from Carnegie Mellon and PhD in pharmacology from MCV. Her IRTA Fellowship at NINDS focused on molecular pharmacodynamics of dopamine responses. She lost an infant to a Ultrarare condition and counsels parents/patients facing similarly risky crossroads. She is recognized in publications on CNL7 (Mila), FUS/ALS (Jaci), Angelman’s Syndrome, and TECPR2: her reduction strategy with the first wave of N=1 oligo’s INDs formed the basis of the FDA N=1 Nonclinical Guidance.






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