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Approach to Drafting Regulatory Submissions and Challenges of Institutional Implementation

April 24, 2023 12:30-1:30 PM US EST

The N=1 Collaborative Institutional Implementation workgroup will present their activities thus far in their approach to drafting regulatory submissions. Margot Cousin, PhD and Roger J Paxton, PhD will provide a brief introduction using several illustrated examples the workgroup has compiled including protocols, informed consent, and pre-IND & IND applications.

Roger Paxton, PhD, is the Research Manager for the Precision Medicine Institute at Children’s Hospital Colorado. In this role, Dr. Paxton is responsible for implementing precision medicine diagnostics research and advising on best practices for precision medicine therapeutics at Children’s Colorado. To this end, he manages and oversees several teams of research operations specialists to develop safety and efficacy standards for novel therapeutic trials.

Catherine Douthwright, PhD, is a biomedical researcher who specializes in rare neurological and neuromuscular diseases. She received her PhD from UMass Chan Medical School in 2014 with her doctoral work focusing on the investigation of several proteins involved in the pathogenesis of Amyotrophic Lateral Sclerosis (ALS). After completing her doctoral work, Catherine moved from bench to bedside, focusing her efforts on clinical research investigations. She has contributed to the undertaking of 20 interventional drug studies and 10 observational/natural history studies across several neuromuscular diseases. As Assistant Professor, she currently oversees a small research team and directs the implementation and operation of a multitude of clinical research studies, including natural history studies, a phase 2 platform trial, several phase 3 studies, and a phase 1/2 gene therapy trial.

Margot A. Cousin, Ph.D., is an Associate Consultant in the Dept. of Molecular Medicine at Mayo Clinic and in the High-Definition Therapeutics initiative in the Center Individualized Medicine at Mayo Clinic. Dr. Cousin earned her B.S. in Diagnostic Genetics and Mathematics from Northern Michigan University in 2005 and earned her Ph.D. in Clinical and Translational Science from Mayo Clinic Graduate School of Biomedical Science in 2015. She completed her postdoctoral training in the Translational Omics Program within the Center for Individualized Medicine at Mayo Clinic. Her current research focuses on antisense oligonucleotide therapeutic development for rare genetic disorders.



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