Agenda Details | ASGCT Annual Meeting

Detailed Program

Wednesday, May 17 2023
08:00 AM – 09:45 AM PT

Refinement of NHP usage in gene therapy drug development

With the passage of the FDA Modernization Act this year that ended the FDA mandate requiring experimental drugs to be tested on animals before use in human clinical trials, this session was crafted to highlight the latest advances in large and small animal models available for gene therapy drug development beyond NHPs. In addition, perspectives from the EU where large animals have already begun being limited will be shared.

Session Details

08:00 AM – 09:45 AM - Concourse Hall 152 & 153

Co-Chair

PJ Brooks, PhD. NCATS, NIH, MD

08:00 AM – 09:45 AM - Concourse Hall 152 & 153

Co-Chair

Nicole K.. Paulk, PhD. University of California San Francisco, San Francisco, CA

08:01 AM – 08:25 AM - Concourse Hall 152 & 153

Diversifying away from NHPs

Christine Fuentes, PhD. Dark Horse Consulting, CA

08:25 AM – 08:50 AM - Concourse Hall 152 & 153

In vitro pharmacology study of gene therapy using retina organoids of CLN2 patient-derived induced pluripotent stem cells

Kwi Hye Kim, PhD. REGENXBIO Inc, Rockville, MD

08:50 AM – 09:15 AM - Concourse Hall 152 & 153

Ocular Anatomy and Relevance to Gene Therapy & Alternative Animal Models for Ocular Gene Therapy

Krishna Yekkala, BVSc, PhD, DACVP. Janssen Pharmaceuticals, Spring House, PA

08:50 AM – 09:15 AM - Concourse Hall 152 & 153

Ocular anatomy and relevance to gene therapy & alternative animal models for ocular gene therapy

Timothy K.. MacLachlan, PhD. Novartis, Cambridge, MA

09:15 AM – 09:40 AM - Concourse Hall 152 & 153

Use of Rat Data to Support an IND for a Rare Disease Trial (NGLY1)

Becky Schweighardt, PhD. Grace Science, LLC, Menlo Park, CA