Agenda Details

Learn more about sessions and presentations at the 26th Annual Meeting

Detailed Program

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


PJ Brooks, PhD. NCATS, NIH, MD


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

Diversifying away from NHPs

Christine Fuentes, PhD. Dark Horse Consulting, CA

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

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

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

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

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

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

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

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