Webinars & Presentations Webinar: How to Mitigate Gene Editing Program Risk through Comprehensive Off-Target Safety Profiling & Characterization

Webinar: How to Mitigate Gene Editing Program Risk through Comprehensive Off-Target Safety Profiling & Characterization

Are you engineering a CRISPR-based editing system for application in cell or gene therapy? Learn how to assemble an off-target safety package for your IND submission. For those in earlier stages of development, learn how early assessment of off-target risks can help de-risk your program.

Key topics covered

Guide selection approaches for the discovery stage: Identifying off-target effects remains a critical challenge in gene editing. SeQure DX's two-step, variant-aware assay approach helps you find gene editing off-targets safely and efficiently, setting up your program for regulatory approval down the line. Learn about the latest tools used to generate off-target data for both CRISPR/Cas and base editing systems.
Gene therapies for HSPCs: Papillon Therapeutics presents their program on gene-modified and gene-edited hematopoietic stem and progenitor cells, or HSPCS.
The path to IND submission for Friedreich's ataxia study: Papillon Therapeutics shares their experience collaborating with SeQure DX to advance a Friedreich’s ataxia program toward IND submission, including a preview of their off-target safety data package.

Speakers

Stephanie Cherqui, PhD

Professor in the Department of Pediatrics at University of California San Diego Director of the Gene Therapy Initiative and Co-Founder of Papillon Therapeutics

In addition to her professorship and other leadership roles, Stephanie Cherqui, PhD, is chair of the Cystinosis Stem Cell and Gene Therapy Consortium; member of the Scientific Review Board of the Cystinosis Research Foundation; and scientific council member for the Cure Cystinosis International Registry. Cherqui’s lab has received over $30 million in funding from the California Institute of Regenerative Medicine and NIH towards development of multi-systemic HSPC therapies. She earned her PhD from University René Descartes

Colin Exline, PhD

Director, Research and Development at Papillon Therapeutics

Colin Exline leads the Research and Development efforts at Papillon Therapeutics and has been working in regenerative medicine for over 15 years. He has developed methods to efficiently edit genomes by both virally and non-virally. These methods have been employed while leading projects in HSPC to create anti-viral immune systems and within organ transplantation settings to decrease the risk of rejection for both xeno- and traditional transplantation. He earned his PhD from the University of Iowa studying viral gene expression.

Vijetha Vemulapalli, PhD

Vice President of Data Science at SeQure DX, a MaxCyte Company

Vijetha Vemulapalli earned her PhD in computational bioscience from University of Colorado, where she developed machine learning methods to study genome evolution. Vijetha has extensive leadership experience in the biotech industry spanning early-target discovery, clinical-stage personalized immunotherapies and the application of artificial intelligence in digital health. In her current leadership role, she drives the bioinformatics and data science efforts behind MaxCyte’s off-target product portfolio, focusing on product development, effective delivery of insights to clients and research support.