Webinars & Presentations Reducing Risk in Gene Editing Programs through Robust Off-Target Safety Evaluation

Reducing Risk in Gene Editing Programs through Robust Off-Target Safety Evaluation

Join us live for a webinar on how to assess off-targets for CRISPR-based gene editing applications

Tuesday, June 24, 2025

09:00 PDT; 12:00 EDT; 17:00 BST; 18:00 CEST

If you're engineering a CRISPR-based editing system for application in cell or gene therapy, this session is a must attend. You’ll gain insight into assembling an off-target safety package for your IND submission. For those in earlier stages of development, we’ll highlight how early assessment of off-target risks can help de-risk your program.

The session will feature presentations from Dr. Stephanie Cherqui (Papillon Therapeutics) and Dr. Colin Exline (Papillon Therapeutics), who will share their experience advancing a Friedreich’s Ataxia program toward IND submission, including a preview of their off-target safety data package. Dr. Vijetha Vemulapalli (MaxCyte - SeQure DX) will provide a comprehensive overview of the current tools used to generate robust off-target data for CRISPR/Cas and base editing systems.

Whether you’re in early discovery or preparing for IND-enabling studies, this session offers practical guidance rooted in real-world experience. Join us to gain strategic insights, learn from experts, and strengthen the safety foundation of your CRISPR-based therapeutic program.

Presenters

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.