This meeting will connect a broad network of researchers who share a common goal of using CRISPR-based genome engineering approaches to understand and treat human disease. Specifically, the program will bridge early stage research focused on developing and using CRISPR tools to understand disease mechanisms, through applications of these technologies in preclinical models and current therapeutic strategies for oncology and genetic disease.

Specifically, this conference aims to:

1. Highlight the utility of CRISPR approaches to elucidate disease mechanisms from academic and industry perspectives.
2. Discuss advances in preclinical models that prove safety and efficacy to enable CRISPR strategies to proceed toward clinical applications.
3. Address ongoing scientific and regulatory barriers to getting genome editing-related therapies to patients.
4. Bring together researchers across the spectrum of disease-relevant research to spark new ideas and collaborations with the ultimate goal of accelerating the path of CRISPR approaches to the clinic.

The program will address major strategic challenges that delay or impede the path to the clinic, to provide solutions that will accelerate clinical impacts.  These include: how to focus broader outcomes of a target discovery campaign into a preclinical efficacy and safety effort for a clinic-bound program?  How do lessons from preclinical efforts inform clinical deployment strategies, and how do early-stage clinical data enable “fast-follow” efforts?   What are the avenues for regulatory harmonization to accelerate path to clinic?

Attendees will gain deep insight into how to better approach discovery, development, preclinical and clinical studies to accelerate their path to the clinic.  Specifically, attendees will explore how to:

1. Optimize screen design to accelerate translation to preclinical studies
2. Streamline preclinical studies by de-risking key components early on
3. Design dose-escalation trials using CRISPR-Cas using generalizable principles

Ultimately, attendees will learn how to approach a disease indication and architect an end-to-end “disease to phase 1/2' program using best practices approaches in functional genomics, preclinical studies, and early-stage clinical trial design.