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From Mutation to Dose-able CRISPR Medicine In 3 Months: Obstacles & Actionable Paths Forward

Featuring a Novel Framework for Affordable and Accessible Genetic Therapies

August 28, 2023 | 12:30-1:30 US EDT

From Mutation to Dose-able CRISPR Medicine In 3 Months: Obstacles and Actionable Paths Forward

We will discuss an actionable path towards an academic/nonprofit framework to manufacture and administer a subject-specific CRISPR-Cas treatment within 3 months of diagnosis.

Dr. Fyodor Urnov is a Professor of Molecular Therapeutics at UC Berkeley and a Scientific Director at its Innovative Genomics Institute. He co-developed the toolbox of human genome and epigenome editing and led the team that developed a strategy for genome editing in the hemoglobinopathies, sickle cell disease and beta-thalassemia, that has yielded sustained clinical benefit for subjects in several ongoing clinical trials. At the IGI Fyodor directs efforts to develop scalable CRISPR-based approaches to treat diseases of the immune system, sickle cell disease, neurodegeneration, and neuroinflammation. His recent op-ed in the New York Times describes a major goal for the field of genome editing, and a key focus of Fyodor's work at the IGI - expanding access to CRISPR therapies for N=1 genetic disease.

A Novel Framework for Affordable and Accessible Genetic Therapies

We will discuss a recent Task Force report, in which we convened 30 experts and practitioners from a range of disciplines to explore an alternative framework to the traditional for-profit/VC path with respect to (1) Pricing & Access; (2) Manufacturing and Regulation; (3) Intellectual Property and Licensing; and (4) Organizational Models and Funding. The report lays out existing challenges but also makes specific recommendations that mission-driven organizations and institutions can take to bring down the prices of genomic medicines.

Dr. Manar Zaghlula is the Policy and Engagement Manager at the Innovative Genomics Institute of UC Berkeley. As part of the IGI’s Public Impact team, Manar works with key stakeholders and experts to identify innovative policy and regulatory solutions to make cell and gene therapies - particularly for rare diseases - more accessible and affordable. He is also committed to ensuring scientific evidence is easily accessible to decision-makers in Washington and around the world. Prior to joining the IGI, Manar was a CCST Science and Technology Policy Fellow in the California State Assembly, where he developed and analyzed novel state policies, negotiated legislation with stakeholders, and covered issue areas ranging from health to education to housing. Manar earned his Ph.D. in Translational Biology and Molecular Medicine from Baylor College of Medicine, where he studied novel therapeutic entry points for a rare neurogenetic disorder, and holds a B.S. in Biology and Chemistry from the George Washington University.

Abstract of a keynote presentation at the 2023 Cold Spring Harbor meeting on Genome Engine
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