Advance Beyond: A Live Case Study Symposium

July 2, 2025 by Aldevron

From Concept to Commercial, Gene Editing the Cure for Beta Thalassemia and Sickle Cell Disease

In 2012, a groundbreaking discovery showed how the CRISPR-Cas9 system could be programmed to cut DNA at specific locations in a test tube. This powerful gene editing tool, described in a paper published by Nobel Prize winners Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, and their teams began a 13-year journey to the cure for beta thalassemia and sickle cell disease, advances celebrated in this case study symposium, recorded June 12, 2025.

Covering the discovery of CRISPR, the identification of the dissection point on the gene, how the gene edited cell therapy product went from discovery to the clinic, and the regulatory approval pathway. We end with Victoria Gray, the woman at the end of this amazing success story — the first person to have their cells edited by CRISPR.

To tell the story of this success, Aldevron gathered several experts who helped make this treatment possible, including:

• Fyodor Urnov, PhD; Professor of Molecular Therapeutics and UC Berkley and Scientific Director at Innovative Genomics Institute
• Daniel Bauer, MD, PhD; Boston Children’s Hospital
• Tirtha Chakraborty, PhD; CSO and Head of Tech Ops at Vor Bio
• Tom Foti; VP/General Manager Protein, Aldevron
• Kimberly Benton, PhD; Master Principal & Head of Regulatory, Dark Horse Consulting
• Victoria Gray, the first person to have their cells edited by CRISPR

Moderated by Sadik Kassim, PhD, CTO of Genomic Medicines for Danaher, this informative and inspiring event shows the impact of CRISPR and how it can help develop cures for diseases previously thought to be incurable.

Watch the Full Symposium (Total running time: 4 hours)

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