Engineering Next-Generation Cas9s to Advance Rare Disease Therapy Portfolio

• Explore how engineered Cas9 variants are expanding precision, efficiency, and safety in gene editing applications
• Review strategies for tailoring Cas9 platforms to address the unique challenges of rare disease indications
• Learn from case studies on building a diversified rare disease pipeline through Cas9- based therapeutic development

Speakers:

Salman Mustfa

Senior Scientist

AstraZeneca