Challenges for CNS directed gene therapies for rare neurogenetic disease

CNS-directed gene therapies face unique hurdles due to complex delivery requirements, limited patient populations, and high safety standards. Souad Messahel, Head of Clinical Operations at Elpida Therapeutics, will discuss strategies to navigate clinical operations, optimize trial design, and address operational and translational challenges in rare neurogenetic disorders. 

Key Takeaways:  

• Examine delivery challenges specific to CNS-targeted gene therapies 

• Evaluate strategies to ensure patient safety and manage adverse events 

• Discuss operational considerations for clinical trials in rare neurogenetic diseases 

• Explore approaches to optimize trial design and patient recruitment 

• Assess translational challenges and approaches to improve therapeutic efficacy