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ASGCT 2021 | Challenges in developing gene therapies for rare diseases & CNS disorders

Kathrin Meyer, PhD, Nationwide Children’s Hospital, Columbus, OH, explains how the small number of patients suffering from ultra rare diseases and challenges in obtaining funding makes it difficult for companies to justify the high costs associated with the development, manufacture and market approval of gene therapies. Furthermore, many rare diseases lack natural history data, rendering clinical trial design difficult, as both patient variability and disease evolution must be considered. The development of therapies for rare diseases affecting the central nervous system (CNS) can be challenging due to the size of the CNS and protection by the blood-brain barrier, making it difficult for homogenous drug action to be achieved across the system. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.


Kathrin Meyer has received royalties from Batten Disease Gene Therapy programs licensed to Amicus. She has also received sponsored research funding and scientific advisory and consultancy fees from Alcyone Therapeutics.