1. In Vivo

CGT Is In Its Infancy For CNS Disorders, But Technical Strides Being Made

 
• By David Wild

Technical challenges and lackluster investor sentiment about the near-term commercial potential of cell and gene therapies in neurological disorders may be holding the field back.

3D illustration male nervous system, medical concept. - Illustration
The CNS Remains A Challenging Target For Cell and Gene Therapy • Source: Shutterstock

Neurological disorders account for roughly 45% of rare diseases; and 85% of rare and ultra-rare disease trace back to a single gene defect. Given this, the potential for regenerative therapies to benefit patients with central nervous system-based disorders is vast. 

But the task at hand for companies in the CNS cell and gene therapy space is formidable. 

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