Novel Therapeutic Interventions to Address the Root Genetic Cause of Neurodevelopmental Disorders

A global leader in therapies for rare genetic diseases seeks novel approaches to address the root genetic cause of neurodevelopmental disorders. The company is particularly interested in disease-modifying interventions for specific developmental epileptic encephalopathies and neurodevelopmental disorders (CDKL5 deficiency disorder, Fragile X Syndrome, Rett Syndrome, SYNGAP1 disorder, and STXBP1 disorder).

Approaches of interest:

• Allele-specific knockdown
• Protein replacement/enhancement/stabilization
• Modulation of endogenous gene regulation and expression
• Nonviral approaches, mRNA, oligonucleotides
• Small molecules and protein replacement/substitution/stabilization

Out of scope:

• Viral gene therapy approaches
• Disease indications not specifically listed

Developmental stage of interest: Opportunities with supporting data demonstrating disease modification are of high interest. Basic and preclinical research assessing treatment feasibility is also within scope.