Innovative Therapies for Genetic Mitochondrial Disorders and Leukodystrophy

A genetic disease-focused pharmaceutical company is seeking novel therapeutic targets for genetic mitochondrial diseases and leukodystrophies, as well as innovative drug discovery and development platforms tailored to these disease areas.

Approaches of interest:

• Target molecules in brain cells for mitochondrial diseases
• Mechanisms improving mitochondrial function
• Target molecules in oligodendrocytes or OPCs for leukodystrophies
• Mechanisms promoting myelination or inhibiting demyelination
• Modalities including small molecules, peptides, antibodies, and gene therapy
• Novel drug discovery and development platforms

Out of scope:

• Cell therapy or medical devices
• Unvalidated or widely adopted discovery/development platforms

 Developmental stage of interest: 

Opportunities from basic research to preclinical stage, with proof-of-concept. Validation in human disease lines preferred; animal disease models also considered.