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. The team is also interested in innovative drug discovery and development platforms tailored to these disease areas. 

Approaches of Interest:

• Submission for mitochondrial diseases should focus on: (i) Target molecules expressed in brain cells (neurons, microglia, astrocytes, oligodendrocytes) (ii) Mechanisms that improve oxygen consumption, NAD⁺/NADH ratio, or overall mitochondrial function (other MoAs will be considered) (iii) Demonstrated improvement in survival or behavior in in vivo disease models is preferred (iv) Priority diseases include Leigh syndrome, MELAS, MERRF, NARP, and KSS (other diseases will be considered) 
• Submissions for leukodystrophies should focus on: (i) Target molecules in oligodendrocytes or oligodendrocyte progenitor cells (OPCs) (targets in other brain cell types will be considered) (ii) Mechanisms promoting myelination, OPC differentiation, or inhibiting demyelination (other MoAs will be considered) (iii) Priority diseases include Pelizaeus-Merzbacher disease, Metachromatic leukodystrophy, and Krabbe disease (other diseases will be considered) 
• Modalities of interest include small molecules, peptides, antibodies and gene therapy
• A highly novel and original drug discovery and development platform, with demonstrated validation and use in real-world drug discovery 

Out of Scope:

• Opportunities relating to cell therapy or medical devices 
• Discovery or development platforms that are unvalidated or widely adopted, established platforms 

Developmental Stages of Interest:

• Opportunities from basic research phase to preclinical stage, with proof-of-concept 
• Opportunities with validation in human disease lines are preferred; validation in animal disease models will also be considered 

Submission Information

Submission of one-page, 200–300-word briefs is encouraged, along with any optional supplementary information e.g. relevant publications. Our client is also open to novel research proposals which can be outlined using this submission form. In submitting to this campaign, you confirm that your submission contains only non-confidential information. 

Opportunity for Collaboration

Our client is open to a range of collaboration opportunities, with the most appropriate outcome being decided on a case-by-case basis. Example outcomes include licensing assets, project/PhD funding, and research collaborations.

Please check your organization’s external collaboration policy and/or guidelines before submitting to this anonymous campaign, which may result in introduction and subsequent partnering discussions with the client company. Please get in touch with campaigns@inpart.io if you have any queries.