Genetic Therapy Solutions for Severe Neurological Disorders

Neurogene is a global biotechnology company whose mission is to advance genetic medicines for the treatment of neurological disorders. Neurogene is interested in engaging with new academic partners focused on the use of genetic medicines to treat severe neurological disorders, including neurodevelopmental, neurodegenerative, and neuroinflammatory diseases as well as genetic epilepsies.

Our client wishes to engage with academic partners that utilize traditional gene replacement via a vectorized delivery as well as novel technological approaches for diseases not well served by traditional gene replacement therapy.

Further information

Our client wishes to focus on adeno-associated virus (AAV) delivered genetic treatments (gene transfer, shRNA, miRNA, etc.) for relevant neurologic indications.

Neurogene is also focused on novel technologies that overcome key limitations of current gene therapy approaches and address the following criteria: 

• Allow for delivery/treatment of large genes, genes sensitive to dosage, and/or genes sensitive to ectopic expression
• Allow for large-scale multi-exon gene correction 
• Address dominant and recessive inheritance patterns 
• Avoid permanent genome modification (focus on RNA level approaches) 

Non-viral delivery approaches (e.g. lipid nanoparticles) are also of interest.

Out of scope:

• Lentiviral delivery approaches (unless transformed for use in AAV) 

Development stage:

Basic research to pre-clinical research is in scope and will be considered. Novel technologies at any stage and AAV-delivered approaches with in vivo validation are of highest priority. 

Submission Information

• Applicants may use this template when preparing a response outlining an academic, research project, technology, spinout or centre of excellence.