BioMarin Collaborates with Allen Institute to Develop Gene Therapies for Rare Brain Diseases

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Shots:

  • BioMarin will receive an exclusive license to each program for research, development, and commercialization of the gene therapies
  • The collaboration will utilize Allen institute technologies to develop new gene therapies for rare genetic diseases of CNS by targeting gene expression to specific cells
  • Allen Institute has developed modified engineering AAVs to carry genes on specific types of neurons or other cells in the brain

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