Development of a PK/PD Model for Gene Therapy for a Rare Neurodegenerative Disease

Allucent’s client requested support to bring an AAV gene therapy for a rare neurodegenerative disease into the clinic for a Phase 1b study in patients. Dosescaling from pharmacology and toxicology studies by brain volume alone for administration directly into the brain by MRI-Guided Convection-EnhancedDelivery led to human equivalent doses that lacked a margin of safety.

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