Award
NIH Reporter #2R44NS134446-03
Gene Therapy for Idiopathic Parkinson's Disease: In Vivo Validation
Recipient
Mark Therapeutics, Inc.
Award Amount
$862,457.00
Ceiling
$862,457.00
Awarded
August 28, 2026
Identifier
2R44NS134446-03
Award to Mark Therapeutics, Inc. for developing gene therapy for idiopathic Parkinson's Disease, focusing on preclinical validation and safety testing using AAV vectors.
Description
This project aims to develop a disease-modifying gene therapy utilizing Adeno-Associated Viral (AAV) vectors to treat idiopathic Parkinson’s Disease (idPD). The therapy targets the loss of PLA2g6L protein, restoring cellular functions and halting dopaminergic neuron loss. The research includes optimizing mouse models, validating therapeutic efficacy and safety in animal models, including non-human primates, and preparing for clinical translation.