Award
Eunice Kennedy Shriver National Institute of Child Health and Human Development 1R43HD114328-01
Morquio A therapy integrating gene transfer with lectin-enhanced enzyme delivery to treat multisystemic clinical impairments of rare metabolic childhood diseases
Recipient
BIOSTRATEGIES, LC
Award Amount
$295,923.00
Ceiling
$295,923.00
Awarded
September 20, 2023
Identifier
1R43HD114328-01
Award for developing a gene therapy combining gene transfer and lectin-enhanced enzyme delivery to treat Morquio A syndrome, a rare metabolic childhood disease.
Description
This project focuses on developing a delivery-enhanced gene therapy for Morquio A syndrome, utilizing an RTB:GALNS fusion to improve enzyme delivery to hard-to-treat tissues, with the goal of addressing unmet medical needs in patients with this rare disease.