Award
NIH Reporter #3U19NS132301-04S1
LEAD PROJECT 1: PHENYLKETONURIA (PKU)
Recipient
Children's Hospital of Philadelphia, Philadelphia, PA, United States
Award Amount
$5,455,342.00
Ceiling
$5,455,342.00
Awarded
August 19, 2026
Identifier
3U19NS132301-04S1
Award for development of lipid nanoparticle-based adenine base-editing treatments for PKU, funded by NIH with $5.45 million, project period 2026-2028.
Description
The project involves development of lipid nanoparticle (LNP)-based adenine base-editing treatments for PKU or UCDs. The project aims to file IND applications and conduct a phase I/II clinical trial. Funding is from NIH, with a total award amount of approximately $5.45 million. The project period is from August 1, 2026, to July 31, 2028.