Award
NIH Reporter #2U01HL148681-03
cGMP Manufacture Of FVIII-Expressing Placental Cells For Hemophilia A
Recipient
Wake Forest University Health Sciences
Award Amount
$816,782.00
Ceiling
$816,782.00
Awarded
August 27, 2026
Identifier
2U01HL148681-03
This award funds the development and manufacturing of a cell/gene therapy product for Hemophilia A, focusing on prenatal treatment to induce lifelong tolerance and reduce treatment burdens, with plans for clinical trials.
Description
The project aims to optimize and scale-up a clinical cell/gene therapy product for Hemophilia A, proven safe and efficacious as a prenatal treatment, and to define mechanisms of therapeutic effect following prenatal delivery, paving the way for a phase one clinical trial.