BioMarin Reports FDA’s Hold on P-I/II Phearless Trial of BMN 307 in Adults with PKU
Shots: The FDA’s clinical hold is based on interim safety findings from a pre-clinical study which showed that 6/7 animals treated with BMN 307 (2e14 Vg/kg) had tumors on liver necropsy @52wks. after dosing with evidence for integration of portions of AAV vector into the genome No lesions were observed in any mice @24wks. 5 […]