Roberto T Zori, MD

This is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria…

Investigator

Roberto T Zori

Status

Accepting Candidates

Ages

18 Years - 65 Years

Sexes

All

Investigator

Roberto T Zori

Status

Accepting Candidates

The goal of Parts A and B of this Phase 1, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD) and multiple (MAD) ascending doses of oral JNT-517 in healthy participants. In Part C, the…

Investigator

Roberto T Zori

Status

Accepting Candidates

Ages

18 Years - 65 Years

Sexes

All