SAR444836 / Sanofi, MediciNova - LARVOL DELTA

PHASE 1/2 OPEN-LABEL STUDY DESIGN TO EVALUATE SAFETY, TOLERABILITY, AND EFFICACY OF SAR444836, AN AAV-MEDIATED GENE TRANSFER IN PKU PATIENTS (SSIEM 2024) - P1/2 | "Background: Patients with phenylketonuria (PKU) require lifelong treatment with dietary Phenylalanine (Phe) restriction, tetrahydrobiopterin (BH4), and/or PEGylated phenylalanine ammonia lyase (Pegvaliase). Discussion/Conclusion Targeting the unmet need in the treatment of PKU, this innovative trial will investigate whether AAV gene therapy can provide a substantial treatment advantage over existing therapy. NCT number: NCT05972629 Palavras-chave : Phenylketonuria, Safety, Efficacy, Study design, AAV-mediated gene transfer"

Clinical • P1/2 data • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: Sanofi | Not yet recruiting ➔ Recruiting

Enrollment open • Viral vector • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria (clinicaltrials.gov) - P1/2 | N=32 | Not yet recruiting | Sponsor: Sanofi

New P1/2 trial • Viral vector • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases