HMI-102 / Homology Medicines - LARVOL DELTA

pheNIX: Gene Therapy Clinical Study in Adult PKU (clinicaltrials.gov) - P1/2 | N=10 | Terminated | Sponsor: Homology Medicines, Inc | N=28 ➔ 10 | Active, not recruiting ➔ Terminated; Homology Medicines has discontinued the development of this program.

Enrollment change • Gene therapy • Trial termination • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

pheNIX: Gene Therapy Clinical Study in Adult PKU (clinicaltrials.gov) - P1/2 | N=28 | Active, not recruiting | Sponsor: Homology Medicines, Inc | Suspended ➔ Active, not recruiting

Enrollment closed • Gene therapy • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

pheNIX: Gene Therapy Clinical Study in Adult PKU (clinicaltrials.gov) - P1/2 | N=28 | Suspended | Sponsor: Homology Medicines, Inc | Trial completion date: Sep 2023 ➔ Jun 2023 | Recruiting ➔ Suspended | Trial primary completion date: Jun 2023 ➔ Jan 2023

Gene therapy • Trial completion date • Trial primary completion date • Trial suspension • Gene Therapies • Metabolic Disorders • Phenylketonuria • Rare Diseases

"PAH is the name of the gene delivered by HMI-102 (for PKU)." (@sanjayofSF)

Long-Term Follow Up Study of Subjects Previously Administered HMI 102 (clinicaltrials.gov) - P; N=21; Enrolling by invitation; Sponsor: Homology Medicines, Inc

Clinical • New trial • Metabolic Disorders • Phenylketonuria • Rare Diseases

10th World Orphan Drug Congress (WODC) (November 12-14, 2019 - Barcelona, Spain). (PubMed, Drugs Today (Barc)) - "A co-conference on Cell and Gene Therapy was also organized. This rare disease conference addressed many different challenges in the field with numerous discussions on how to improve cross-border communications, how to better identify patients and shorten their diagnosis time using new tools such as artificial intelligence and machine learning, as well as how to improve usage of patient data and patient empowerment."

Journal • Orphan drug