BMN 331 / BioMarin - LARVOL DELTA

HAErmony-1: A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (clinicaltrials.gov) - P1/2 | N=44 | Active, not recruiting | Sponsor: BioMarin Pharmaceutical | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene therapy • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Hereditary Angioedema

Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema. (PubMed, J Allergy Clin Immunol Pract) - "Donidalorsen is an investigational GalNAc-conjugated antisense oligonucleotide that binds to prekallikrein mRNA in the liver and reduces the expression of prekallikrein...ADX-324 is a GalNAc-conjugated siRNA being investigated in HAE...NTLA-2002 is an investigational in vivo clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-based therapy designed to knock out the prekallikrein-coding KLKB1 gene in hepatocytes; a phase 1/2 study is ongoing. Findings from these and other ongoing studies are highly anticipated with the expectation of expanding the array of treatment options in HAE."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Hereditary Angioedema

HAErmony-1: A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (clinicaltrials.gov) - P1/2 | N=34 | Recruiting | Sponsor: BioMarin Pharmaceutical | Initiation date: Nov 2021 ➔ Feb 2022

Trial initiation date • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Hereditary Angioedema

HAErmony-1: A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (clinicaltrials.gov) - P1/2; N=34; Recruiting; Sponsor: BioMarin Pharmaceutical; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Hereditary Angioedema

HAErmony-1: A Phase 1/2 Safety, Tolerability & Efficacy Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (clinicaltrials.gov) - P1/2; N=34; Not yet recruiting; Sponsor: BioMarin Pharmaceutical

Clinical • New P1/2 trial • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Hereditary Angioedema