P-KLKB1-101 / Roche - LARVOL DELTA

Novel Cyclohexane Based Ionizable Lipids (CHILs) for Non-viral Liver Delivery of Complex Nucleic Acid Genetic Medicine Payloads (ASGCT 2025) - "Data demonstrating the evolution of this novel class of CHILs from conception through the identification and integration of HBC365 into P-KLKB1-101 and P-FVIII-101 programs will be covered in this presentation. Disease Focus of Abstract:None"

Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hereditary Angioedema • Rare Diseases

HIGHLY SPECIFIC NON-VIRAL GENE EDITING WITH P-KLKB1-101 FOR HEREDITARY ANGIOEDEMA (ACAAI 2024) - "P-KLKB1-101 offers high specificity via high-fidelity targeted gene editing while potentially enabling durable reduction in kallikrein activity. These preliminary data support further development for HAE, and the potential for other future high-fidelity genetic medicines."

Late-breaking abstract • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema