Drug intelligence / Profile preview

LNP-delivered Cas12n (alphaCas) targeting KLKB1

Development stage
Preclinical
Lead developer
Castalysis Bioscience
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Intravenous
01

Overview

**LNP-delivered Cas12n (alphaCas) targeting KLKB1** is an investigational, liver-directed in vivo genome-editing therapy from Castalysis Bioscience for **hereditary angioedema due to C1 esterase inhibitor deficiency**. The product uses a proprietary lipid nanoparticle carrying mRNA encoding the ultracompact AcCas12n editor, also called alphaCas, together with a KLKB1-targeted single-guide RNA. Following intravenous delivery, alphaCas is intended to disrupt hepatic **KLKB1**, reducing production of plasma prekallikrein and thereby suppressing kallikrein-driven bradykinin generation, the principal mediator of swelling attacks in C1-INH hereditary angioedema. Reported preclinical mouse and nonhuman-primate findings describe durable KLKB1 editing and sustained reduction of plasma prekallikrein after a single administration.

02

Targets

KLKB1 (Plasma kallikrein B1)

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