Poseida Therapeutics Advances Non-Viral Gene Editing Therapy for Hereditary Angioedema

P-KLKB1-101:
Poseida's lead liver-directed non-viral gene editing therapy for hereditary angioedema (HAE), which uses the Cas-CLOVER nuclease system for high-fidelity site-specific gene editing.

Preclinical Data:
Studies in non-human primates (NHPs) demonstrated favorable tolerability and liver editing approaching the desired therapeutic range. In humanized mouse models, a single dose achieved a 58% reduction in kallikrein levels, with effects lasting at least 180 days.

Cas-CLOVER Technology:
Poseida's proprietary high-fidelity nuclease system, engineered for high specificity and minimal off-target editing, which has shown significant capabilities for site-specific insertion of genes.

Non-Viral Approach:
Poseida's fully non-viral approach to genetic medicine employs differentiated gene delivery, gene editing, and gene insertion technology, offering advantages such as low immunogenicity, lower oncogenic risks, and integrated and stable expression.

Pipeline Focus: The company is focused on advancing its two fully non-viral lead programs in rare diseases with significant unmet patient need:
P-KLKB1-101 for HAE and P-FVIII-101 for Hemophilia A.