Laverock Therapeutics Transthyretin program

Laverock Therapeutics is developing a programmable gene silencing therapy, often identified as LVK02, for the treatment of transthyretin-mediated amyloidosis (ATTR). The program utilizes the company's proprietary GEiGS (Gene Editing induced Gene Silencing) platform, which employs CRISPR-based engineering to redirect endogenous microRNAs (miRNAs) to silence specific target genes. In this application, the technology is used to silence the transthyretin (TTR) gene in the liver, thereby reducing the production of the TTR protein and preventing the formation of amyloid deposits that cause organ damage in ATTR patients. This approach is designed to provide a durable, stable, and potentially tunable method of gene knockdown compared to traditional RNA interference (RNAi) or antisense oligonucleotide (ASO) therapies.

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Laverock Therapeutics ATTR program