Lacerta Therapeutics is a biotechnology company based in Alachua, FL, dedicated to the discovery and development of AAV-based gene therapies. With a focus on the nervous system, the company leverages its expertise in proprietary AAV capsid technologies and scalable manufacturing platforms to provide novel therapeutic options across three modalities: gene replacement, vectorized antibody delivery, and artificial microRNA-mediated modulation of gene expression.
The company's mission is to address various genetic disorders, including Pompe Disease, Mucopolysaccharidosis Type IIIB, Friedreich's Ataxia, and Glioblastoma, by utilizing AAV capsids with desired properties to improve patient quality of life. Lacerta Therapeutics also boasts platform technologies such as the OneBac Manufacturing Platform, which provides a cost-effective system for developing high-quality material for gene therapy applications, and the rAAV Vector Platform, which enhances AAV capsids to improve tissue tropism and immune escape profiles.
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