UF Innovate | Sid Martin Biotech resident client Lacerta Therapeutics, a US-based gene therapy startup of University of Florida, received $30m in funding from biopharmaceutical firm Sarepta Therapeutics.
Founded in 2017, Lacerta Therapeutics is developing gene therapy programs for rare, inherited metabolic conditions, known as lysosomal storage disorders, such as Pompe disease and the fatal Sanfilippo syndrome type B. The startup is also working on treatments for central nervous system diseases including neurodegenerative conditions.
The platform creates virus protein shells known as capsids that possess certain characteristics associated with the adeno-associated virus, which causes a very mild immune response, to gain an advantage over malignant cells.
The funding will sustain development of Lacerta’s clinical pipeline.