Lacerta Therapeutics, a US-based gene therapy spinout of University of Florida, received $30m in funding from biopharmaceutical firm Sarepta Therapeutics yesterday.
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 spinout is also working on treatments for central nervous system (CNS) diseases including neurodegenerative conditions.
The platform creates virus protein shells known as capsids that possess certain…
