Lacerta Therapeutics, a US-based gene therapy startup spun out 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 company’s 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, which includes drug candidates for central nervous system diseases including neurodegenerative conditions.
Sarepta has licensed Lacerta’s treatment for Pompe disease and secured options on two further drug candidates as part of a strategic partnership. It will also gain access to the startup’s capsid screening and manufacturing systems.
Lacerta will lead preclinical development of the assets before handing them over to Sarepta as they reach the clinic. Lacerta stands to receive single-digit royalties on net sales and will be entitled to collect payments on development and sales-based milestones.
Lacerta is based at the UF Innovate Sid Martin Biotech incubator, operated by University of Florida’s commercialisation arm, UF Innovate.
– The original version of this artcle appeared on our sister site, Global University Venturing.