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 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.
Sarepta has licensed Lacerta’s treatment for Pompe disease and secured options on two further drug candidates as part of a strategic partnership and will gain access to the startup’s capsid screening and manufacturing systems.
As part of that agreement, Lacerta will lead pre-clinical development before handing over to Sarepta as assets 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.
Joseph Reddy, president and chief executive of Lacerta, said: “[This investment] has also been facilitated by being a resident company at Sid Martin Biotech. I joined the company a little over a year ago and immediately decided to establish laboratory operations at Sid Martin.
“Unquestionably, without the support of the incubator’s staff and startup programs, closing this investment round would have been more difficult to accomplish in such a short timeframe.”
