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Florida CNS gene therapy biotech shuts down

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Seven-year-old gene therapy startup Lacerta Therapeutics has folded and sold its assets, a board member confirmed to Endpoints News.

The University of Florida spinout, based in Alachua, Florida, had forged partnerships with Sarepta Therapeutics, UCB and Eli Lilly’s Prevail.

But it “fell victim to a very difficult funding environment in the last couple of years for early stage gene therapy,” said Jackson Streeter, Lacerta board member and director of UF Innovate Ventures, in an email to Endpoints.

“Lacerta is not operational and the assets have been sold to an undisclosed buyer,” Streeter said.

The biotech downsized last year, and Sarepta terminated a license agreement in May 2023 and took a $30 million impairment loss, according to its annual report from February. A Lilly spokesperson said in an email that the pharma giant has “no information to disclose on this matter.”

The company was developing a clinical-stage AAV-based gene therapy for Pompe disease and had other programs for mucopolysaccharidosis type III (or Sanfilippo syndrome), Friedreich’s ataxia, glioblastoma and other CNS indications.

Lacerta’s CEO, Edgar Rodriguez-Lebron, left in April and is now CEO of another Florida-based biotech that he co-founded. That company, Andante Biologics, is working on an immunotherapy for chronic stress, according to his LinkedIn bio.

The shutdown arrives as multiple large drugmakers pull back from gene therapies and smaller biotechs struggle to secure financing for the complex, capital-intensive class of medicines. A few CNS gene therapy biotechs have still emerged recently, including Latus Bio.

Roche’s Spark Therapeutics last month also scrapped development of an experimental Pompe disease gene therapy amid a restructuring. Other drugmakers like Bayer’s AskBio and Astellas are exploring gene therapies for the rare, muscle-weakness genetic disorder.

It’s been a particularly rough stretch for the life sciences community in the town of Alachua. Biomanufacturer Resilience laid off workers in the area last year, Endpoints reported in December. Thermo Fisher Scientific has also downsized in the Florida town. Meanwhile, gene therapy biotech Beacon Therapeutics sold its manufacturing facility in the town to Ascend Advanced Therapies earlier this year.


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