Sarepta Therapeutics gains rights to University of Florida-resident startup’s programs for central nervous system diseases.
Lacerta Therapeutics closed on the $30 million investment round, made by Cambridge, Massachusetts-based Sarepta Therapeutics, which gives Sarepta rights to multiple Lacerta central nervous system gene therapy programs. The announcement came from UF Innovate | Sid Martin Biotech, a biotechnology incubator at the University of Florida in Gainesville, where Lacerta is resident. Lacerta had previously raised $2.3 million in a Fast-track Phase I&II Small Business Technology Transfer from the National Cancer Institute.
Lacerta is developing adeno-associated virus vector (AAV) technologies for the treatment of CNS and lysosomal storage diseases. The company’s CNS pipeline includes therapies in development for aromatic L-amino acid decarboxylase deficiency, neurodegenerative proteinopathies, spinocerebellar ataxias and the brain cancer glioblastoma, according to its website. Its lysosomal storage disorder pipeline includes programs focused on Pompe disease and Sanfilippo syndrome Type B.
Sarepta won accelerated approval from the Food and Drug Administration in 2016 for Exondys 51 (eteplirsen), the first drug to win FDA approval for Duchenne muscular dystrophy. The latter company is publicly traded and worth about $8 billion.
Gene therapies that correct genetic defects have been touted as carrying significant potential for treating and possibly even curing rare diseases.
On Monday, Orchard Therapeutics announced it had closed a Series C funding round worth $150 million, just four months after acquiring a portfolio of treatments for rare diseases from GlaxoSmithKline, including Strimvelis, a gene therapy approved by the European Medicines Agency in 2016 for adenosine deaminise severe combined deficiency. The US Food and Drug Administration approved Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl) for biallelic RPE65 mutation-associated retinal dystrophy, a rare, inherited form of blindness. Spark is also developing AAV-based therapies for hemophilia, as is BioMarin, while bluebird bio is developing Lentiglobin, a lentiviral vector-based therapy, for beta-thalassemia and sickle cell disease.
Still, the therapies are highly complex and expensive, carrying price tags in the hundreds of thousands of dollars. Luxturna costs $850,000.