Lacerta Therapeutics, Inc. is a clinical-stage gene therapy company developing treatments for central nervous system and lysosomal storage diseases using a constellation of adeno-associated virus (AAV) vector technologies. In August 2018, Sarepta Therapeutics Inc. (NASDAQ:SRPT) - formerly dba AVI BioPharma- and itself an SBIR-involved firm until 2008 - licensed exclusive worldwide rights to Lacerta Therapeutics Inc.'s gene therapy candidate for Pompe disease - an autosomal recessive metabolic disorder which damages muscle and nerve cells. Paying an initial $30M to the smaller firm, Sarepta also has options to license two additional neuro-focused candidates from Lacerta's pipeline.