Lacerta Therapeutics

Developing a constellation of gene-based therapies.

Charting a path to tomorrow’s health.

Our Mission

Lacerta Therapeutics is dedicated to the discovery and development of AAV-based gene therapies. We aim to provide novel therapeutic options by leveraging our expertise in the nervous system, proprietary AAV capsid technologies and scalable manufacturing platforms across three modalities: gene replacement, vectorized antibody delivery and artificial microRNA-mediated modulation of gene expression.

CNS Targeted Therapies

Lysosomal Storage Diseases

POMPE DISEASE

Classically defined by the buildup of lysosomal glycogen, Pompe Disease is a genetic disorder that is caused by a mutation in the GAA gene. This gene encodes for the enzyme acid alpha-glucosidase, that is responsible for breaking down lysosomal glycogen. Accumulation of glycogen has a profoundly negative impact on heart and neuromuscular function, but symptoms vary depending on the age at which the disease appears. Pompe disease covers a wide phenotypic spectrum where patients may be diagnosed as infants, adolescents, or adults. Our approach incorporates AAV capsids that carry desired properties to address each disease component and improve the patient’s quality of life.

MUCOPOLYSACCHARIDOSIS TYPE IIIB (MPSIIIB)

Also known as Sanfilippo Syndrome B, is a disorder caused by mutations in the NAGLU gene and is characterized by progressive motor and cognitive deterioration. Our patent-pending strategy uses a proprietary capsid to deliver a functional copy of NAGLU to the cells affected by this disease.

Ataxias

FRIEDREICH’S ATAXIA​
(FRDA OR FA)

FA is a rare mitochondrial disorder caused by mutations in the FXN gene and is characterized by ataxia, cardiomyopathy, and diabetes. FA affects 1 in every 40,000 people, with symptoms typically presenting between 5-15 years of age. Our patent-pending therapeutic strategy is designed to deliver an enhanced copy of FXN via a proprietary capsid that displays the desired tropism for the central nervous system and heart.

Solid Tumors

GLIOBLASTOMA (GBM)

Glioblastoma is the most common and aggressive cancer of the brain. Symptoms include loss of cognitive function, numbness in the face, arms, or legs, trouble with balance, nausea, vomiting, and headaches. Current standard treatments exert a large physical and financial toll on patients. Our approach uses select AAV capsid variants with high precision for glioblastoma cells to target key master regulators driving tumorigenesis.

platform technologies

OneBac Manufacturing Platform

Our proprietary OneBac platform uses a baculovirus expression system to infect cells derived from the Spodoptera frugiperda larval moth. Using a chemically defined medium, our system provides a platform to deliver superior yields and purification of infectious AAV for use in gene therapy applications. These key advantages are accompanied by a reduction in the requirement for manufacturing components, providing a cost-effective system to develop the highest quality material for patients.

AAV Capsid Engineering

rAAV Vector Platform: Our proprietary process uses multiple methodologies to enhance both naturally occurring and engineered AAV capsids to improve tissue tropism and provide immune escape profiles. Our strategy has the advantage of targeting the variable regions of capsids, resulting in libraries with superior complexity compared to traditional shuffled serotype libraries.

Leadership in AAV gene therapy

Founded in 2017 by leaders in the AAV gene therapy community, Lacerta currently occupies over 12,000 sq ft of R&D space in a brand new, state-of-the-art building in the City of Alachua, Florida.

Lacerta Therapeutics is pleased to announce our new collaboration with UCB. This brings the potential to drive a fundamental change in how diseases are treated—by moving from treating symptoms, to disease modification, and eventually towards a cure.

Take a moment to listen to the news directly from Dr. Dhavalkumar Patel, UCB’s Chief Scientific Officer and our own Dr. Edgardo Rodriguez-Lebron, President and Chairman of the Board at Lacerta Therapeutics