Leveraging decades of expertise to develop next generation therapies
Our therapeutic programs are focused on exploiting our proprietary capsid technology and scalable manufacturing platform to advance AAV-based therapies via three primary approaches: gene replacement, vectorized antibody, and vectorized micro-RNA modulation. We invite you to look at our team and our work to see our continued growth — and what the future promises.
Lysosomal Storage Diseases
Classically defined by the buildup of lysosomal glycogen, Pompe Disease is 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.
(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.