Details

Our gene therapy product candidate, PBGM01, utilizes an AAVhu68 viral vector to deliver a codon-optimized gene sequence encoding functional β-gal enzyme. The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection), with the goal of increasing β-gal enzyme activity levels in both the central nervous system and periphery.

Details

Our gene therapy product candidate, PBFT02, an AAV1 viral vector to deliver a modified DNA encoding the granulin gene (GRN) to a patient’s cells. The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection). The goal of this vector and delivery approach is to provide higher-than-normal levels of the progranulin protein (PGRN) to the CNS to overcome the progranulin deficiency in GRN gene mutation carriers.

Details

We are developing PBAL05 to treat amyotrophic lateral sclerosis (ALS), a motor neuron disease characterized by rapid degeneration of upper and lower motor neurons, leading to progressive weakness and premature death. We are targeting ALS patients who have a gain-of-function mutation in the C9orf72 gene. Most cases of ALS are sporadic with an unknown etiology, but there are also genetic forms of the disease inherited in an autosomal dominant fashion. Mutations in the C9orf72 gene are the most common genetic defects implicated in ALS, accounting for approximately 34% of familial ALS cases and approximately 5% of sporadic ALS cases.