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Find Clinical Drug Pipeline Developments & Deals by Passage Bio
PBFT02 is an adeno-associated virus delivery gene therapy. It is being evaluated in phase 1/2 clinical trials for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.
PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual beta-galactosidase enzyme activity and subsequent neurodegeneration.
PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual beta-galactosidase enzyme activity and subsequent neurodegeneration.
PBFT02 utilizes an AAV1 viral vector to deliver, through ICM administration, a functional GRN gene to patients with mutations in the gene that encodes for progranulin (PGRN).
PBML04, an adeno-associated virus -delivery gene therapy to treat MLD, a recessive lysosomal storage disease caused by genetic mutations that result in demyelination of fibers of central and peripheral nervous systems, leading to progressive motor and cognitive impairments.
The data show that the low dose of PBGM01 is well-tolerated, has a favorable safety profile and exerts a biological effect that appears to translate to improvement in developmental milestones for these children.
Preclinical results, report marked improvements in both disease progression and key biomarkers in large and small animal models of Krabbe disease following a single administration of PBKR03, with no observed dose-limiting toxicities.
Interim safety data at three and nine months in Imagine-1 showed low dose PBGM01, a gene therapy for GM1 gangliosidosis well tolerated with no serious adverse events reported and no evidence of dorsal root ganglion toxicity.
PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C).
PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C).