GM1 Gangliosidosis Drug Receives Rare Pediatric Disease Designation

Passage Bio has been granted the Rare Pediatric Disease designation for their GM1 gangliosidosis treatment, PBGM01. This designation means that Passage will receive a priority review voucher, and it is meant to be an incentive for creating serious, rare illnesses that primarily affect children. Passage Bio is now looking forward to clinical trials of PBGM01, which should start by the end of this year.

About GM1 Gangliosidosis

GM1 gangliosidosis is a rare disorder that is characterized by the destruction of neurons in the brain and spinal cord. There are three forms, all defined by the age at which symptoms appear. Infantile is the most severe, followed by juvenile and adult. All three types share symptoms, which include developmental regression, exaggerated reactions, enlarged liver and spleen, seizures, skeletal abnormalities, clouded corneas, cardiomyopathy, a cherry-red spot, intellectual disability, and loss of vision. The infantile form experiences all of these symptoms, while the latter forms may only experience some. A mutated GLB1 gene causes this disorder, as the mutation results in a lack of the beta-galactosidase enzyme, which then leads to an inability to break down GM1 ganglioside. It accumulates and causes the neurons in the brain and spinal cord to die off. This mutation is inherited in an autosomal recessive pattern, meaning both parents must pass it down.

About PBGM01

This treatment is a gene therapy that uses the AAV-delivery system. It is a treatment for the infantile version of GM1 gangliosidosis. This therapy is given through intra-cisterna magna in order to deliver a functional GLB1 gene, which can then break down GM1 ganglioside and reverse neural toxicity.

Preclinical studies showed that the central nervous system and peripheral organs were affected by this treatment. It is this data that earned PBGM01 the Orphan Drug designation along with the Rare Pediatric Disease designation. Passage Bio hopes to continue these positive results in their Phase 1/2 trial, which is slated to begin in the last quarter of this year.

As those affected by GM1 gangliosidosis face an unmet need for therapies, researchers are very excited by this designation and their trial results. They hope that PBGM01 will offer a viable treatment option.

Find the source article here.

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