P

PBD Project

Founded in 2021
501(c)(3)

https://pbdproject.org/

PBD Project is funding medical research with the objective to provide a meaningful, positive clinical impact for patients with Peroxisome Biogenesis Disorders (PBD), with a focus on PEX10 mutations.

Therapeutics of interest

Gene Therapy (AAV)
Other RNA Therapy
Antisense Oligonucleotide (ASO)
Stem Cell Transplant

Therapeutics in development

Drug Repurposing or Drug Screening

Members

AL

Andrew Longenecker

AW

Albert Wang

Assistant

Peroxisome Biogenesis Disorders (PBD)

Epidemiology

  • 1 in 50000 prevalence
  • 100 patients engaged

Disease categories

Unknown

Causal genes

PEX10

No Conditions Yet

This organization is not associated with any conditions yet.

Research Plans

Title & DescriptionStage of Activity
Theraputic-Agnostic

PBD pipeline

Disease Models