P
PBD Project
Founded in 2021
Peroxisome Biogenesis Disorders (PBD)
We have a focus on Zellweger Spectrum Disorder (ZSD) caused by mutations to the PEX10 gene. Research on PBD has implications for COVID-19, West Nile, Zika, HIV, Alzheimer's, Parkinson's, diabetes, and cancer.
Patient population
- 1 in 100000 prevalence
- 100 patients engaged
Disease categories
Loss-of-Function
Causal genes
PEX10
PEX1
PEX5
PEX3
PEX26
About us
PBD Project's mission is to incubate and advance peroxisome science, transforming discovery into treatment by convening leading scientists, funding bold research, and catalyzing the translation of biology into medicines that patients urgently need.
Website
Therapeutics of interest
Gene Therapy (AAV)
Other RNA Therapy
Antisense Oligonucleotide (ASO)
Stem Cell Transplant
Therapeutics in development
Drug Repurposing or Drug Screening
Gene editing
Small molecule
Enzyme or protein replacement
Antisense oligonucleotide (ASO)
Gene therapy (AAV)
Gene therapy (non-AAV)
Drug repurposing
Members
AL
Andrew Longenecker
Research plans
Title | Research strategy | Stage | Status | Partners | Links | Number of projects | Description |
|---|---|---|---|---|---|---|---|
PBD pipeline | 7 published projects |
