# PROJECT 3: MUCOPOLYSACCHARIDOSIS TYPE 1 (MPS1)

> **NIH NIH U19** · CHILDREN'S HOSP OF PHILADELPHIA · 2024 · $688,528

## Abstract

PROJECT SUMMARY
Mucopolysaccharidosis type I (MPSI, Hurler syndrome) is an autosomal recessive lysosomal storage disease in
which pathology begins before birth and untreated children die of cardiorespiratory complications by 10 years of
age. Established treatments are ineffective against preexisting pathology and include costly, lifelong,
immunogenic enzyme replacement therapy and hematopoietic stem cell transplantation. A GàA mutation
(W402X), amenable to correction via adenine base editing, accounts for 40% of patients and has a strong
genotype-phenotype correlation—all homozygous W402X patients exhibit severe disease. Project 3 will focus
on an AAV-based adenine base editing postnatal treatment of MPSI, with the aim to file an IND application
and begin a clinical trial, and prenatal base editing treatment of MPSI, with the aim of performing preclinical
studies during the five-year funding period to enable an eventual IND application if the postnatal clinical trial
proves successful.

## Key facts

- **NIH application ID:** 10896264
- **Project number:** 5U19NS132301-02
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** Beverly L. Davidson
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $688,528
- **Award type:** 5
- **Project period:** 2023-08-01 → 2028-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10896264

## Citation

> US National Institutes of Health, RePORTER application 10896264, PROJECT 3: MUCOPOLYSACCHARIDOSIS TYPE 1 (MPS1) (5U19NS132301-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10896264. Licensed CC0.

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