# Grace Science, LLC

> Grace Science, LLC is a Palo Alto-based biotechnology company founded in 2017 by Silicon Valley entrepreneur Matt Wilsey and Nobel laureate chemist Dr. Carolyn Bertozzi to develop therapies built on the biology of NGLY1, an enzyme central to protein quality control. The company grew out of a father's search for a treatment after his daughter Grace was diagnosed with ultra-rare NGLY1 Deficiency. Its lead program, GS-100, is an AAV9 gene replacement therapy now in a Phase 1/2/3 clinical trial and holds FDA RMAT designation. Grace Science also pursues small-molecule and biomarker work that extends NGLY1 biology into oncology and neurodegeneration.

- **Founded:** 2017
- **Headquarters:** Palo Alto, California, United States
- **Founders:** Matt Wilsey (Co-founder & CEO), Carolyn Bertozzi (Co-founder & Scientific Advisory Board Member)
- **Team size:** ~15 employees
- **Products:** GS-100, Small-molecule programs, Biomarker development
- **Notable:** FDA granted RMAT (Regenerative Medicine Advanced Therapy) designation to GS-100 in April 2026, Selected by the FDA for the START pilot program for GS-100, Dosed the first patient in the Phase 1/2/3 trial of GS-100 (2024)

## Products & services

- **GS-100** — An investigational AAV9 gene replacement therapy for NGLY1 Deficiency, in an open-label Phase 1/2/3 clinical trial. Holds FDA RMAT designation and was selected for the FDA START pilot program.
- **Small-molecule programs** — Discovery-stage small-molecule inhibitors targeting the NGLY1 pathway, with potential applications in immuno-oncology and neurodegeneration.
- **Biomarker development** — NGLY1 pathway biomarkers (including GNA-based biomarkers) to support diagnosis, patient stratification, and clinical development.

## Achievements

- FDA granted RMAT (Regenerative Medicine Advanced Therapy) designation to GS-100 in April 2026
- Selected by the FDA for the START pilot program for GS-100
- Dosed the first patient in the Phase 1/2/3 trial of GS-100 (2024)
- Reported motor and cognitive improvements in patients treated with GS-100 for at least 52 weeks
- Co-founded with 2022 Nobel Prize in Chemistry laureate Carolyn Bertozzi
- Presented early clinical activity data at ASGCT 2026

## Latest updates

- **2026-04** — FDA granted RMAT designation to GS-100 for NGLY1 Deficiency.
- **2026-05** — Presented early GS-100 activity data at ASGCT 2026.
- **2024-06** — Selected for FDA START pilot program; announced successful treatment of second patient.
- **2024-03** — Announced first patient treated in Phase 1/2/3 trial of GS-100.
- **2024-04** — Partnered with Andelyn Biosciences for suspension-process AAV manufacturing tech transfer.

## Links

- Website: https://www.gracescience.com
- LinkedIn: https://www.linkedin.com/company/grace-science-llc
- YouTube: https://www.youtube.com/@gracesciencefoundation9799

---

Profile page: https://yespress.io/grace-science-llc
Published by YesPress — https://yespress.io
Last updated: 2026-08-05