What Grace Science does

Grace Science, LLC is a privately held clinical-stage biotechnology company founded in 2018 by Matt Wilsey and Nobel Laureate Dr. Carolyn Bertozzi to develop therapeutics based on NGLY1 biology, the product of over a decade of research initiated through the Grace Science Foundation starting in 2013. Headquartered in Menlo Park, California, the company operates with a lean team of 11–50 employees and targets both ultra-rare genetic disease (NGLY1 Deficiency) and larger indications in oncology and neurodegeneration that converge on the same NGLY1/NFE2L1 axis.

The pipeline is built around four programs: GS-100, an AAV9 single-stranded viral vector gene therapy delivering the full-length human NGLY1 protein, currently in a Phase 1/2/3 trial (NCT06199531) for NGLY1 Deficiency; GS-220 and GS-221, small molecule NGLY1 inhibitors designed to indirectly suppress the NFE2L1 transcription factor for undisclosed oncology indications; and GS-310, a small molecule program for neurodegenerative diseases such as Parkinson's disease. The underlying technology platform is anchored by more than a decade of NGLY1 pathway research, including a 2017 breakthrough linking NGLY1 to cancer and neurodegeneration, and a foundation-funded network of nearly 75 scientists across 20 medical centers in 5 countries.

Grace Science is pre-revenue and has not disclosed pricing or a commercial model. The intended revenue mechanism is eventual commercialization of ultra-rare disease gene therapies following regulatory approval, typically priced above $1 million per treatment; the addressable population for the lead indication is approximately 12 US births per year. Patient access is currently restricted to clinical trial enrollment (no expanded access program offered). The company has secured FDA Orphan Drug Designation (June 2021), IND clearance (November 2023), FDA START Pilot Program selection (June 2024), and RMAT designation (April 2026) for GS-100, and operates a strategic collaboration with Takeda. Only one funding round of $7 million (February 2017) has been disclosed, and the company faces an FDA-request bottleneck that could delay gene therapy development.

Name

Grace Science

Legal name

Grace Science, LLC

Website

https://gracescience.com

Company type

Private

Founded year

2013

Operating status

Operating

Headcount range

11–50 employees

Short description

Grace Science, LLC is a clinical-stage biotechnology company developing AAV9 gene therapy (GS-100) and small molecule therapeutics (GS-220, GS-221, GS-310) based on NGLY1 biology, targeting the ultra-rare NGLY1 Deficiency as well as oncology and neurodegenerative diseases.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

akta.pro secondary industry

Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)

Keywords

• Rare disease therapeutics
• AAV9 gene therapy
• Small molecule inhibitors
• Oncology drug development
• Neurodegenerative disease research

Headquarters

HQ city

San Francisco

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Supply Chain

Revenue model

1. Gene Therapy Commercialization: Pre-revenue clinical stage biotech company developing therapies for ultra-rare diseases. Revenue generation model would be through eventual therapy commercialization following regulatory approval, likely with premium pricing typical of orphan/rare disease gene therapies.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Grace Science is a clinical-stage biopharmaceutical company developing therapeutics based on NGLY1 biology. Its lead asset GS-100 is an AAV9 gene therapy delivering the full-length human NGLY1 protein to treat the ultra-rare genetic disease NGLY1 Deficiency, currently in a Phase 1/2/3 clinical trial. The company is also advancing small molecule programs (GS-310 for neurodegeneration and GS-220/GS-221 for oncology) that target NGLY1 to indirectly shut down the NFE2L1 transcription factor.

Product overview

Grace Science is a biotechnology company developing a pipeline of therapeutics based on NGLY1 biology. The company's portfolio includes GS-100, an AAV9 gene therapy for NGLY1 Deficiency (the lead program currently in Phase 1/2/3 clinical trials), and multiple small molecule programs including GS-310 for neurodegenerative diseases and GS-220/GS-221 for oncology. The company leverages its deep understanding of the NGLY1 pathway to develop treatments across rare and common diseases in Oncology and Neurodegeneration. The company is agnostic to therapeutic modality, developing both gene therapies and small molecules.

Differentiator

Problem solved

Functional benefit

Products and services

• GS-100 An AAV9 single-stranded viral vector gene therapy that encodes the full-length human NGLY1 protein, being developed to treat NGLY1 Deficiency, an ultra-rare autosomal recessive disease that devastates the central nervous system. Patients in the ongoing Phase 1/2/3 trial (NCT06199531) treated for at least 52 weeks have demonstrated improvements in motor function (ability to sit, stand, or walk with assistance), cognitive skills, and caregiver interaction.
• GS-310 A small molecule program for neurodegenerative diseases leveraging research showing that NGLY1 touches many of the same pathways impacted in common neurodegenerative diseases such as Parkinson's disease.
• GS-220 A small molecule inhibitor program being developed to treat undisclosed cancers. The target of inhibition is NGLY1, which in turn shuts down NFE2L1, a critical transcription factor that is difficult to inhibit directly.
• GS-221 A small molecule inhibitor program being developed to treat undisclosed cancers. The target of inhibition is NGLY1, which in turn shuts down NFE2L1, a critical transcription factor that is difficult to inhibit directly.

Quantifiable outcome

• Patients treated with GS-100 for at least 52 weeks demonstrated improvements in motor function including ability to sit, stand, or walk with assistance, as well as improvements in cognitive skills and caregiver interaction

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

One partnership is on record.

• TakedacoreStrategic or Co-development PartnerTakeda collaboration mentioned on Grace Science website as a key partnership, with video content titled 'IT TAKES ONE LITTLE SPARK' highlighting the collaboration. Nature of collaboration not detailed in available sources but represents a significant pharma partnership for a biotech of this stage.

Scale indicators3 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Regenxbio: AAV gene therapy platform company with approved and clinical-stage assets for rare diseases; comparable in AAV vector technology and rare disease gene therapy development focus, including CNS indications similar to GS-100.
• Ultragenyx Pharmaceutical: Commercial and clinical-stage rare disease company with multiple modalities including gene therapy; comparable in ultra-rare genetic disease focus, regulatory designation strategy, and platform approach across multiple rare indications.
• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments with CNS-focused programs; comparable in AAV vector platform technology and neurodegenerative disease pipeline overlap with Grace Science's GS-310 program.
• Taysha Gene Therapies: Clinical-stage AAV gene therapy company focused on rare and ultra-rare CNS diseases; directly comparable in CNS-targeted AAV gene therapy, ultra-rare patient population, and clinical execution profile.
• uniQure: Clinical-stage AAV gene therapy company developing treatments for rare genetic diseases including CNS-targeted programs; comparable in modality (AAV9-class vectors), ultra-rare indication focus, and regulatory milestones like RMAT.
• Sarepta Therapeutics: Commercial-stage gene therapy company targeting ultra-rare genetic neuromuscular diseases with AAV-based modalities (Elevidys); directly comparable in regulatory pathway, patient population dynamics, and orphan pricing model for an AAV gene therapy.
• Solid Biosciences: Clinical-stage gene therapy company developing AAV-based treatments for rare neuromuscular genetic diseases; comparable in AAV modality, ultra-rare patient population, and clinical-stage development profile.

Broad incumbents

• BioMarin Pharmaceutical: Established rare-disease biotech with approved enzyme replacement and gene therapies; multiple Grace Science leaders (CSO, SAB, Senior Advisor) came directly from BioMarin's rare disease and gene therapy programs, making it the most comparable incumbent operating model.
• Pfizer: Major pharmaceutical company with active gene therapy programs (including rare disease AAV-based assets from the Beam/Sangamo collaborations and prior rare disease acquisitions); comparable as a broad incumbent that competes for rare disease gene therapy assets and partnerships.
• Takeda Pharmaceutical: Global pharmaceutical company with a significant rare disease and gene therapy franchise and an active strategic collaboration with Grace Science; comparable as both a partnership counterparty and as a broader incumbent that has invested in rare disease gene therapy.

Market position

Strengths4 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/grace-science-llc

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds1 record

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Grace Science