What Grace Science Foundation does

Grace Science Foundation is a private nonprofit research foundation founded in 2013 in San Francisco by Matt and Kristen Wilsey after their daughter Grace was diagnosed with NGLY1 Deficiency, an ultra-rare genetic disorder. The foundation's mission is to coordinate and fund research aimed at curing NGLY1 Deficiency, an endeavor that traditional biomedical funding mechanisms have neglected due to the disease's extreme rarity — approximately 130 patients have been confirmed worldwide as of February 2024. The foundation operates as a patient-driven convening organization, building a global research network of 20+ teams and ~75 scientists across 20 medical centers in the USA, Canada, Germany, Italy, and Japan. Its core "products" are the NGLY1 Patient Registry, a family community platform, research grantmaking, and a translational for-profit affiliate — Grace Science, LLC — co-founded with Nobel Laureate Dr. Carolyn Bertozzi to convert academic discoveries into drug candidates.

The foundation's technology base is biomedical rather than digital: it supports research using iPS cell platforms (Gladstone/Dr. Yamanaka), glycan biochemistry (RIKEN/Dr. Suzuki), Drosophila and C. elegans models (Baylor, Harvard/MGH), mouse models (Jackson Laboratory), high-throughput genetic interaction mapping (University of Toronto), and stem cell biology (Salk, Stanford). The foundation channels donations into these academic collaborations and into the Takeda T-CiRA industrial drug discovery program, with no commercial revenue of its own. The 100%-of-donations-to-research commitment is a primary donor acquisition claim, marketed through patient stories, blog content, earned media in Wired, Nature, The New Yorker, CNN, and Stanford, and community events such as 'Run Across Tennessee.'

Revenue is entirely donation-based and not publicly disclosed. The foundation is led by Chairman/President/Co-founder Matt Wilsey, a Stanford-educated entrepreneur and former KKR and CardSpring executive; Co-founder Kristen Wilsey; Chief Scientific Officer Kevin Lee; and Clinical Research & Patient Support Manager Gracie Morrison. The organization is structurally a patient advocacy foundation with a translational for-profit arm, positioning it at the intersection of nonprofit research coordination and venture-style drug development.

Name

Grace Science Foundation

Legal name

Grace Science Foundation

Website

https://gracescience.org

Company type

Private

Founded year

2013

Operating status

Operating

Headcount range

51–100 employees

Short description

Grace Science Foundation is a San Francisco-based nonprofit founded in 2013 that funds and coordinates a global research network of 20+ teams to develop treatments for NGLY1 Deficiency, an ultra-rare genetic disorder affecting approximately 130 patients worldwide.

Ownership category

akta.pro rank

Product category

Rare Disease Research Foundation

NAICS

Scientific Research and Development Services (5417), Voluntary Health Organizations (813212)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Research & Science Grantmaking Foundations (BPAGAKAI)

akta.pro secondary industries

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA), Health & Medical Research Grantmaking Foundations (BPAGAKAL)

Keywords

• Rare disease research
• Genetic disorder research
• Patient registry services
• Medical research funding
• Drug development coordination

Headquarters

HQ city

San Francisco

HQ country

United States

HQ region

North America

Markets served

GTM type

B2C

Offering type

Services

Cost components

Technology or R&D, Personnel

Revenue model

1. Donations and Charitable Contributions: The foundation is funded entirely through donations, with 100% of donations going directly to finding a cure for NGLY1 Deficiency. No commercial revenue streams.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Grace Science Foundation is a nonprofit medical research foundation that coordinates and funds a global research network of scientists and clinicians to find treatments and a cure for NGLY1 Deficiency, an ultra-rare genetic disorder. It operates an NGLY1 Patient Registry and natural history study, supports roughly 75 scientists across 20 medical centers in 5 countries, and bridges basic discovery to therapeutic development via a translational entity (Grace Science LLC) co-founded with Nobel Laureate Dr. Carolyn Bertozzi. The foundation is funded entirely through donations, with a stated commitment that 100% of contributions go directly to research.

Product overview

Grace Science Foundation operates as a research-focused nonprofit organization rather than a traditional software company. Its primary offerings include: (1) an NGLY1 Patient Registry that collects patient data for clinical research; (2) Research Coordination Services funding over 20 research teams globally; (3) an NGLY1 Family Community Platform connecting patients and families; (4) Educational Resources and Blog providing disease and research information; and (5) a Donation Platform directing 100% of contributions to research. The foundation does not offer a unified software product but rather a constellation of programs supporting NGLY1 research and patient advocacy.

Differentiator

Problem solved

Functional benefit

Products and services

• NGLY1 Patient Registry A patient registry platform that collects and maintains longitudinal data from individuals diagnosed with NGLY1 Deficiency to support clinical research, natural history studies, and trial-readiness. Built for the global NGLY1 patient and family community (~130 confirmed patients worldwide as of February 2024).
• Research Coordination Services Coordination and direct funding of academic and industry research teams working on NGLY1 Deficiency biology, animal models, drug screening, and therapeutic development. The foundation has funded 20+ research teams at 20 medical centers in 5 countries (USA, Canada, Germany, Italy, Japan), supporting approximately 75 scientists.
• NGLY1 Family Community Platform Community support platform connecting families affected by NGLY1 Deficiency, providing peer support, patient stories, family resources, and a sense of community for an ultra-rare disease population spread across multiple countries.
• Donation Platform Online donation portal enabling supporters to contribute directly to NGLY1 research funding, with itemized giving levels (e.g., $100 funds an RNA-Seq library preparation, $250 funds 10 western blot protein gels, $500 funds 2 antibodies). The foundation states that 100% of donations go directly to research with no overhead deduction.

Quantifiable outcome

• 130 patients diagnosed and in registry as of February 2024
• +2 more outcomes

Named customers4 records

Segments1 record

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Twelve partnerships are on record, tiered core.

• Gladstone InstitutescoreStrategic or Co-development PartnerAcademic research partnership with Dr. Shinya Yamanaka (Nobel Laureate) and Dr. Deepak Srivastava. Dr. Yamanaka's iPS cell technology provides a platform for studying NGLY1 Deficiency using patient-derived cells. The Finkbeiner Lab collaborated on NGLY1 Deficiency phenotypes research.
• Stanford UniversitycoreStrategic or Co-development PartnerMultiple research collaborations with Stanford labs including Bertozzi Lab (Dr. Carolyn Bertozzi, Nobel Laureate), Dr. Greg Enns (Biochemical Genetics), Dr. Katherine Mackenzie (Pediatric Neurology), Dr. Michael Snyder (Genetics), and Dr. Maria Grazia Roncarolo (Stem Cell Transplantation).
• RIKENcoreStrategic or Co-development PartnerCollaboration with Glycometabolome Team at RIKEN led by Dr. Tadashi Suzuki. Research focuses on NGLY1 enzyme function, free-glycans analysis, and drug screening for ENGase inhibitors. Dr. Chengcheng Huang and Dr. Haruhiko Fujihira conduct research at RIKEN.
• Takeda Pharmaceutical (T-CiRA)coreStrategic or Co-development PartnerT-CiRA is a joint program of Takeda, CiRA, and RIKEN. Dr. Ryuichi Tozawa, Dr. Takayuki Kamei, and Dr. Makoto Asahina work on NGLY1 research through this partnership, focusing on drug discovery and therapeutic development.
• Baylor College of MedicinecoreStrategic or Co-development PartnerResearch collaboration with Dr. Hamed Jafar-Nejad using Drosophila models to understand NGLY1's role in animal development and identify drug targets. Dr. Ashutosh Pandey also conducts research at BCM.
• Massachusetts General Hospital (Harvard)coreStrategic or Co-development PartnerDr. Gary Ruvkun's lab conducts research on protein degradation mechanisms and NGLY1's role. Dr. Nicolas Lehrbach studies genetic modifiers of proteasome function in NGLY1 mutant C. elegans models.
• UT Southwestern Medical CentercoreStrategic or Co-development PartnerDr. Nan Yan's lab investigates innate immune responses associated with NGLY1 deficiency. Dr. Kun Yang characterizes immune phenotypes and explores therapeutic mechanisms.
• Virginia Commonwealth UniversitycoreStrategic or Co-development PartnerDr. Senthil Radhakrishnan's lab studies cellular protein degradation pathways with focus on the Nrf1-proteasome axis and translates findings into therapies for NGLY1 disease.
• The Jackson LaboratorycoreStrategic or Co-development PartnerMouse model development for human neuromuscular diseases. Dr. Orsolya Kiraly develops and optimizes mice models for NGLY1 research. Dr. Lorin Roiphe contributes genetics expertise.
• University of TorontocoreStrategic or Co-development PartnerDr. Charlie Boone's lab conducts high-throughput systems level genetic analyses and genetic interaction network mapping relevant to NGLY1 research.
• Salk InstitutecoreStrategic or Co-development PartnerDr. Rusty Gage's lab models human neurological disease using stem cells and studies genomic mosaicism relevant to NGLY1 Deficiency research.
• Grace Science LLCcoreStrategic or Co-development PartnerTranslational entity co-founded by Matt Wilsey and Nobel Laureate Dr. Carolyn Bertozzi to translate scientific discoveries into real-world treatments, bridging the gap between discovery and patient access.

Scale indicators4 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Cure SMA: Family-driven rare disease foundation that funded research leading to FDA-approved spinal muscular atrophy therapies (Spinraza, Zolgensma, Evrysdi). Direct peer for Grace Science given shared ultra-rare pediatric genetic disease model and proven pipeline-to-treatment track record.
• Chordoma Foundation: Patient-founded foundation funding and coordinating research for an ultra-rare cancer, including translational research programs and partnerships with academic medical centers. Structurally similar to Grace Science in coordinating fragmented research for a low-prevalence disease.
• Parent Project Muscular Dystrophy (PPMD): Patient-driven nonprofit funding Duchenne muscular dystrophy research globally, with parallel translational and advocacy operations. Highly comparable to Grace Science in mission, structure, and operating model for ultra-rare genetic disease research coordination.
• Alex's Lemonade Stand Foundation: Pediatric cancer research foundation funding research grants and clinical trials. Comparable to Grace Science in philanthropic fundraising model, research coordination, and pediatric disease focus, though operating at substantially greater scale and a longer funding history.
• cureSanfilippo Foundation: Parent-led foundation funding research to cure Sanfilippo Syndrome, an ultra-rare pediatric genetic disorder. Comparable to Grace Science in mission, scale, parent-driven origin story, and direct research funding approach.
• Foundation for Prader-Willi Research: Family-driven rare disease research foundation funding therapeutic development for Prader-Willi syndrome. Highly comparable to Grace Science in ultra-rare genetic disease focus, parent-led origin, and research grant-making operating model.

Broad incumbents

• Global Genes: Advocacy and support organization for the broader rare disease community, providing resources, networking, and educational content across many conditions. Functionally comparable to Grace Science in rare disease community-building and awareness, though operates as an umbrella rather than single-disease funder.
• Cystic Fibrosis Foundation: Large disease-focused foundation that funded foundational CF research and partnered with Vertex to develop transformative therapies, generating substantial royalty revenues. A scaled-up version of Grace Science's intended research-to-treatment translation model, though operating at far greater scale and financial footprint.
• National Organization for Rare Disorders (NORD): Umbrella advocacy organization for rare diseases in the US, providing patient assistance programs and policy advocacy. Comparable in rare disease mission space and policy influence but operates across many conditions rather than a single disease.

Emerging players

• RARE-X: Nonprofit data platform focused on building federated patient registries for rare diseases. Comparable to Grace Science's patient registry initiative but operates as an infrastructure platform across many conditions rather than for a single disease.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• YouTubeyoutube.com/channel/UCXT577tGg3sLkwnakcDKXHA
• Xx.com/gracescience
• Facebookfacebook.com/CureNGLY1
• LinkedInlinkedin.com/company/grace-science-foundation

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Grace Science Foundation