The Charlotte and Gwenyth Gray Foundation

What The Charlotte and Gwenyth Gray Foundation does

The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease is a federally recognized 501(c)(3) public charity founded in 2015 by Kristen and Gordon Gray after their daughters Charlotte and Gwenyth were diagnosed with CLN6 Batten disease. The foundation exists to improve and save the lives of children affected by Batten disease and other neurodegenerative disorders, with a primary focus on the ultra-rare CLN6 variant. Its core programmatic asset is the CLN6 Gene Therapy Program, an investigational treatment that uses a self-complementary adeno-associated virus serotype 9 (AAV9) vector to deliver the human CLN6 gene via intrathecal injection into cerebrospinal fluid, enabling the therapeutic to cross the blood-brain barrier and reach affected brain and spinal cord tissue. The program is supplemented by broader Batten disease research funding across cellular therapy, stem cell transplantation, and small molecule approaches, and includes a Building Blocks Scholarship Fund that provides financial assistance to families for therapies, equipment, education, and rehabilitation.

The foundation coordinated and funded a first-of-its-kind Phase 1/2 clinical trial at Nationwide Children's Hospital from 2016 to 2020, which treated 15 children with the investigational AAV9-CLN6 gene therapy (AT-GTX-501) and demonstrated safety, tolerability, and reported stabilization of motor and language function. Building on those findings, the foundation announced a partnership in April 2025 with Rady Children's Health (formerly CHOC) to revitalize and enhance the CLN6 program, and in April 2026 established a manufacturing partnership with Genezen, a gene therapy contract development and manufacturing organization (CDMO), to produce GMP-grade viral vector supply for a new Phase 1/2 trial scheduled to begin in April 2026.

The foundation operates on a donation-based revenue model with no commercial product sales. Funding sources include individual donations (one-time and recurring) through the Zeffy online platform, a tiered corporate sponsorship program (Bronze $250, Silver $500, Gold $1,000, Platinum $2,500 per year), monthly child sponsorship programs for clinical trial participants, and high-visibility celebrity-driven fundraising campaigns supported by figures including Dwayne Johnson, Jennifer Garner, Julia Roberts, and Anne Hathaway. The foundation's Scientific Advisory Board includes David A. Pearce (President, Sanford Research), Clive Svendsen (Director, Cedars-Sinai Regenerative Medicine Institute), and Jill Weimer (Chief Science Officer, Amicus Therapeutics). It maintains affiliations with NORD, BDSRA Foundation, Global Genes, and emerging international CLN6 patient organizations. As of the latest reporting, the foundation has raised $11.1M cumulatively since inception, organized 115 community events, and operates with 1-10 employees from Pacific Palisades, California.

Name

The Charlotte and Gwenyth Gray Foundation

Legal name

The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease

Website

https://www.curebatten.org/

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

1–10 employees

Short description

The Charlotte and Gwenyth Gray Foundation is a 501(c)(3) nonprofit founded in 2015 that funds AAV9-based gene therapy research and provides financial support to families affected by CLN6 Batten disease, a fatal ultra-rare neurodegenerative disorder affecting children.

Ownership category

akta.pro rank

Product category

Pediatric Rare Disease Research Foundation

NAICS

Voluntary Health Organizations (813212)

SIC

Services-Social Services (8300)

akta.pro primary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

Keywords

• Rare disease research
• Gene therapy funding
• Patient family support
• Nonprofit fundraising
• Clinical trial sponsorship

Headquarters

HQ city

Santa Monica

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Others

Revenue model

1. Individual Donations: Public donations through online platform (Zeffy), one-time and recurring gift options for general foundation operations and research funding.
2. Corporate Sponsorship Program: Tiered sponsorship program (Bronze $250, Silver $500, Gold $1000, Platinum $2500) with varying benefits including social media mentions, website recognition, and event participation.
3. Patient Support Programs: Building Blocks Scholarship Fund providing financial assistance to children with Batten CLN6 for therapies, equipment, and rehabilitation programs.
4. Clinical Trial Sponsorship: Monthly sponsorship program for families to fund pre-clinical costs, equipment rentals, housing, and travel for children participating in clinical trials.

Pricing tiers

Go-to-market motion1 record

Distribution channels3 records

Marketing channels6 records

Core offering

The foundation funds and coordinates scientific research into treatments for CLN6 Batten disease, most notably an investigational AAV9-mediated gene therapy delivered intrathecally. It complements this research mission with direct financial support programs for affected families, including the Building Blocks Scholarship Fund covering therapies, equipment, education, and rehabilitation, as well as The Gray Academy, an educational facility for children with neurological challenges.

Product overview

The foundation operates as a single unified charitable mission rather than a platform architecture. The core offerings center on the CLN6 Gene Therapy Program—an investigational AAV9-mediated gene therapy for CLN6 Batten disease—supported by broader Batten Disease Research Funding across cellular, small molecule, and gene therapy modalities. These are complemented by family support services including the Building Blocks Scholarship Fund and The Gray Academy educational facility. The Sponsorship Program enables charitable giving through tiered recognition levels.

Differentiator

Problem solved

Functional benefit

Products and services

• CLN6 Gene Therapy Program Investigational gene therapy program using adeno-associated virus serotype 9 (AAV9) vector to deliver the human CLN6 gene for treatment of CLN6 Batten disease, a rare neurodegenerative lysosomal storage disorder. Delivered via intrathecal injection into cerebrospinal fluid to target brain and spinal cord tissues.
• Batten Disease Research Funding Funding and coordination of scientific research across cellular therapy, small molecule therapy, and gene therapy approaches to identify treatments for Batten disease beyond the CLN6 variant. Includes validation of novel therapeutics using FDA-approved drugs, GRAS nutraceuticals, and designer molecules tested on patient cells.
• Family Financial Support Programs Family Financial Support Programs Building Blocks Scholarship Fund and related financial assistance programs providing financial support to families of children with Batten CLN6 for therapies, equipment, education, rehabilitation, and overall patient wellbeing. Includes a monthly sponsorship program funding pre-clinical costs, equipment rentals, housing, and travel for clinical trial participants.
• The Gray Academy The Gray Academy Educational facility launched by the foundation for children with neurological challenges, providing therapeutic treatments on site and customized learning plans.
• Corporate Sponsorship Program Corporate Sponsorship Program Tiered annual corporate sponsorship program offering Bronze ($250), Silver ($500), Gold ($1,000), and Platinum ($2,500) levels with benefits including social media mentions on Facebook and Instagram, website business name and logo placement, URL redirect at Gold and Platinum, email mentions at Platinum, and special event recognition. Valid for one year.

Quantifiable outcome

• 15 children treated through investigational gene therapy clinical trial (2016-2020)
• +3 more outcomes

Named customers3 records

Segments2 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

14 partnerships are on record, tiered core, major and supporting.

• GenezencoreStrategic or Co-development Partner · 28 April 2026Manufacturing partnership for CLN6 gene therapy Phase 1/2 clinical trial. Genezen, a gene therapy CDMO, used its viral vector manufacturing platform and regulatory expertise to support rapid progression from proof of concept to GMP clinical supply. Direct FDA engagement to expedite path to clinic.
• Rady Children's HealthcoreStrategic or Co-development Partner · 1 April 2025Partnership with Rady Children's Health (formerly CHOC - Children's Hospital of Orange County) to revitalize and enhance the CLN6 gene therapy program. Builds on Phase 1/2 clinical trial findings from 2016-2020. Announced as part of foundation's 10-year milestone celebration.
• Nationwide Children's HospitalcoreStrategic or Co-development PartnerPrevious clinical trial partner (2016-2020) for initial Phase 1/2 clinical trial evaluating AT-GTX-501 (AAV9-CLN6 gene therapy). Trial demonstrated safety and efficacy with single intrathecal injection.
• Cedars-Sinai Medical CentermajorStrategic or Co-development PartnerAcademic medical center partner with Scientific Advisory Board member Clive Svendsen (Director of Board of Governors Regenerative Medicine Institute). Supports research in regenerative medicine approaches.
• Sanford ResearchmajorStrategic or Co-development PartnerResearch partner with Scientific Advisory Board members David A. Pearce (President) and Jill Weimer (Senior Director of Therapeutic Development). Dr. Weimer's lab initiated first gene therapy trials for CLN3 and CLN6 Batten disease.
• Amicus TherapeuticsmajorStrategic or Co-development PartnerBiotechnology company developing gene therapies. Scientific Advisory Board member Jill Weimer serves as Chief Science Officer. Company working on Phase 2 program following foundation-funded Phase 1 trial.
• BDSRA FoundationsupportingStrategic or Co-development PartnerBatten Disease Specialized Research Association partner providing resources and support for families affected by Batten disease. Co-affiliated organization in the Batten disease community.
• National Organization for Rare Disorders (NORD)supportingStrategic or Co-development PartnerAffiliation with major rare disease advocacy organization providing resources, advocacy support, and credibility for foundation activities.
• Global GenessupportingStrategic or Co-development PartnerRare disease advocacy organization partner providing resources and community support for families affected by rare diseases including Batten.
• University of Otago, Department of BiochemistrysupportingTechnology or IntegrationAcademic research partner from New Zealand contributing to Batten disease research efforts.
• Washington UniversitysupportingTechnology or IntegrationAcademic research partner contributing to Batten disease scientific research and development.
• Vanguard ClinicalsupportingTechnology or IntegrationClinical services partner supporting foundation activities and patient care programs.
• Munchkin Inc.supportingGTM or Marketing PartnerConsumer products company partnership announced to support foundation mission and raise awareness for Batten disease.
• The Gray AcademysupportingStrategic or Co-development PartnerEducational facility launched by foundation for children with neurological challenges, providing therapeutic treatments on site and customized learning plans.

Scale indicators7 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Batten Disease Support and Research Association (BDSRA): Largest US Batten disease patient advocacy and research funding organization; the Gray Foundation lists BDSRA as a co-affiliated partner, and both organizations fundraise for Batten families and CLN research.
• Cure SMA: Disease-specific 501(c)(3) nonprofit funding spinal muscular atrophy research and providing direct family support — same parent-founded, research-grant-plus-family-services model the Gray Foundation uses for Batten CLN6.
• Parent Project Muscular Dystrophy: Parent-led 501(c)(3) driving Duchenne muscular dystrophy research funding and patient advocacy; structurally analogous in mission, fundraising tactics, and ultra-rare disease focus.
• Friedreich's Ataxia Research Alliance (FARA): Parent- and patient-driven 501(c)(3) funding Friedreich's ataxia research with coordinated clinical trial sponsorship and family support programs — a near-identical operational template to the Gray Foundation.
• Children's Tumor Foundation: Long-standing 501(c)(3) funding neurofibromatosis research with clinical trial networks, family support, and direct-to-donor fundraising — comparable mission and donor engagement model.
• CureSearch for Children's Cancer: Pediatric disease-specific 501(c)(3) that funds research and supports families through childhood cancer treatment — comparable family-services-plus-research funding structure.

Broad incumbents

• National Organization for Rare Disorders (NORD): Umbrella advocacy organization for rare diseases with research grants, patient support programs, and policy work — listed by the Gray Foundation as an affiliation partner.
• Global Genes: Rare disease advocacy and education organization with RARE Conifer Grants and patient support programs; listed as an affiliation partner of the Gray Foundation.
• Amicus Therapeutics: Biotech developing gene therapies for rare diseases including Batten CLN6; Jill Weimer (Gray Foundation SAB) is its CSO, and Amicus is advancing a Phase 2 program that builds on foundation-funded Phase 1 data.
• V Foundation for Cancer Research: Celebrity-driven (ESPN/Jim Valvano) cancer research nonprofit that pioneered the high-profile celebrity-endorsement fundraising model the Gray Foundation applies to Batten disease.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/curebatten
• Facebookfacebook.com/fundacionbattencostarica

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about The Charlotte and Gwenyth Gray Foundation