Beyond Batten Disease Foundation
Beyond Batten Disease Foundation is a 501(c)(3) nonprofit that funds research and advances Batten-1 (miglustat) clinical development to treat juvenile (CLN3) Batten disease, serving 800+ affected families through 11+ family-directed funds and pharmaceutical partnerships with THX Pharma and Biocodex.
- Company typePrivate
- Founded2008
- HeadquartersAustin, United States
- Headcount1–10
- GTM typeB2C
- OfferingServices
What Beyond Batten Disease Foundation does
Beyond Batten Disease Foundation (BBDF), founded in August 2008 in Austin, Texas, is a 501(c)(3) nonprofit foundation dedicated to eradicating juvenile (CLN3) Batten disease, a fatal childhood neurodegenerative disorder with no FDA-approved treatment. The foundation serves three interlocking constituencies: affected Batten families (800+ families supported through programs and 11+ family-directed sub-funds), Batten disease researchers, and prospective parents seeking genetic risk information. Its core technology assets include a comprehensive next-generation-sequencing carrier screening panel — co-developed with the National Center for Genome Resources covering Batten disease plus 400–600+ other rare genetic conditions and recognized as a Time Magazine Top 10 Medical Research Breakthrough in 2012 — and the largest-ever induced pluripotent stem cell collection for juvenile Batten disease (24 individuals), created in partnership with the New York Stem Cell Foundation.
BBDF's primary drug development focus is Batten-1 (miglustat), a clinical-stage treatment for CLN3 Batten disease developed in partnership with THX Pharma (formerly Theranexus). Batten-1 has produced positive Phase I/II results demonstrating preserved visual acuity and a 33% reduction in serum neurofilament light chain biomarker at 18 months, with FDA and EMA endorsement for Phase III planning. In February 2026, THX Pharma and Biocodex announced a strategic licensing agreement covering Batten-1 plus two other rare diseases, formalizing a commercialization pathway.
BBDF's revenue model is donation-based: it raises funds through individual and corporate gifts, grassroots community events (galas, golf tournaments, relay runs), crowdfunding pages, and directed family funds. Cumulative research investment exceeds $35 million since inception, funded by an estimated run-rate in the low-to-mid single-digit millions annually (exact figures undisclosed). The foundation operates with a lean four-person professional staff and is governed by an eight-member Board of Directors chaired by co-founder T. Craig Benson. Its go-to-market is community-led, anchored by family-volunteer fundraising networks distributed across multiple US states and, increasingly, international geographies through partnerships.
Beyond Batten Disease Foundation firmographics
Firmographics- Name
- Beyond Batten Disease Foundation
- Legal name
- Beyond Batten Disease Foundation
- Website
- https://beyondbatten.org
- Company type
- Private
- Founded year
- 2008
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Beyond Batten Disease Foundation is a 501(c)(3) nonprofit that funds research and advances Batten-1 (miglustat) clinical development to treat juvenile (CLN3) Batten disease, serving 800+ affected families through 11+ family-directed funds and pharmaceutical partnerships with THX Pharma and Biocodex.
- Ownership category
- akta.pro rank
Beyond Batten Disease Foundation industry classification
Industry- Product category
- Rare Disease Research Foundation
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Services-Commercial Physical & Biological Research (8731)
- akta.pro primary industry
- Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)
- akta.pro secondary industry
- Health & Medical Research Grantmaking Foundations (BPAGAKAL)
Keywords
Where Beyond Batten Disease Foundation is headquartered
LocationHeadquarters
- HQ city
- Austin
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Beyond Batten Disease Foundation business model
Business model- GTM type
- B2C
- Offering type
- Services
- Cost components
- Personnel, Operations, Marketing or Sales, Technology or R&D, Others
Revenue model
- Donations and Fundraising: Voluntary donations from individuals, corporations, and foundations. Fundraising through events (galas, golf tournaments, relay runs), crowdfunding pages, and major gift campaigns.
- Carrier Screening Test Sales: Proceeds from sales of the comprehensive carrier screening genetic test panel, which is designed to generate sustainable funding for research efforts.
- Directed Family Funds: Affiliated family funds (Will Herndon Fund, Hugs for Hudson, Tyler’s Mission, Chase the Cure, etc.) that raise dedicated funds for Batten disease research through their own community efforts.
Go-to-market motion1 record
Distribution channels3 records
Marketing channels8 records
Beyond Batten Disease Foundation product offering
Product offeringCore offering
Beyond Batten Disease Foundation is a 501(c)(3) nonprofit foundation that funds research, advances drug development (notably Batten-1/miglustat through THX Pharma), and supports families affected by juvenile (CLN3) Batten disease. It also developed a comprehensive genetic carrier screening test panel covering Batten disease and 400-600+ rare genetic conditions through its partnership with the National Center for Genome Resources.
Product overview
Beyond Batten Disease Foundation is a non-profit organization dedicated to eradicating juvenile Batten disease through research funding and drug development. The foundation's primary focus is advancing Batten-1 (miglustat), a drug candidate currently in clinical trials for CLN3 Batten disease treatment. Additionally, BBDF operates The Be Project fundraising campaign and the Batten Disease Global Research Initiative grant program. The organization has historically supported development of a carrier screening test for genetic mutation detection. BBDF partners with pharmaceutical companies including THX Pharma (formerly Theranexus) and Biocodex for drug development and commercialization.
Differentiator
Problem solved
Functional benefit
Brands
- Bind Up Batten: Family fund established by Jo and Mike Klein in 2018 for Batten disease research
- Chase the Cure
- Delaney's Dream
- Hugs for Hudson
- Iron Will
- The Isla Edwards Research Fund
- Nathan's Batten Battle
- Rare Sisters
- Sofia's Dream
- Tyler's Mission
- Wheeler's Warriors Research Fund
- Will Herndon Fund (HOPE)
- Batten-1
- BE Project
Products and services
- Carrier Screening Test
- Batten-1 (miglustat)
Quantifiable outcome
- FDA Approved Clinical Trial for Batten-1 treatment
- +3 more outcomes
Companies that use Beyond Batten Disease Foundation
Customer profileNamed customers13 records
Segments3 records
Ideal customer profiles4 records
Beyond Batten Disease Foundation technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Beyond Batten Disease Foundation partnerships and signals
Strategic signalPartnerships
Eight partnerships are on record, tiered core and minor.
- BiocodexcoreBiocodex is an independent international pharmaceutical group and long-time pioneer in the treatment of rare pediatric diseases. In February 2026, THX Pharma and Biocodex announced a strategic licensing agreement covering three rare diseases including Batten disease with Batten-1, and Gaucher disease and Niemann-Pick disease type C with TX01. BBDF is a key partner in this arrangement for the Batten disease component.
- THX Pharma (formerly Theranexus)coreTHX Pharma (formerly Theranexus) is a biopharmaceutical company specializing in rare neurological diseases. BBDF has partnered with THX Pharma since 2020 to develop and advance Batten-1 (miglustat) through clinical trials. THX Pharma rebranded from Theranexus in September 2025 as part of strategic evolution toward commercialization. The partnership includes positive Phase I/II trial results and ongoing Phase III trial planning with FDA and EMA endorsement.
- National Organization for Rare Disorders (NORD)minorNORD’s Batten Disease Premium & Copay Patient Assistance Program provides financial support to eligible Batten patients for out-of-pocket healthcare costs. BBDF promotes this assistance program to families.
- Batten Disease Global Research Initiative (BDGRI)minorBDGRI announced inaugural research grant recipients in April 2025, awarding $320,000 to six groundbreaking projects worldwide. BBDF participates in this collaborative research funding initiative with other Batten disease organizations.
- New York Stem Cell Foundation (NYSCF)corePartnership to develop the largest-ever collection of induced pluripotent stem (iPS) cells for juvenile Batten (CLN3) disease research. NYSCF created stem cell lines from 24 individuals including affected patients and family members using the NYSCF Global Stem Cell Array technology. The collection is available to researchers worldwide for studying disease mechanisms and screening compounds.
- Texas Children’s Hospital / Jan and Dan Duncan Neurological Research InstitutecoreBBDF partnered with Texas Children’s Hospital and Baylor College of Medicine to establish research programs at the Jan and Dan Duncan Neurological Research Institute. In 2009, BBDF contributed $500,000 (combined with $2M from Cherie and James Flores) to bring Dr. Andrea Ballabio and Dr. Marco Sardiello from Italy to conduct research. The partnership has led to key discoveries including the TFEB master gene and advancement of Batten disease research.
- National Center for Genome Resources (NCGR)corePartnership with NCGR in Santa Fe, New Mexico to develop the comprehensive carrier screening test using next-generation DNA sequencing technology. NCGR led technical development of the test that can detect mutations for Batten disease and 400-600+ other rare genetic conditions. BBDF invested $1.5 million with NCGR for test development.
- Batten Disease Support & Research Association (BDSRA) FoundationcoreBBDF works with BDSRA (both US and Australia chapters) for family support, research coordination, and advocacy. Dr. Ineka Whiteman serves as both BBDF Scientific Consultant and Head of Research and Medical Affairs for BDSRA Australia.
Scale indicators5 records
Recent moves6 records
Expansion highlights5 records
Beyond Batten Disease Foundation competitors and assessment
Company assessmentMarket position
Competitive moat4 records
Key risks6 records
Key highlights7 records
Customer concentration
Beyond Batten Disease Foundation social profiles
Digital presenceBeyond Batten Disease Foundation financial estimates
Financial estimateRevenue estimate
Valuation estimate
Beyond Batten Disease Foundation leadership team
Management profileNumber of profiles
Profiles12 records
Beyond Batten Disease Foundation funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Beyond Batten Disease Foundation M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Beyond Batten Disease Foundation
What does Beyond Batten Disease Foundation do?
Beyond Batten Disease Foundation is a 501(c)(3) nonprofit foundation that funds research, advances drug development (notably Batten-1/miglustat through THX Pharma), and supports families affected by juvenile (CLN3) Batten disease. It also developed a comprehensive genetic carrier screening test panel covering Batten disease and 400-600+ rare genetic conditions through its partnership with the National Center for Genome Resources.
Is Beyond Batten Disease Foundation a public or private company?
Beyond Batten Disease Foundation is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was Beyond Batten Disease Foundation founded?
Beyond Batten Disease Foundation was founded in 2008. It employs 1 to 10 people.
Where is Beyond Batten Disease Foundation based?
Beyond Batten Disease Foundation is headquartered in Austin, United States, in the North America region.
How does Beyond Batten Disease Foundation make money?
Three revenue lines are on record. Donations and Fundraising is the primary driver. The others are carrier Screening Test Sales and directed Family Funds.
Does Beyond Batten Disease Foundation have an API?
No public API is recorded for Beyond Batten Disease Foundation.
What industry is Beyond Batten Disease Foundation in?
Beyond Batten Disease Foundation's product category is Rare Disease Research Foundation. Its primary akta.pro industry code is BPAGACAA, Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS), with a secondary code of BPAGAKAL, Health & Medical Research Grantmaking Foundations. Its NAICS code is 541714 and its SIC code is 8731.