Cure SMC1A Foundation
Cure SMC1A Foundation is a 501(c)(3) nonprofit founded in 2021 by parents of children with SMC1A-related Developmental and Epileptic Encephalopathy, operating patient registries, biosample coordination, and seed research grants to accelerate treatments for this ultra-rare genetic disorder.
- Company typePrivate
- Founded2021
- HeadquartersSan Francisco, United States
- Headcount1–10
- GTM typeB2C
- OfferingServices
What Cure SMC1A Foundation does
Cure SMC1A Foundation is a 501(c)(3) nonprofit charitable organization (EIN 84-3406755), legally named SMC1A Foundation, founded in 2021 by parents of children diagnosed with SMC1A-related Developmental and Epileptic Encephalopathy (DEE) — an X-linked genetic disorder characterized by loss-of-function in the SMC1A gene and resulting in intractable seizures and developmental impairment. The foundation operates from San Francisco, California with a European Chapter in Pisa, Italy, and serves two primary constituents: SMC1A patient families (who participate in registries, community programs, and fundraising) and SMC1A researchers (who access de-identified patient data, biosamples, and disease models). Its core product is not a commercial offering but a coordinated suite of research-enablement services: the SMC1A Patient Registry, the Natural History Registry, the Family Contact Registry, and a Community Needs Survey, built on third-party infrastructure including QuestionPro, CoRDS (Coordination of Rare Diseases at Sanford), and Ciitizen (Rare Patient Network).
The foundation's technology layer extends to biosample coordination, facilitating researcher access to patient-derived lymphoblastoid cell lines (LCLs), iPSCs, fibroblasts, brain organoids, and SMC1A knockout mouse models through partnerships with CHOP, Coriell, NIGMS Repository, GemPharmaTech, and Jax Lab. It operates a For Researchers portal and a Foundation-managed Data Manager role focused on transforming registry data into 'decision-grade' assets for global pharmaceutical partners. To date, it has funded two disclosed seed research grants: a $31,000 award to Seattle Children's/UW for molecular etiology work and iPSC/NPC disease modeling, and a $182,935 award to UC Davis for CRISPR-based interventional genetics and gene therapy approaches, the latter co-funded by two patient families.
The business model is philanthropy-only: the foundation generates no commercial revenue, with 100% of donations directed toward SMC1A research and operations. Fundraising is conducted through GoFundMe, PayPal Giving Fund, an eBay storefront, ShopRaise, Bonfire merchandise, employer matching, and social-media-native fundraisers. Go-to-market is community-led, acquiring supporters through Facebook, Twitter/X, LinkedIn, Instagram, YouTube, and TikTok, and converting them via email communications and a gated Facebook parent community. It earned a 2026 Candid Platinum Seal of Transparency, indicating the highest tier of nonprofit accountability. Headcount is reported at 1–10, with all team members being parents of affected children serving in largely volunteer or part-time capacities, including a Medical Advisory Board from UCSF and the Keck School of Medicine of USC.
Cure SMC1A Foundation firmographics
Firmographics- Name
- Cure SMC1A Foundation
- Legal name
- SMC1A Foundation
- Website
- https://smc1a-epilepsy.org
- Company type
- Private
- Founded year
- 2021
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Cure SMC1A Foundation is a 501(c)(3) nonprofit founded in 2021 by parents of children with SMC1A-related Developmental and Epileptic Encephalopathy, operating patient registries, biosample coordination, and seed research grants to accelerate treatments for this ultra-rare genetic disorder.
- Ownership category
- akta.pro rank
Cure SMC1A Foundation industry classification
Industry- Product category
- Patient Advocacy and Rare Disease Research Foundation
- NAICS
- Voluntary Health Organizations (813212), Scientific Research and Development Services (5417)
- SIC
- Services-Membership Organizations (8600), Services-Commercial Physical & Biological Research (8731), Services-Misc Health & Allied Services, Nec (8090)
- akta.pro primary industry
- Health & Medical Research Grantmaking Foundations (BPAGAKAL)
- akta.pro secondary industries
- Patient Registries & Chronic Disease Registry Analytics (HLACAHAH), Biobank & Biospecimen Management LIMS (HLACAMAG)
Keywords
Where Cure SMC1A Foundation is headquartered
LocationHeadquarters
- HQ city
- San Francisco
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Cure SMC1A Foundation business model
Business model- GTM type
- B2C
- Offering type
- Services
- Cost components
- Others, Technology or R&D, Marketing or Sales, Personnel, Operations
Revenue model
- Donations and Fundraising: The foundation operates as a 501(c)3 nonprofit that collects donations entirely dedicated to funding SMC1A research studies. Fundraising channels include GoFundMe, PayPal Giving Fund, eBay merchandise sales, ShopRaise, Facebook fundraisers, TikTok fundraisers, Instagram fundraisers, and employer matching programs. All donations are tax-deductible.
Go-to-market motion1 record
Distribution channels6 records
Marketing channels12 records
Cure SMC1A Foundation product offering
Product offeringCore offering
The SMC1A Foundation operates patient registries (Natural History Registry, Family Contact Registry, Community Survey) built on third-party platforms to collect disease progression, family engagement, and community needs data for SMC1A-DEE research. The foundation funds seed grants for SMC1A gene therapy and precision medicine studies at academic research institutions, and facilitates researcher access to biosamples (cell lines, iPSCs, brain organoids, animal models) and de-identified patient data.
Product overview
SMC1A Foundation is a patient-led non-profit organization (501(c)3) focused on advancing research for SMC1A-related Developmental and Epileptic Encephalopathy. Rather than a software product company, the Foundation operates a suite of patient registry and community services built on third-party platforms including QuestionPro, CoRDS (Sanford Research), and Ciitizen. The core offerings consist of the SMC1A Patient Registry (collecting baseline disease data), Natural History Registry (longitudinal progression tracking), Family Contact Registry (direct family engagement), and Community Survey (needs assessment). These interconnected registries enable researchers to access de-identified patient data while facilitating the Foundation's mission to accelerate research, build an active research pipeline, and develop meaningful treatments for SMC1A disorders. The Foundation also coordinates bio sample access, maintains a researcher portal, and fosters community connection among affected families worldwide.
Differentiator
Problem solved
Functional benefit
Products and services
- SMC1A Patient Registry
Quantifiable outcome
- Funded $31,000 seed grant for iPSC and NPC disease modeling study at Seattle Children's/UW
- +2 more outcomes
Companies that use Cure SMC1A Foundation
Customer profileNamed customers5 records
Segments3 records
Ideal customer profiles3 records
Cure SMC1A Foundation technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Cure SMC1A Foundation partnerships and signals
Strategic signalPartnerships
Eleven partnerships are on record, tiered core and supporting.
- CoRDS (Coordination of Rare Diseases at Sanford)coreLargest centralized international patient registry for all rare diseases. CoRDS collects long-term disease data tracking individuals from childhood to adulthood. SMC1A Foundation participates to advance international patient data collection for research.
- Ciitizen (Citizen Health)corePlatform advancing DEE research by collecting and analyzing data extracted from medical records. US families only. Collects records, de-identifies data, and organizes for researcher/pharmaceutical sharing.
- CHOP (Children's Hospital of Philadelphia)supportingLCLs (Lymphoblastoid Cell Lines) donated to Dr. Ian D. Krantz for biobanking and research distribution.
- CNR Italy (Institute of Technologies Biomedical, National Research Council)coreDr. Antonia Musio leads European research partnership studying dysregulated genes in SMC1A epileptic girls and investigating Ataluren safety/efficacy. Located in Pisa, Italy.
- UW/Seattle Children's (ISCRM)coreDr. Xinxian Deng's lab at ISCRM generated iPSCs and disease-mimicking brain organoids from patient cells. Core recipient of foundation seed grants.
- Coriell InstitutesupportingBiobank providing iPSCs and fibroblast cell lines for SMC1A research upon request.
- GemPharmaTechsupportingDistributes conditional SMC1A knockout mouse strain for preclinical disease research.
- Jax LabsupportingProvides SMC1A mouse model for research studies on disease aspects.
- Rare Epilepsy NetworksupportingNetwork partner for rare epilepsy patient advocacy and research collaboration.
- NIGMS RepositorysupportingResearch biobank collecting blood/tissue samples and creating cell lines and DNA for scientists. Accepts SMC1A family donations.
- UC Davis Regenerative Medicine Stem Cell ProgramcoreKyle Fink, PhD and Julian Halmai, PhD conducting interventional genetics research for SMC1A gene therapy and x-activation approaches. Co-funded by foundation, family Lucia Riestra, and Ricardo Javier Rodriguez.
Scale indicators4 records
Recent moves6 records
Expansion highlights6 records
Cure SMC1A Foundation competitors and assessment
Company assessmentBroad incumbents
- RARE-X: Federated rare disease data platform that aggregates patient-reported data across multiple disease foundations. SMC1A Foundation's Ciitizen partnership overlaps with RARE-X's mission, making it a broad incumbent in the patient-data layer the foundation depends on.
- Genetic Alliance: Network of disease-specific foundations supporting genetic disease research and patient engagement. Comparable as a peer ecosystem organization that connects ultra-rare disease foundations like SMC1A to shared infrastructure and policy.
- National Organization for Rare Disorders (NORD): Umbrella advocacy and research-funding organization for the broader rare disease community. Comparable as a macro-level counterpart that competes for philanthropic dollars and provides grant programs (e.g., Rare Disease Day) that rare disease foundations like SMC1A participate in.
- EveryLife Foundation for Rare Diseases: Rare disease policy and advocacy foundation that works to accelerate therapeutic development across many individual rare disease communities. Comparable as a policy/advocacy counterpart to SMC1A Foundation's awareness and ICD-10 coding efforts.
Others
- Orphan Disease Center (University of Pennsylvania): Academic center that administers rare disease pilot grants and therapeutic development programs. Comparable as a counterpart funder of seed-stage SMC1A-relevant research and a potential co-funder or partner for the foundation's grant programs.
- Coriell Institute for Medical Research: Biobank and research institute that provides iPSC and fibroblast cell lines to SMC1A Foundation researchers. Comparable as a critical infrastructure partner that supplies the biosamples the foundation helps distribute.
Direct peers
- SCN1A Foundation: Foundation focused on SCN1A-related disorders, another severe genetic developmental and epileptic encephalopathy. Comparable as a small, parent-led rare epilepsy foundation that funds research and builds patient registries.
- Dravet Syndrome Foundation: Foundation dedicated to Dravet syndrome, a severe genetic developmental and epileptic encephalopathy. Directly comparable to SMC1A Foundation in disease scope (DEE), patient registry infrastructure, and seed grantmaking for genetic epilepsies.
- Cure SMA: Patient-led nonprofit that funded and accelerated the SMA therapeutic pipeline (including Spinraza and Zolgensma). Closely comparable to SMC1A Foundation in model: rare genetic disease, parent-driven, combines patient registry with seed grantmaking for curative therapies.
- CdLS Foundation: Foundation for Cornelia de Lange Syndrome, a related cohesinopathy that overlaps with SMC1A mutations. Comparable as a sibling-disease foundation sharing scientific frameworks, researchers, and historically some of the same donor families.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks6 records
Key highlights7 records
Customer concentration
Cure SMC1A Foundation social profiles
Digital presenceCure SMC1A Foundation financial estimates
Financial estimateRevenue estimate
Valuation estimate
Cure SMC1A Foundation leadership team
Management profileNumber of profiles
Profiles9 records
Cure SMC1A Foundation funding detail
Funding detailFunding overview
Funding rounds
Investors
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Cure SMC1A 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 Cure SMC1A Foundation
What does Cure SMC1A Foundation do?
The SMC1A Foundation operates patient registries (Natural History Registry, Family Contact Registry, Community Survey) built on third-party platforms to collect disease progression, family engagement, and community needs data for SMC1A-DEE research. The foundation funds seed grants for SMC1A gene therapy and precision medicine studies at academic research institutions, and facilitates researcher access to biosamples (cell lines, iPSCs, brain organoids, animal models) and de-identified patient data.
Is Cure SMC1A Foundation a public or private company?
Cure SMC1A Foundation is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was Cure SMC1A Foundation founded?
Cure SMC1A Foundation was founded in 2021. It employs 1 to 10 people.
Where is Cure SMC1A Foundation based?
Cure SMC1A Foundation is headquartered in San Francisco, United States, in the North America region.
How does Cure SMC1A Foundation make money?
One revenue line is on record: donations and Fundraising.
Who are Cure SMC1A Foundation's main competitors?
Broad incumbents on record are RARE-X, Genetic Alliance, National Organization for Rare Disorders (NORD) and EveryLife Foundation for Rare Diseases. Others are Orphan Disease Center (University of Pennsylvania) and Coriell Institute for Medical Research. Direct peers are SCN1A Foundation, Dravet Syndrome Foundation, Cure SMA and CdLS Foundation.
Does Cure SMC1A Foundation have an API?
No public API is recorded for Cure SMC1A Foundation.
What industry is Cure SMC1A Foundation in?
Cure SMC1A Foundation's product category is Patient Advocacy and Rare Disease Research Foundation. Its primary akta.pro industry code is BPAGAKAL, Health & Medical Research Grantmaking Foundations, with a secondary code of HLACAHAH, Patient Registries & Chronic Disease Registry Analytics. Its NAICS code is 813212 and its SIC code is 8600.