Cure Rare Disease
Cure Rare Disease is a 501(c)(3) nonprofit biotechnology organization developing customized CRISPR, AAV, and antisense oligonucleotide gene therapies for ultra-rare neuromuscular and neurodegenerative diseases, serving patient populations too small for traditional pharmaceutical development.
- Company typePrivate
- Founded1998
- HeadquartersBoston, United States
- Headcount1–10
- GTM typeB2B and B2C
- OfferingServices
What Cure Rare Disease does
Cure Rare Disease (CRD) is a 501(c)(3) nonprofit clinical-stage biotechnology organization developing customized genetic therapies for ultra-rare neuromuscular and neurodegenerative diseases. Founded in 2017 (with the Horgan family's DMD journey originating in 1998) and headquartered in Woodbridge, Connecticut with a satellite presence in Boston, CRD operates with a 1–10 person core staff augmented by a deep network of academic and industry collaborators. Its mission is to address the structural market failure in rare disease drug development: an estimated 95% of the 7,000+ rare diseases have no treatment because commercial pharma cannot justify development costs for populations as small as 50 patients globally. CRD's pipeline targets Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophy subtypes 2b, 2g, and 2i/R9, spinocerebellar ataxia types 3 and 7, and ADSSL1-related myopathy.
The company runs a four-phase drug development engine spanning Research & Development, Prototype Development, Testing & Clinical Trials (FDA IND submissions), and Licensing/Approval, designed to compress timelines from years to months. Its technology stack is built on three complementary platforms: CRISPR/Cas9 gene editing (applied to DMD mutation correction), next-generation AAV gene therapy vectors featuring novel liver-detargeting, muscle-tropic capsids that reduce off-target hepatic distribution, and antisense oligonucleotides (ASOs) using steric-blocking splice-switching (e.g., exon 10 exclusion in ATXN3 for SCA3). CRD's DMD program received FDA approval for a first-in-human clinical trial; both SCA3 and LGMD2i/R9 programs hold FDA Orphan Drug Designation. The model combines academic research institutions (Yale, UCLA, Boston Children's Hospital, Leiden, University of Utah, VCU, SickKids Toronto) with industry service providers (Charles River, Axolabs, Mass Biologics, Form Bio) and patient foundations (LGMD2L Foundation, KOMD, Heal DMD).
CRD's revenue model is donation- and grant-based rather than commercial. Cumulative disclosed grant funding exceeds $20.79 million across recent awards from the California Institute for Regenerative Medicine (CIRM), the LGMD2L Foundation, the Muscular Dystrophy Association, HRSA, and the Hospital for Sick Children. Patient access occurs exclusively through FDA-regulated clinical trials and compassionate use, with no marketed products. The 2024 opening of a 10,000 sq ft laboratory facility in Woodbridge—partially funded by a $2M HRSA grant—has expanded internal R&D capacity while the broader 25,000 sq ft site includes space leased to other life sciences tenants. Go-to-market is community-led: patient families and foundations both fund programs and provide patient access, eliminating traditional pharma sales channels in favor of direct patient-researcher relationships.
Cure Rare Disease firmographics
Firmographics- Name
- Cure Rare Disease
- Legal name
- Cure Rare Disease
- Website
- https://cureraredisease.org
- Company type
- Private
- Founded year
- 1998
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Cure Rare Disease is a 501(c)(3) nonprofit biotechnology organization developing customized CRISPR, AAV, and antisense oligonucleotide gene therapies for ultra-rare neuromuscular and neurodegenerative diseases, serving patient populations too small for traditional pharmaceutical development.
- Ownership category
- akta.pro rank
Cure Rare Disease industry classification
Industry- Product category
- Rare Disease Genetic Therapeutics Development
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Voluntary Health Organizations (813212)
- SIC
- Services-Misc Health & Allied Services, Nec (8090)
- akta.pro primary industry
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)
- akta.pro secondary industries
- Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)
Keywords
Where Cure Rare Disease is headquartered
LocationHeadquarters
- HQ city
- Boston
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Cure Rare Disease business model
Business model- GTM type
- B2B and B2C
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Supply Chain, Marketing or Sales, Infrastructure
Revenue model
- Donations and Charitable Contributions: CRD operates as a 501(c)(3) nonprofit organization relying entirely on donations from individuals, families, and supporters. The organization hosts fundraising events and accepts standard donations, cryptocurrency donations, and donor advised fund contributions.
- Foundation and Grant Funding: CRD receives substantial grant funding from organizations including CIRM (California Institute for Regenerative Medicine), Muscular Dystrophy Association, and patient-led foundations like the LGMD2L Foundation. These grants fund specific therapeutic development programs.
- Collaborative Research Agreements: CRD partners with academic institutions and industry collaborators through sponsored research agreements. These collaborations may include funding from academic partners, in-kind contributions, and shared resource allocation for therapeutic development.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| Other | Other | No commercial products - all therapies are investigational |
Go-to-market motion1 record
Distribution channels2 records
Marketing channels5 records
Cure Rare Disease product offering
Product offeringCore offering
Cure Rare Disease is a nonprofit drug development organization that develops customized genetic therapies for ultra-rare neuromuscular and neurodegenerative diseases. It operates a four-phase development engine spanning research and development, prototype development, FDA-regulated clinical trials, and eventual licensing, using three core technology platforms: CRISPR gene editing, next-generation AAV (adeno-associated virus) vectors, and antisense oligonucleotides (ASOs). Patient access to investigational therapies occurs exclusively through clinical trials and compassionate use programs.
Product overview
Cure Rare Disease operates as a clinical-stage nonprofit biotechnology organization with a unified drug development platform designed to accelerate therapies for ultra-rare neuromuscular and neurodegenerative diseases. The organization employs three core technology platforms—CRISPR gene editing, next-generation AAV (adeno-associated virus) gene therapy vectors, and antisense oligonucleotides (ASOs)—which work together with their collaborative ecosystem model connecting academic researchers, patient families, and industry partners. Their pipeline includes therapeutic programs targeting Duchenne & Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy types 2b/2g/2i, Spinocerebellar Ataxia types 3 and 7, and ADSSL1 myopathy. The organization also provides preclinical research services including patient cell line development and CRISPR testing.
Differentiator
Problem solved
Functional benefit
Products and services
- CRD Drug Development Platform A nonprofit drug development engine that streamlines and de-risks the development of therapies for ultra-rare diseases through a collaborative model connecting academic researchers, patient families, rare disease organizations, and biotech/pharma partners, structured across four phases: Research & Development, Prototype Development, Testing & Clinical Trials, and Licensing/Approval.
- DMD CRISPR Cell Line Testing Service Preclinical research service establishing patient cell lines from muscle biopsies for Duchenne Muscular Dystrophy to test genome editing therapies and measure dystrophin protein restoration. Used to support CRD's DMD therapeutic programs and academic collaborators (including SickKids Toronto).
Quantifiable outcome
- Developed treatment for founder's brother's rare DMD mutation in 3 years
- +3 more outcomes
Companies that use Cure Rare Disease
Customer profileNamed customers1 record
Segments4 records
Ideal customer profiles4 records
Cure Rare Disease technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
Cure Rare Disease partnerships and signals
Strategic signalPartnerships
Twelve partnerships are on record, tiered core and supporting.
- LGMD2L FoundationcoreMulti-year partnership backed by $7.65 million in funding from the LGMD2L Foundation to develop a novel gene replacement therapy for Anoctamin 5 (ANO5)-related disease. The program supports therapeutic design, preclinical studies, manufacturing scale-up, and a first-in-human clinical trial. This represents a landmark effort by patient-led organizations to advance treatments for rare diseases overlooked by traditional pharmaceutical pipelines.
- The Hospital for Sick Children (SickKids)coreThree-year collaboration with over $500,000 in funding from CRD to SickKids in Toronto for developing CRISPR-based genome editing therapies for Duchenne muscular dystrophy patients. The project aims to correct gene duplications that cause DMD by restoring full-length dystrophin, testing feasibility with cells from four CRD patients.
- UCLAcoreAcademic collaboration for clinical development of SCA3 program. Dr. Susan Perlman serves as clinical partner, Director of Ataxia Center at UCLA Medical Center.
- Yale Medical SchoolcoreAcademic collaboration led by Dr. Monkol Lek for DMD gene therapy research. Dr. Lek's lab leads translational research and cell line development for DMD programs.
- Boston Children's HospitalcoreAcademic collaboration with Dr. Alan Beggs serving as Scientific Director. Dr. Beggs is Director of the Manton Center for Orphan Disease Research and has pioneered gene therapy development for congenital myopathies and muscular dystrophies.
- Leiden University Medical CentercoreCollaboration with the Neuro-D Lab of Dr. Willeke van Roon-Mom for SCA3 ASO therapeutic development. Dr. van Roon-Mom leads translational research on neurodegenerative diseases and co-directs the Dutch Center of RNA Therapeutics.
- Charles River LaboratoriescoreIndustry collaborator providing preclinical testing and development services. Charles River is a leading CRO supporting drug development across multiple therapeutic areas.
- AxolabssupportingIndustry collaborator providing specialized expertise supporting CRD's ASO and oligonucleotide therapeutic development programs.
- Mass BiologicssupportingIndustry collaborator providing manufacturing and biologics development expertise.
- Form BiosupportingIndustry collaborator supporting CRD's development programs.
- Virginia Commonwealth UniversitycoreAcademic collaboration with Dr. Nick Johnson, division chief of neuromuscular medicine, focused on inherited neuromuscular disorders including limb girdle muscular dystrophies.
- University of UtahcoreAcademic collaboration with Dr. Russell Butterfield leading translational research in muscular dystrophies through the Utah Program in Inherited Neuromuscular Disorders (UPIN).
Scale indicators4 records
Recent moves7 records
Expansion highlights6 records
Cure Rare Disease competitors and assessment
Company assessmentBroad incumbents
- BioMarin Pharmaceutical: Large rare disease-focused biotech with multiple approved products and a gene therapy pipeline — represents the established commercial model CRD's nonprofit approach is designed to complement or compete with for ultra-rare indications.
- Sarepta Therapeutics: Commercial leader in DMD gene therapy with FDA-approved Elevidys and a portfolio of approved and clinical-stage treatments — the dominant commercial player in CRD's largest therapeutic indication.
- uniQure: Commercial-stage gene therapy company with FDA-approved Hemgenix and a pipeline targeting rare diseases including neuromuscular indications — operates in CRD's therapeutic space with substantially greater resources.
- Regenxbio: Established AAV gene therapy platform company with clinical programs across multiple rare disease indications, including neuromuscular targets — a competitor for CRD's AAV-based programs and a potential licensing partner for capsid technology.
Emerging players
- Solid Biosciences: Clinical-stage gene therapy company developing AAV-based treatments for Duchenne muscular dystrophy and other neuromuscular diseases — directly competing with CRD's DMD and LGMD programs.
- Verve Therapeutics: Clinical-stage gene editing company developing in vivo CRISPR-based therapies — uses similar CRISPR platform technology as CRD but targets cardiovascular indications, providing a comparable technology benchmark.
- Beam Therapeutics: Clinical-stage precision genetic medicine company using base editing — operates in the same gene editing modality space as CRD's CRISPR programs and provides a comparable benchmark for platform-stage gene editing companies.
Direct peers
- Parent Project Muscular Dystrophy: Leading nonprofit advocacy and research organization focused on Duchenne and Becker muscular dystrophy, directly overlapping CRD's primary indication area and competing for the same patient/foundation donor base.
- n-Lorem Foundation: Nonprofit foundation that develops individualized antisense oligonucleotide (ASO) therapies for patients with ultra-rare diseases — a directly analogous N-of-1 nonprofit biotech model targeting the same market gap CRD addresses.
Others
- Muscular Dystrophy Association: Major voluntary health organization that funds muscular dystrophy research and is both a CRD grant funder and a competitor for charitable donations in the same therapeutic space.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat4 records
Key risks6 records
Key highlights6 records
Customer concentration
Cure Rare Disease social profiles
Digital presenceCure Rare Disease financial estimates
Financial estimateRevenue estimate
Valuation estimate
Cure Rare Disease leadership team
Management profileNumber of profiles
Profiles10 records
Cure Rare Disease funding detail
Funding detailFunding overview
Funding rounds4 records
Investors2 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Cure Rare Disease 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 Rare Disease
What does Cure Rare Disease do?
Cure Rare Disease is a nonprofit drug development organization that develops customized genetic therapies for ultra-rare neuromuscular and neurodegenerative diseases. It operates a four-phase development engine spanning research and development, prototype development, FDA-regulated clinical trials, and eventual licensing, using three core technology platforms: CRISPR gene editing, next-generation AAV (adeno-associated virus) vectors, and antisense oligonucleotides (ASOs). Patient access to investigational therapies occurs exclusively through clinical trials and compassionate use programs.
Is Cure Rare Disease a public or private company?
Cure Rare Disease is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was Cure Rare Disease founded?
Cure Rare Disease was founded in 1998. It employs 1 to 10 people.
Where is Cure Rare Disease based?
Cure Rare Disease is headquartered in Boston, United States, in the North America region.
How does Cure Rare Disease make money?
Three revenue lines are on record. Donations and Charitable Contributions are the primary driver. The others are foundation and Grant Funding and collaborative Research Agreements.
Who are Cure Rare Disease's main competitors?
Broad incumbents on record are BioMarin Pharmaceutical, Sarepta Therapeutics, uniQure and Regenxbio. Emerging players are Solid Biosciences, Verve Therapeutics and Beam Therapeutics. Direct peers are Parent Project Muscular Dystrophy and n-Lorem Foundation. Muscular Dystrophy Association is listed as an others.
Does Cure Rare Disease have an API?
No public API is recorded for Cure Rare Disease.
What industry is Cure Rare Disease in?
Cure Rare Disease's product category is Rare Disease Genetic Therapeutics Development. Its primary akta.pro industry code is HLAAACAE, Gene Therapy for Rare/Monogenic Diseases, with a secondary code of HLAIAIAN, Rare Pediatric & Congenital Disorder Therapies. Its NAICS code is 541714 and its SIC code is 8090.