RARE-X

What RARE-X does

RARE-X is a 501(c)(3) rare disease technology nonprofit that operates a federated, cloud-native Data Collection Platform purpose-built to let patient communities gather, structure, govern, and share de-identified health data with qualified researchers, clinicians, and drug developers. The core product is the RARE-X Data Platform, comprising a Data Collection Program (validated surveys including a Health & Development Survey and Level 2 instruments, genetic report uploads, EHR data) layered onto a federated analysis infrastructure originally developed at the Broad Institute of MIT and Harvard for projects such as Count Me In. Built on this platform are several programs: the Research Readiness Program (clinical trial readiness assessments), the Diversity, Equity, and Inclusion Program (DEIP), Xcelerate RARE (an Open Science Data Challenge hosted on Synapse focused on rare pediatric neurodevelopmental diseases), and the CNVs Commission. Patients retain control of consent and participate in data access governance.

The organization serves three distinct user constituencies: patient communities and caregivers (free access to data collection and registry tools), academic and biotech researchers (data access via federated analysis or Xcelerate RARE challenges), and biopharma industry partners (sponsorship, Corporate Advisory Council membership, and trial-readiness services). Patient-controlled consent and an open-science framing differentiate RARE-X from for-profit registry vendors. Since February 2023, RARE-X has operated as a research program of Global Genes following the completion of a merger.

RARE-X's business model is a nonprofit, grant- and sponsorship-funded model rather than a fee-for-service SaaS model. The platform is offered at no cost to patient communities; revenue derives from biopharma grants and corporate commitments (Genentech, Travere Therapeutics, Roche, Datavant, Horizon, RTW Charitable Foundation) directed at named programs (DEIP, Xcelerate RARE, data standards development). Go-to-market is community-led and channel-partner driven, executed through patient advocacy organizations, the Corporate Advisory Council, events (Exchange Forum, summits), webinars, podcasts (RARE-Xtra), and the Xcelerate RARE challenge mechanism. Specific grant amounts are not disclosed, and the organization operates with 1–10 employees.

Name

RARE-X

Legal name

RARE-X

Website

https://rare-x.org

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

1–10 employees

Short description

RARE-X is a 501(c)(3) nonprofit (and research program of Global Genes) that operates a federated, Broad Institute-derived data collection and sharing platform serving rare disease patient communities, academic researchers, and biopharma sponsors, funded by grants and corporate sponsorships rather than user fees.

Ownership category

akta.pro rank

Product category

Rare Disease Data Platform

NAICS

Web Search Portals and All Other Information Services (519290), Other Computer Related Services (541519)

SIC

Services-Misc Health & Allied Services, Nec (8090), Services-Health Services (8000)

akta.pro primary industry

Disease Registries (Cancer, Immunization, Rare Disease, etc.) (HLAJAJAE)

akta.pro secondary industry

Multi-Omics Precision Medicine Platforms (genomics/proteomics/metabolomics) (HLAAANAF)

Keywords

• Rare disease data
• Patient data platform
• Federated data sharing
• Patient registries
• Clinical trial readiness

Headquarters

HQ city

Aliso Viejo

HQ country

United States

HQ region

North America

Markets served

GTM type

B2B and B2C

Offering type

Software

Cost components

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

Revenue model

1. Grants from biopharma partners: RARE-X is a 501(c)(3) nonprofit that funds programs via grant support from biopharmaceutical companies. Genentech and Travere Therapeutics provided grant support to launch the Diversity, Equity, and Inclusion Program in January 2021.
2. Corporate sponsorship and funding commitments: RARE-X secures funding commitments from biopharma partners (e.g., Travere Therapeutics' February 2021 funding commitment) to support patient community data collection, data standards development, and platform enhancements.
3. Open Science Data Challenge sponsorship: Xcelerate RARE is sponsored by named partners including Datavant, Horizon, Roche, and RTWCF, providing program funding for the open science challenge.

Pricing tiers

Go-to-market motion3 records

Core offering

RARE-X provides a federated, cloud-native Data Collection Platform (built with the Broad Institute of MIT and Harvard) that lets rare disease patient communities capture, structure, govern, and share de-identified health data — including validated patient-reported surveys, genetic reports, EHR records, imaging, and wearable data — with qualified global researchers, clinicians, and drug developers. Surrounding the platform are programs such as Xcelerate RARE (open science data challenge), the Research Readiness Program (clinical trial readiness assessment), and the Diversity, Equity, and Inclusion Program, all operated as a research program of Global Genes.

Product overview

RARE-X is a 501(c)(3) rare disease technology nonprofit and research program of Global Genes that operates a single unified platform-plus-programs offering built around the RARE-X Data Platform. The RARE-X Data Platform is the core product, comprising the Data Collection Program (DCP) for structured patient-reported and clinical data ingestion (including genetic reports and validated surveys) and the federated data analysis platform (developed with the Broad Institute) for governance, consent, and data sharing. Built on top of this platform are a portfolio of programs and initiatives, including Xcelerate RARE (an Open Science Data Challenge for AI-driven analysis of rare neurodevelopmental disease data), the Research Readiness Program (clinical trial readiness assessment and data collection mobilization), the Diversity, Equity, and Inclusion Program (DEIP), the RARE-Xtra podcast, the Xplore news and insights hub, and specialized rare disease community initiatives such as the CNVs Commission. Together these offerings form RARE-X's effort to build the largest collaborative, patient-driven, open-data access project for rare diseases globally.

Differentiator

Problem solved

Functional benefit

Brands

• Xcelerate RARE: A Rare Disease Open Science Data Challenge bringing together researchers and data scientists in a collaborative and competitive environment to make the best use of patient-provided data to solve big unknowns in healthcare.
• RARE-X Research Readiness Program

Products and services

• RARE-X Data Platform A collaborative, federated data sharing and analysis platform (originally developed at the Broad Institute of MIT and Harvard) that enables rare disease patient communities to gather, structure, govern, and securely share de-identified health data with global researchers, clinicians, and drug developers. Combines a Data Collection Program with a federated data analysis platform and patient-controlled consent.
• Xcelerate RARE Open Science Data Challenge A Rare Disease Open Science Data Challenge that brings together academic and biopharmaceutical researchers and data scientists to analyze patient-provided data from the RARE-X Data Platform in a pre-competitive environment, with anticipated outcomes including shortening the diagnostic odyssey through predicted diagnosis and identifying previously unrecognized disease symptoms.
• Research Readiness Program A program that helps rare disease patient communities systematically assess their clinical trial readiness, identify research gaps, galvanize data collection on the RARE-X Data Collection Platform, and prioritize investments by connecting patient advocacy groups with key researchers and mentors from RARE-X's Corporate Alliance Council.
• Diversity, Equity, and Inclusion Program (DEIP) A program designed to ensure the RARE-X data platform is demographically representative of the rare disease community by engaging underrepresented populations through community-based participatory research, with a DEI Advisory Council, scoping study led by Teneasha Washington, and a published strategic recommendations report.

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Integration1 record

AI capability5 records

Feature6 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Genetic Alliance: Long-standing patient advocacy umbrella running registry and data-sharing initiatives across many disease areas; offers overlapping capability with RARE-X but operates as a broader advocacy organization rather than a specialized rare disease data platform.
• NORD (National Organization for Rare Disorders): US rare disease advocacy organization operating the IAMRARE patient registry platform and other data programs; competes for the same patient communities and biopharma partnerships while serving a wider advocacy mandate.

Direct peers

• NIH RDCA-DAP (Rare Disease Cures Accelerator-Data and Analytics Platform): NCATS-led federally funded platform aggregating rare disease data for drug developers; competes for the same researcher and biopharma data-access dollars as RARE-X and offers strong credibility with NIH.
• Sage Bionetworks (Synapse): Operates the Synapse open data sharing platform that RARE-X uses to host Xcelerate RARE challenge data; shares RARE-X's federated, open-science approach to biomedical research data.
• PatientsLikeMe: Patient-driven health data sharing platform historically focused on outcome reporting across conditions; a comparable patient-controlled data model for research use, including rare disease communities.
• CoRDS (Coordination of Rare Diseases at Sanford): Sanford Health's rare disease patient registry and natural history platform; a direct competitor for patient community onboarding and biopharma-sponsored registry use, and a listed RARE-X collaboration partner.

Others

• Datavant: Health data connectivity and de-identification company sponsoring Xcelerate RARE; an ecosystem enabler that sits adjacent to RARE-X's platform by linking disparate clinical and research datasets.

Emerging players

• Ciitizen: Health data infrastructure platform that converts patient medical records into computable, research-ready datasets; a RARE-X integration partner with overlapping capability in rare disease data structuring and patient record digitization.
• Invitae: Genetic testing company with a rare disease franchise that aggregates patient-level genetic and clinical data; comparable in rare disease data ambition but commercial and testing-led rather than registry-led.

Regional players

• EURORDIS (Rare Diseases Europe): European rare disease umbrella organization with rare disease registries and data initiatives; comparable in mission and data-sharing orientation but focused on the European regulatory and patient landscape.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/Rare_x_
• YouTubeyoutube.com/channel/UCtO-tNbC1G1cO7z0fe7Wh-Q
• Facebookfacebook.com/Data4Rare
• LinkedInlinkedin.com/company/rare-x
• Instagraminstagram.com/_rare.x_

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about RARE-X