IFCR

What IFCR does

International Foundation for CDKL5 Research (IFCR) is a 501(c)(3) nonprofit patient advocacy foundation incorporated in 2009 in Ohio to serve families affected by CDKL5 Deficiency Disorder (CDD), a rare developmental epileptic encephalopathy with an incidence of approximately 1 in 40,000-60,000 live births. IFCR's mission is to accelerate treatment development and improve clinical care for CDD through research funding, clinical infrastructure, and family support. The foundation operates a portfolio of interconnected programs: the Connect CDKL5 global patient contact registry (500+ patients across 45+ countries), 10 multidisciplinary CDKL5 Centers of Excellence at major US academic medical centers, the International CDKL5 Clinical Research Network (ICCRN), the co-stewarded International CDKL5 Disorder Database (ICDD, 350+ patients across 20+ countries), the Family Support Representatives peer-to-peer program, and the CDKL5 Variant Tool developed with the Broad Institute.

Name

IFCR

Legal name

International Foundation for CDKL5 Research

Website

https://cdkl5.com

Company type

Private

Founded year

2009

Operating status

Operating

Headcount range

1–10 employees

Short description

IFCR is a 501(c)(3) nonprofit patient advocacy foundation serving global CDKL5 Deficiency Disorder families through research funding, a 10-site US Centers of Excellence network, and patient registries enabling clinical trial readiness.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy

NAICS

Voluntary Health Organizations (813212), Scientific Research and Development Services (5417)

SIC

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

akta.pro primary industry

Clinical Data Repositories (CDR) & Longitudinal Patient Records (HLACAIAH)

akta.pro secondary industry

Clinical Trial Monitoring Support (On-site/Remote) (HLAGACAG)

Keywords

• Patient advocacy services
• Rare disease research
• Clinical trial readiness
• Family support programs
• Patient registry platform

Headquarters

HQ city

Wadsworth

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

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

Revenue model

1. Donations and Fundraising: The primary revenue source is donations from individuals, families, and supporters. The organization hosts fundraising events including the annual Move CDKL5 Forward event, family-hosted fundraisers, corporate partnerships, and Facebook/Instagram fundraising tools. Memorial and tribute gifts are also accepted.
2. Corporate Sponsorships: Corporate partners provide financial or in-kind support for IFCR programs and events, including conference sponsorships and program funding.
3. Stock Gifts and Securities: The organization accepts gifts of securities (stock transfers) as an investment vehicle for supporting CDKL5 treatments and research.
4. Monthly Giving Program: Recurring monthly contributions from donors providing sustained support for the organization.

Go-to-market motion1 record

Distribution channels4 records

Marketing channels10 records

Core offering

IFCR is a 501(c)(3) patient advocacy foundation that supports families affected by CDKL5 Deficiency Disorder through a portfolio of programs: the Connect CDKL5 global patient contact registry, the CDKL5 Variant Tool for genetic mutation visualization, the C.A.R.E. Binder caregiver resource, an educational Resource Library, and Family Support Representatives providing peer outreach. The foundation also funds scientific research (mouse/iPSC models, biomarkers, outcome measures), operates the ICCRN clinical trial readiness network alongside 10 CDKL5 Centers of Excellence, and coordinates the ICDD natural history database in partnership with Telethon Kids Institute.

Product overview

The International Foundation for CDKL5 Research (IFCR) is a nonprofit patient advocacy organization that operates a portfolio of interconnected programs and tools supporting CDKL5 Deficiency Disorder families and research. The core offerings include Connect CDKL5 (a global patient registry), the CDKL5 Variant Tool (a mutation visualization tool), the Resource Library (educational guides and videos), Family Support Representatives (peer support), and 10 CDKL5 Centers of Excellence (multidisciplinary clinical care). The organization also coordinates the International CDKL5 Clinical Research Network (ICCRN) and International CDKL5 Disorder Database (ICDD) for clinical trial readiness, and hosts annual events including the Family Educational Conference and Move CDKL5 Forward awareness campaign.

Differentiator

Problem solved

Functional benefit

Products and services

• Connect CDKL5
• CDKL5 Variant Tool
• C.A.R.E. Binder
• CDKL5 Resource Library
• CDKL5 Centers of Excellence
• Family Support Representatives (FSRs)
• Clinical Trial Readiness Program
• CDKL5 Family Educational Conference
• International CDKL5 Clinical Research Network (ICCRN)
• International CDKL5 Disorder Database (ICDD)

Quantifiable outcome

• First FDA-approved treatment (ZTALMY/Ganaxolone) for CDKL5 Deficiency Disorder achieved March 2022
• +3 more outcomes

Named customers2 records

Segments4 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

17 partnerships are on record, tiered core, major and minor.

• Marinus PharmaceuticalscoreGTM or Marketing Partner · 1 January 2022Marinus Pharmaceuticals received FDA approval for ZTALMY (ganaxolone) for CDKL5 Deficiency Disorder in March 2022. IFCR supported this development through patient advocacy and community engagement, and provides patient resources for ZTALMY.
• Broad InstitutemajorTechnology or Integration · 1 January 2020Partnership with Dr. Dennis Lal's research team to develop the CDKL5 Variant Tool, a web-based widget for accurate diagnosis of Variants of Uncertain Significance (VOUS) in CDKL5 and other monogenic disorders.
• Loulou FoundationcoreStrategic or Co-development Partner · 1 January 2019Co-hosted the 2019 Patient-Focused Drug Development Meeting with FDA, gathering patient perspectives on CDKL5 Deficiency Disorder. The Loulou Foundation has been instrumental in supporting CDKL5 research and advocacy efforts, including gene therapy development programs.
• Child Neurology FoundationmajorStrategic or Co-development Partner · 1 January 2019Collaborated to create the Family Support Representative (FSR) peer-to-peer support program, providing training and support for volunteers reaching out to CDKL5 families and caregivers.
• Harvard Brain BankmajorStrategic or Co-development Partner · 1 January 2019Collaboration established to allow brain and tissue donation samples from CDKL5 patients to be safely stored for future investigation, supporting post-mortem research into the disorder.
• Rocky Mountain Rett AssociationminorStrategic or Co-development Partner · 1 January 2013Partnered with Rocky Mountain Rett Association to establish the inaugural CDKL5 Center of Excellence at Children's Colorado in 2013, pioneering the COE model.
• Telethon Kids InstitutecoreStrategic or Co-development Partner · 1 January 2012Partnership established in 2012 with researchers at Telethon Kids Institute (Perth, Australia) to create the International CDKL5 Disorder Database (ICDD). This natural history database has collected parent-reported and genetic data from 350+ individuals across 20+ countries, informing over 25 publications. The collaboration expanded in 2019 to form the International CDKL5 Clinical Research Network (ICCRN).
• International Rett Syndrome Foundation (IRSF)majorStrategic or Co-development Partner · 1 January 2010Co-funded the CDKL5 Knock-Out Mouse Model in 2010 with Dr. Cornelius Gross at EMBL, creating the first animal model missing its entire CDKL5 gene. Continued partnership on related research including MeCP2 phosphorylation studies and drug therapy research.
• Boston Children's HospitalcoreStrategic or Co-development PartnerEstablished CDKL5 Center of Excellence at Boston Children's Hospital (2013) with Dr. Heather Olson. The COE provides multidisciplinary care and conducts clinical research including EEG studies and ketogenic diet trials.
• Children's Hospital ColoradocoreStrategic or Co-development PartnerInaugural CDKL5 Center of Excellence established in 2013 at Children's Colorado with Dr. Tim Benke. Serves as a key site for the ICCRN and clinical trials readiness research.
• Children's Hospital of PhiladelphiacoreStrategic or Co-development PartnerCDKL5 Center of Excellence established in 2019 at CHOP with Dr. Eric Marsh. Key clinical research site participating in the ICCRN and outcome measure validation studies.
• Longboard Pharmaceuticals (now Lundbeck)majorGTM or Marketing PartnerLongboard Pharmaceuticals conducted DEEp Ocean Study (LP352/bexicaserin) and PACIFIC Study for developmental and epileptic encephalopathies including CDKL5. IFCR facilitates patient awareness and participation in these trials.
• Praxis Precision MedicinesmajorGTM or Marketing PartnerConducted the Emerald Study evaluating relutrigine for DEEs including CDKL5 Deficiency Disorder. IFCR facilitates patient engagement and provides updates to the community on trial progress.
• UltragenyxmajorGTM or Marketing PartnerDeveloping gene therapy UXO55 (ELJ-101) for CDKL5 Deficiency Disorder. IFCR provides community education through webinars and resources on gene therapy developments.
• UCB (formerly Zogenix)majorGTM or Marketing PartnerZogenix conducted the GEMZ Study evaluating fenfluramine for CDKL5-related epilepsy before being acquired by UCB. IFCR facilitated patient participation and provides study information to families.
• University of California San DiegomajorStrategic or Co-development PartnerPartnership with Dr. Alysson Muotri's lab to develop the first iPS CDKL5 cell lines from patient skin fibroblasts in 2011, enabling study of CDKL5 Deficiency in various cell types including neurons.
• University of PennsylvaniamajorStrategic or Co-development PartnerLong-standing partnership with Dr. Zhaolan (Joe) Zhou's lab for multiple CDKL5 mouse model development projects including Knock-In Mouse Model R59X, genetic dissection research, and CDKL5 antibodies.

Scale indicators9 records

Recent moves18 records

Expansion highlights5 records

Broad incumbents

• National Organization for Rare Disorders (NORD): Umbrella rare disease advocacy organization representing 300+ disease-specific foundations including IFCR peers. Operates the IAMRARE patient registry platform—comparable infrastructure and policy advocacy role at a multi-disease level.
• Child Neurology Foundation: Child neurology advocacy and education organization that partnered with IFCR to create the Family Support Representative peer-to-peer program. Comparable as a pediatric neurology support organization with broader scope.
• Epilepsy Foundation: Largest general epilepsy advocacy organization in the US operating across all seizure disorders. Comparable as a patient-facing advocacy and research funding body, but at much broader scope than IFCR's CDKL5-specific focus.

Direct peers

• Lennox-Gastaut Syndrome Foundation: Rare pediatric epilepsy advocacy foundation and C.A.R.E. Binder co-development partner. Comparable in mission, fundraising model, and clinical care network approach to IFCR.
• Cure SMA: Spinal muscular atrophy advocacy organization widely cited as the gold-standard rare disease advocacy model with research funding, Centers of Excellence, and a patient registry. Comparable as a rare pediatric neurological disease foundation that catalyzed approved therapies.
• Loulou Foundation: Private foundation dedicated to CDKL5 Deficiency Disorder that has co-funded gene therapy development programs and co-hosted the 2019 FDA Patient-Focused Drug Development meeting with IFCR. Directly comparable as a CDD-specific research and advocacy funder.
• Dravet Syndrome Foundation: Rare epileptic encephalopathy advocacy organization that co-developed the C.A.R.E. Binder with IFCR. Highly comparable as a small-disease patient advocacy foundation that funds research, runs a patient registry, and supports centers of excellence.
• Parent Project Muscular Dystrophy (PPMD): Duchenne muscular dystrophy advocacy organization that pioneered the Certified Duchenne Care Center model and substantial patient registry. Highly comparable operating model to IFCR's COE network and Connect CDKL5 registry.
• International Rett Syndrome Foundation (IRSF): Long-standing IFCR partner that co-funded the original CDKL5 Knock-Out Mouse Model in 2010. Operates as a rare neurodevelopmental disorder advocacy and research funder with overlapping scientific advisory networks and similar center-of-excellence infrastructure.
• TSC Alliance: Tuberous Sclerosis Complex advocacy organization and C.A.R.E. Binder co-development partner. Operates a mature clinic network, research grants, and patient registry—comparable infrastructure model to IFCR for a different rare epilepsy.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/CDKL5USA
• YouTubeyoutube.com/channel/UCim6I5JIN2i_yu8LuEihlSw
• Facebookfacebook.com/CDKL5foundation
• LinkedInlinkedin.com/company/international-foundation-for-cdkl5-research
• Instagraminstagram.com/cdkl5_ifcr

Revenue estimate

Valuation estimate

Number of profiles

Profiles18 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about IFCR