What KIF1A.ORG does

KIF1A.ORG is a registered 501(c)(3) nonprofit patient advocacy and research foundation founded in 2017 to serve individuals and families affected by KIF1A Associated Neurological Disorder (KAND), a progressive and currently untreatable neurodegenerative condition. The organization functions as the sole global convening body for the KAND community, connecting 550+ documented diagnosed patients across multiple countries with a research network of 50+ institutions. Its core deliverables are research infrastructure rather than commercial products: the confidential global KIF1A Patient Registry; the ASCEND Natural History Study (online longitudinal surveys, launched 2017 with Chung Lab); the KOALA Study (in-person clinical assessments launched 2022 establishing FDA-grade endpoints); an iPSC biobank managed via Coriell Institute; the Treatment Accelerator Program (TAP) with NeuCyte producing patient-derived disease models and brain organoids; plus disease-specific studies in epilepsy (Columbia University, 2022) and speech (MCRI Australia, 2022). KIF1A.ORG also runs community programs (private family resources, newly diagnosed meetups, quarterly calls) and convenes the annual KIF1A Family and Scientific Engagement Conference.

The organization's revenue model is donations- and grant-funded: tiered individual donations ($25-$2,500, plus monthly sustaining donors recognized on the League of Sidekicks Wall of Fame), event fundraising (including the 2027 Boston conference), and research grants administered for partner labs. Documented grants include a 4-year NIH R01 (2020), a 4-year Australian NHMRC grant (starting 2022), the XSeed Award from Deerfield Management (March 2022), the Rising Tides grant from Child Neurology Foundation (2020), and a 3-year Australian government grant for epilepsy research (2021). Pharmaceutical and biotech partners include Ovid Therapeutics (aptamer/RNAi drug development with Chung Lab) and NeuCyte (TAP); key academic partners are Chung Lab at Boston Children's Hospital (primary research partner since 2017), Christodoulou Lab at MCRI, Gennerich Lab at Albert Einstein College of Medicine, The Jackson Laboratory, and Coriell Institute.

Name

KIF1A.ORG

Legal name

KIF1A.ORG

Website

https://kif1a.org

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

KIF1A.ORG is a 501(c)(3) nonprofit foundation that serves as the global convening body for KIF1A Associated Neurological Disorder (KAND) patients and families, operating the sole worldwide patient registry, natural history studies, and a 50+ institution research network accelerating therapeutic development for the ultra-rare disease.

Ownership category

akta.pro rank

Product category

Rare Disease Research Foundation

NAICS

Scientific Research and Development Services (5417), Other Scientific and Technical Consulting Services (541690)

SIC

Services-Commercial Physical & Biological Research (8731), Services-Social Services (8300)

akta.pro primary industry

Site Network Operators & SMOs (HLAGACAA)

akta.pro secondary industry

Site Identification & Feasibility (HLAGACAB)

Keywords

• Rare disease advocacy
• Patient registry services
• Neurological research funding
• Natural history studies
• Therapeutic development acceleration

Headquarters

HQ city

New York

HQ country

United States

HQ region

North America

Markets served

GTM type

B2B and B2C

Offering type

Services

Cost components

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

Revenue model

1. Charitable Donations: KIF1A.ORG is a registered 501(c)(3) nonprofit organization. Its primary revenue stream is charitable donations from individuals, including one-time gifts, monthly sustaining donations (recognized through the League of Sidekicks Wall of Fame), and donations made in honor of specific KIF1A superheroes. The organization also accepts donations with optional coverage of the 3% transaction processing fee.
2. Research Grants and Foundation Funding: KIF1A.ORG receives and administers grant funding for KAND research, including awards from the Child Neurology Foundation (Rising Tides grant, 2020) and co-funds projects with government agencies and industry partners. Research grants fund the Treatment Accelerator Program, Natural History Study, and specific lab collaborations.
3. Fundraising Events: The organization raises funds through events and campaigns, including the 2027 KIF1A Family and Scientific Engagement Conference in Boston, corporate giving programs, and general fundraising initiatives.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels8 records

Core offering

KIF1A.ORG operates a confidential global patient registry and longitudinal natural history studies (ASCEND online surveys and KOALA in-person clinical assessments) to characterize KIF1A Associated Neurological Disorder (KAND). The foundation runs a Treatment Accelerator Program that builds patient-derived iPSC disease models (co-cultures and brain organoids) for drug screening in partnership with NeuCyte. It convenes a KIF1A Research Network of more than 50 institutions and provides community support, educational resources, and direct research funding to accelerate therapeutic development for KAND.

Product overview

KIF1A.ORG is a nonprofit patient advocacy and research organization (registered 501(c)(3)) rather than a traditional technology company. The organization operates a portfolio of research programs and community services including: the Patient Registry tracking KAND diagnoses globally; the ASCEND Natural History Study collecting longitudinal symptom data through online surveys; the KOALA Study conducting in-person clinical assessments to prepare for trials; the KAND Epilepsy Study analyzing EEG patterns; the International KAND Speech Study; and the Treatment Accelerator Program (TAP) partnering with NeuCyte for drug screening using patient-derived stem cells. The organization also maintains the KIF1A Research Network connecting over 50 research institutions, provides educational resources including the KIF1A Glossary and Research Simplified content, and organizes the annual KIF1A Family and Scientific Engagement Conference.

Differentiator

Problem solved

Functional benefit

Products and services

• KIF1A Patient Registry A confidential global registry tracking individuals diagnosed with KAND worldwide, used to inform family, scientific, medical, pharmaceutical, regulatory, and governmental stakeholders about the community's collective need for treatments.
• ASCEND Natural History Study A longitudinal study conducted by Chung Lab in partnership with KIF1A.ORG that collects online interviews and surveys from KAND families to understand how KAND symptoms develop and change over time.
• KOALA Study KIF1A Outcome measures, Assessments, Longitudinal And endpoints Study - An in-person clinical assessment program preparing the KAND community for clinical trials by establishing standardized outcome measures and endpoints.
• KAND Epilepsy Study A study launched with Columbia University researchers Dr. Jennifer Bain and Dr. Tristan Sands to analyze EEGs and identify patterns in epilepsy and seizures associated with KAND.
• International KAND Speech Study A study partnered with MCRI in Melbourne, Australia to identify and characterize speech and non-verbal communication in KAND patients, and identify communication aides, therapies, and speech biomarkers.
• KIF1A Research Network A collaborative network of over 50 researchers, clinicians, industry leaders and innovators from around the world working to accelerate treatment for KAND, led by KIF1A.ORG.
• Treatment Accelerator Program (TAP) A drug development program partnering with NeuCyte to create cell-based models of KAND using patient-derived iPSCs for drug screening to identify and test potential therapeutics.
• Brain Donation Program A partnership between KIF1A.ORG and Autism BrainNet providing a compassionate process for KAND families to advance research through brain donation.

Quantifiable outcome

• KIF1A.ORG has grown from an unnamed disorder in 2017 to a globally recognized rare disease community with 550+ documented patients and representation across multiple countries.
• +5 more outcomes

Named customers4 records

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Integration1 record

Feature7 records

Partnerships

33 partnerships are on record, tiered core, major and supporting.

• Chung Lab at Boston Children's HospitalcoreStrategic or Co-development Partner · 1 January 2017Led by Wendy Chung, MD, PhD, Chung Lab is the central research institution for KAND. It manages the global patient registry, natural history studies (ASCEND and KOALA), patient-derived iPSC lines, animal and cell disease models, biomarker studies, and clinical disease characterization. Dr. Chung has been KIF1A.ORG's primary research partner since the organization's founding in 2017, serving as scientific advisor and principal investigator for most funded projects.
• Murdoch Children's Research Institute (MCRI)coreStrategic or Co-development PartnerMCRI's Christodoulou Lab, led by Professor John Christodoulou and Dr. Simranpreet Kaur, operates a high-throughput drug screening platform to test FDA-approved drugs for repurposing to improve KIF1A molecular function. MCRI also collaborated on the KAND Speech Study and produces the annual KAND Impact and Involvement Report. Awarded government grants for KAND epilepsy research.
• Ovid TherapeuticsmajorStrategic or Co-development PartnerBiopharmaceutical company focused on rare neurological disorders, developing genetic-based treatments for KAND through aptamer protein targeting and RNAi gene targeting in collaboration with Chung Lab. Supported by KIF1A.ORG funding and data.
• NeuCytemajorStrategic or Co-development PartnerBiotechnology company solely focused on neurological diseases, engaged by KIF1A.ORG to build the Treatment Accelerator Program. NeuCyte uses its proprietary SynFire iPSC-derived neural cell platform to create KAND disease models, co-cultures, and brain organoids for target identification, drug efficacy testing, and neurotoxicity assessment. All models are made openly available to the KIF1A Research Network.
• Gennerich Lab at Albert Einstein College of Medicine and AtomwisemajorStrategic or Co-development PartnerLed by Arne Gennerich, PhD, in collaboration with Hernando Sosa, PhD, and AI drug discovery company Atomwise. Combines cryo-electron microscopy (cryo-EM) to resolve high-resolution KIF1A protein structures with AI-based virtual screening to identify small-molecule drug candidates for KAND. Awarded XSeed Award from Deerfield Management in 2022.
• BioLoomicssupportingTechnology or IntegrationPreclinical biotechnology company using 'evolution-in-a-microscope' technology to build high-throughput drug discovery tools. BioLoomics' KIF1A program creates improved assays for measuring therapeutic candidate effects and identifies novel targets for KAND therapeutic development.
• Christodoulou Lab at MCRIcoreStrategic or Co-development PartnerLed by Professor John Christodoulou and Dr. Simranpreet Kaur, this lab created a high-throughput drug screening platform to test FDA-approved drugs that can be repurposed to improve KIF1A molecular function for KAND. Awarded grants from the Australian government (NHMRC and 3-year epilepsy research grant). Collaborates with University of Sydney and Columbia University.
• The Jackson Laboratory (JAX)coreStrategic or Co-development PartnerNonprofit biomedical research institution that developed multiple KIF1A mouse models and a variety of genetically engineered KIF1A variant cell lines with isogenic controls, all made available to the KIF1A Research Network. JAX also collaborates with Miralinc Pharma and Barinka Lab on HDAC6 inhibition testing in KIF1A mouse models.
• Coriell Institute for Medical ResearchcoreStrategic or Co-development PartnerNonprofit biomedical research center that manages a biobank of induced pluripotent stem cells (iPSCs) derived from KAND patient blood samples in collaboration with KIF1A.ORG. These iPSCs are available to researchers in the KIF1A Research Network for therapeutic development.
• Chitnis Lab at National Institutes of Health (NIH)supportingStrategic or Co-development PartnerLed by Ajay Chitnis, MBBS, PhD, at the NIH's Section on Neural Developmental Dynamics. Created a zebrafish model of KAND that is available to the KIF1A Research Network for studying nervous system development and KIF1A biology.
• Niwa Lab at Tohoku UniversitysupportingStrategic or Co-development PartnerLed by Shinsuke Niwa, PhD, studying molecular mechanisms of cellular morphogenesis, kinesin, and microtubule networks. Created multiple C. elegans (roundworm) models of KAND available to the KIF1A Research Network for disease modeling and drug screening.
• Bain Lab and Sands Lab at Columbia UniversitymajorStrategic or Co-development PartnerDr. Jennifer M. Bain, MD, PhD (neurology and pediatrics) and Dr. Tristan Sands, MD, PhD (pediatric and neonatal neurologist and epileptologist) co-lead the KAND Epilepsy and EEG Study, analyzing EEG data and natural history data to identify electrophysiological patterns in KAND, improve epilepsy diagnosis, evaluate existing treatments, and develop new seizure therapies. Supported by KIF1A.ORG funding.
• Holzbaur Lab at University of PennsylvaniasupportingStrategic or Co-development PartnerLed by Erika Holzbaur, PhD, studying molecular motors like KIF1A, cell biology of neurons, and neurodegeneration. Project led by Jayne Aiken, PhD, investigates how disease-causing KIF1A mutations alter neuronal function to identify rescue strategies and assess viability of gene-transfer approaches for specific mutation classes.
• Silverman Lab at Simon Fraser UniversitysupportingStrategic or Co-development PartnerLed by Michael Silverman, PhD, cellular neurobiologists studying microtubule-based organelle transport. Characterizes microtubule-based cargo transport in KAND patient-derived induced neurons to improve understanding of KIF1A biology and identify therapeutic pathways.
• Frontier Research Institute for Interdisciplinary Sciences, Tohoku UniversitysupportingStrategic or Co-development PartnerKumiko Hayashi, PhD, leads a project to develop a non-invasive force measurement method to study KIF1A axonal transport in human iPSC-derived neurons. This work aims to identify biomarkers for therapeutic development.
• Barinka Lab and Miralinc Pharma (formerly HNF Pharma)supportingStrategic or Co-development PartnerCyril Barinka, PhD, at the Institute of Biotechnology of the Czech Academy of Sciences collaborates with Miralinc Pharma to investigate HDAC6 inhibition as a potential therapeutic for KAND and related disorders such as Charcot-Marie-Tooth (CMT). Miralinc provides the novel brain-penetrant HDAC6 inhibitor WT36-87 for testing in the KIF1A leg-dragger mouse model at The Jackson Laboratory.
• McKenney Lab at University of California, DavissupportingStrategic or Co-development PartnerLed by Richard McKenney, PhD, using state-of-the-art biochemical and biophysical methods to uncover molecular basis of defects in KIF1A motor proteins. Focused on characterizing KIF1A mutations including P305L using single-molecule microscopy to visualize KIF1A movement characteristics and inform therapeutic strategies.
• Curia (formerly Albany Molecular Research Inc. / AMRI)supportingTechnology or IntegrationContract research organization (CRO) engaged by KIF1A.ORG to conduct in silico (computer-based) modeling to identify potential strategies and targets for KAND therapeutic development. Project involves modeling the KIF1A crystal structure to understand mutation effects and explore existing kinesin-binding compounds. Results are openly available to the KIF1A Research Network.
• Vallee Lab at Columbia UniversitysupportingStrategic or Co-development PartnerLed by Richard Vallee, PhD, investigating the role of KIF1A in brain development, specifically during basal nuclear migration and in BDNF transport and secretion, which markedly affects neuronal migration. Collaborator on NIH grant with Chung Lab and Gennerich Lab.
• Verhey Lab at University of MichigansupportingStrategic or Co-development PartnerLed by Kristen Verhey, PhD, dedicated to uncovering how the interplay between microtubules and molecular motors facilitates intracellular transport in mammalian cells. Contributions have dramatically shaped understanding of KIF1A through multiple collaborative projects with the KIF1A research community.
• Hancock Lab at The Pennsylvania State UniversitysupportingStrategic or Co-development PartnerLed by William O. Hancock, PhD, dedicated to elucidating underlying mechanisms of motor proteins (kinesin and dynein) that contribute to cell function. Biochemical characterization of KIF1A chemomechanical cycle through studying kinetic rates of transition states.
• Columbia UniversitycoreStrategic or Co-development PartnerMultiple collaborative labs at Columbia University (Bain Lab, Sands Lab, Vallee Lab) contribute to KAND research including epilepsy studies, brain development research, and natural history studies. Columbia's College of Physicians and Surgeons hosts the KIF1A gene variant classification program.
• The Jackson Laboratory (JAX)coreStrategic or Co-development PartnerNonprofit biomedical research institution and pioneer in using laboratory mice as models for human disease. JAX's KIF1A program includes multiple mouse models and genetically engineered KIF1A variant cell lines with isogenic controls, freely available to the KIF1A Research Network.
• University of California, DavissupportingStrategic or Co-development PartnerUC Davis Biology Department hosts the McKenney Lab researching molecular basis of KIF1A motor protein defects through biophysical and microscopic methods, contributing to understanding of KIF1A mutation mechanisms.
• Boston Children's HospitalcoreStrategic or Co-development PartnerHome to the Chung Lab and primary site for KAND clinical research, including patient evaluations, natural history studies (ASCEND and KOALA), and the global patient registry. KIF1A.ORG co-funded research programs with the hospital.
• Coriell Institute for Medical ResearchcoreStrategic or Co-development PartnerNonprofit biomedical research center managing the iPSC biobank of KAND patient samples, making patient-derived stem cells available for research and therapeutic development across the KIF1A Research Network.
• NCATS (National Center for Advancing Translational Sciences, NIH)supportingStrategic or Co-development PartnerNCATS supports KIF1A.ORG as part of the Rare As One Network, a collaborative initiative supporting rare disease patient organizations in their research and drug development efforts.
• NORD (National Organization for Rare Disorders)supportingGTM or Marketing PartnerKIF1A.ORG is a Platinum member of NORD, participating in broader rare disease advocacy, information sharing, and policy efforts. NORD provides resources and a platform for ultra-rare disease organizations to amplify their voices.
• Child Neurology FoundationsupportingGTM or Marketing PartnerAwarded KIF1A.ORG the Rising Tides grant during COVID-19. Supports organizational capacity and enables research partnerships. KIF1A.ORG participates in CNF's broader advocacy and educational programs for pediatric neurological disorders.
• Rare Foundation AlliancesupportingGTM or Marketing PartnerAdvocacy organization (affiliated with Global Genes) that provides a network for rare disease foundations, connecting KIF1A.ORG with other patient groups and resources for awareness and advocacy campaigns.
• The MightysupportingGTM or Marketing PartnerHealth community platform serving as a content and advocacy partner, helping KIF1A.ORG reach broader audiences of patients, caregivers, and advocates through storytelling and peer support content.
• Rare As One Network (Coriell/NCATS)supportingStrategic or Co-development PartnerKIF1A.ORG participates in the Rare As One Network, a collaborative initiative supporting rare disease patient organizations in building research capacity, developing treatments, and advancing scientific understanding through shared tools and best practices.
• Autism BrainNetsupportingStrategic or Co-development PartnerThrough a partnership between KIF1A.ORG and Autism BrainNet, a compassionate process has been established for KAND families who wish to advance research through brain donation after death. This enables post-mortem tissue studies critical for understanding KAND neuropathology.

Scale indicators11 records

Recent moves6 records

Expansion highlights5 records

Broad incumbents

• Rare Foundation Alliance (Global Genes): Network of rare disease foundations offering shared advocacy, awareness, and capacity-building resources — KIF1A.ORG participates as a member for cross-rare-disease support and amplification.
• EveryLife Foundation for Rare Diseases: Broader rare disease advocacy and policy organization supporting patient foundations across many conditions — comparable advocacy function but at industry-association scale rather than single-disease focus.
• National Organization for Rare Disorders (NORD): Umbrella rare disease advocacy organization of which KIF1A.ORG is a Platinum member; provides shared infrastructure, policy advocacy, and grantmaking across many rare diseases rather than focusing on a single disorder.

Direct peers

• Parent Project Muscular Dystrophy: Nonprofit driving Duchenne muscular dystrophy research through funded labs, a patient registry, clinical endpoint development, and biotech partnerships — analogous operating model for a progressive neuromuscular rare disease.
• Children's Tumor Foundation: Nonprofit running patient registries (NF Registry), funding research networks, and coordinating multi-institution preclinical and clinical research programs for neurofibromatosis — analogous rare disease research infrastructure model.
• Cure SMA: Patient advocacy and research nonprofit for Spinal Muscular Atrophy that funds translational research, operates a patient registry, and built a research network that drove approval of multiple SMA therapies — directly comparable operating model to KIF1A.ORG.
• Cystic Fibrosis Foundation: Disease-focused nonprofit that funded transformative CF therapeutic development, established patient registries, and realized financial returns through drug royalty arrangements — the canonical model KIF1A.ORG's structure resembles at smaller scale.
• Rett Syndrome Research Trust: Focused rare neurological disease foundation funding translational research, biomarkers, and natural history studies — comparable disease area, research infrastructure approach, and scale.
• Charcot-Marie-Tooth Association: Patient organization funding research, building patient registries, and partnering with academic and industry researchers for a heterogeneous inherited neurological disorder — closely comparable structure and disease-class focus.

Emerging players

• Alex's Lemonade Stand Foundation: Childhood disease research nonprofit funding grants, running a research database, and supporting clinical trials — comparable model at larger scale in a different therapeutic area.

Market position

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xtwitter.com/KIF1A
• Facebookfacebook.com/KIF1A
• LinkedInlinkedin.com/company/kif1a.org
• Instagraminstagram.com/kif1a

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about KIF1A.ORG