The KIF1A Research Network is a collaborative network of researchers, clinicians, industry leaders and innovators from around the world working to accelerate treatment for this generation of patients affected by KIF1A Associated Neurological Disorder (KAND). Led by KIF1A.ORG and centered in our mission to improve the lives of KAND patients and families, the KIF1A Research Network works in collaboration to fill gaps in knowledge and pursue multiple therapeutic strategies for KAND.

With this neurodegenerative disorder, every day matters. We have no time to waste working alone or in sequential order. By joining forces with KIF1A.ORG, the KIF1A Research Network is doing more—faster—to bring life-changing and life-saving treatments to KAND patients.

Over 50 institutions from around the world have joined the KIF1A Research Network. If your institution or company wants to collaborate with a relentless, science-driven community of families, researchers, clinicians and innovators, we want to hear from you. Contact KIF1A.ORG’s Chief Science Director, Dr. Dylan Verden, to join our mission and learn how KIF1A.ORG de-risks and supports therapeutic development for KAND.

Therapeutic Collaboration Highlights

Core KIF1A Research Network Members

Therapeutic Development Collaborators

Chung Lab, Boston Children’s Hospital

KIF1A.ORG-Sponsored Collaboration

Led by Wendy Chung, MD, PhD, Chung Lab manages the world’s most comprehensive pre-clinical KIF1A research program, including a natural history and endpoint-enabling study, cell and animal modeling and clinical disease characterization. Dr. Chung leads and advises multiple therapeutic development collaborations with KIF1A.ORG.

Ovid Therapeutics

Ovid is a biopharmaceutical company focused on developing medicines for patients and families living with rare neurological disorders. In collaboration with Chung Lab, Ovid is developing genetic-based treatments for KAND through aptamer protein targeting and RNAi gene targeting.

NeuCyte

KIF1A.ORG-Sponsored Collaboration

NeuCyte is a biotechnology company solely focused on neurological diseases known for their cell-based target identification and drug discovery platforms. Their KIF1A program is creating cell-based models of KAND to better understand the disorder, identify treatment strategies and test potential therapeutics.

Gennerich Lab, Albert Einstein College of Medicine and Atomwise

KIF1A.ORG-Sponsored Collaboration

In collaboration with Hernando Sosa, PhD, and the AI drug discovery company Atomwise, Arne Gennerich, PhD, is combining cryo-electron microscopy (cryo-EM) and artificial intelligence to improve our structural knowledge of the KIF1A protein and search for small molecule compounds to treat KAND.

BioLoomics

BioLoomics is a preclinical biotechnology company that uses their “evolution-in-a-microscope” technology to build high throughput drug discovery tools. Their KIF1A program is creating improved assays (a special type of test that can be used to measure effects of therapeutic candidates) and identifying novel targets for KAND therapeutic development.

Christodoulou Lab, Murdoch Children’s Research Institute

KIF1A.ORG-Sponsored Collaboration

Led by Professor John Christodoulou and Simran Kaur, PhD, the Christodoulou Lab is creating a high throughput drug screening platform to test FDA-approved drugs that can be repurposed to improve KIF1A molecular function. In November 2021, the lab was also awarded a three-year grant from the Australian government to study epilepsy treatments for KAND.

Disease Modeling Collaborators

The Jackson Laboratory

JAX is a nonprofit biomedical research institution that pioneered the use of laboratory mice as models for human disease to discover treatments. JAX’s KIF1A program includes multiple mouse models and a variety of genetically engineered KIF1A variant cell lines with isogenic control that are available to the KIF1A Research Network.

Coriell Institute for Medical Research

Coriell is a nonprofit biomedical research center dedicated to the study of the human genome. In collaboration with KIF1A.ORG, Coriell manages a biobank of induced pluripotent stem cells (iPSCs) derived from KAND patient blood samples that can be used in research and therapeutic development.

Chitnis Lab, National Institutes of Health

Led by Ajay Chitnis, MBBS, PhD, the Chitnis Lab in the Section on Neural Developmental Dynamics at the National Institutes of Health specializes in studying zebrafish models to better understand the nervous system. Their KIF1A program has created a zebrafish model of KAND that is available to the KIF1A Research Network.

Niwa Lab, Tohoku University

Led by Shinsuke Niwa, PhD, the Niwa Lab studies the molecular mechanism of cellular morphogenesis, kinesin and microtubule networks. Their KIF1A program has created a variety of C. elegans models of KAND (roundworms) that are available to the KIF1A Research Network.

Additional Collaboration Highlights

Bain Lab, Columbia University

KIF1A.ORG-Sponsored Collaboration

Jennifer M. Bain, MD, PhD, is an assistant professor of neurology and pediatrics at Columbia University Irving Medical Center and has collaborated closely with KIF1A.ORG and Dr. Wendy Chung since 2017. Most recently Dr. Bain is co-leading a KAND epilepsy study with Dr. Tristan Sands to provide crucial insights into epilepsy diagnosis, evaluate existing treatments, and help develop new treatments.

Sands Lab, Columbia University

KIF1A.ORG-Sponsored Collaboration

Tristan Sands, MD, PhD, is an assistant professor of neurology at Columbia University Irving Medical Center and is a pediatric and neonatal neurologist and epileptologist. Dr. Sands is co-leading a KAND epilepsy study with Dr. Jennifer Bain and providing his expert interpretation of EEGs to search for electrophysiological patterns in KAND to help improve diagnosis and treatment.

Holzbaur Lab, University of Pennsylvania

KIF1A.ORG-Sponsored Collaboration

Led by Erika Holzbaur, PhD, the Holzbaur Lab studies molecular motors like KIF1A, the cell biology of neurons and neurodegeneration. In the lab, Jayne Aiken, PhD, is leading a project to study how disease-causing mutations of KIF1A alter neuronal function to search for rescue strategies and determine whether gene-transfer approaches are viable for specific classes of mutations.

Silverman Lab, Simon Fraser University

KIF1A.ORG-Sponsored Collaboration

Led by Michael Silverman, PhD, the Silverman Lab is a group of cellular neurobiologists who study the process of microtubule-based organelle transport. Their KIF1A program is characterizing microtubule-based cargo transport in KAND patient-derived induced neurons to improve our understanding of KIF1A biology and identify therapeutic pathways.

Frontier Research Institute for Interdisciplinary Sciences, Tohoku University, Japan

KIF1A.ORG-Sponsored Collaboration

Kumiko Hayashi, PhD, is leading a project to develop a non-invasive force measurement method to study KIF1A axonal transport in human iPSC derived neurons. This work will improve our understanding of the effects of KIF1A mutations on axonal transport and help identify biomarkers that can used in therapeutic development.

Barkina Lab & Miralinc Pharma, Inc.

KIF1A.ORG-Sponsored Collaboration

In collaboration with Miralinc Pharma (formerly HNF Pharma) and The Jackson Laboratory, the Barinka Lab at the Institute of Biotechnology of the Czech Academy of Sciences in the Czech Republic is investigating the use of HDAC6 inhibition as a potential therapeutic for KAND and related disorders such as Charcot-Marie-Tooth (CMT) disorder.

McKenney Lab, University of California, Davis

Previous KIF1A.ORG-Sponsored Collaboration

Led by Richard McKenney, PhD, the McKenney Lab studies how dysfunction in motor proteins like KIF1A leads to human diseases such as KAND. Their KIF1A program uses microscopy to visualize KIF1A proteins and their movement characteristics to help identify therapeutic strategies for KAND.

Curia

Previous KIF1A.ORG-Sponsored Collaboration

KIF1A.ORG began working with Curia (formerly AMRI) in 2020 to use in silico (or computer-based) modeling to identify potential strategies and targets for KAND therapeutic development. The results of this project are openly available to the KIF1A Research Network.

Vallee Lab, Columbia University

Richard Vallee, PhD, has a long-standing research interest in biological phenomena involving motor proteins. Dr. Vallee and team investigate the role of KIF1A in brain development, specifically during basal nuclear migration and in BDNF transport and secretion, which, in turn, markedly affects neuronal migration.

Verhey Lab, University of Michigan

The Verhey Lab is dedicated to uncovering how the interplay between microtubules and molecular motors facilitate intracellular transport in mammalian cells. Headed by Dr. Kristen Verhey, PhD, the Verhey Lab’s contributions have dramatically shaped our current understanding of KIF1A through a multitude of projects with heavy emphasis on collaboration with the KIF1A research community.

Hancock Lab, The Pennsylvania State University

The Hancock Lab is dedicated to elucidating the underlying mechanisms of motor proteins, kinesin and dynein, that contribute to cell function. By studying the kinetic rates of KIF1A’s transition states, Dr. William O. Hancock and team have been able to biochemically characterize the KIF1A chemomechanical cycle.