KIF1A.ORG Community,

Today we’re highlighting the exciting work we’re doing with Albany Molecular Research Inc. (AMRI) to support KIF1A.ORG’s therapeutic development efforts. This project was made possible by a Rising Tides grant awarded by The Child Neurology Foundation to support organizations during the COVID-19 pandemic.

When COVID-19 forced academic labs around the world to abruptly shut down in Spring 2020, we took immediate action to limit the disruption to KIF1A research. What was first an emergency response has now become a permanent strategy of our organization to protect and accelerate the progress of KIF1A research and development of treatment. In addition to strengthening and expanding our research network, KIF1A.ORG has engaged private labs and contract research organizations (CROs) like AMRI to ensure our assets remain safe during possible pandemic closures in labs. Relationships with organizations like AMRI ensure ongoing KIF1A research activities do not slow down.

AMRI is a reliable and innovative CRO with a range of services that support therapeutic development. This team works collaboratively with other KIF1A researchers in our network. Our first project with AMRI is an in silico docking study using existing compounds to support the hit-to-lead process and model KIF1A crystal structure to understand the location and possible effects of known gain-of-function and loss-of-function mutations. What does that mean? Let me borrow an explanation from KIF1A.ORG’s Chief Science Officer, Dr. Dominique Lessard:

“Unlike a team of scientists working on a lab bench to do experiments, the team at AMRI is using sophisticated computer technology to conduct computational experiments that help us understand the shape and structure of the KIF1A protein. Data from the KIF1A natural history study and other published KIF1A studies have informed much of this work. This allows us to apply a clinical understanding of KAND to the project, increasing our understanding of reported KIF1A variants. The goal with this project is to help identify certain drugs, or compounds, that fit well into KIF1A’s unique shape.”

Mutation mapping on a crystal structure of KIF1A created by AMRI

KIF1A.ORG is making the results of this work freely available to the scientific community to accelerate drug development for KAND. This is another powerful example of our current projects leveraging the foundational Tools for Development that the KIF1A.ORG patient community has built over the last three years. This advancement in KIF1A research would not be possible without donations to support the mission of KIF1A.ORG and engagement in research from KIF1A families. Learn more about this and other projects on our Funded Projects page, and donate today to accelerate our urgent mission.


Kathryn Atchley
President, KIF1A.ORG

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