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KIF1A.ORG Community,

KIF1A.ORG is excited to expand our pipeline of therapeutic development projects for KIF1A Associated Neurological Disorder (KAND) through our partnership with Rarebase. Rarebase is a public benefit corporation pioneering a new approach to biotech by partnering with patient communities like ours. They share our sense of urgency and optimism in today’s science to unlock treatments for rare disorders like KAND. Together, we can expedite the path to treatment for KAND and beyond.

It’s not just the robust scientific capabilities of Rarebase we’re impressed with—it’s the people. Rarebase was founded by Onno Faber and Omid Karkouti, two biotech leaders impacted by rare disease. You can learn more about them in this blog post from Onno, but I think this particular part of their founders’ story will stand out to you as it did to me:

“When we first met, we realized that we shared the same vision – that translating science into therapies needs more than just technology, we need a different business model.

We need a company that cares about exactly the same thing as the patient community: a tangible treatment as fast and reliably as possible, not contingent on its novelty or ability to be commercialized.”

Onno Faber, Co-founder and CEO of Rarebase

This is the approach and commitment we need to swiftly bring treatment options to people living with KAND. Our project with Rarebase aims to develop an antisense oligonucleotide (ASO) to reduce pathological axonal degeneration. As this form of degeneration is seen in many neurological disorders, such as KAND and hereditary spastic paraplegia, this approach can be applied to a broader spectrum of diseases that are characterized by neurodegeneration. The initial scope of work for this project began in March 2021, and includes iPSC differentiation, identification of markers for neurodegeneration, and ASO testing in KIF1A motor neurons.

This project would not be possible without the entire KIF1A.ORG community, including patients, families, researchers and donors, who work together to advance our mission. Learn more about this and other projects on our Funded Projects page, and donate today to help KIF1A.ORG accelerate and expand our projects to rapidly develop treatment for KAND.


Kathryn Atchley
President, KIF1A.ORG

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