NeuCyte Logo with tagline: "Translatable Neuroscience"

KIF1A.ORG Community,

Earlier this week, we announced NeuCyte as the first partner of KIF1A.ORG’s Treatment Accelerator Program to fast-track therapeutic discovery for KIF1A Associated Neurological Disorder (KAND). This collaboration will make significant leaps in our understanding of KAND and drug discovery capabilities. Because the KIF1A.ORG community rallied to fund this initiative, we’re able to make this resource openly available to the scientific community to speed up and expand our search for treatments.

In April 2021, we launched a campaign to kickstart the Treatment Accelerator Program (TAP). Why? Over the last few years, we’ve identified several potential therapeutic options for KAND. But we can’t just give these drugs to KAND patients or even start a clinical trial until we do the preclinical work to show their effectiveness. Through TAP, KIF1A.ORG will hire Contract Research Organizations (CROs) to design and manage systems to model KAND in cells and test potential therapeutics. (CROs are specialized labs that pharmaceutical companies often use to execute research and development.)

Illustration of TAP: DIFFERENTIATE: Create neurons with KIF1A mutations to model the disease. PHENOTYPE: Collect data on observable characteristics of healthy neurons vs. KAND mutations. SCREEN: Test promising treatments for KAND, using insights from phenotyping to look for measurable improvements. IDENTIFY THERAPEUTIC CANDIDATES FOR CLINICAL TRIALS

With guidance from key members of the KIF1A Research Network, the KIF1A.ORG team has spent the last few months exploring the capabilities of several CROs to identify our first TAP partner. We’re excited about the possibilities of teaming up with these organizations to leverage their efficiency and expertise—just like a pharmaceutical company would.

NeuCyte immediately stood out as a high-performing biotechnology company with the capabilities and experience we need. Specializing in neurological diseases and drug discovery optimization, they are a perfect partner to help advance KIF1A.ORG’s therapeutic development strategy.

As we have come to know the team behind NeuCyte, including Tao Huang, PhD, JD, President and CEO, and Ji Wu, VP of Business Development and Operations, their commitment to accelerating science to improve the lives of KAND patients and their families has impressed us just as much as their scientific capabilities. If you missed the press release announcing our collaboration, check it out here.

To each and every donor who made this initiative possible: thank you. Because of you, we’re able to expand KIF1A.ORG’s Tools for Development. We are literally saving time and money—two of our biggest obstacles to KAND treatment—by investing in the scientific tools that our academic and industry collaborators need to advance KIF1A science and discover treatment. We cannot depend on every academic lab or biotech to create their own model system specifically designed for KIF1A. That would be an inefficient use of time and resources. In collaboration, we can work so much smarter and faster.

Just last month at the 2021 KAND Family & Scientific Engagement Conference, one of our collaborators shared a powerful example of how our community’s Tools for Development work. In his update on Rarebase’s therapeutic development program for KAND, Rarebase Co-Founder and COO Omid Karkouti reported that KIF1A.ORG’s Tools for Development saved Rarebase six months—SIX MONTHS!—in our development timeline.

Patients and families in this community know that every single day matters in our effort to outpace the ticking degeneration of KAND. With no treatment options and time running out for our friends living with KAND, we must work together with urgency. 

Please help me send our sincere thanks to the NeuCyte team for joining our mission to discover treatments and ultimately a cure for KAND. We have an aggressive timeline as NeuCyte executes the first module over the next few months, characterizing iPSCs and producing induced neurons. We’ll keep the KIF1A community, including families and scientists, up to date along the way and freely share our learnings.


Kathryn Atchley
President, KIF1A.ORG

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