KIF1A.ORG’s 12th Research Roundtable meeting, “Research Updates with KIF1A.ORG Therapeutic Development Partners,” was presented by Omid Karkouti, Co-Founder and COO of Rarebase, and Dr. Douglas Chapnick, Founder and CEO of BioLoomics.

Attendance

31 RESEARCHERS, CLINICIANS, & BIOTECH REPS

16 RESEARCH INSTITUTIONS & INDUSTRY PARTNERS/ORGS

6 KIF1A.ORG REPS

Who Is 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. 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. Learn more about our collaboration here.

Who Is BioLoomics?

BioLoomics is a preclinical stage biotechnology company with a unique evolution-in-a-microscope technology to build high throughput drug discovery tools that detect candidate therapeutics for KAND, as well as identify novel drug targets. As we work relentlessly to outpace the ticking clock of neurodegeneration, we are grateful to the team at BioLoomics for joining our mission to bring treatment to this generation of people affected by KAND. BioLoomics uses this tech to make drug discovery tools, biosensing drugs, and gene therapies. Their unique approach requires less knowledge about a disease’s mechanism of action to find effective treatments than most drug discovery strategies and expands the search potential far beyond 96-well formats. Our collaborative project began in June 2021. Learn more about our collaboration here.

Summary

Rarebase

  • Rarebase walked us through their therapeutic discovery roadmap, highlighting ways in which they are working to target KAND-related neurodegeneration.
  • We were updated on their progress with ASO development. So far, their delivery and target controls, which can be thought of as ways to confirm that their system is working, are looking very good. Preliminary data of their neurodegenerative target being inhibited by ASO is also looking very promising.
  • As a next step, Rarebase is actively working on moving this work into neurons derived from cells isolated from KAND patients.
  • We had a great discussion in the Q&A session following this talk about translational medicine and how researchers can bridge the gap between basic science ideas and therapeutic discovery.

BioLoomics

  • BioLoomics walked us through their evolution-in-a-microscope technology, detailing how they are using biosensors to help find targets for KAND therapeutic development.
  • We were updated on their progress with target identification revealing that, in the first round of experimentation, they had identified many biosensors for further investigation and validation.
  • As a next step, BioLoomics is moving their findings forward through various stages of thorough target validation.
  • Our Research Network members had many great questions about this impressive technology. We even identified some areas of overlap between Research Network members that facilitated some great brainstorming!

Main Takeaways

  • Our therapeutic development partners are making great progress on their respective projects and therapeutic approaches.
  • Though these approaches are different, they often hinge around the same questions and gaps in knowledge regarding KIF1A/KAND.
  • Having our partners present at Research Roundtable meetings is a great way for our Research Network to provide feedback and promote collaborative discussion to improve the efficacy and speed of KAND therapeutic discovery!

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