sRNA-FIND uncovers biomarkers that track progression of Huntington’s
disease 20 years before symptomatic onset
BOSTON–(BUSINESS WIRE)–The FDA’s Center for Drug Evaluation and Research (CDER) Biomarker
Qualification Program issued a Letter
of Support to sRNAlytics
encouraging the use and development of the sRNA-FIND platform, and a
biomarker panel it discovered, that monitors Huntington’s disease
progression 20 years before symptomatic onset.
Current drug programs targeting Huntington’s disease utilize subjective
measures to evaluate a patient’s overall physical and neurological
health to measure efficacy. However, symptoms such as motor and
cognitive impairment can vary from day-to-day. “The use of objective
biomarkers to assess Huntington’s Disease in clinical trials will enable
us to more accurately determine if treatments are effective. sRNAlytics’
discovery of an objective biomarker will help transform the way we think
about therapeutic development and treating patients,” said Dr.
Blair Leavitt, Professor at the University of British Columbia.
In this recent study, sRNAlytics used their sRNA-FIND platform to
discover small RNA biomarkers in the brain tissue of deceased
Huntington’s patients. In collaboration with researchers at the Boston
University Medical School, they showed that these biomarkers could
monitor Huntington’s disease progression 20 years prior to symptomatic
onset in a liquid biopsy. Additional work showed correlations with
neuronal cell death and loss of motor functions in patients. Dr.
Richard Myers, a Professor of Neurology and study lead commented:
“The small RNA profiles that sRNAlytics identified represent the most
promising approach to test efficacy that I have seen to date. The FDA’s
recognition of these findings is an important step forward in validating
their technology and accelerating their future use in clinical trials.”
sRNAlytics is initiating partnerships with Huntington’s drug developers
to identify new efficacy and patient selection markers using sRNA-FIND.
“Receiving the Letter of Support is a major milestone in the development
of our platform. In a few years, we’ll be able to look back and know
that we played a role in delivering game-changing therapies for
patients,” said Dr. David W. Salzman, the company’s CEO.
is an AI-powered, computational platform that analyzes small RNA
sequencing data to uncover new insights around the genes and molecular
pathways driving disease. The platform includes a proprietary suite of
algorithms that have cataloged over 4.2 million small RNAs used to fuel
its strong power to discover. One of the outputs of sRNA-FIND are
biomarkers that translate across species and biological matrixes and can
be tracked as they move from a diseased tissue into the periphery via
exosomes. Applications of sRNA-FIND have been validated across CNS,
Oncology and Immunology disease areas.
is at the forefront of small RNA research. They offer technology and
services to drug developers to facilitate drug discovery and development
by providing deep analysis of disease pathways within a drug’s mechanism
of action. Their analytical approach and lab protocols combined with
sRNA-FIND and a growing database of over 15,000 patient samples is
focused on accelerating their partners’ programs.