QurAlis Announces Publication in Molecular Biology of the Cell of Next Generation Drug Screening Platform for ALS Developed by Company Founders

CELLXPEDITE platform enables discovery of molecules and therapeutics that counteract the multi-faceted pathological features of diseased cellular activity in ALS

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QurAlis

Dec 23, 2021, 07:45 ET

CAMBRIDGE, Mass., Dec. 23, 2021 /PRNewswire/ -- QurAlis Corporation, a biotech company developing breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, today announced research published in Molecular Biology of the Cell that described the discovery of CELLXPEDITE, a new-generation drug screening platform for ALS.

QurAlis founders Kasper Roet, PhD, (QurAlis CEO; Boston Children's Hospital), Clifford Woolf, MD, PhD, (Boston Children's Hospital), and Kevin Eggan, PhD, (Harvard University), and Bruno Boivin (Boston Children's Hospital), together with Anne Carpenter, PhD, and other scientists from the Broad Institute, developed CELLXPEDITE, a high-throughput cloud-based image processing and unbiased multiparametric activity profiling analytic platform to guide the development of therapeutics that counteract the multifaceted pathological features of diseased cellular activity in ALS.

"The work described in the Molecular Biology of the Cell paper highlights the existence of complex and previously unexploited facets of the ALS excitability phenotype and the discovery of compounds that robustly correct many parameters of this phenotype simultaneously," said Dr. Roet, CEO of QurAlis and co-first study author. "Here we combine advances in human stem cell-derived neuronal models, fluorescent reporters, high throughput live cell imaging systems, and cloud-based analysis platforms, to enable the discovery of molecules that can transform a complex disease phenotype to a healthy phenotype."

Patient stem cell-derived models enable imaging of complex disease phenotypes and the development of scalable drug discovery platforms. Traditional high throughput screens focus on one parameter but using patient derived stem cell models in combination with the newly developed cloud-based image processing and analysis platform CELLXPEDITE enables the discovery of molecules that correct 153 parameters that define the complex ALS motor neuron disease phenotype. Most drugs for neurodegenerative diseases fail in clinical trials due to poor efficacy or unforeseen side effects, which is a health risk for patients and costly for the pharmaceutical industry. A multiparametric understanding of drug candidates is likely to increase the probability of clinical success. CELLXPEDITE is a computationally efficient and deterministic approach, one that can be launched by the push of a button.

"We call it CELLXPEDITE for its ability to process large volumes of cellular imaging data, thereby accelerating drug screening, and make it open source for use by the neuroscience community. The CELLXPEDITE platform can be easily adapted to define complex activity disease phenotypes for other disorders and for the subsequent discovery of molecules that correct them. This discovery, coupled with the sensitive cloud-based high-throughput multiparametric framework for capturing and analyzing subtle changes in cellular activity, represents a substantial leap forward for comprehensive disease profiling and drug screening in the pursuit of novel treatments for ALS and other diseases," added Dr. Roet.

About CELLXPEDITE
CELLXPEDITE is a cloud-based high-throughput image processing and analysis platform that captures the intricate activity profile revealed by GCaMP fluorescent recordings of intracellular calcium changes and enables the discovery of molecules that correct 153 parameters that define the ALS motor neuron disease phenotype. This platform can guide the development of therapeutics that counteract the multifaceted pathological features of diseased cellular activity.

Once adjusted for the type of cells being studied and the recording frequency, the CELLXPEDITE platform approach does not require human intervention and can be applied to any number of recordings, provides comprehensive activity profiles, and is entirely reproducible. This is particularly useful when studying complex genetic diseases such as ALS, since it allows for the comparison of the activity profiles of different mutations under the same treatment.

CELLXPEDITE is available as an open-source software (https://github.com/brunoboivin/cellxpedite). It is currently designed for deployment on Amazon Web Services, but it is also compatible with local clusters and other cloud computing service providers with minimal changes to the deployment scripts.

About QurAlis Corporation
QurAlis is trailblazing the path to conquering amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets with next-generation precision medicines. QurAlis' proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a deep pipeline of antisense oligonucleotides and small molecule programs including addressing sub-forms of ALS that account for the majority of ALS patients. For more information, please visit www.quralis.com or follow us on Twitter @QurAlisCo.

SOURCE QurAlis