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

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CELLXPEDITE platform enables discovery of molecules and therapies that counteract the multifaceted pathological features of diseased cell activity in ALS

CAMBRIDGE, Mass., 23 December 2021 / PRNewswire / – QurAlis Corporation, a biotechnology company developing revolutionary precision drugs for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, today announced research published in Molecular Biology of the Cell which describes the discovery of CELLXPEDITE, a new next-generation drug screening platform for ALS.

Logo of QurAlis Corporation (PRNewsfoto / QurAlis)

Founders of QurAlis Kasper Roet, PhD, (CEO of QurAlis; 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), in collaboration with Anne Carpentier, PhD, and other scientists at the Broad Institute, have developed CELLXPEDITE, an analytical platform for high-throughput, cloud-based image processing and unbiased multiparametric activity profiling to guide the development of therapies that counteract Multifaceted pathological features of diseased cell activity in ALS.

“The work described in the article on the molecular biology of the cell highlights the existence of complex and previously untapped facets of the excitability phenotype of ALS and the discovery of compounds that robustly correct many parameters of this. phenotype simultaneously, ”said Dr. Roet, CEO of QurAlis. and co-first author of the study. “Here, we combine advances in neural models derived from human stem cells, fluorescent reporters, high-throughput living cell imaging systems and cloud-based analysis platforms, to enable the discovery of molecules capable of transforming a complex disease phenotype into a healthy phenotype. “

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

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

About CELLXPEDITE
CELLXPEDITE is a high-throughput, cloud-based image processing and analysis platform that captures the complex activity profile revealed by fluorescent GCaMP 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 therapies that counteract the multifaceted pathological features of diseased cell activity.

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

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

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

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