QurAlis Corporation today announced the presentation of preclinical findings from its STATHMIN-2 (STMN2) program,
| Connection between STATHMIN-2, TDP-43, and ALS cell biology led to discovery of Company’s approach and lead development candidate in ALS Updated data on TDP-43 biomarker identification informed Company’s fully developed assay for ALS and other neurodegenerative diseases including Alzheimer’s Disease and Parkinson’s disease CAMBRIDGE, Mass., March 15, 2022 /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 the presentation of preclinical findings from its STATHMIN-2 (STMN2) program, including novel biology linking TDP-43 loss to impaired Golgi trafficking through STMN2 function and rescue by QurAlis’ antisense oligonucleotides (ASOs), as well as updated research on TDP-43 biomarker identification in oral presentations at AD/PD 2022, Alzheimer’s and Parkinson’s Diseases Conference being held March 15-20, 2022 in Barcelona, Spain.
Details of the presentations are as follows: Title: The Impacts of TDP-43 Loss on STATHMIN-2 Expression, Golgi Apparatus Morphology, and Neurite Outgrowth, in Human Cortical and Motor Neurons STATHMIN-2 is a well-known protein that plays an important role in axonal growth and maintenance and is the most significantly regulated gene by TDP-43 exclusively in humans. STATHMIN-2 expression is significantly decreased in nearly all ALS patients and it is the most consistently decreased gene in all sporadic ALS patient data sets. TDP-43 is an RNA binding protein involved in regulation of RNA splicing. In addition to nearly all ALS patients, TDP-43 pathology is also associated with other neurodegenerative diseases including frontotemporal dementia (FTD) which is the second most common form of dementia, Alzheimer’s Disease, and Parkinson’s disease. We and others have discovered that the rescue of STMN2 even in the presence of TDP-43 pathology is enough to restore neurodegenerative phenotypes including neuronal processes and Golgi transport. “Our research shows for the first time, the connection between STMN2, TDP-43, and important cell biology of ALS. These findings validated our discovery approach and led to our clinical candidate QRL-201, for the recovery of STMN2 expression in patients with ALS,” said Dr. Elbaum, QurAlis’ chief scientific officer, and study co-author. Title: Biomarkers for Patient Stratification and Target Engagement in Patients with TDP-43 Pathology “TDP-43 pathology has been described in nearly all ALS patients, up to 50 percent of FTD, approximately 30 percent of Alzheimer’s Disease, and about one-tenth of patients with Parkinson’s disease. Patient identification and stratification, as well as early identification of target engagement can be crucial to the development of effective therapies. We have developed biomarker assays that for the first time detect STMN2 protein and RNA in human biofluids and characterized these biomarkers in ALS patients and healthy individuals. These assays could be used to identify patients with TDP-43 proteinopathy and help with the research and discovery of treatments for ALS, FTD, and other neurodegenerative diseases,” said Dr. Hinckley, senior director, head of discovery at QurAlis, and study co-author. Presentations will be available on-demand on the conference website on March 15, 2022 at 09:00 CEST. The presentations will also be available on the QurAlis website at the conclusion of the conference. The Link Between STATHMIN-2 and TDP-43 About QurAlis Corporation
SOURCE QurAlis |
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