STMN2

Arbor Biotechnologies to Present Data Supporting Therapeutic Programs in PH1 and ALS, and the Discovery of a Novel Type V Nuclease at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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Monday, April 22, 2024
CRISPR, Impact, Compact, Cell, CGT, PCSK9, PH1, STMN2, Liver, Gene, Society, ALS, Messenger, HAO1, NHP, Engineering, Medical device

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced four upcoming presentations at the 2024 American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, taking place May 7-11 in Baltimore, Maryland.

Key Points: 
  • Arbor will present in vivo NHP data supporting clinical development of ABO-101, its most advanced gene editing therapeutic candidate designed to address primary hyperoxaluria type 1 (PH1) through inactivation of the HAO1 gene in the liver.
  • The company also will present data supporting its end-to-end nuclease development platform in a third presentation outlining the discovery and optimization of a unique, compact nuclease, termed ABR-004.
  • Proof-of-concept data in non-human primates show potent, therapeutically relevant silencing of PCSK9 in vivo with the novel nuclease, signaling opportunities for broader therapeutic applications.
  • Together, the suite of presentations will demonstrate the utility of Arbor’s proprietary discovery and development approach for enabling efficient identification and optimization of novel nucleases.

QurAlis and Unlearn Announce Collaboration to Accelerate and Optimize ALS Clinical Trials With Generative Artificial Intelligence Technologies

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Tuesday, June 27, 2023
STMN2, Isabella Tree, Clinical trial, Patient, Machine learning, SAN, Artificial intelligence, Digital, AI, Bias, ALS, Pharmaceutical industry, Medical imaging, Medical device

CAMBRIDGE, Mass. and SAN FRANCISCO, June 27, 2023 /PRNewswire/ -- QurAlis Corporation, a clinical-stage biotechnology company developing breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, and Unlearn, a pioneering technology company innovating machine learning to revolutionize medical research, today announced they have entered into a collaboration to accelerate and optimize QurAlis' clinical program in ALS with Unlearn's advanced generative artificial intelligence (AI) technology.

Key Points: 
  • Unlearn develops digital twins of clinical trial patients that are predictions of individual health outcomes under the control treatment over time.
  • Digital twins are employed in randomized controlled trials (RCTs) called TwinRCTs to run more efficient trials that produce regulatory-suitable evidence .
  • The collaboration aims to reduce variability and increase the study power in QurAlis' clinical trials for QRL-201 and QRL-101, the Company's lead product candidates in ALS.
  • QRL-201 rescues STMN2 loss of function in QurAlis ALS patient-derived motor neuron disease models in the presence of TDP-43 pathology.

QurAlis Presents at ALS Drug Development Summit 2022

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Wednesday, May 25, 2022
Dementia, FTD, Death, Phenotype, KCNQ2, Disease, Neurodegeneration, Drug development, Parkinson's disease, RNA splicing, Motor neuron, Gene, Alzheimer's disease, GWAS, Cytoplasm, Patient, Multimedia, View, Paralysis, Amyotrophic lateral sclerosis, Research, Frontotemporal dementia, Genetics, Harvard University, Biomarker, KvLQT2, Human, Mouse, RNA, HIV disease progression rates, Pathology, Maintenance, Therapy, CMO, Peripheral neuropathy, Harvard Medical School, STMN2, Electrophysiology, ALS, CEO, Pharmaceutical industry, Medical device, Medical imaging, Agriculture, Golgi

CAMBRIDGE, Mass., May 25, 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 that Kasper Roet, Ph.D., founder and chief executive officer (CEO) and Angela Genge, M.D., chief medical officer (CMO), will deliver presentations at the ALS Drug Development Summit being held May 24-26, 2022 in Boston, Massachusetts.

Key Points: 
  • "The ALS Drug Development Summit brings together ALS experts, researchers, and industry leaders to discuss recent clinical developments, research and development trends, and breakthrough innovations to transform ALS drug development.
  • "Biomarkers are critical to the successful development of therapeutics for patients with ALS.
  • For information about the ALS Drug Development Summit, visit www.als-drug-development.com .
  • QurAlis' proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations.

QurAlis Presents Data About STATHMIN-2 Role in Neuronal Disease Biology and TDP-43 Biomarker Identification at AD/PD™ 2022

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Tuesday, March 15, 2022
Harvard Medical School, Disease, FTD, Dementia, Human, Paralysis, Patient, Alzheimer's disease, Asos, Conference, Multimedia, RNA splicing, Frontotemporal dementia, Motor neuron, Parkinson's disease, Axon, Neuron, Vance DeGeneres, Amyotrophic lateral sclerosis, Neurodegeneration, Cell, Biomarker, Research, STMN2, Cell biology, View, Harvard University, ALS, Golgi apparatus, Microtubule, Biology, Maintenance, Pathology, Nature Neuroscience, RNA, Pharmaceutical industry, Medical device, Golgi

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.

Key Points: 
  • 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.
  • "Our research shows for the first time, the connection between STMN2, TDP-43, and important cell biology of ALS.
  • Patient identification and stratification, as well as early identification of target engagement can be crucial to the development of effective therapies.
  • QurAlis' proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations.

QurAlis Presents New Data Confirming Role of STATHMIN-2 in Neuronal Disease Biology at 32nd International Symposium on ALS/MND

Retrieved on: 
Tuesday, December 7, 2021
Central nervous system, Microtubule, RNA splicing, Research, Population, Alzheimer's disease, Axon, Frontotemporal dementia, Temperature, Neurodegeneration, Motor neuron, Cell death, Drug discovery, Disease, Harvard University, Long-term impact of alcohol on the brain, View, Patient, Cell biology, Vance DeGeneres, Biomarker, Asos, Parkinson's disease, Golgi, Harvard Medical School, Amyotrophic lateral sclerosis, STMN2, Paralysis, CNS, Multimedia, The Human-Induced Earthquake Database, RNA, Symposium, Nature Neuroscience, Survival, Doctor of Philosophy, ALS, Golgi apparatus, Cell, Dementia, Vaccine, Pharmaceutical industry

CAMBRIDGE, Mass., Dec. 7, 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 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 research to enable central nervous system (CNS) drug discovery in poster sessions at the 32nd International Symposium on ALS/MND being held virtually, December 7-10, 2021.

Key Points: 
  • Our research shows for the first time the connection between STMN2, TDP-43, and important cell biology of ALS.
  • Dr. Hinckley stated, "One of the challenges in the research of neurodegenerative diseases, such as ALS, is the availability of neuronal cells.
  • Our approach is incredibly enabling from a drug discovery perspective to help neuronal research move more rapidly."
  • QurAlis' proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations.