Efferent neurons

Cell Reports Publishes Discovery Data Supporting the Importance of Ion Channel, Kv7.2/7.3 as a Therapeutic Target in ALS

Retrieved on: 
Tuesday, June 8, 2021
Research, Genetics, Clinical trials, Other Health, Biotechnology, Managed care, Pharmaceutical, Health, Science, Other Science, Neurological disorders, Branches of biology, Neurodegenerative disorders, Efferent neurons, Amyotrophic lateral sclerosis, Motor neuron, Neuron, Kv7.2/7.3 Ion Channel Openers, QRL-101 (QRA-244), QurAlis Corporation, KV7.2/7.3 ION CHANNEL OPENERS, QRL-101 (QRA-244), QURALIS CORPORATION

The publication describes a live-cell screening strategy targeting abnormal electrophysiological properties to reveal targets that modulate the intrinsic hyperexcitability of ALS motor neurons.

Key Points: 
  • The publication describes a live-cell screening strategy targeting abnormal electrophysiological properties to reveal targets that modulate the intrinsic hyperexcitability of ALS motor neurons.
  • This unbiased screen using human ALS motor neurons identified Kv7.2/7.3 as a strongly overrepresented drug target.
  • Previous research has identified Kv7.2/7.3 as an ALS drug target and the unbiased nature of this screen further emphasizes the importance of Kv7.2/7.3 in ALS motor neuron dysfunction.
  • We are advancing three antisense and small molecule programs addressing sub-forms of ALS that account for the majority of ALS patients.

QurAlis' Selective Kv7 Opener, a Drug Candidate for ALS, Demonstrates up to 20-Fold Increase in Potency and Dramatic Reduction in Side Effects Compared to Retigabine in Preclinical Models

Retrieved on: 
Friday, December 11, 2020
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Neurological disorders, Neuroscience, Branches of biology, Rare diseases, Motor neuron diseases, Efferent neurons, Motor system, Amyotrophic lateral sclerosis, Retigabine, Motor neuron, QRA-244, ALS, QurAlis Corporation

QRA-244 is a Kv7.2/7.3 ion channel opener optimized for both safety and efficacy as a potential treatment for motor neuron hyperexcitability-induced neurodegeneration in ALS patients.

Key Points: 
  • QRA-244 is a Kv7.2/7.3 ion channel opener optimized for both safety and efficacy as a potential treatment for motor neuron hyperexcitability-induced neurodegeneration in ALS patients.
  • Importantly, QRA-244 showed dramatic reductions in side effects associated with retigabine including fatigue/somnolence, dizziness and bladder retention in head-to-head pre-clinical studies.
  • Degeneration of motor neurons in ALS patients leads to loss of innervation of muscles, which leads to paralysis and eventually fatality.
  • Together with a world-class network of thought leaders, drug developers and patient advocates, our team is rising to the challenge of conquering ALS.