KvLQT2

QurAlis Presents at ALS Drug Development Summit 2022

Retrieved on: 
Wednesday, May 25, 2022

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.

Knopp Biosciences Receives Rare Pediatric Disease Designation for Kv7 Activator KB-3061 for Treatment of KCNQ2 Epileptic Encephalopathy

Retrieved on: 
Wednesday, July 15, 2020

Food and Drug Administration(FDA) forits therapeutic candidate KB-3061, an activator of voltage-gated Kv7.2/7.3 potassium channels, for the treatment of KCNQ2 epileptic encephalopathy (KCNQ2-EE).

Key Points: 
  • Food and Drug Administration(FDA) forits therapeutic candidate KB-3061, an activator of voltage-gated Kv7.2/7.3 potassium channels, for the treatment of KCNQ2 epileptic encephalopathy (KCNQ2-EE).
  • The granting of this designation from the FDA represents a major milestone for Knopp Biosciences, said Michael Bozik, M.D., Chief Executive Officer.
  • The Rare Pediatric Disease Designation by FDA for KB-3061 advances our mission of promoting research and supporting families of children with KCNQ2 developmental and epileptic encephalopathy, added Jim Johnson, president of the KCNQ2 Cure Alliance .
  • Knopps preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain.

KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) - Pipeline Insight, 2020 - ResearchAndMarkets.com

Retrieved on: 
Friday, July 3, 2020

The "KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) - Pipeline Insight, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) - Pipeline Insight, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • A detailed picture of the KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) pipeline landscape is provided, which includes the disease overview and KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) treatment guidelines.
  • The assessment part of the report embraces in-depth KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) commercial assessment and clinical assessment of the KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) pipeline products from the pre-clinical developmental phase to the marketed phase.
  • It comprises of detailed profiles of KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) therapeutic products with key coverage of developmental activities, including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product-related details
    Detailed KCNQ2 Developmental and Epileptic Encephalopathy (KCNQ2-DEE) research and development progress and trial details, results wherever available, are also included in the pipeline study.

Knopp Biosciences Presents Data Further Characterizing Its Lead Kv7.2 Activator Drug Candidate for the Treatment of KCNQ2 Epileptic Encephalopathy at the American Epilepsy Society’s 2019 Annual Meeting

Retrieved on: 
Monday, December 9, 2019

The disease is caused by dominant-negative mutations in the KCNQ2 gene, which produces a potassium channel, Kv7.2, critical to early brain development.

Key Points: 
  • The disease is caused by dominant-negative mutations in the KCNQ2 gene, which produces a potassium channel, Kv7.2, critical to early brain development.
  • Data presented at AES by Knopp scientists and its academic collaborators support the continued development of KB-3061 as a potential treatment for children with KCNQ2-EE.
  • Nanomolar brain concentrations of KB-3061 conferred significant seizure protection in the maximal electroshock model (MES) of epilepsy with minimal impact on neurobehavior.
  • Follow Knopp Biosciences on Twitter via @KnoppBio and keep up to date with AES Annual Meeting news and updates using the hashtag #AES2019.

Knopp Biosciences Receives Year 4 Renewal of NIH Blueprint Grant Award for Advancing the Kv7 Activator KB-3061 in a Rare Neonatal Epilepsy

Retrieved on: 
Wednesday, September 25, 2019

Knopp Biosciences LLC today announced the renewal of its grant award for Year 4 from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN) to advance its lead Kv7 potassium channel activator, KB-3061, in rare neonatal epilepsy.

Key Points: 
  • Knopp Biosciences LLC today announced the renewal of its grant award for Year 4 from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN) to advance its lead Kv7 potassium channel activator, KB-3061, in rare neonatal epilepsy.
  • The grant has a total potential direct award value of $2.4 million over four years.
  • The disease is caused by dominant-negative mutations in the KCNQ2 gene, which produces a potassium channel, Kv7.2, critical to early brain development.
  • The content of this announcement is solely the responsibility of Knopp and does not necessarily represent the views of the NIH.

Knopp Biosciences Reports Data Demonstrating Restoration of Function by KB-3061 in Cellular Model of Gene Variants Causing KCNQ2 Epileptic Encephalopathy

Retrieved on: 
Tuesday, September 17, 2019

When treated with KB-3061, the function of the mutated channels was fully restored, producing normal Kv7.2 channel current density.

Key Points: 
  • When treated with KB-3061, the function of the mutated channels was fully restored, producing normal Kv7.2 channel current density.
  • The disease is caused by dominant-negative mutations in the KCNQ2 gene, which normally produces a potassium channel, Kv7.2, critical to early brain development.
  • The results suggest that restoring suppressed Kv7.2 channel activity may ameliorate the dysfunction caused by KCNQ2 gene mutations.
  • As previously announced by Knopp, KB-3061 has also demonstrated potent seizure control in an in vivo model of epilepsy.

Knopp Biosciences to Present Data on Its Potassium Channel Activator Program at 2019 Antiepileptic Drug and Device Trials XV Conference

Retrieved on: 
Wednesday, May 15, 2019

KCNQ2-NEE, a rare genetic epilepsy, results from mutations in genes encoding the Kv7.2/Kv7.3 ion channel, leading to frequent seizures and abnormal brain development.

Key Points: 
  • KCNQ2-NEE, a rare genetic epilepsy, results from mutations in genes encoding the Kv7.2/Kv7.3 ion channel, leading to frequent seizures and abnormal brain development.
  • Among ion channel targets, potassium channels play a key role in the pathophysiology of epilepsy and other unmet needs in diseases of the nervous system.
  • Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need.
  • Knopp's pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration.