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Neuroene Therapeutics Announces Orphan Drug Designation Granted by FDA for Dravet Syndrome Treatment - NT102

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Friday, October 29, 2021
ADD, Dravet syndrome, Phenotype, Mutation, Research, Orphan Drug Act of 1983, University, Dravet Syndrome Foundation, Disease, Myoclonus, DRS, Drug development, History, Patient, FDA, Lists of diseases, SCN1A, Seizure, Parkinson's disease, Fever, Brain, National, Degenerative disease, Multimedia, Therapy, Temperature, Epilepsy, Encephalopathy, Nav1.1, Orphan, Pharmaceutical industry

CHARLESTON, S.C., Oct. 29, 2021 /PRNewswire/ -- Neuroene Therapeutics announced that the FDA has granted orphan drug designation for the company's proprietary compound: NT102.

Key Points: 
  • CHARLESTON, S.C., Oct. 29, 2021 /PRNewswire/ -- Neuroene Therapeutics announced that the FDA has granted orphan drug designation for the company's proprietary compound: NT102.
  • NT102 has broad seizure protection in several preclinical animal models and in particular has excellent protection against seizures in Dravet syndrome a rare childhood syndrome marked by severe encephalopathy and epilepsy.
  • "This is an exciting development for Neuroene Therapeutics, and patients and families of those suffering Dravet syndrome," said Drs.
  • Neuroene's pipeline of candidates include NT102, which was granted Orphan Drug Designation from the FDA for the treatment of Dravet syndrome, a rare pharmacoresistant childhood epilepsy.

Stoke Therapeutics Announces Publication of Preclinical Data on STK-001 in the Journal Science Translational Medicine that Demonstrate Significant Improvements in Survival and Reductions in Seizure Frequency in a Dravet Syndrome Mouse Model

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Wednesday, August 26, 2020
FDA, Health, Other Health, Clinical trials, Pharmaceutical, Biotechnology, Epilepsy, Branches of biology, Medicine, Health, Nav1.1, Dravet syndrome, Oligonucleotide, Sense, Sudden unexpected death in epilepsy, STK-001, Dravet Syndrome, TANGO, Stoke Therapeutics

Data published today in the journal Science Translational Medicine also showed that STK-001 achieved target engagement, pharmacologic activity and efficacy by selectively increasing Scn1a gene and Nav1.1 protein expression.

Key Points: 
  • Data published today in the journal Science Translational Medicine also showed that STK-001 achieved target engagement, pharmacologic activity and efficacy by selectively increasing Scn1a gene and Nav1.1 protein expression.
  • STK-001 is an antisense oligonucleotide (ASO) that was created using Stokes proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach.
  • Antisense Oligonucleotides Increase Scn1a Expression and Reduce Seizures and SUDEP Incidence in a Mouse Model of Dravet Syndrome, is now available online at: https://stm.sciencemag.org/lookup/doi/10.1126/scitranslmed.aaz6100 .
  • Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome.

Stoke Therapeutics Presents Preclinical Data on the Biodistribution, Target Engagement and Safety of STK-001 in Non-Human Primates That Support the Planned Clinical Development of STK-001 for the Treatment of Dravet Syndrome

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Sunday, December 8, 2019
Biotechnology, Pharmaceutical, Genetics, Health, Neurological disorders, Organ systems, Epilepsy, Dravet syndrome, SCN1A, Sodium channels, Nav1.1, Ataxia, STK-001, Dravet Syndrome, Stoke Therapeutics

The effects of Dravet syndrome go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances.

Key Points: 
  • The effects of Dravet syndrome go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances.
  • These results will be included in our planned IND submission and provide additional confidence in our clinical plans for STK-001.
  • Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous mutations in the SCN1A gene, resulting in 50% of normal Nav1.1 protein expression.
  • Stoke selected two dose levels of STK-001 for this non-GLP study in order to evaluate safety, brain biodistribution, target engagement and Nav1.1 protein expression.

Stoke Therapeutics Presents Preclinical Data From Studies of STK-001 That Showed Improvements in Survival and Reductions in Seizure Frequency in a Mouse Model of Dravet Syndrome

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Saturday, December 7, 2019
Biotechnology, Health, Stem cells, Pharmaceutical, Clinical trials, Branches of biology, Medicine, Clinical medicine, Dravet syndrome, Sodium channels, SCN1A, Electrophysiology, Nav1.1, Haploinsufficiency, Epilepsy, Epileptic seizure, Electroencephalography, STK-001, Dravet Syndrome, Stoke Therapeutics

EEG is a highly sensitive measure of seizure activity, which enables the detection of seizures that may not be otherwise visible.

Key Points: 
  • EEG is a highly sensitive measure of seizure activity, which enables the detection of seizures that may not be otherwise visible.
  • What is particularly remarkable is that these data were generated from a spontaneous seizure model, which we believe accurately reflects the clinical situation in people with Dravet syndrome.
  • Dravet syndrome is a severe and progressive genetic epilepsy that begins within the first year of life.
  • In the studies presented in todays poster, the efficacy of STK-001 was evaluated in a Scn1a-linked mouse model of Dravet syndrome that results in Nav1.1 haploinsufficiency.

Stoke Therapeutics to Present New Preclinical Data on STK-001 at the American Epilepsy Society Annual Meeting

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Monday, November 25, 2019
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Organ systems, Medicine, Health, SCN1A, Dravet syndrome, Sodium channels, Nav1.1, Sudden unexpected death in epilepsy, Epilepsy, Oligonucleotide, Lori L. Isom, Epilepsy-intellectual disability in females, STK-001, Dravet Syndrome, Stoke Therapeutics

Data will be presented from preclinical studies demonstrating the effects of STK-001, a proprietary antisense oligonucleotide (ASO), in the Scn1a-linked Dravet syndrome mouse model and in non-human primates.

Key Points: 
  • Data will be presented from preclinical studies demonstrating the effects of STK-001, a proprietary antisense oligonucleotide (ASO), in the Scn1a-linked Dravet syndrome mouse model and in non-human primates.
  • Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous loss of function mutations in the SCN1A gene, resulting in 50% Nav1.1 protein expression.
  • Stoke has generated preclinical data demonstrating proof-of-mechanism for STK-001.
  • Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP.

Stoke Therapeutics Enrolls First Patient in an Observational Study of Children and Adolescents Living with Dravet Syndrome

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Tuesday, August 20, 2019
Children, Genetics, Clinical trials, Biotechnology, Pharmaceutical, Consumer, Health, Organ systems, Epilepsy, Health, Branches of biology, Dravet syndrome, SCN1A, Nav1.1, Mutation, Epilepsy-intellectual disability in females, the BUTTERFLY Study, STK-001, Stoke Therapeutics

Our goal is to develop the first medicine to treat the underlying cause of Dravet syndrome, said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics.

Key Points: 
  • Our goal is to develop the first medicine to treat the underlying cause of Dravet syndrome, said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics.
  • Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous loss of function mutations in the SCN1A gene.
  • The BUTTERFLY study is an observational study of children and adolescents ages 2 to 18 who have been diagnosed with Dravet syndrome as a result of an SCN1A gene mutation.
  • Stoke Therapeutics, the Stoke logo and all product names are trademarks of Stoke Therapeutics, Inc.
    View source version on businesswire.com: https://www.businesswire.com/news/home/20190820005258/en/

Stoke Therapeutics Granted FDA Orphan Drug Designation for STK-001, an Investigational New Treatment for Dravet Syndrome

Retrieved on: 
Tuesday, August 6, 2019
Biotechnology, FDA, Health, Pharmaceutical, Epilepsy, Organ systems, Dravet syndrome, SCN1A, Nav1.1, Health, Nervous system, Dravet Syndrome, STK-001, Stoke Therapeutics

Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product candidate, STK-001, an investigational new treatment for Dravet syndrome.

Key Points: 
  • Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product candidate, STK-001, an investigational new treatment for Dravet syndrome.
  • Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous loss of function mutations in the SCN1A gene.
  • Our goal with STK-001 is to slow or even stop disease progression by treating the underlying cause of Dravet syndrome.
  • Stoke Therapeutics, the Stoke logo and all product names are trademarks of Stoke Therapeutics, Inc.
    View source version on businesswire.com: https://www.businesswire.com/news/home/20190806005791/en/

Global Dravet Syndrome Market: Industry Analysis & Outlook (2019-2023) - Attractive Orphan Drug Policies & Rising Strategic Alliances are Key Drivers - ResearchAndMarkets.com

Retrieved on: 
Tuesday, February 5, 2019
Health, Pharmaceutical, Neurological disorders, Medicine, Epilepsy, Sodium channels, Dravet syndrome, SMEI, Nav1.1, Epileptic seizure, Myoclonus, Dravet Syndrome Foundation, Charlotte Dravet

The "Global Dravet Syndrome Market: Industry Analysis & Outlook (2019-2023)" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Dravet Syndrome Market: Industry Analysis & Outlook (2019-2023)" report has been added to ResearchAndMarkets.com's offering.
  • Currently, the U.S. and Europe are holding the potential growth for Dravet Syndrome market due to increasing R&D into new drug development for Dravet syndrome and approval for these drugs in these regions.
  • Dravet syndrome, formerly known as severe myoclonic epilepsy of infancy (SMEI), is a severe kind of epilepsy which causes seizures.
  • Patients with Dravet syndrome are not able to produce sufficient levels of functional Nav1.1 sodium channel, preventing inhibitory neurons from firing properly.