Temporal lobe epilepsy

uniQure Announces 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Wednesday, February 28, 2024
Acquisition, Fabry disease, TMS, Natural history, CSL Behring, ALS, Research, FDA, Partnership, Cerebrospinal fluid, Food, CSL, Etranacogene dezaparvovec, Sale, TFC, Conference, SOD1, Unified, Information technology, AFL, Immunosuppression, Patient, Disease, Phase, Hand, IND, CGMP, State, Temporal lobe epilepsy, Pharmaceutical industry

LEXINGTON, Mass. and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year 2023 and highlighted recent progress across its business.

Key Points: 
  • and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year 2023 and highlighted recent progress across its business.
  • “We are pleased with the progress made across the company in 2023 and are now laser-focused on execution across multiple clinical programs,” stated Matt Kapusta, chief executive officer of uniQure .
  • By the end of 2024, the Company expects to have greater clarity regarding a potential approval pathway for AMT-130.
  • AMT-191 for the treatment of Fabry disease – In November 2023, the Company announced the clearance of an IND for the Phase I/IIa clinical study of AMT-191.

Ovid Therapeutics to Present Five Abstracts Supporting its Epilepsy Programs at the 77th American Epilepsy Society Annual Meeting (2023)

Retrieved on: 
Friday, December 1, 2023
Neuroprotection, Benzodiazepine, Temporal lobe epilepsy, Kainic acid, Brain, KCC2, AES, American Epilepsy Society, Seizure, PD, Annual general meeting, Epilepsy, Dentate gyrus, Patient, DSM-IV codes, Pharmaceutical industry

and Chief Medical Officer of Ovid Therapeutics.

Key Points: 
  • and Chief Medical Officer of Ovid Therapeutics.
  • “GABA-AT is a validated target in seizure reduction that has been limited in clinical use to date.
  • Title: Blocking of GABA-AT Activity by OV329 Selectively Alters Tonic and Phasic Inhibition in Dentate Gyrus Granule Cells.
  • Title: Low, Repeat Dosing of OV329 Enhances GABA-AT Inhibition in Rodent Brain: Relationship between Pharmacokinetic (PK) and Pharmacodynamic (PD) Effects.

IAMA Therapeutics to Present New Preclinical Data at Upcoming American Epilepsy Society 2023 Annual Meeting

Retrieved on: 
Friday, December 1, 2023
Health, Neurology, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, CNS, Dravet syndrome, American Epilepsy Society, Annual general meeting, Therapy, Treatment, Refractory, Patient, NKCC1, Management of drug-resistant epilepsy, Seizure, Central nervous system disease, IAMA, Epilepsy, Temporal lobe epilepsy, DSM-IV codes, Spectrum, AES, Pharmaceutical industry

IAMA Therapeutics, a pharmaceutical company focused on the discovery, development, and commercialization of novel medicines for children affected by brain disorders, today announced that it will present a poster titled “Selective NKCC1 Inhibitors for the Treatment of Refractory Epilepsy” at the American Epilepsy Society (AES) 2023 Annual Meeting, taking place December 1-5 in Orlando, FL.

Key Points: 
  • IAMA Therapeutics, a pharmaceutical company focused on the discovery, development, and commercialization of novel medicines for children affected by brain disorders, today announced that it will present a poster titled “Selective NKCC1 Inhibitors for the Treatment of Refractory Epilepsy” at the American Epilepsy Society (AES) 2023 Annual Meeting, taking place December 1-5 in Orlando, FL.
  • “The altered expression of chloride importer NKCC1, which can lead to an imbalance in brain excitation and inhibition, is implicated in several neurodevelopmental and neurological conditions, including drug-resistant epilepsies like Mesial Temporal Lobe Epilepsy (MTLE) and Dravet syndrome,” said Annalisa Savardi, Team Leader Neuropharmacology at IAMA Therapeutics.
  • “In the upcoming presentation, preclinical data show that IAMA’s lead program IAMA-6, a small molecule targeting NKCC1 for the treatment of autism and epilepsy, effectively reduces epileptic activity in an animal model of MTLE and, in preliminary data, reduces the number of seizures in a model of Dravet syndrome.
  • “We look forward to sharing more with the healthcare providers, scientists, advocates, and other industry professionals dedicated to better outcomes for people with epilepsy at AES 2023.”

uniQure Announces Third Quarter 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Tuesday, November 7, 2023
Temporal lobe epilepsy, FDA, CSL, Research, Sale, IND, SOD1, Food, Chief scientific officer, ALS, Fabry disease, Workforce, Acquisition, Phase, Neuroscience, Hand, Nobel Prize, Safety, ARD International Music Competition, Investment, France, Patient, Immunosuppression, Food and Drug Administration Amendments Act of 2007, Pharmaceutical industry, Management, Cryptocurrency, EU

and AMSTERDAM, Nov. 07, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2023 and highlighted recent progress across its business.

Key Points: 
  • and AMSTERDAM, Nov. 07, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2023 and highlighted recent progress across its business.
  • Screening and patient enrollment is expected to begin in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter of 2024.
  • The Company expects to initiate patient screening in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter of 2024.
  • AMT-191 for the treatment of Fabry disease – The Company expects to submit an IND in the fourth quarter of 2023 and to begin patient dosing in 2024.

uniQure Announces Major Presence at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting

Retrieved on: 
Tuesday, October 24, 2023
Alzheimer's disease, AAV, Control, SR, Chief scientific officer, Seed, Research, Poster, Cell, Science, Gene, APOE, Annual general meeting, ESGCT, Chemistry, European Society of Gene and Cell Therapy, GRIK2, Patient, Temporal lobe epilepsy, Pharmaceutical industry, Management, Tutoring

LEXINGTON, Mass.

Key Points: 
  • LEXINGTON, Mass.
  • and AMSTERDAM, Oct. 24, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting being held October 24-27 in Brussels, Belgium.
  • “Our multiple presentations at ESGCT demonstrate uniQure’s continued leadership in advancing important research within the field of gene therapy, as well as highlight our industry-leading expertise in AAV gene therapy manufacturing,” stated Richard Porter, Ph.D., Chief Business and Scientific Officer at uniQure .
  • “We look forward to sharing these presentations that feature our technology and platform experience across numerous programs.”
    Specific details on uniQure’s presentations taking place at ESGCT include:
    Presenter: Richard Porter, Chief Business and Scientific Officer, uniQure
    Session Title and Location: CNS & sensory diseases II, Session 10a, Shed 2A
    Session Title and Location: CNS & sensory diseases I, Session 2c, Maison de la Poste
    Title: Pivotal safety studies for AMT-260, a novel AAV9-dual microRNA-based vector targeting GRIK2 for the treatment of temporal lobe epilepsy (Poster #433)
    Presenter: Nick Pearson, Sr. Director Toxicology & Translational Safety, Non-Clinical, uniQure
    Poster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2
    Poster Session Date and Time: Wednesday, October 25, 18:15 – 19:30, Thursday October 26, 19:30 – 20:30 GMT+2
    Poster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2
    Title: Silence and replace as potential treatment for Alzheimer Disease: in concert lowering toxic APOE and augmenting protective APOE (Poster #049)
    Poster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2
    Poster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2
    Poster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2
    Presenter: Erich Ehlert, Director – Process Development – Chemistry, Manufacturing and Controls, uniQure
    Poster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2
    Poster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2
    Poster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2
    Presenter: Zandra Félix Garza, Project Manager- BPU – Cells, Seeds and Research Supply, uniQure
    Poster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2

Neurona Therapeutics Announces Publication in Cell Stem Cell Reporting the Development of Investigational Novel Regenerative Cell Therapy Strategy for Drug-resistant Focal Epilepsy

Retrieved on: 
Thursday, October 5, 2023
Interneuron, Neuropathology, Temporal lobe epilepsy, RNA, Clinical, Cell, GABAergic, B cell, DSM-IV codes, Survival, MGE, Generalized tonic–clonic seizure, Epilepsy, Transplant, Translation, GMP, Cell Stem Cell, Therapy, GABA, Seizure, Clinical trial, History, SAN, Tissue, Behavior informatics, Patient, Pharmaceutical industry, Vaccine, Sarcocystis neurona

SAN FRANCISCO, Oct. 05, 2023 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, today announced the publication of the development and characterization of a highly purified population of human stem cell-derived, pallial-specific MGE inhibitory interneurons for the potential treatment of focal epilepsy. In a preclinical study, administration of a single dose of the cells in a chronic model of drug-resistant MTLE resulted in durable and consistent suppression of focal seizures, as well as improvements in neuropathology and an increase in survival of the model. The study also demonstrated a potentially broad, safe and effective dosing range in the preclinical model. The data support the development of Neurona Therapeutics’ regenerative cell therapy candidate, NRTX-1001, which is being evaluated in an ongoing Phase I/II clinical trial of a one-time dose in subjects with drug-resistant MTLE (NCT05135091). The preclinical data were published today in Cell Stem Cell as a Clinical and Translational Report titled “Human pallial MGE-type GABAergic interneuron cell therapy for chronic focal epilepsy” by first authors Bershteyn, Bröer, Parekh, Maury, and colleagues and is available online.

Key Points: 
  • The data support the development of Neurona Therapeutics’ regenerative cell therapy candidate, NRTX-1001, which is being evaluated in an ongoing Phase I/II clinical trial of a one-time dose in subjects with drug-resistant MTLE ( NCT05135091 ).
  • The preclinical data were published today in Cell Stem Cell as a Clinical and Translational Report titled “Human pallial MGE-type GABAergic interneuron cell therapy for chronic focal epilepsy” by first authors Bershteyn, Bröer, Parekh, Maury, and colleagues and is available online .
  • “We are thrilled to announce this landmark publication, which builds the foundation for NRTX-1001 and exemplifies the phenomenal science being conducted by Neurona employees.
  • The data support the development of Neurona’s cell therapy candidate NRTX-1001, which is being evaluated in an ongoing clinical trial ( NCT05135091 ) in adults with drug-resistant MTLE.

uniQure Announces Strategic Reorganization to Reduce Operating Expenses and Support Advancement of Multiple Clinical-Stage Programs

Retrieved on: 
Thursday, October 5, 2023
Neuroscience, ALS, Acquisition, Temporal lobe epilepsy, CSL Behring, CSL, Chief scientific officer, Risk, Fabry disease, IND, Patient, FDA, GMP, Alzheimer's disease, Workforce, Phase, AAV, Rich Porter, MA, CNS, Research, Genetic distance, Construction, Management, Pharmaceutical industry, Fabry

LEXINGTON, Mass. and AMSTERDAM, Oct. 05, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced a strategic reorganization that will significantly reduce operating expenses while supporting focused execution to rapidly advance multiple clinical-stage programs to proof-of-concept.

Key Points: 
  • and AMSTERDAM, Oct. 05, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced a strategic reorganization that will significantly reduce operating expenses while supporting focused execution to rapidly advance multiple clinical-stage programs to proof-of-concept.
  • “At uniQure, our highest priority is to deliver innovative, life-changing therapies to patients with significant unmet needs.
  • To accomplish our mission and generate near-term value for our stakeholders, we will implement a strategic restructuring of our business,” stated Matt Kapusta, chief executive officer of uniQure .
  • “We are taking important actions today to cut operating expenses while ensuring that we have the necessary resources to advance our prioritized clinical-stage programs as rapidly as possible to proof-of-concept.

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-260 Gene Therapy for Refractory Mesial Temporal Lobe Epilepsy

Retrieved on: 
Tuesday, September 5, 2023
Seizure, Clinical trial, GRIK2, MicroRNA, Investigational New Drug, Patient, POC, FDA, Temporal lobe epilepsy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Safety, Pharmaceutical industry

and AMSTERDAM, Sept. 05, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-260, the Company’s gene therapy candidate for refractory mesial temporal lobe epilepsy ( MTLE ).

Key Points: 
  • and AMSTERDAM, Sept. 05, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-260, the Company’s gene therapy candidate for refractory mesial temporal lobe epilepsy ( MTLE ).
  • AMT-260 comprises an AAV9 vector that locally delivers two engineered miRNAs designed to degrade the GRIK2 gene and suppress the aberrant expression of glutamate receptor subtype GLUK2 that is believed to trigger seizures in patients with refractory MTLE.
  • The second part is expected to be a randomized, controlled trial to generate proof of concept (POC) data.
  • The clinical trial is expected to begin patient screening in the fourth quarter of 2023.

From Trump to Winnie the Pooh: how we use diagnosis as a narrative tool to make sense of dysfunction and deviance

Retrieved on: 
Monday, April 17, 2023
Infection, Psychosis, Depression, AC 25.1309-1, Friends, Differential diagnoses of anorexia nervosa, Tigger, Politics, Whodunit, Policy, Pathology, ADHD, Diagnosis, COVID, Omicron, Psychopathy, Temporal lobe epilepsy, Alpha, Delta, Spectrum, Chronic fatigue syndrome, Tic disorder, Narcissism, Schizophrenia, Facial nerve paralysis, Fibromyalgia, Winnie-the-Pooh, Bacteria, House, Character, Bipolar disorder, Personality disorder, Disease, Medical imaging, Nursing, Dietary supplement, Vaccine, Entertainment, Medical device

Being diagnosed with COVID makes sense of symptoms, determines what we should do about them, and shapes our collective responsibility to the community.

Key Points: 
  • Being diagnosed with COVID makes sense of symptoms, determines what we should do about them, and shapes our collective responsibility to the community.
  • You have symptoms, the doctor examines or tests you, you get a name for what ails you.
  • Even a diagnosis as seemingly clear-cut as COVID is more than just a label stuck to a virus.
  • Read more:
    COVID testing led to new techniques of disease diagnosis: progress mustn’t stop now

Diagnosis as storytelling

    • Diagnosis is so important to understanding our lives and those around us that it’s often applied outside of the health setting.
    • TV shows such as House use diagnostic mysteries to underpin plots – less Whodunit and more Whatisit.
    • A diagnosis is a story, in and of itself.
    • You have an infection of your lungs, probably caused by bacteria or a virus and possibly triggered by that cold you had last week.

Stories and deviance

    • Diagnostic stories are explanations of deviance.
    • By “deviance” we mean the sociological sense of the term: an inability to meet social expectations of behaviour, belief or experience.
    • To explain deviance, we often defer to diagnosis.
    • More than 150 scientific authors have thrown themselves at finding a diagnosis to explain his deviance.

Medicalising experiences

    • These stories say more about us, the diagnosers, and our contemporary views, than the lives of those they seek to describe.
    • By using diagnosis to explain people, we medicalise our experience of the world and shut down other avenues of explanation.
    • Just as explaining an imaginary character via diagnosis means we’ve lost faith in stories.

Ovid Therapeutics to Present Data on Its Epilepsy Programs at American Epilepsy Society Annual Meeting (2022)

Retrieved on: 
Friday, December 2, 2022
Takeda Pharmaceutical Company, Lennox–Gastaut syndrome, Epilepsy, COVID-19, Safety, Vigabatrin, Seizure, Brain, Dravet syndrome, KCC2, Therapy, Benzodiazepine, CKM, MES, American Epilepsy Society, Security (finance), Annual general meeting, AES, Risk, Temporal lobe epilepsy, MB, Pharmaceutical industry, Takeda

If successful, these programs will provide hope for people with treatment-resistant epilepsies, said Jeremy Levin, D. Phil, MB BChir and Chairman and Chief Executive Officer of Ovid Therapeutics.

Key Points: 
  • If successful, these programs will provide hope for people with treatment-resistant epilepsies, said Jeremy Levin, D. Phil, MB BChir and Chairman and Chief Executive Officer of Ovid Therapeutics.
  • Our pipeline of potential medicines is designed with unique mechanisms of action to address the diverse, underlying pathophysiology of seizures.
  • Our data suggest OV329 to be a potent, next-generation GABA-AT inhibitor with the potential for seizure reduction with chronic, low dosing.
  • Data presented on OV350 support the therapeutic opportunity associated with activating KCC2, a novel target in epilepsy.