GABAergic

Encoded Therapeutics Presents Preclinical Data Across its Gene Therapy Portfolio at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Dienstag, Mai 7, 2024
Biotechnology, Neurology, Health, Genetics, Pharmaceutical, DRG, SCN9A, Seizure, Risk, Angelman syndrome, Locomotion, UBE3A, Human, Neurodevelopmental disorder, Abstract, Code, AAV, Epilepsy, Ataxia, CNS, GABAergic, Disease, Incidence, IPSC, Spinal cord, MAPT, Intellectual disability, Central nervous system, DSM-IV codes, Neuron, NHP, Therapy, Gene, MicroRNA, Vaccine

“We are thrilled to share multiple advancements across our portfolio of precision genetic medicines, showcasing substantial progress across various fronts.

Key Points: 
  • “We are thrilled to share multiple advancements across our portfolio of precision genetic medicines, showcasing substantial progress across various fronts.
  • These data underscore our platform’s potential to deliver precision therapies for a range of CNS diseases,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded.
  • Encoded’s vector engineering platform has enabled the development of GABA-selective AAV-mediated gene therapies designed to potentiate GABAergic neurotransmission and modulate the circuit dysfunction underlying refractory epilepsies.
  • These data support further development of our candidate gene therapy for the treatment for refractory epilepsies.

Encoded Therapeutics Provides Pipeline Updates From Its Vector Engineering Platform Ahead of Four Preclinical Presentations at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Dienstag, April 23, 2024
Biotechnology, Genetics, Health, Factorization of polynomials, Lennox–Gastaut syndrome, Brain, DRG, DSM-IV codes, SCN9A, Dorsal root ganglion, Dravet syndrome, ETF, Central nervous system disease, Gene silencing, LGS, Transgene, Neuroscience, Abstract, Code, CNS, GABAergic, Engineering, Res, Interneuron, MAPT, Angelman syndrome, Neuropathic pain, Central nervous system, Therapy, MicroRNA, Vaccine

Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.

Key Points: 
  • Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.
  • “Our ASGCT presentations showcase the depth and versatility of our vector engineering platform to develop highly specific precision therapies for CNS disorders with high unmet need,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded.
  • “Combining our novel regulatory elements and transgenes provides control of expression in target cells, potentially enabling treatment of devastating, intractable diseases.
  • Engineered transgenes include transcription factors (eTFs) that upregulate the expression of endogenous genes as well miRNA sequences derived from Encoded’s miRNA discovery platform.

Encoded Therapeutics Announces US IND Clearance and Australian CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101

Retrieved on: 
Dienstag, Februar 6, 2024
Children, Biotechnology, FDA, Health, General Health, Pharmaceutical, Neurology, Health Technology, Genetics, Consumer, Clinical Trials, CTA, Patient, IND, Central nervous system, GABAergic, Dravet syndrome, CNS, Food, SCN1A, Disease, Therapy, Pharmaceutical industry

Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome.

Key Points: 
  • Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome.
  • Dravet syndrome is primarily caused by loss-of-function variants in the SCN1A gene, accounting for over 85% of cases.
  • ETX101 is an AAV9-mediated candidate gene regulation therapy designed to selectively upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons and potentially address the underlying cause of the disease.
  • Encoded has received clearance for its Investigational New Drug (IND) application from the US Food and Drug Administration and approval under the Clinical Trial Approval (CTA) scheme from the Australia Therapeutic Goods Administration to initiate clinical trials of its gene therapy candidate, ETX101.

Scientists at King's College London and bit.bio Collaborate to Develop Multi-Cell Models of the Human Brain With Optimised Open-Source Protocols for Use by Any Researcher

Retrieved on: 
Freitag, November 24, 2023
Research, Mental Health, Genetics, Stem Cells, Biotechnology, University, Pharmaceutical, Health, Science, Education, Schizophrenia, Brain, Microglia, Psychology, Disease, Neurodevelopmental disorder, DSM-IV codes, Genomics, GABAergic, Multi, Neuron, Dementia, Epilepsy, Drug discovery, Cell, Central nervous system, Royal Historical Society, Spectrum, 3D printing, Fine chemical, Vaccine

The cells have been precision reprogrammed from induced pluripotent stem cells (iPSCs) using bit.bio’s opti-ox™️ technology , meaning they are consistent at scale.

Key Points: 
  • The cells have been precision reprogrammed from induced pluripotent stem cells (iPSCs) using bit.bio’s opti-ox™️ technology , meaning they are consistent at scale.
  • The human brain is made up of numerous different cell and sub-cell types.
  • Subtle defects in one cell type can unbalance this highly-connected system and give rise to neuronal and psychiatric disorders.
  • Multi-cell models are therefore essential to reveal some of the more complex aspects of a disease that cannot be studied in mono-culture systems.

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: 
Donnerstag, Oktober 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.

Unlocking the Potential: Global Dystonia Drugs Market Set to Grow at 5.3% CAGR by 2027 - Analyzing Emerging Markets and Healthcare Advancements - ResearchAndMarkets.com

Retrieved on: 
Mittwoch, September 27, 2023
Health, Pharmaceutical, Growth, Torticollis, Pharmacist, Dystonia, Drug, Research, Pharmacy, GABAergic, Pharmaceutical industry

Among these, GABAergic agents have the largest market share, attributed to their effectiveness in treating dystonia, especially cervical dystonia.

Key Points: 
  • Among these, GABAergic agents have the largest market share, attributed to their effectiveness in treating dystonia, especially cervical dystonia.
  • The pharmaceutical industry's discovery of highly effective GABAergic agent medications for dystonia treatment is further driving the growth of this segment.
  • The injectable segment holds a significant market share due to innovations in parenteral dosing and increased research and development for botulinum injections.
  • The drug stores and retail pharmacies segment dominates the market, benefiting from its extensive distribution network and longstanding presence as a convenient channel for drug distribution.

Beyond Air® Announces Positive Preclinical Data on Role of Nitric Oxide in Autism Spectrum Disorder (ASD)

Retrieved on: 
Montag, August 7, 2023
Faculty, Al-Quds, Hebrew University of Jerusalem, Medical school, Autism spectrum, GABAergic, HUJI, Nitric oxide, DSM-IV codes, ESN, Mutation, Brain, Research, NOS1, Assistant, ISN, UCL, CNTNAP2, NO, Poster, Patient, ASD, European Society for Neurochemistry, School, Spectrum, Mouse, European Society for Primary Immunodeficiencies, Yissum Research Development Company of the Hebrew University, Pharmaceutical industry, Pharmacy, Medicine

GARDEN CITY, N.J., Aug. 07, 2023 (GLOBE NEWSWIRE) -- Beyond Air, Inc. (NASDAQ: XAIR) (“Beyond Air” or the “Company”) a commercial stage medical device and biopharmaceutical company focused on harnessing the power of endogenous and exogenous nitric oxide (NO) to improve the lives of patients suffering from respiratory illnesses, neurological disorders and solid tumors (through its affiliate Beyond Cancer, Ltd. (“Beyond Cancer”)), today announced the presentation of data that support the ongoing preclinical development of selective neuronal nitric oxide synthase (nNOS) inhibibitors for the treatment of Autism Spectrum Disorder (ASD). These data will be presented by the Amal Lab from the Hebrew University of Jerusalem (HUJI) in a poster at the Annual International Society of Neurochemistry (ISN) and the European Society of Neurochemistry (ESN) meetings, which are being held in Portugal from August 8th - 11th.

Key Points: 
  • Research conducted recently at HUJI has shown that mutation in the CNTNAP2 gene increases NO formation in the brain of the ASD mouse model.
  • These data demonstrate that the inhibition of NO production may reverse ASD phenotypes, and provide further evidence that NO is a pathologic factor in autism.
  • These results have led researchers to believe that NO is a novel target for the treatment of ASD and other neurological disorders.
  • The preclinical data developed thus far show that this exciting novel approach to treating ASD has the potential to address a significant unmet medical need.

bit.bio is Industrialising Human Cell Manufacturing, Unleashing the Potential of Synthetic Biology for Reproducible Research and Affordable Regenerative Medicines

Retrieved on: 
Donnerstag, Juni 15, 2023
Other Manufacturing, Research, Genetics, Manufacturing, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, SAB, Charles River Laboratories, DNA, CRISPR, Heart, Neuron, Partnership, Cell biology, FX Palo Alto Laboratory, Drug development, University, IPSC, Stanford University, ISSCR, GABAergic, 3D, ARM, B cell, Wellcome–MRC Cambridge Stem Cell Institute, Systems biology, Brain, Medicine, Cell, Department of Biochemistry, University of Oxford, Regenerative medicine, Breakthrough Prize in Life Sciences, Computational biology, Parity, Stanford University School of Medicine, Professor, Computation, Animal, Patient, Pathology, Machine learning, Acorn Computers, Drug discovery, Biology, International Society for Stem Cell Research, Meat, SVP, Gene, Synthetic biology, International Society, Vaccine, Management, Fine chemical, Microscope, Pharmaceutical industry, Cryptocurrency, Illumina

bit.bio, a synthetic biology company focused on human cells, has achieved a milestone in the manufacture of human cells.

Key Points: 
  • bit.bio, a synthetic biology company focused on human cells, has achieved a milestone in the manufacture of human cells.
  • The data that will be presented at the International Society for Stem Cell Research (ISSCR) conference show an unparalleled level of consistency with regards to multiple human cell products for research use.
  • I am proud of this fantastic achievement by the team.”
    bit.bio's human cell manufacturing technology is set to reshape the future of scientific research and biomedicine.
  • Data for multiple cell types - glutamatergic neurons, sensory neurons and GABAergic neurons - will be presented at ISSCR, showing the approach may be generalisable to any human cell type.

RespireRx Pharmaceuticals Inc. Discloses Novel Structural Analogs of its GABAA Receptor Potentiator, KRM-II-81 with Antiseizure Efficacy and Antitremor Effects without Sedation.

Retrieved on: 
Montag, April 3, 2023
Epilepsy, GABA, Anticonvulsant, Tremor, GABAergic, OTC Markets Group, DRS, KPP, University of Wisconsin–Milwaukee, KRM, Essential tremor, Pain, Patient, University, Blood, Neuropathic pain, Indiana University–Purdue University Indianapolis, Irena Kosmowska, Drug discovery, CFO, ACS Omega, IND, RespireRx, Pharmaceutical industry

Like KRM-II-81, these analogs enhance GABAergic neurotransmission and function as orally-bioavailable anti-seizure agents in the absence of the sedation typically associated with GABA augmentation.

Key Points: 
  • Like KRM-II-81, these analogs enhance GABAergic neurotransmission and function as orally-bioavailable anti-seizure agents in the absence of the sedation typically associated with GABA augmentation.
  • In addition KRM-II-81 is active in relieving acute, chronic, and neuropathic pain in a number of models without developing tolerance or producing sedation.
  • 385:50-61 document the preclinical effects and tolerability of structural analogs of KRM-II-81.
  • In established rat models predicting efficacy in essential tremor and in tremor associated with Parkinson’s Disease, MP-III-024 was an effective anti-tremor compound.

PharmaTher Holdings Submits for FDA Orphan Drug Designation for Ketamine to Treat Rett Syndrome

Retrieved on: 
Mittwoch, Januar 18, 2023
Disease, CWRU, Case Western Reserve University, Mental health, Depression, Laboratory, School, Rett syndrome, FDA, Ketamine, Marketing, Medical school, X chromosome, Orphan Drug Act of 1983, Disorder, BDNF, Orphan drug, GABAergic, Female, MECP2, Pharmaceutical industry, Rett, Medicine

According to the Rett Syndrome Foundation, Rett syndrome is a rare genetic neurological disorder that occurs almost exclusively in girls.

Key Points: 
  • According to the Rett Syndrome Foundation, Rett syndrome is a rare genetic neurological disorder that occurs almost exclusively in girls.
  • Prominent features of Rett syndrome include near constant repetitive hand movements and loss of purposeful hand use.
  • The Orphan Drug Act grants special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor.
  • The FDA grants orphan status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics.