Tauopathy

Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2024 Financial Results

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Giovedì, Maggio 9, 2024
Biotechnology, Neurology, Health, Genetics, Pharmaceutical, STAC, FDA, CTA, Haemophilia, Enzyme replacement therapy, Kite, Investment, Nav1.7, Fabry disease, Poster, Disease, ERT, Sigma-Aldrich, STAAR, Health care, MAA, Partnership, Sangamo Therapeutics, MINT, AAV, Ligand Pharmaceuticals, IND, Tauopathy, Novartis, DNA, Patient, Pivotal, DSM-IV codes, EMA, Prion, BBB, Genome, BLA, MHRA, Manuscript, BioRxiv, Capsid, Modularity, Biogen, Therapy, Food and Drug Administration, European Medicines Agency, Gene, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.
  • “We are pleased with the progress being made in business development discussions across our portfolio, including our Fabry disease program.
  • An IND submission is expected in the fourth quarter of 2024.
  • Revenues for the first quarter ended March 31, 2024 were $0.5 million, compared to $158.0 million for the same period in 2023.

Amylyx Pharmaceuticals Reports First Quarter 2024 Financial Results

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Giovedì, Maggio 9, 2024
Research, FDA, Neurology, Clinical Trials, Biotechnology, Health, Pharmaceutical, Other Science, Science, Investment, Webcast, IND, WFS1, TDP43, AMX0035, Diabetes, Samuel, CSF, Biomarker, Astellas Pharma, Disclosure, Brain, Food, Amylyx Pharmaceuticals, Endoplasmic reticulum, Health Canada, Tauopathy, ALS, Eli Lilly and Company, FCCP, Medication, Merck, Cerebrospinal fluid, Safety, PB, Neurological disorder, Genetic linkage, Week, Wolfram syndrome, C-peptide, DSM-IV codes, Drug development, Clinical trial, Walking, Division, MD, Patient, Disease, Regulation Fair Disclosure, Principal, Regulation, PGIC, Metabolism, Interim, FACP, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the first quarter ended March 31, 2024.

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the first quarter ended March 31, 2024.
  • This decision was informed by topline PHOENIX trial results, engagement with regulatory authorities, and discussions with the ALS community.
  • Amylyx anticipates reporting topline data from all 12 participants at Week 24 in the fall of 2024.
  • Amylyx’ management team will host a conference call and webcast today, May 9, 2024, at 8:00 a.m.

Voyager Therapeutics to Present Broad Set of Translational Data Supporting IV-Delivered, CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting

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Lunedì, Aprile 22, 2024
TRACER, Histology, Mouse, Phenotype, Data science, Cell, AAV, CNS, Machine learning, Senior, Tauopathy, Gene, Process, Society, BBB, ALS, Central nervous system, Public, HEK293, Therapy, Centrifuge, SOD1, Vaccine

“Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.

Key Points: 
  • “Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.
  • Three of those programs now have development candidates selected, and we see the potential for them to enter clinical trials next year,” said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics.
  • ET
    Intravenous administration of BBB-penetrant, MAPT-Silencing, AAV gene therapy provides broad and robust CNS Tau lowering in tauopathy mouse models (#1602).
  • ET
    Intravenous delivery of AAV gene therapy for the treatment of SOD1-ALS provides broad SOD1 lowering in NHP (#1647).

Sangamo Therapeutics to Present Neurology-Focused Pre-Clinical Data From Its Epigenetic Regulation, Capsid Delivery and Genome Engineering Platforms at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

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Lunedì, Aprile 22, 2024
Research, Infectious Diseases, Neurology, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, Science, STAC, Neuron, Central nervous system disease, Dravet syndrome, Central nervous system, MD, Sangamo Therapeutics, Cell, AAV, Tauopathy, CNS, Engineering, DNA, Charcot–Marie–Tooth disease, Patient, Gene, Society, DSM-IV codes, Prion, Angelman syndrome, ALS, Genome, Capsid, Therapy, Powder metallurgy

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.
  • Presentations will focus on the progression of Sangamo’s neurology-focused pre-clinical pipeline, including data supporting innovations in zinc finger epigenetic regulation, advances in AAV capsid engineering, and discovery of next-generation integrase technology.
  • These data will showcase a novel AAV capsid, STAC-BBB, that exhibited robust and widespread central nervous system transduction in cynomolgus macaques after intravenous delivery, as well as another novel AAV capsid, STAC-150, engineered to accelerate the discovery of potent and highly specific epigenetic regulators.
  • Sangamo will also present additional data from its AAV capsid engineering platform SIFTER and its efforts for integrase evolution and utilization.

Amylyx Pharmaceuticals Announces Formal Intention to Remove RELYVRIO®/ALBRIOZA™ from the Market; Provides Updates on Access to Therapy, Pipeline, Corporate Restructuring, and Strategy

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Giovedì, Aprile 4, 2024
Health, FDA, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, Cell death, Medication, Survival, Wolfram syndrome, ALS, Partnership, Metabolism, Patient, AMX0035, Research, American Academy, OLE, Tauopathy, Health Canada, Biology, AAN, Clinical trial, Endoplasmic reticulum, Palsy, Workforce, Amylyx Pharmaceuticals, Division, Publication, Food, Vance DeGeneres, Pharmaceutical industry

Amylyx will continue to evaluate and share learnings from PHOENIX to help inform future ALS research.

Key Points: 
  • Amylyx will continue to evaluate and share learnings from PHOENIX to help inform future ALS research.
  • At this time, Amylyx intends to continue to collect available data on survival at the encouragement of ALS specialists.
  • “Our pipeline is supported by compelling clinical and preclinical science demonstrating the potential of AMX0035 and AMX0114 in neurodegenerative diseases.
  • “We are so thankful and grateful to our Amylyx team for their contributions and steadfast dedication,” said Cohen and Klee.

Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Mercoledì, Marzo 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.

Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs

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Mercoledì, Marzo 13, 2024
Research, Neurology, Genetics, Clinical Trials, Health, Pharmaceutical, General Health, Other Science, Science, STAC, AAV, Sangamo Therapeutics, Gene, Brain, Capsid, Gene expression, ALS, BBB, Central nervous system, Novartis, CNS, CTA, DRG, Traffic barrier, Prion, NHP, Tropism, IND, Nav1.7, Neuron, Biogen, Tauopathy, Excipient, Therapy, DSM-IV codes, Spinal cord, Liver, Animal, Vaccine

Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.

Key Points: 
  • Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.
  • In NHP studies when administered intravenously at clinically relevant doses, STAC-BBB demonstrated its potential to be a leading neurotropic capsid.
  • Exhibited 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the study.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.

Biosplice Therapeutics Announces Department of Defense (DoD) Award to Fund Collaboration with The Roskamp Institute to Advance its Neurology Program

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Venerdì, Marzo 8, 2024
CFO, SAN, ALS, Roskamp Institute, TBI, DYRK, Therapy, Patient, DSM-IV codes, CLK, CBO, Research, Tauopathy, Pharmaceutical industry

As part of the collaboration, the DoD TBIPHRP Award will fund Roskamp to undertake preclinical development in their well characterized TBI models using Biosplice’s DYRK inhibitors.

Key Points: 
  • As part of the collaboration, the DoD TBIPHRP Award will fund Roskamp to undertake preclinical development in their well characterized TBI models using Biosplice’s DYRK inhibitors.
  • The data from this collaboration are anticipated to inform the development of Biosplice’s DYRK inhibitor class in the treatment of degenerative neurological conditions, including various tauopathies.
  • This collaboration with Roskamp will help accelerate the development of our novel neurology therapies, ranging from TBI to ALS to Alzheimer’s disease.”
    This award builds upon more than four years of scientific collaboration between Roskamp and Biosplice.
  • Previous research conducted by Roskamp has demonstrated efficacy in TBI animal models with Biosplice’s first-generation DYRK inhibitors.

CervoMed Announces Presentation of Biomarker Data from the AscenD-LB Phase 2a Trial and Preclinical Data Supporting Potential of Neflamapimod in Tau-Mediated Disease at AD/PD ™ 2024

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Martedì, Marzo 5, 2024
Axonal transport, Tauopathy, Neuron, CRVO, Cell biology, DLB, Plasma, Medical laboratory, MB, Phosphorylation, Overalls, FTD, GS, Vrije Universiteit Amsterdam, Publication, MAPK, Glial fibrillary acidic protein, GFAP, Axon, Brain, CNR, IW, EM, SDL, UCL, RAF kinase, Alpha-synuclein, Conference, Biomarker, AG, Lewy body, GBA, MD, Neurochemistry, PDF, JC, CP, Patient, Dementia, Basal forebrain, JA, Medical imaging

BOSTON, March 05, 2024 (GLOBE NEWSWIRE) -- CervoMed Inc. (NASDAQ: CRVO), a clinical stage company focused on developing treatments for degenerative diseases of the brain, today announced the presentation of biomarker data from the AscenD-LB Phase 2a trial of neflamapimod in patients with dementia with Lewy bodies (DLB), demonstrating that neflamapimod reduces plasma levels of glial fibrillary acidic protein (GFAP) compared placebo, and that the effects of neflamapimod on GFAP were inversely correlated to change in CDR-SB (reduction in GFAP associated with improvement in CDR-SB, and increase in GFAP associated with worsening in CDR-SB). These data will be featured in a poster presentation at the 18th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™) 2024, being held both virtually and in Lisbon, Portugal from March 5–9, 2024. In addition, academic researchers from UCL will be presenting data in a separate poster at the meeting demonstrating that p38MAPK inhibition, including with neflamapimod specifically, improves tau-induced axonal transport defects both in vitro and in a tauopathy mouse model.

Key Points: 
  • These data will be featured in a poster presentation at the 18th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™) 2024, being held both virtually and in Lisbon, Portugal from March 5–9, 2024.
  • “The effects on GFAP, particularly the association between GFAP response and clinical outcomes, further support that neflamapimod is clinically efficacious in patients with DLB,” said John Alam, MD, Chief Executive Officer of CervoMed.
  • A PDF copy of the GFAP poster presentation will be available on the “ Presentations and Publications ” section of the CervoMed website.
  • Title: NEW INSIGHTS IN THE DEVELOPMENT OF BIOMARKERS, IMAGING, AND THERAPY OF ALPHA-SYNUCLEIN, LRKK2, AND GBA PATHOLOGIES

The Rainwater Charitable Foundation Announces 2024 Rainwater Prize Winners, Dr. Virginia Man-Yee Lee and Dr. Cristian Lasagna-Reeves, for Ongoing Contributions to Scientific Understanding of Tau in the Brain

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Giovedì, Febbraio 15, 2024
Yee, DNA-binding protein, Tauopathy, BSN, ALS, Research, Dementia, TAR, Nature Neuroscience, PSP, Rain, Pathology, Alpha-synuclein, Neurodegenerative disease, Conference, Frontotemporal dementia, University, Medical laboratory, Drug discovery, Innovation, Associate, Movement, Biology, Brain, Pharmaceutical industry

FORT WORTH, Texas, Feb. 15, 2024 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative research that enables field-advancing programs, resources and breakthrough solutions for primary tauopathies, today announced Virginia Man-Yee Lee, Ph.D., University of Pennsylvania Perelman School of Medicine, will be awarded the 2024 Outstanding Innovation in Neurodegenerative Disease Research Prize of $400,000, and Cristian Lasagna-Reeves, Ph.D., M.S., Indiana University School of Medicine will be awarded the Rainwater Prize for Innovative Early-Career Scientist of $200,000. The prizes will be presented during the Tau2024 Global Conference on March 25-26, 2024, in Washington, D.C.

Key Points: 
  • The Rainwater Prize Program , now in its fifth year, aims to highlight and support scientific progress toward addressing critical gaps for neurodegenerative diseases associated with the accumulation of the tau protein in the brain.
  • Currently, Dr. Lasagna-Reeves' lab focuses on understanding the role of tau in Alzheimer's disease and related dementias.
  • Dr. Lasagna-Reeves winning this year's Early-Career Prize recognizes the scientific discoveries that could lead to a potential new therapeutic approach for tauopathies.
  • We're proud to support their research and provide resources that may contribute to potential future breakthroughs in our understanding of tau."