Cerebrospinal fluid

Metabolon and Cardiff University Centre for Neuropsychiatric Genetics and Genomics Forge Alliance to Propel Multiple Sclerosis Research

Retrieved on: 
tisdag, mars 12, 2024
Plasma, Metabolomics, Disease, Multiple sclerosis, CSF, Partnership, Central nervous system, Biomedical Research, Patient, Biomarker, Research, Parkinson's disease, Cerebrospinal fluid, Medicine, Cardiff University, Alzheimer's disease, Pathology, Pharmaceutical industry

MORRISVILLE, N.C., March 12, 2024 /PRNewswire/ -- Metabolon, Inc., the global leader in providing metabolomics solutions advancing a wide variety of life science research, diagnostic, therapeutic development, and precision medicine applications, today announced a partnership with Cardiff University Centre for Neuropsychiatric Genetics and Genomics, a renowned institution at the forefront of biomedical research. This collaboration is poised to discover new biomarkers to accelerate advancements in the understanding and treatment of multiple sclerosis (MS).

Key Points: 
  • This collaboration is poised to discover new biomarkers to accelerate advancements in the understanding and treatment of multiple sclerosis (MS).
  • MS is a neuroinflammatory disorder that affects the central nervous system, giving rise to a range of physical and neuropsychological challenges.
  • For years, the Cardiff University Centre for Neuropsychiatric Genetics and Genomics has been following MS patients and developing cutting-edge tools for clinicians and researchers.
  • To learn more about Metabolon's research and development efforts in neurodegeneration diseases, including Alzheimer's, Parkinson's, and MS, visit https://www.metabolon.com/applications/neuroscience/

Ventyx Biosciences Reports Clinical Data for its NLRP3 Inhibitor Portfolio and Provides Pipeline Updates at Virtual Investor Event

Retrieved on: 
måndag, mars 11, 2024
SAN, Ulcerative colitis, Week, CDAI, IC50, Solipsism syndrome, Peripheral, Obesity, Cerebrospinal fluid, Cardiovascular disease, Treatment, MACE, NLRP3, Volunteering, Inflammation, Modulation, QD, Safety, Biomarker, UC, CAPS, Pharmacokinetics, Webcast, Patient, Molina Healthcare, TYK2, CSF, PIPE, Plasma, FCAS, Risk, Pharmaceutical industry

SAN DIEGO, March 11, 2024 (GLOBE NEWSWIRE) -- Ventyx Biosciences, Inc. (Nasdaq: VTYX) (“Ventyx”), a clinical-stage biopharmaceutical company focused on advancing novel oral therapies that address a broad range of inflammatory diseases with significant unmet medical need, will provide clinical and pipeline updates today during its virtual investor event.

Key Points: 
  • We believe these data support the potential for VTX3232 to emerge as a best-in-class CNS-penetrant NLRP3 inhibitor for the treatment of neuroinflammatory diseases.
  • We believe these data establish compelling clinical proof of concept for our peripheral NLRP3 inhibitor VTX2735.
  • At our virtual investor event, we will present data from the ongoing Phase 2 open-label extension.
  • Ventyx will host a virtual investor event today, Monday, March 11, 2024 from 11:00AM to 12:30PM ET.

Acumen Pharmaceuticals Presents Sabirnetug (ACU193) Fluid Biomarker and Target Engagement Analyses from Phase 1 INTERCEPT-AD Study in Early Alzheimer’s at the AD/PD™ 2024 Annual Meeting

Retrieved on: 
fredag, mars 8, 2024
ABOS, Immunotherapy, Pathology, Cerebrospinal fluid, Plaque, Neurogranin, Conference, Biomarker, DSM-IV codes, Disease, Trial of the century

CHARLOTTESVILLE, Va., March 08, 2024 (GLOBE NEWSWIRE) -- Acumen Pharmaceuticals, Inc. (NASDAQ: ABOS), a clinical-stage biopharmaceutical company developing a novel therapeutic that targets soluble amyloid beta oligomers (AβOs) for the treatment of Alzheimer’s disease (AD), today presented cerebrospinal fluid (CSF) biomarker data from the sabirnetug (ACU193) Phase 1 INTERCEPT-AD trial in an oral presentation at the International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD) in Lisbon, Portugal, and online. Acumen also presented a poster showcasing the method used to develop a first-of-its-kind assay to measure target engagement of an AβO-selective antibody.

Key Points: 
  • Acumen also presented a poster showcasing the method used to develop a first-of-its-kind assay to measure target engagement of an AβO-selective antibody.
  • Acumen’s sabirnetug is the first humanized monoclonal antibody to clinically demonstrate selective target engagement of AβOs, a soluble and highly toxic form of Aβ that accumulates early in AD and triggers synaptic dysfunction and neurodegeneration.
  • Positive topline results from 62 participants dosed in the Phase 1 INTERCEPT-AD trial (NCT04931459) were reported in July 2023 , and additional insights from exploratory analyses have further confirmed sabirnetug’s proof-of-mechanism and broad therapeutic potential as a next-generation treatment for early AD.
  • The novel assay configuration was tailored to selectively detect sabirnetug-AβO complex in CSF as a direct measure of target engagement, showing clear dose-related increases in target engagement across all cohorts.

Vaxxinity Announces Positive Target Engagement Data from Phase 1 Clinical Trial for Parkinson’s Disease at AD/PD™ 2024

Retrieved on: 
torsdag, mars 7, 2024
PD, Research, Doctor of Philosophy, SVP, Cerebrospinal fluid, Conference, MJFF, Mayo Clinic, Clinical professor, Patient, CSF, Synucleinopathy, Pharmaceutical industry

UB-312 is the first active immunotherapy candidate to show reduction of pathological alpha-synuclein (aSyn) in cerebrospinal fluid (CSF) of PD patients.

Key Points: 
  • UB-312 is the first active immunotherapy candidate to show reduction of pathological alpha-synuclein (aSyn) in cerebrospinal fluid (CSF) of PD patients.
  • UB-312 is designed to target aggregated forms of aSyn, the toxic species that underlies PD and other synucleinopathies.
  • As part of the randomized, double-blind, placebo-controlled Phase 1 clinical trial, The Michael J.
  • “Currently, there are no treatments that address the underlying conditions of Parkinson’s, and we are very excited about this target engagement data.

Mustang Bio Announces Publication in Nature Medicine of Data from Phase 1 Trial Evaluating MB-101 IL13Rα2-targeted CAR T-Cells in High-Grade Glioma

Retrieved on: 
torsdag, mars 7, 2024
T cell, Glioblastoma, Arms, ICT, ICV, Traffic barrier, Toxicity, Headache, Biopsy, DSM-IV codes, Cerebrospinal fluid, City, Cancer, Central nervous system, CXCL10, TME, Cerebral edema, Dual, Hope, GBM, Safety, CXCL9, Fatigue, Hypertension, Translation, CNS, Tumor microenvironment, Patient, Arm, Spinal cord, Survival, Brain, Nature Medicine, Infrared spectroscopy correlation table, Vaccine

WORCESTER, Mass., March 07, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced Phase 1 clinical data were published in Nature Medicine that demonstrated the promising safety and clinical activity of Mustang’s MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of patients with recurrent and refractory malignant glioma, including glioblastoma.

Key Points: 
  • MB-101 was developed by City of Hope, one of the largest cancer research and treatment organizations in the United States, and exclusively licensed to Mustang.
  • Central nervous system (CNS) increases in inflammatory cytokines, including IFNγ, CXCL9, and CXCL10, were associated with CAR T-cell administration and bioactivity.
  • Primary endpoints were safety and feasibility, with secondary endpoints measuring therapy-related cytokine dynamics, CAR T-cell persistence and clinical outcomes.
  • Dr. Brown has a financial interest in Mustang and has previously been a paid consultant for the company.

NodThera’s NLRP3 Inhibitor NT-0796 Reverses Neuroinflammation in Parkinson’s Disease Phase Ib/IIa Trial

Retrieved on: 
torsdag, mars 7, 2024
Queen Square, Doctor of Philosophy, Aes, TREM, CXCL8, CXCL1, Blood, IL-6, Cerebrospinal fluid, Inflammation, Neuroinflammation, NLRP3, Șaeș, Fibrinogen, Volunteering, Risk, Human, Biomarker, AFL, CRP, Patient, Pharmacokinetics, Disease, DSM-IV codes, MRCP, MBBS, Brain, CCL2, Mouse, Face, Pharmaceutical industry

IL-1β, IL-6, CCL2, CXCL1 and CXCL8) over 28 days compared to baseline to levels approximating those of healthy elderly controls, demonstrating reversal of NLRP3-mediated neuroinflammation.

Key Points: 
  • IL-1β, IL-6, CCL2, CXCL1 and CXCL8) over 28 days compared to baseline to levels approximating those of healthy elderly controls, demonstrating reversal of NLRP3-mediated neuroinflammation.
  • In addition, reductions in neurodegenerative markers were also observed following oral dosing of NT-0796, including NfL and soluble TREM (sTREM2).
  • The correlation between Parkinson’s disease and neuroinflammation is well-documented, with alpha-synuclein fibrils triggering microglial NLRP3 activation, leading to neuroinflammation and subsequent neurodegeneration.
  • This is the inaugural demonstration of an NLRP3 inhibitor’s potential to not only address Parkinson’s disease but also offer a broader impact on neurodegenerative diseases.

Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
onsdag, mars 6, 2024
Therapy, Facial muscles, Pharmacology, Central nervous system, Research, GSK, MD, Liver disease, Dystrophin, Obesity, Cerebrospinal fluid, N-Acetylgalactosamine, HD, Weight loss, DMD, Duchenne muscular dystrophy, Lof, Exercise, Lipolysis, Exon, MBA, Conference, Wave, Activin and inhibin, AATD, Health, Safety, Biomarker, RNA, Greenshoe, Genetics, Pharmacokinetics, Webcast, Patient, Clinical trial, HTT, Q&A, Small RNA, Serum, Episcopal conference, Human, Coronary artery disease, Pharmaceutical industry

In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.

Key Points: 
  • In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.
  • Revenue was $29.1 million for the fourth quarter of 2023 as compared to $1.2 million in the prior year quarter.
  • Net loss was $16.3 million for the fourth quarter of 2023 as compared to $43.7 million in the prior year quarter.
  • ET to review the fourth quarter and full year 2023 financial results and pipeline updates.

Fortress Biotech and Cyprium Therapeutics Announce $4.1 Million Grant from NINDS to Further Development of AAV-ATP7A Gene Therapy for Menkes Disease

Retrieved on: 
måndag, mars 4, 2024
Bangladesh Technical Education Board, Natural history, Gene, Nationwide, NIH, ATPase, FDA, Fortress Biotech, Food, Cerebrospinal fluid, Hospital, Principal investigator, Gene therapy, Human, Research institute, Therapy, Menkes disease, Patient, X-linked recessive inheritance, Cyprium, CSF, Clinical trial, Pediatrics, Fortification, Male, Genetics, Mutation, Spinal muscular atrophy, Copper, NDA, Orphan, AAV, DSM-IV codes, Research, Mouse, Pharmaceutical industry

MIAMI, March 04, 2024 (GLOBE NEWSWIRE) -- Cyprium Therapeutics, Inc. (“Cyprium”), a majority-owned subsidiary of Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), today announced that the National Institute of Neurological Disorders and Stroke (“NINDS”) of the National Institutes of Health (“NIH”) has awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Stephen G. Kaler, M.D., M.P.H., to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial to advance adeno-associated virus (“AAV”)-ATP7A gene therapy, also known as AAV-ATP7A, for the treatment of Menkes disease.

Key Points: 
  • Often lethal if untreated, Menkes disease is an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase.
  • “By combining CUTX-101 with working copies of ATP7A delivered by AAV, we hope to enhance clinical outcomes in Menkes disease, a fatal rare pediatric disease.
  • Preclinical studies have demonstrated a synergistic effect of AAV-ATP7A and CUTX-101 in a reliable mouse model of Menkes disease.
  • In early studies, cerebrospinal fluid (“CSF”)-directed AAV gene therapy rescued 22-53% of mice with a mutation in the human Menkes disease homolog (mottled-brindled) when combined with CSF or subcutaneous copper.

Cassava Sciences Reports Full-year 2023 Financial Results and Corporate Updates

Retrieved on: 
onsdag, februari 28, 2024
Research, ARIA, FLNA, Drug development, ADAS, Exercise, ADAS-Cog, Private law, Mass spectrometry, Criterion, CDR, Bawskee 3.5, Protein, Intellectual property, Patient, Plasma, Cassava Sciences, PET, MRI, City University, Hyperkinesia, Cerebrospinal fluid, Edema, Cassava, Memory, Insulin, G&A, Tablet, Brain, City, CUNY, SAP, Calendar, Completion, Safety, Clinical, Knowledge, Alzheimer's disease, Magnetic resonance imaging, Blood, FDA, Ageing, Food, Data, Lymphocyte, Medicine, DSMB, Pharmaceutical industry

AUSTIN, Texas, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company focused on Alzheimer’s disease, today reported financial and operating results for the full year ended December 31, 2023 and presented corporate updates.

Key Points: 
  • AUSTIN, Texas, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company focused on Alzheimer’s disease, today reported financial and operating results for the full year ended December 31, 2023 and presented corporate updates.
  • Net cash used in operations full-year 2023 was $82.0 million, consistent with previous guidance.
  • (This amount is not included in the above cash and cash equivalents at December 31, 2023.)
  • Recent corporate highlights include the following:
    In January 2024, we completed a dividend distribution of common stock warrants to shareholders.

uniQure Announces 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
onsdag, februari 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.