Cerebrospinal fluid

Wave Life Sciences Reports First Quarter 2024 Financial Results and Provides Business Update

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torsdag, maj 9, 2024
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ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., May 09, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Revenue was $12.5 million for the first quarter of 2024, as compared to $12.9 million in the first quarter of 2023.
  • Net loss was $31.6 million for the first quarter of 2024, as compared to $27.4 million for the first quarter of 2023.
  • ET to review the first quarter 2024 financial results and pipeline updates.

Denali Therapeutics Reports First Quarter 2024 Financial Results and Business Highlights

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tisdag, maj 7, 2024
FDA, Amyloid beta, Part, Total, PIPE, CDER, Transferrin receptor, Nerve, BBB, CSF, Biomarker, Gamma, Enzyme replacement therapy, Traffic barrier, Hunting, OTV, PTV, Food, MGH, IDS, Conference, MAPT, ALS, Therapy, Cerebrospinal fluid, SAN, MPS II, Hospital, Safety, AFL, SGSH, UC, Alpha-synuclein, LUMA, SNCA, Clinical trial, Glycogen storage disease, Neurodegenerative disease, MPS, COMPASS, Patient, Alfa, Acquisition, ETV, ATV, Hunter syndrome, Sanofi, Massachusetts General Hospital, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the first quarter ended March 31, 2024, and provided business highlights.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the first quarter ended March 31, 2024, and provided business highlights.
  • There was no collaboration revenue for the quarter ended March 31, 2024, compared to $35.1 million for the quarter ended March 31, 2023.
  • Total research and development expenses were $107.0 million for the quarter ended March 31, 2024, compared to $128.8 million for the quarter ended March 31, 2023.
  • General and administrative expenses were $25.2 million for the quarter ended March 31, 2024, compared to $27.1 million for the quarter ended March 31, 2023.

Creyon Bio to Present Clinical Data on Rapid AI-Enabled Engineering of Oligonucleotide-Based Medicines at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting

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tisdag, maj 7, 2024
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SAN DIEGO and RESEARCH TRIANGLE PARK, N.C., May 07, 2024 (GLOBE NEWSWIRE) -- Creyon Bio, Inc. ("Creyon"), a clinical stage drug development company that engineers Oligonucleotide-Based Medicines (OBMs) with industry-leading efficiency creating novel, best-in-class gene-centric medicines to treat rare and common diseases, today announced encouraging clinical data highlighting the use of its custom designed data set paired with artificial intelligence (AI) to rapidly engineer an investigational antisense oligonucleotide (ASO) therapy. Within one year of project initiation in partnership with The TNPO2 Foundation, Creyon developed a novel allele-selective Locked Nucleic Acid (LNA) treatment candidate for an ultra-rare and severe neurological disease caused by a single nucleotide variant in the Transportin-2 (TNPO2) receptor, and an investigator-initiated clinical trial received approval to dose a patient. Initial data from this trial demonstrate that the investigational treatment successfully reduced seizures, restored developmental milestones, and was well-tolerated over nine months. Creyon will share these results during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore on Friday, May 10, 2024, between 5:15-5:30 p.m. EDT in Room 307-308 of the Baltimore Convention Center.

Key Points: 
  • Initial data from this trial demonstrate that the investigational treatment successfully reduced seizures, restored developmental milestones, and was well-tolerated over nine months.
  • Creyon will share these results during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore on Friday, May 10, 2024, between 5:15-5:30 p.m. EDT in Room 307-308 of the Baltimore Convention Center.
  • “We developed predictive models that guide our molecular engineering from the outset and help mitigate potential safety concerns.
  • “Inflammatory reactions to oligonucleotide therapies have caused serious adverse reactions in other early clinical-stage therapies delivered directly to the central nervous system.

Amylyx Pharmaceuticals Reports First Quarter 2024 Financial Results

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torsdag, maj 9, 2024
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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.

QIAGEN expands tuberculosis portfolio with new NGS Panel to support real-time surveillance and combat antimicrobial resistance

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onsdag, april 24, 2024
TST, Genetically modified bacteria, WHO, Virtual reality, CDC, Multidrug-resistant tuberculosis, Bacteria, AMR, ESCMID, QIAGEN, ECCMID, MDR-TB, WGS, Doctor of Philosophy, Research, Workflow, Sputum, NGS, PCR, Disease, Patient, Panel, Mycobacterium tuberculosis, QGEN, Gold, Infection, D20, Emerging, Tuberculosis, Cerebrospinal fluid, Vaccine

The QIAseq xHYB Mycobacterium tuberculosis Panel represents a major advancement in solving the problem of bacterial WGS from complex host samples, using next-generation sequencing (NGS).

Key Points: 
  • The QIAseq xHYB Mycobacterium tuberculosis Panel represents a major advancement in solving the problem of bacterial WGS from complex host samples, using next-generation sequencing (NGS).
  • By eliminating the need for a 4-to-6-week bacterial culture, the panel significantly accelerates the process of obtaining results.
  • “The introduction of the QIAseq xHYB Mycobacterium tuberculosis Panel reflects our ongoing commitment to address global health challenges like TB and antimicrobial resistance,” said Nitin Sood, Vice President, Head of the Life Sciences Business Area at QIAGEN.
  • The QIAseq xHYB Mycobacterium tuberculosis Panel also plays a vital role in the detection and management of antimicrobial resistance (AMR), a growing concern in TB treatment.

Cognition Therapeutics KOL Event Provided Platform for Valuable Discussion of Current Therapeutic Options and Need for Additional Options to Treat Alzheimer’s Disease

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måndag, april 22, 2024
Amyloid, Alzheimer's disease, Jort, University, MCI, Inflammation, Oxidative stress, ARIA, News, Research, Neuroinflammation, Cognition, Doctor of Philosophy, ADAS, START, KOL, Patient, SHINE, Perception, Neuron, Antibody, Therapy, Plaque, Cerebrospinal fluid

“The KOL roundtable we conducted featured a panel of Alzheimer’s disease expert clinicians and researchers who highlighted the need to find Alzheimer’s disease treatments that are less burdensome for patients and their care partners,” said Lisa Ricciardi, Cognition’s president and CEO .

Key Points: 
  • “The KOL roundtable we conducted featured a panel of Alzheimer’s disease expert clinicians and researchers who highlighted the need to find Alzheimer’s disease treatments that are less burdensome for patients and their care partners,” said Lisa Ricciardi, Cognition’s president and CEO .
  • The SHINE study enrolled participants in the U.S. and several countries in Europe including The Netherlands.
  • Dr. Vijverberg reviewed the design of the SHINE study and the results of the preliminary analysis of the first 24 patients who finished six months of treatment.
  • An archive of the KOL roundtable discussion is available on the News & Events page of the Investors section of the Cognition website or may be accessed directly at the following URL: https://lifescievents.com/event/cognition/ .

Vigil Presents Key Findings from ILLUMINATE & IGNITE Studies in ALSP at the 2024 American Academy of Neurology Annual Meeting

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onsdag, april 17, 2024
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WATERTOWN, Mass., April 17, 2024 (GLOBE NEWSWIRE) -- Vigil Neuroscience, Inc. (Nasdaq: VIGL), a clinical-stage biotechnology company committed to harnessing the power of microglia for the treatment of neurodegenerative diseases, today announced the presentation of multiple oral and poster presentations on the Company’s lead clinical candidate iluzanebart at the 2024 American Academy of Neurology (AAN) Annual Meeting.

Key Points: 
  • “We are excited to see our enthusiasm for iluzanebart matched by clinical leaders in the ALSP community.
  • The interim findings from both the IGNITE Phase 2 and ILLUMINATE Natural History studies support the potential of iluzanebart to become the first, disease-modifying therapy for those living with ALSP,” said Petra Kaufmann, M.D., F.A.A.N, Chief Medical Officer of Vigil.
  • “Both trials have led to an incredible step forward – not only in understanding ALSP disease progression, but also in drawing parallels between biomarkers and correlating clinical outcomes.
  • Data on clinical measures, biomarkers, and MRI from ILLUMINATE were presented at the conference.

Alterity Therapeutics Parkinson’s Disease and Multiple System Atrophy Data Featured at the American Academy of Neurology (AAN) 2024 Annual Meeting

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onsdag, april 17, 2024
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MELBOURNE, Australia and SAN FRANCISCO, April 17, 2024 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that three posters were presented at the American Academy of Neurology (AAN) 2024 Annual Meeting taking place April 13-18, 2024, in Denver, Colorado, USA. Featured presentations described the Company’s work in Parkinson’s disease and Multiple System Atrophy (MSA), including initial biomarker data and baseline characteristics from the ATH434-201 Phase 2 clinical trial.

Key Points: 
  • Featured presentations described the Company’s work in Parkinson’s disease and Multiple System Atrophy (MSA), including initial biomarker data and baseline characteristics from the ATH434-201 Phase 2 clinical trial.
  • At AAN we reported the baseline characteristics from our ATH434-201 Phase 2 trial including fluid biomarkers and neuroimaging data.
  • The data showed increased iron in areas of pathology and elevated plasma Neurofilament Light Chain (NfL) levels at baseline that correlated significantly with disease severity.
  • Plasma NfL significantly increased over 12 months, and both plasma and CSF NfL were associated with disease progression in MSA.

Acumen Pharmaceuticals Presents First Comprehensive Clinical and Biomarker Data for Sabirnetug (ACU193) at American Academy of Neurology 2024 Annual Meeting

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tisdag, april 16, 2024
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Acumen is developing sabirnetug as a potential best-in-class antibody treatment for early AD.

Key Points: 
  • Acumen is developing sabirnetug as a potential best-in-class antibody treatment for early AD.
  • Acumen remains on track to initiate the ALTITUDE-AD placebo-controlled Phase 2 trial of sabirnetug in the first half of 2024.
  • Based on safety, target engagement and biomarker data from the INTERCEPT-AD trial, Acumen has determined sabirnetug doses of 35 mg/kg Q4W and 50 mg/kg Q4W in ALTITUDE-AD.
  • Acumen also plans to initiate a Phase 1 bioavailability study to support a subcutaneous dosing option of sabirnetug in mid-2024, as announced in November 2023 .

Alzheon Announces First Patient Dosed in Long-Term Extension of APOLLOE4 Phase 3 Trial of Oral ALZ-801/Valiltramiprosate and Launches 52-Week Extension of Phase 2 Biomarker Trial in Patients with Early Alzheimer’s Disease

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tisdag, april 30, 2024
Mental Health, Health, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, Amyloid, MCI, APOE4, Risk, Safety, Cognition, MD, Doctor of Philosophy, ADAS, Disease, MB, Week, Atrophy, Patient, Master of Science, Alzheimer's disease, CSF, Genotype, Biomarker, Neurotoxicity, Cerebrospinal fluid, Plasma, Pharmaceutical industry

Early AD includes patients with mild cognitive impairment due to AD (MCI) and mild AD.

Key Points: 
  • Early AD includes patients with mild cognitive impairment due to AD (MCI) and mild AD.
  • ALZ-801/valiltramiprosate is an investigational oral disease-modifying therapy in Phase 3 development for the treatment of Early AD.
  • In mechanism of action studies, ALZ-801 fully blocked the formation of neurotoxic soluble beta amyloid oligomers at the Phase 3 clinical dose.
  • “Given these encouraging results, we are compelled to extend the trial for an additional year, bringing the treatment period to four years for these patients.