SNCA

Denali Therapeutics Reports First Quarter 2024 Financial Results and Business Highlights

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화요일, 5월 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.

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

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화요일, 2월 27, 2024
MPS II, Hearing, ARIA, Antibody, LRRK2, PTV, IDS, Transferrin receptor, MAPT, Traffic barrier, ALS, Hunting, Dermatan sulfate, Therapy, BBB, SAN, Total, Amyloid-related imaging abnormalities, Alpha-synuclein, COMPASS, ETV, Hunter syndrome, Neurodegenerative disease, Conference, MPS, Safety, Biomarker, OTV, LUMA, AFL, SNCA, Amyloid beta, CSF, PIPE, Glycogen storage disease, Research, ATV, FDA, Food, Risk, Sanofi, UC, CD98, Patient, Cognition, Oligonucleotide, Science, Alfa, Pharmaceutical industry

Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.

Key Points: 
  • Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.
  • Sanofi intends to present the detailed efficacy and safety results of the ALS Phase 2 HIMALAYA study at a future scientific forum.
  • There was no collaboration revenue for the quarter ended December 31, 2023, compared to $10.3 million for the quarter ended December 31, 2022.
  • Further, for the quarter ended December 31, 2023, there was also a decrease in other unallocated research and development expenses as a result of reduced facility costs.

Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases

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월요일, 1월 8, 2024
CSF, Lysosomal storage disease, Cerebrospinal fluid, Risk, Sanfilippo syndrome, Amyloid, BBB, UC, Sanfilippo, Manuscript, ETV, ALS, OTV, SNCA, IDS, Asos, Conference, Cognition, RIPK1, Fluid mechanics, Neuroinflammation, Premedication, Glycogen storage disease, Amyloid beta, SGSH, Therapy, COMPASS, Traffic barrier, Hunting, N-sulfoglucosamine sulfohydrolase, Hunter syndrome, ISR, NewCo, Amyloid-related imaging abnormalities, Inflammation, CNS, Neurodegenerative disease, Takeda, Biomarker, Alpha-synuclein, K2, Biology, DSM-IV codes, LUMA, Brain, MPS, Investigational New Drug, PTV, ARIA, Nerve, AFL, Pathology, MPS II, Interim, BioRxiv, Plaque, MAPT, LRRK2, Elevator mechanic

The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.

Key Points: 
  • The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.
  • Upon completion of the ongoing Phase 1/2 study, and together with data from COMPASS, this combined data package is intended to support registration.
  • Increased RIPK1 activity in the CNS is hypothesized to drive neuroinflammation and cell necroptosis and to contribute to neurodegeneration.
  • Denali will maintain ownership of and continue to advance its current portfolio of clinical stage small molecule programs.

WaveBreak Presents New Preclinical Data at MDS 2023 Demonstrating the Efficacy of its First-in-Class, Small-Molecule Inhibitors of Toxic Oligomers for Blocking Progression of Both α-Synuclein Oligomers and Aggregates in Models of Parkinson's Disease

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수요일, 8월 30, 2023
Therapy, Disease, Protein folding, MDS, Movement, Protein, Parkinson's disease, Drug discovery, Poster, ALS, M83, Neurotoxicity, Alzheimer's disease, Pharmaceutical industry, Fine chemical, SNCA

BOSTON, Aug. 30, 2023 /PRNewswire/ -- WaveBreak announced today the presentation of new preclinical data for its first-in-class, oral inhibitors of α-synuclein oligomer generation demonstrating efficacy for blocking the progression of both α-synuclein oligomers and aggregates in cell and mouse models of Parkinson's disease.

Key Points: 
  • BOSTON, Aug. 30, 2023 /PRNewswire/ -- WaveBreak announced today the presentation of new preclinical data for its first-in-class, oral inhibitors of α-synuclein oligomer generation demonstrating efficacy for blocking the progression of both α-synuclein oligomers and aggregates in cell and mouse models of Parkinson's disease.
  • The data were presented in a poster presentation today at the MDS International Congress of Parkinson's Disease and Movement Disorders® taking place August 27–31 in Copenhagen.
  • Highlights of the presentation follow, and the poster is available on WaveBreak's website:
    A WaveBreak oral, small-molecule inhibitor of α-synuclein oligomer generation—WTX-A—demonstrated potent reduction of α-synuclein oligomers and aggregates in cell and mouse models of Parkinson's disease.
  • WaveBreak's technology platform enables analysis of the inhibition of the source mechanisms that produce oligomer intermediates in complex disease processes.

Seelos Therapeutics Announces Initiation of Preclinical Study of SLS-004 in Parkinson’s Disease

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목요일, 5월 28, 2020
Branches of biology, Biology, Proteins, Peripheral membrane proteins, Genome editing, Immune system, Lewy body dementia, Neuropathology, Alpha-synuclein, Synuclein, SNCA, Parkinson's disease

Seelos is constructing a bimodular viral system harboring an endogenous alpha-synuclein (-synuclein) transgene and inducible regulated repressive CRISPR/Cas9-unit to achieve constitutive activation and inducible suppression of PD-related pathologies.

Key Points: 
  • Seelos is constructing a bimodular viral system harboring an endogenous alpha-synuclein (-synuclein) transgene and inducible regulated repressive CRISPR/Cas9-unit to achieve constitutive activation and inducible suppression of PD-related pathologies.
  • There has been a high level of interest in the alpha-synuclein approach to Parkinson's and beginningfurtherwork on our first gene therapy program is exciting, said Raj Mehra, Ph.D., Chairman and CEO of Seelos.
  • The SNCA gene, which encodes -synuclein expression, has been implicated as a highly significant risk factor for PD.
  • The group's goal was to lay out a roadmap to advance drugs targeting alpha-synuclein proteins and increase their odds of success.

Seelos Therapeutics Reports Q2 2019 Pipeline Update

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목요일, 8월 1, 2019
Branches of biology, Proteins, Peripheral membrane proteins, Biology, Lewy body dementia, Neuropathology, Alpha-synuclein, Synuclein, SNCA, Parkinson's disease

NEW YORK, Aug. 01, 2019 (GLOBE NEWSWIRE) -- Seelos Therapeutics, Inc. (NASDAQ: SEEL), a clinical-stage biopharmaceutical company, today released its second quarter pipeline update.

Key Points: 
  • NEW YORK, Aug. 01, 2019 (GLOBE NEWSWIRE) -- Seelos Therapeutics, Inc. (NASDAQ: SEEL), a clinical-stage biopharmaceutical company, today released its second quarter pipeline update.
  • Additional preclinical studies are underway for SLS-007 to further characterize the peptides potential in targeting alpha-synuclein pathology in Parkinsons Disease.
  • Seelos acquired exclusive worldwide licensing of a gene therapy program targeting the regulation of the SNCA gene, which encodes alpha-synuclein expression,from Duke University.
  • Seelos announced the receipt of a grant from Team Sanfilippo Foundation to be used towards funding of the SLS-005 program.