N-sulfoglucosamine sulfohydrolase

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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Monday, January 8, 2024
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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.

Denali Therapeutics Reports Third Quarter 2023 Financial Results and Business Highlights

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Tuesday, November 7, 2023
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SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (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 third quarter ended September 30, 2023, and provided business highlights.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (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 third quarter ended September 30, 2023, and provided business highlights.
  • Denali has selected five ASO targets for further development and is focused on advancing two OTV candidates towards clinical development.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were approximately $1.12 billion as of September 30, 2023.
  • General and administrative expenses were $25.3 million and $23.3 million for the three months ended September 30, 2023 and 2022, respectively.

Denali Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Business Highlights

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Monday, February 27, 2023
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In February 2023, at the WORLDSymposiumTM, Denali reported additional interim data from the open-label, single-arm Phase 1/2 study of DNL310.

Key Points: 
  • In February 2023, at the WORLDSymposiumTM, Denali reported additional interim data from the open-label, single-arm Phase 1/2 study of DNL310.
  • The Phase 2/3 COMPASS study continues to enroll up to 54 participants with MPS II with and without neuronopathic disease.
  • In October 2022, Denali raised net proceeds of approximately $296.2 million through a public offering of its common stock.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were $1.34 billion as of December 31, 2022.

Denali Therapeutics Announces New Interim Data from DNL310 Phase 1/2 Study for MPS II and DNL126 Preclinical Data for MPS IIIA at WORLDSymposium™

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Wednesday, February 22, 2023
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DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II.

Key Points: 
  • DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II.
  • The interim Phase 1/2 data are being presented at the 19th Annual WORLDSymposium™ in Orlando, Florida, February 22-26, 2023.
  • “As the study progresses and enrollment continues for the global Phase 2/3 COMPASS study, I am excited to learn more about the potential of DNL310 to offer meaningful benefit for the entire MPS II patient population."
  • The safety profile of DNL310 remains consistent with standard of care, now with data up to two years of treatment with DNL310.

Lysogene Provides Clinical Update on AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPS IIIA at the 25th Annual ASGCT Meeting

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Wednesday, May 18, 2022
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Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, provided updated clinical data from the ongoing AAVance phase 2/3 clinical trial with the investigational gene therapy LYS-SAF302 for the treatment of MPS IIIA (NCT03612869) during the 25th Annual Meeting of the ASGCT (American Society for Gene & Cell Therapy) in Washington, DC.

Key Points: 
  • Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, provided updated clinical data from the ongoing AAVance phase 2/3 clinical trial with the investigational gene therapy LYS-SAF302 for the treatment of MPS IIIA (NCT03612869) during the 25th Annual Meeting of the ASGCT (American Society for Gene & Cell Therapy) in Washington, DC.
  • These results, which suggest that younger patients are more responsive to the gene therapy treatment, need to be corroborated and confirmed by the final statistical analyses.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).
  • Lysogene is also developing an innovative AAV gene therapy approach for the treatment of Fragile X syndrome, a genetic disease related to autism.

Lysogene Provides Update on Efficacy, Safety and Timelines of the AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPSIIIA

Retrieved on: 
Thursday, February 24, 2022
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Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today provides an update on clinical results and timelines of the ongoing AAVance phase 2/3 clinical trial with the investigational gene therapy LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

Key Points: 
  • Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today provides an update on clinical results and timelines of the ongoing AAVance phase 2/3 clinical trial with the investigational gene therapy LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).
  • LYS-SAF302 is intended to deliver a functional copy of the SGSH gene and allow the brain to secrete the missing enzyme.
  • In terms of safety, white matter abnormalities were observed near injection sites of patients treated in AAVance.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).