Latest news with #WORLDSymposium™2025
Associated Press
30-01-2025
- Business
- Associated Press
Denali Therapeutics Announces Upcoming Presentations on Hunter Syndrome (MPS II) and TransportVehicle™ Enabled Investigational Therapeutic Tividenofusp Alfa at the 2025 WORLDSymposium™
SOUTH SAN FRANCISCO, Calif., Jan. 30, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced upcoming oral (platform) and poster presentations at the 21st Annual WORLD Symposium ™, which will be held February 3-7, 2025, in San Diego, California. The oral presentation includes clinical results related to its Hunter syndrome (MPS II) investigational therapeutic, tividenofusp alfa (DNL310). Tividenofusp alfa is enabled by the Denali TransportVehicle™ platform, which is designed to effectively deliver enzyme, oligonucleotide, or antibody therapeutics to all tissues in the body, including the brain by crossing the blood-brain barrier. The presentation schedule at WORLD Symposium ™ 2025 is as follows: Title: Interim Analysis of Efficacy and Safety of Weekly Intravenous Tividenofusp Alfa in Mucopolysaccharidosis Type II (MPS II) – Platform Presentation Session: Clinical Applications Platform Presentations Date: Thursday, February 6, 2025 Session Time: 8:30 AM Pacific Time Title: Unmet Needs in the Treatment and Care of Somatic Manifestations in People with Mucopolysaccharidosis Type II (Hunter Syndrome): A Targeted Literature Review – Poster #44 Session: Translational Research – Poster Session II Date: Thursday, February 6, 2025 Session Time: 3:30 – 5:30 PM Pacific Time Title: Age-Dependent Reference Intervals for Cerebrospinal Fluid (CSF) and Urine Heparan Sulfate (HS) and Dermatan Sulfate (DS) and CSF Gangliosides – Poster #132 Session: Clinical Applications – Poster Session III Date: Thursday, February 6, 2025 Session Time: 3:30 – 5:30 PM Pacific Time Denali is also sponsoring the following satellite symposium event at WORLD Symposium ™ 2025: Title: Voices in Unison – Insights into the Unmet Needs in MPS II from Patient Community and Physician Perspectives (Chair: Barbara K. Burton, M.D.; Speakers: Joseph Muenzer, M.D., Ph.D. and Kristin McKay, President and Executive Director of Project Alive) Date: Wednesday, February 5, 2025 Time: 6:45 – 7:45 AM Pacific Time About Denali Therapeutics Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for neurodegenerative diseases and lysosomal storage diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the blood-brain barrier and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San Francisco. For additional information, please visit Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements by Denali Therapeutics Inc. ('Denali' or the 'Company') regarding Denali's planned presentations and events at the 2025 WORLD Symposium ™ and expectations related to Denali's TransportVehicle™ (TV) platform. Actual results are subject to risks and uncertainties and may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to: Denali's dependence on successful development of its BBB platform technology and TV-enabled product candidates; Denali's ability to initiate and enroll patients in its current and future clinical trials; Denali's ability to conduct or complete clinical trials on expected timelines; Denali's reliance on third parties for the manufacture and supply of its product candidates for clinical trials; the potential for clinical trial results to differ from preclinical, early clinical, preliminary or expected results; the risk of significant adverse events, toxicities, or other undesirable side effects; the risk that results from early clinical biomarker studies will not translate to clinical benefit in late clinical studies; the risk that product candidates may not receive regulatory approval necessary to be commercialized; developments relating to Denali's competitors and its industry, including competing product candidates and therapies; Denali's ability to obtain, maintain, or protect intellectual property rights; and other risks and uncertainties. In light of these risks, uncertainties, and assumptions, the forward-looking statements in this press release are inherently uncertain and may not occur, and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Accordingly, you should not rely upon forward-looking statements as predictions of future events. Denali's product candidates are investigational, and their safety and efficacy profiles have not yet been established. No Denali product candidates have been approved by any health authority for any use. Information regarding additional risks and uncertainties may be found in Denali's Annual and Quarterly Reports filed on Forms 10-K and 10-Q filed with the Securities and Exchange Commission (SEC) on February 28, 2024, and November 6, 2024, respectively, and Denali's future reports to be filed with the SEC. Denali does not undertake any obligation to update or revise any forward-looking statements, to conform these statements to actual results or to make changes in Denali's expectations, except as required by law. Laura Hansen, Ph.D. (650) 452-2747 Media Contact Rich Allan FGS Global (503) 851-0807
Associated Press
27-01-2025
- Health
- Associated Press
Immusoft to Present Positive Data from the First Engineered B Cell in a Human Clinical Trial at the 21st Annual WorldSymposium™ 2025
Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system. Results from a first-in-human clinical trial in subjects with MPS I SEATTLE, Jan. 27, 2025 /PRNewswire/ -- Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming WORLDSymposium™ 2025, held in San Diego, February 3-7, 2025. Immusoft will present the following: Oral Presentation: Friday, February 7th, 9:00 AM PST, Dr. Paul Orchard, MD. Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I Presenter: Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School Poster Presentation: Wednesday, February 5th, 2026, 3:30-5:30 PM PST, Kiosk 27-B. Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I Presentation Category: Translational Research & Late-Breaking Science Posters (LB-35 to LB-62) Immusoft is developing B cells as biofactories for therapeutic protein delivery. This new modality has the potential to be both durable and redosable. The Company's lead drug candidate, ISP-001, is the first ever engineered B cell in a human clinical trial and a first-in-class investigational treatment for mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disease. ISP-001 is an autologous B cell product engineered to express human alpha-L-iduronidase (IDUA) for the treatment of MPS I. In this first-in-human clinical trial, the patient was dosed without the need for a preconditioning regimen (required for gene-modified stem cells) or immunosuppression (required for most systemic virus-delivered gene therapy), both of which can be associated with severe toxicities. There is a significant unmet need for new therapies with improved efficacy and convenience in MPS I, where the current standard of care is enzyme replacement therapy (ERT) or hematopoietic stem cell transplant (HSCT). Due to their capacity for high-level protein production, their natural ability to engraft in the bone marrow without the need for toxic preconditioning, and their ability to manufacture a multitude of protein types, B cells have the potential to address unmet needs across numerous therapeutic indications. About Immusoft Immusoft is a cell therapy company focused on developing novel approaches to treat rare diseases by sustained delivery of protein therapeutics from a patient's own B cells. The company is developing a technology platform called Immune System Programming (ISP™), which modifies a patient's B cells and instructs the cells to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients for many years. The company is based in Seattle, WA. For more information, visit Contact Media
Yahoo
27-01-2025
- Health
- Yahoo
Immusoft to Present Positive Data from the First Engineered B Cell in a Human Clinical Trial at the 21st Annual WorldSymposium™ 2025
Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system. Results from a first-in-human clinical trial in subjects with MPS I SEATTLE, Jan. 27, 2025 /PRNewswire/ -- Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming WORLDSymposium™ 2025, held in San Diego, February 3-7, 2025. Immusoft will present the following: Oral Presentation: Friday, February 7th, 9:00 AM PST, Dr. Paul Orchard, MD. Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I Presenter: Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School Poster Presentation: Wednesday, February 5th, 2026, 3:30-5:30 PM PST, Kiosk 27-B. Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I Presentation Category: Translational Research & Late-Breaking Science Posters (LB-35 to LB-62) Immusoft is developing B cells as biofactories for therapeutic protein delivery. This new modality has the potential to be both durable and redosable. The Company's lead drug candidate, ISP-001, is the first ever engineered B cell in a human clinical trial and a first-in-class investigational treatment for mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disease. ISP-001 is an autologous B cell product engineered to express human alpha-L-iduronidase (IDUA) for the treatment of MPS I. In this first-in-human clinical trial, the patient was dosed without the need for a preconditioning regimen (required for gene-modified stem cells) or immunosuppression (required for most systemic virus-delivered gene therapy), both of which can be associated with severe toxicities. There is a significant unmet need for new therapies with improved efficacy and convenience in MPS I, where the current standard of care is enzyme replacement therapy (ERT) or hematopoietic stem cell transplant (HSCT). Due to their capacity for high-level protein production, their natural ability to engraft in the bone marrow without the need for toxic preconditioning, and their ability to manufacture a multitude of protein types, B cells have the potential to address unmet needs across numerous therapeutic indications. Additional information regarding the WORLDSymposium™ presentation can be found at: About Immusoft Immusoft is a cell therapy company focused on developing novel approaches to treat rare diseases by sustained delivery of protein therapeutics from a patient's own B cells. The company is developing a technology platform called Immune System Programming (ISP™), which modifies a patient's B cells and instructs the cells to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients for many years. The company is based in Seattle, WA. For more information, visit Contact MediaSusan RobertsRoberts Communications202-779-0929sr@ View original content to download multimedia: SOURCE Immusoft Sign in to access your portfolio
