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Denali Therapeutics Announces Key Anticipated 2023 Milestones for Its Therapeutic Portfolio for Neurodegeneration and Lysosomal Storage Diseases

Four programs expected to progress in late-stage clinical studies for MPS II (Hunter syndrome), ALS, and Parkinson’s diseaseFurther validation and expansion

articleDenali Therapeutics Inc.January 9, 20233/company/denali-therapeutics-inc/news/denali-therapeutics-announces-key-anticipated-2023-milestones-for-its-therapeutic
Denali Therapeutics Announces Key Anticipated 2023 Milestones for Its Therapeutic Portfolio for Neurodegeneration and Lysosomal Storage Diseases

About this update from Denali Therapeutics Inc.

[{"type":"text","content":"Four programs expected to progress in late-stage clinical studies for MPS II (Hunter syndrome), ALS, and Parkinson’s diseaseFurther validation and expansion of Transport Vehicle (TV) technology, including multiple interim clinical data readouts, submission of IND for additional ETV program, and selection of five Oligonucleotide TV (OTV) targetsContinued invention and scientific insights in neurodegeneration biology fuel broad early-stage pipeline of therapeutic candidates SOUTH SAN FRANCISCO, Calif., Jan. 09, 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 neurodegenerative diseases and lysosomal storage diseases, today announced program progress and expected milestones for 2023, which Chief Executive Officer, Ryan Watts, Ph.D., will highlight during a corporate presentation at the 41st Annual J.P. Morgan Healthcare Conference on Tuesday, January 10th, at 10:30 a.m. Pacific Time. “We are excited to kick off 2023 with a broad and diversified therapeutic portfolio of seven programs in clinical development, anticipated progression and expansion of our TV blood-brain barrier platform technology, and a highly productive discovery organization working to capture and translate the potential of new scientific insights in neurodegeneration biology,\" said Dr. Watts. “With four programs expected to be in late-stage development this year, we continue to build commercial capabilities and expand our global footprint to ultimately deliver effective medicines to people living with neurodegenerative and lysosomal storage diseases.\" Denali’s 2023 Outlook Denali’s therapeutic portfolio includes small molecules designed to cross the BBB and biotherapeutics that are enabled to cross the BBB using Denali’s TV technology. Expected progress and key milestones in 2023 across Denali’s therapeutic portfolio are summarized below. TV-ENABLED PROGRAMS DNL310 (ETV:IDS): MPS II (Hunter syndrome) DNL310 is an investigational, Enzyme Transport Vehicle (ETV)-enabled, brain-penetrant iduronate-2-sulfatase (IDS) replacement therapy designed to address the cognitive, behavioral, and physical manifestations of MPS II (Hunter syndrome). Delivery of the IDS enzyme to the brain addresses a significant unmet therapeutic ...

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