Denali Therapeutics Announces Scientific Journal Publication of Preclinical Proof of Concept for Protein Transport Vehicle PTV

Denali Therapeutics Announces Scientific Journal Publication of Preclinical Proof of Concept for Protein Transport Vehicle (PTV)


Pre-clinical research published in Cell highlights new approach to treat FTD-GRN with enhanced brain uptake of progranulin through use of Denali’s Transport Vehicle to cross the blood-brain barrier.

Biopharmaceutical company Denali Therapeutics Inc. (NASDAQ: DNLI) announced today the publication of preclinical proof of concept for using the company’s Protein Transport Vehicle (PTV).

Denali’s PTV is designed to enhance brain uptake of peripherally administered progranulin (PTV:PGRN) and the approach may have utility in treating types of frontotemporal dementia (FTD), especially FTD-GRN caused by progranulin deficiency.

San Francisco based Denali Therapeutics focuses on developing a broad portfolio of therapeutic drug candidates that are engineered and designed to cross the blood-brain barrier (BBB) for neurodegenerative diseases. 

The company pursues new treatments through rigorous assessment of genetically validated targets, engineering delivery across the blood-brain barrier and guiding development through biomarkers that demonstrate pathway and target engagement. 

The preclinical data was published Thursday, August 26th digitally by Cell ahead of the print issue on September 2nd.

You can read the full data report here: Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.

Denali’s preclinical research showed that progranulin replacement therapy with Denali’s PTV:PGRN rescued both neurodegeneration and microglial dysfunction in progranulin-deficient mice. 

The research also provides new insight into the molecular and cellular mechanisms that may contribute to FTD, identifying novel roles of progranulin in lysosomal function and lipid metabolism, as well as lysosome biomarkers with potential clinical utility.

Denali Therapeutics Leadership Comment on Publication in Cell

Joseph Lewcock, Ph.D., Chief Scientific Officer: “This preclinical research demonstrates that our Protein Transport Vehicle can enhance the uptake of peripherally administered progranulin by multiple cell types in the brain, including neurons and microglia. In addition, the improved mechanistic understanding of progranulin’s role in lysosomal function indicates that our therapeutic strategy with PTV:PGRN may be the most direct and effective way to increase progranulin levels in lysosomes for the potential treatment of people with FTD-GRN.”

Ryan Watts, Ph.D., Chief Executive Officer (Denali Therapeutics CEO): “Publication of this research in Cell marks a significant milestone in the development of therapeutics enabled by our Transport Vehicle technology. We are making great progress towards our goal of initiating clinical testing of our lead PTV:PGRN molecule (DNL593) and believe that our unique brain-penetrant progranulin replacement approach has the potential to make a difference for individuals and their families affected by FTD-GRN.”

Denali Therapeutics Leadership Team

About Cell Press

Cell Press is a publisher of more than 50 scientific journals that span health sciences, life sciences, physical sciences, and earth sciences. The publisher works in conjunction with partners at scientific societies along with independent publication support. Today, the company works to bring editorial excellence, commitment to innovation, unparalleled reach and visibility, and passion for advocacy to all areas of scientific exploration as we work to publish and share science that inspires.

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