Clinical trial for antisense oligonucleotide therapy in a patient with ADLD

JumpstartGrant Assistance Program2023ADLD
Written By Million Dollar Bike Ride

Awardee: Margot Cousin

Institution: Mayo Clinic

Grant Amount: $50,000

Funding Period: May 1, 2023 - April 30, 2024

Summary: The long-term research goal is to advance disruptive innovation to transform care for individuals with ADLD through the development of a translational therapeutics program using team science. We hypothesize that a gapmer ASO to knockdown LMNB1 expression will be safe and well tolerated and that it will ultimately improve clinical outcomes in patients with ADLD. The objectives in this application are to develop and execute a first-in-human clinical trial to determine safety, tolerability, and potential clinical benefit of an LMNB1-targeted ASO therapy in a single patient with ADLD.

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Development of 3D ADLD microfiber and organoid models for studying biomarkers and drug testing