BPGbio to present novel drug target for Huntington’s disease and AI discovered biomarkers for Alzheimer’s disease at Society for Neuroscience 2024
- BPGbio will present two transformative studies at the Society for Neuroscience Annual Meeting, Neuroscience 2024, in Chicago.
- The first study focuses on a novel drug target for Huntington’s Disease, showing that modulating UBE2K activity can disrupt mutant Htt aggregation.
- The second study identifies novel liver and immune system protein biomarkers for Alzheimer’s Disease, which can predict cognitive decline.
- Both studies leverage BPGbio’s NAi Interrogative Biology Platform, highlighting its potential in discovering therapeutic targets and early-disease biomarkers.
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Maze Therapeutics initiates phase 1 trial for MZE782 in chronic kidney disease
- Maze Therapeutics has begun dosing participants in a Phase 1 clinical trial for MZE782, targeting chronic kidney disease (CKD).
- MZE782 is an oral, small molecule targeting the solute transporter SLC6A19, potentially benefiting around five million CKD patients in the U.S.
- The trial is a randomized, double-blind, placebo-controlled study in healthy volunteers, assessing safety, tolerability, pharmacokinetics, and pharmacodynamics.
- Initial results, including proof of mechanism, are expected in the second half of 2025.
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Papillon Therapeutics receives orphan drug designation from the U.S. Food and Drug Administration for PPL-002 for the treatment of Danon disease
- Papillon Therapeutics' PPL-002 has been granted Orphan Drug Designation by the FDA for treating Danon disease.
- PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy.
- The therapy aims to express functional Lamp-2 protein, which is deficient in Danon disease patients.
- The designation offers benefits like tax credits for clinical trials and waivers on prescription drug user fees.
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