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FDA Regulation On Gene Therapy | The Brainstorm EP 96
ARK Invest· 2025-08-06 20:30
Welcome to the brainstorm episode 96, closing into our final 100th episode where Jake and I will make the determination whether to continue on or wrap it all up unless we get enough funny comments. So, please com comment away. We've got Shay Wilberg with us from our genomics team.Shay's first time joining us. Shay, do you want to give a a quick intro. >> Sure.So Shay Wilberg, I recently joined here at Arc Invest as a multiomics analyst on the team. Before this, I was working over on the cell side over at Ba ...
Duchenne Muscular Dystrophy (DMD) Pipeline Insight Report 2025, Featuring Analysis of Vamorolone (Santhera), Givinostat (Italfarmaco), and Pamrevlumab (Fibrogen)
GlobeNewswire News Room· 2025-05-08 08:24
Core Insights - The report titled "Duchenne Muscular Dystrophy - Pipeline Insight, 2025" provides a comprehensive overview of the current landscape of Duchenne Muscular Dystrophy (DMD) therapies, focusing on over 75 companies and pipeline drugs [1][2] Pipeline Overview - The report includes detailed profiles of pipeline drugs, covering both clinical and nonclinical stages, and assesses therapeutics by product type, stage, route of administration, and molecule type [2][10] - Approximately 75+ key companies are involved in developing therapies for DMD, with Roche having candidates in the most advanced stage, i.e., Phase III [7] Emerging Drugs - Vamorolone, developed by Santhera, is a first-in-class drug candidate that aims to dissociate efficacy from typical steroid safety concerns, addressing the unmet medical need in DMD patients [4] - Givinostat, an HDAC inhibitor from Italfarmaco, targets pathogenetic events downstream of genetic defects, potentially benefiting the entire DMD and BMD population [5] - Pamrevlumab, developed by FibroGen, is a first-in-class antibody that inhibits connective tissue growth factor (CTGF) and is currently in Phase 2 trials for DMD [6] Therapeutic Assessment - The report categorizes DMD drugs based on various parameters, including route of administration and molecule type, providing insights into the therapeutic landscape [10][11] - The pipeline includes drugs at different phases of clinical development: late-stage (Phase III), mid-stage (Phase II), early-stage (Phase I), as well as preclinical and discovery stage candidates [8] Development Activities - The report highlights collaborations, acquisitions, mergers, and licensing activities related to DMD therapeutics, indicating a dynamic development environment [12] - Key players in the industry include Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd., and FibroGen, among others [15][18]