Drug intelligence / Profile preview

AVB-601

Development stage: Preclinical
Lead developer: Solid Biosciences
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies

Overview

AVB-601 is a preclinical-stage gene therapy candidate developed by Solid Biosciences for the treatment of cardiovascular diseases, specifically cardiomyopathy. The drug utilizes a gene transference mechanism to deliver therapeutic genetic material to cardiac cells, aiming to address the underlying causes of heart muscle dysfunction. The program was part of the pipeline integrated following Solid Biosciences' merger with AavantiBio in late 2022, which focused on expanding the company's portfolio in precision genetic medicines for neuromuscular and cardiac conditions.

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