gene therapy
Using its CRISPR-Chip technology, the San Diego-based startup is commercializing a transistor platform to measure CRISPR-Cas performance in gene editing.
Synbio Company Replay Launches With $55M in Seed Funding
The San Diego and London-based startup aims to tackle gene therapy payload capacity and off-the-shelf cell therapies using its genome medicine technologies.
Dilated Cardiomyopathy Gene Mutations Point to Possible Therapeutic Strategy
Homozygous loss-of-function mutations in the BAG5 gene turned up in a subset of inherited DCM patients, and BAG5 appeared amenable to gene therapy in mouse models.
Investigational CRISPR Therapy Shows Efficacy Against Transthyretin Amyloidosis in Phase I Study
The therapy works by reducing serum concentration of misfolded transthyretin protein, which leads to the life-threatening disease.
Mission Bio Seeks to Increase Drug Trial Collaborations, Improve Gene and Cell Therapy Efficacy
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Mission Bio will use a recent $70 million funding round to develop quality control assays using its Tapestri single-cell analysis platform.