Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) highlight a potential best-in-class profile--The AATD ...
CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform.
Alpha-1 antitrypsin deficiency (AATD) is a hereditary condition characterised by the misfolding of the alpha-1 antitrypsin (AAT) protein, resulting in its hepatic accumulation and compromised protease ...
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Rescued by fat bubbles: UTSW scientists treat rare genetic disease with designer molecule
DALLAS – Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency — a promising leap toward treating humans with this ...
With 17 Patients Dosed in the Phase 1/2 Trial, BEAM-302 Continues to Demonstrate Durable Correction of the Disease-causing Mutation, Restoration of AAT Physiology, and a Well Tolerated Safety Profile ...
CRISPR Therapeutics (CRSP) announced the acceptance of an abstract for oral presentation at the European Society of Gene and Cell Therapy, ESGCT, ...
Korro Bio, Inc., a biopharmaceutical company focused on RNA editing therapies, announced that the European Medicines Agency (EMA) has granted orphan drug designation for its investigational drug ...
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