A patient with transthyretin amyloidosis (ATTR) who received the investigational gene therapy nexiguran ziclumeran (nex-z) in ...

Learn more about whether BioMarin Pharmaceutical Inc. or CRISPR Therapeutics AG is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Genetic engineers can design and assemble sophisticated gene circuits to program cells with new functions, but important ...

Once a drug target is identified, it often takes decades until a chemical compound is developed that can hit the bullseye and ...

The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred.

Technology opens the door to more accurate preclinical testing, which could enable the development of safer and more effective therapies.