News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, ...

AviadoBio has opened the multi-centre Phase I/II ASPIRE-FTD trial in the UK, aimed at assessing the gene therapy candidate ...

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to ...

AGC Biologics has collaborated with Quell Therapeutics to advance the development of several T-regulatory (Treg) cell therapy ...

Lexeo Therapeutics, Inc.’s LXEO share price has surged by 17.04%, which has investors questioning if this is right time to ...

Learn more about whether BioCryst Pharmaceuticals, Inc. or Ultragenyx Pharmaceutical Inc. is a better investment based on ...

The "Biopharmaceutical Market Opportunity, Growth Drivers, Industry Trend Analysis, and Forecast 2025-2034" report has been added to ResearchAndMarkets.com's offering.The Global Biopharmaceutical ...

Although comprehensive genomic profiling (CGP) expands access to clinical trials ... and a mere 9.4% actually transition to that therapy. 1 Trials targeting rare genetic mutations face even ...

The baby, KJ Muldoon of Clifton Heights, Penn., is one of 350 million people worldwide with rare diseases, most of which are ...

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the ...

Ask scientists which gene-editing tool is most needed to advance gene therapy, and they'd probably describe a system that's ...