From cancer biology to proactive health management, customized care is changing patient outcomes, but what are the costs and ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

Gene therapies are unlocking new drug targets that were previously thought undruggable. Covering modalities like ASOs, siRNA, saRNA, mRNA, and ADARs, each with different mechanisms of action, gene ...

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk. When this type of “genetic surgery” is performed on DNA, for ...

Being proactive — especially when it comes to testing, process development and preparing for commercialization — can greatly decrease the risk that your CGT will receive a CRL.

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development and manufacturing. Three major gene therapy approaches to address ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced today that it will present non- ...