Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for ...

Ultragenyx (RARE) announced that it has completed the rolling submission of its biologics license application to the FDA, seeking approval for ...

Serum CXCL6 was higher in ANCA-associated vasculitis with interstitial lung disease and independently predicted ILD ...

Adeno-associated viruses (AAV) have emerged as pivotal delivery vehicles for gene therapy due to their overall safety (less immunogenic), efficiency (broad tropism), and long-term gene expression.

Gene therapy is a promising technique for the treatment of a wide range of diseases at the genetic level, addressing the root cause of many subsequent disorders. Recent advancements have facilitated ...

A Navy investigation into the deadly sinking of a Marine Corps amphibious assault vehicle last year found leadership confusion, poor communications and gaps in training. But ultimately the service ...

After decades of research, gene therapy is finally on track to deliver solutions to myriad diseases once considered untreatable. More than 400 gene therapy trials are currently underway in the United ...

Several quality attributes (QAs) must be monitored to ensure an effective and safe final product when developing adeno-associated virus (AAV) vectors as drug products. Thermal stability, aggregate ...

Below is our list of the 32 largest contracts in MLB history by average annual value (AAV). Please note that if a player was already under contract and signed an extension, only the new money counts.