Bioinformatics combined with stable-isotope labeling helps identify orphan gene products in bacteria. A major challenge of the genomic era is how best to use the immense and growing body of ...

Shares of gene therapy developer uniQure N.V. (QURE) shot up 41% on Thursday afternoon after it announced that the U.S. Food ...

largely because many of these single-gene diseases are rare and occur in small numbers of families (Antonarakis & Beckmann, 2006). Referred to as "orphan" diseases, these relatively uncommon ...

and challenges for Launch Excellence The relative strength and prognosis for the developed and the emerging pharmaceutical markets Orphan drugs launch challenges Cell and Gene therapies ...

CAMBRIDGE, Mass., March 25, 2025 /PRNewswire/ -- AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD ...

We recently compiled a list of the 10 Best Gene Therapy Stocks to Buy Right Now. In this article, we are going to take a look at where bluebird bio, Inc. (NASDAQ:BLUE) stands against the other gene ...