In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are
currently in clinical development, gene therapy holds the promise of a lasting cure with a …

Gene therapies are gaining momentum as promising early successes in clinical studies
accumulate and examples of regulatory approval for licensing increase. Investigators are …

Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have
reported remarkable success in ameliorating disease phenotype in hemophilia A and B …

Haemophilia is a rare disease for which the approved therapeutic options have remained
virtually unchanged for 50 years. In the past decade, however, there has been an explosion …

Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …

Gene therapy is rapidly becoming a new therapeutic strategy for haemophilia A and B
treatment. In the 1990s, studies in animal models showed that adeno‐associated vectors …

While gene transfer using recombinant adeno-associated viral (rAAV) vectors has shown
success in some clinical trials, there remain many tissues that are not well transduced …

Recombinant adeno-associated virus (rAAV) vectors are a promising platform for in vivo
gene therapy. The presence of neutralizing antibodies (Nab) against AAV capsids …

The power of adeno-associated viral (AAV)-directed evolution for identifying novel vector
variants with improved properties is well established, as evidenced by numerous …

Recent clinical trials have demonstrated the potential of adeno-associated virus (AAV)-
based vectors for treating rare diseases. However, significant barriers remain for the …