The Latest Developments on the Gene Therapy Frontier
In this Trendline, dive into the evolution of gene therapy as well as the most recent clinical breakthroughs.
Gene therapy is once again at the forefront of biomedical research, catalyzed by advances in safer delivery of genes to cells. Science may also move quickly past gene replacement therapy to gene editing via CRISPR and other methods, an approach in which the unknowns are even greater and clinical results are just beginning to emerge.
Included in this Trendline:
- Pfizer hemophilia gene therapy arrives in US to uncertain future
- Orchard sets out to sell world’s priciest gene therapy
- New CMS pilot to test payment scheme for pricey sickle cell gene therapies
