And, despite the pandemic, 2020 was a landmark year for the 24-year-old, from County Durham, who became the first person in the UK to receive a revolutionary new gene therapy on the NHS.
Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an ...
The new father was born with retinitis pigmentosa (RP) and knew even from a young age that it would mean a total loss of vision.
The gene therapy comes seven years after another research group at the university helped come up with the first FDA approved ...
Student Zoe Davis, 20, was just weeks into her junior year when she landed back in the hospital with severe sickle cell pain ...
This story first appeared in Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. The deep financial restructuring ...
CRISPR-Cas is used broadly in research and medicine to edit, insert, delete or regulate genes in organisms. TnpB is an ...
Swiss-based DiNAQOR has developed a revolutionary gene therapy platform which combines gene-vector-promoter constructs with loco-regional delivery to the heart for the treatment of cardiovascular ...
BridgeBio slashes gene therapy budget after early-phase data fall short of 'transformational' target
BridgeBio Pharma is slashing its gene therapy budget and pulling back from the modality after seeing the results of a phase 1/2 clinical trial. CEO Neil Kumar, Ph.D., said the data “are not yet ...
The Asia-Pacific gene therapy market was valued at $349.1 million in 2020 and is expected to grow at a compound annual growth rate (CAGR) of 36.8% to reach nearly $7 billion in 2030. This ...
Soo-Kyung Lee hoped to change that through gene therapy. However, some had doubts that because of FOXG1’s role in brain development in utero, administering a therapy after birth may not have a ...
Unfortunately, gene therapy is not this simple. One major obstacle facing effective gene therapy involves finding the best way to deliver therapeutic genes into target cells within the body.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback