The biotech company Verve Therapeutics launched the study with the aim of using base editing to treat a genetic condition that causes high cholesterol and increases a person's risk of developing cardiovascular disease.
This year’s crop of winning products features many with a clinical focus and others that represent significant advances in sequencing, single-cell analysis, and more.
The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.
Danielle Gerhard, PhD | Mar 22, 2024 | 10+ min read
With the help of directed evolution, scientists inch closer to developing viral vectors that can cross the human blood-brain barrier to deliver gene therapy.