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.

A new wave of research is recruiting patients and other members of the public to serve as equal partners, bringing fresh perspectives to research on diseases and other conditions.

First, the genetically engineered cells became CD8⁺ killer T cells that wiped out his leukemia. Then they transformed into a stable population of CD4⁺ helper T cells that continue to circulate in his body.

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.

Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.

Now more than ever before, recruiting patients for a research study doesn’t have to mean getting them to leave their homes.

The company voluntarily paused its studies, including one in Phase 3, after an unexplained illness in a patient.

Although scientists debate the ethics of deliberately infecting volunteers with SARS-CoV-2, plenty of consenting participants have been exposed to all sorts of pathogens in prior trials.

Upon seeing pregnant women sick with COVID-19 at a University of Pennsylvania hospital, researchers there wrote trial protocols for blood transfusions to treat the disease that include expecting mothers.

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

YODA, a program facilitated by Yale University researchers, has successfully distributed clinical trial records from Johnson & Johnson and Medtronic to external researchers since 2013.

OpenTrials.net seeks to increase transparency and make clinical research more accessible to the public.

Thoughts on how to redesign clinical trials for traumatic brain injury

Scientists identify a human leukocyte antigen gene linked to immune protection from HIV following vaccination.

Enrolling the right patient population could be key to a successful clinical trial.