After receiving an intraocular injection of the gene for a light-sensitive protein, a 58-year-old man diagnosed with the neurodegenerative eye disease retinitis pigmentosa was able to locate objects on a table using engineered goggles.

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.

Immunization had up to 77 percent efficacy in a Phase 2 clinical study of 450 children in Burkina Faso.

Many major biopharmaceutical companies are developing or acquiring drugs that target the NLRP3 inflammasome, a large intracellular complex that researchers say can spark inflammation and stoke diseases of lifestyle and aging.

Following on the success of CAR T cells used to treat cancers of the blood, researchers have launched a Phase 1 clinical trial of genetically modified macrophages to target solid tumors.

The vaccine was 79 percent effective at blocking symptomatic infections, according to data from a Phase 3 trial in the US, Chile, and Peru. A US safety oversight board says the data might be incomplete.

The cancer researcher and executive vice president of AstraZeneca’s oncology research and development is well known for his role in the development of pivotal breast cancer therapies.

Patients who received the drug fared better cognitively and functionally than those taking placebo, but still experienced losses in performance.

Mix-and-match shots could simplify vaccine rollout and stimulate more-robust immune responses. Ongoing clinical trials will soon give answers.

The decision adds to evidence that the benefits of plasma infusions from COVID-19 survivors may be limited, but advocates say the case isn’t closed.

Geeta Swamy of Duke University School of Medicine speaks with The Scientist about starting what she considers a long-overdue clinical study of COVID-19 vaccines in pregnant women.

Two out of three people who received noninvasive ultrasound appear to have gained some level of consciousness, according to preliminary trial results.

Few peer-reviewed clinical papers on the pandemic contained original data, and many of those that did had poor experimental design.

From new treatments to an investigation into the virus’s origins, here are some of the developments we can expect this year.

The world was rocked by the COVID-19 pandemic this year, but researchers rose to all manner of challenges.

It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye.

The Past, Present, and Future of Gene Therapy: How to Scale-up Successfully

The third FDA approval of an RNAi-based therapeutic marks the growing success of a technique that took two decades to get off the ground.

As COVID-19 therapies get emergency-use green lights, the Biden administration must organize a therapeutic review board to help identify what’s working and what’s not.