In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) - a blockage that results in lack of oxygen and painful episodes - for at least one year, and 100% were able to remain hospitalization-free for the same length of time.

Researchers have described the optimal timing for COVID-19 patients to take the antiviral, Paxlovid, to get the most benefit from the treatment, according to a study published April 16 in eLife.

The findings suggest that taking Paxlovid three to five days after COVID-19 symptoms emerge may maximise the drug's ability to reduce viral loads, minimise viral spread and reduce viral rebound.

Cancer is the monster of our society. Last year alone, more than 600,000 people in the United States died from cancer, according to the American Cancer Society. The relentless pursuit of understanding this complex disease has shaped medical progress on developing treatment procedures that are less invasive while still highly effective.

Immunotherapy is on the rise as a possible solution. Immunotherapy involves harnessing the power of the body's immune system to fight against cancer cells.

A novel approach to drug discovery is enabling University of Birmingham researchers to overcome the 'valley of death', where projects fail due to the funding gap between original research and commercial investment.

The approach, detailed in a feature published in the April issue of Drug Discovery Today, has attracted more than £4m in industry funding, grants and industry awards, on the back of just £0.2m investment from the University's Dynamic Investment Fund (DIF).

Alzheimer's disease (AD) currently afflicts nearly seven million people in the U.S. With this number expected to grow to nearly 13 million by 2050, the lack of meaningful therapies represents a major unmet medical need. Scientists at Sanford Burnham Prebys have now identified promising real-world links between common HIV drugs and a reduced incidence of AD. The study, led by Jerold Chun, M.D., Ph.D., was published in Pharmaceuticals.

Chun's new research builds on his lab's landmark publication in Nature in 2018 that described how somatic gene recombination in neurons can produce thousands of new gene variants within Alzheimer's disease brains.

Antisense oligonucleotides (ASOs) are next-generation drugs that can treat disease by blocking the transfer of harmful messages from our genes. In people with cancer, ASOs have the potential to block messages that encourage the growth and spread of the tumor. However, ASOs aren’t used for treating cancer yet. They must first get delivered inside cancer cells, but the cancer cells won’t let them in.

Finding an effective ASO delivery system is a major challenge. Cancer cells have gatekeeper molecules that stop unwanted substances from entering.

Researchers have developed a functional precision medicine approach that targets cancer by combining genetic testing with a new way to test individual drugs on tumor samples. The results of the clinical study were published today in Nature Medicine.

This combined approach, developed by Florida International University cancer researcher Diana Azzam, was used successfully for the first time to guide treatment of relapsed pediatric cancer patients in collaboration with First Ascent Biomedical and Dr. Maggie Fader at the Helen & Jacob Shaham Cancer & Blood Disorders Institute at Nicklaus Children's Hospital in Miami.