Research led by Associate Professor Duc Dong, Ph.D., has shown for the first time that the effects of Alagille syndrome, an incurable genetic disorder that affects the liver, could be reversed with a single drug. The study, published in the Proceedings of the National Academy of Sciences, has the potential to transform treatment for this rare disease and may also have implications for more common diseases.

Scientists are harnessing a new way to turn cancer cells into potent, anti-cancer agents. In the latest work from the lab of Khalid Shah, MS, PhD, at Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, investigators have developed a new cell therapy approach to eliminate established tumors and induce long-term immunity, training the immune system so that it can prevent cancer from recurring.

Numerous studies have shown health benefits of time-restricted eating including increase in life span in laboratory studies, making practices like intermittent fasting a hot topic in the wellness industry. However, exactly how it affects the body on the molecular level, and how those changes interact across multiple organ systems, has not been well understood.

Vaccination and boosting, especially when recent, helped to limit the spread of COVID-19 in California prisons during the first Omicron wave, according to an analysis by researchers at UC San Francisco that examined transmission between people living in the same cell.

An enzyme that defends human cells against viruses can help drive cancer evolution towards greater malignancy by causing myriad mutations in cancer cells, according to a study led by investigators at Weill Cornell Medicine. The finding suggests that the enzyme may be a potential target for future cancer treatments.

Pfizer Inc. (NYSE: PFE) today announced positive top-line results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B.

GSK plc (LSE/NYSE: GSK) has announced the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) authorised Sanofi's SARS-CoV-2 spike protein (B.1.351 strain) vaccine for the prevention of COVID-19 disease in adults aged 18 and above in Great Britain, following the European Commission licence last month, which included Northern Ireland.

High levels of ammonia in tumors leads to fewer T cells and immunotherapy resistance in mouse models of colorectal cancer, new findings from the University of Michigan Rogel Cancer Center revealed. Researchers found that ammonia inhibits the growth and function of T cells, which are vital for anti-tumor immunity. The findings appear in Cell Metabolism.

Mergers and acquisitions are expected to be a key theme in the pharmaceutical industry in 2023.

Pfizer Inc. (NYSE: PFE) announced that the U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) for etrasimod for individuals living with moderately-to-severely active ulcerative colitis (UC). The FDA's decision is expected in the second half of 2023. The European Medicines Agency (EMA) has also accepted the Marketing Authorization Application (MAA) for etrasimod in the same patient population with the decision anticipated in the first half of 2024.

New research from the University of Virginia School of Medicine speaks to the benefits of a COVID-19 booster.

The new findings shed light on how mRNA boosters - both Pfizer and Moderna - affect the durability of our antibodies to COVID-19. A booster, the researchers report, made for longer-lasting antibodies for all recipients, even those who have recovered from a COVID-19 infection.

Mergers and acquisitions are expected to be a key theme in the pharmaceutical industry in 2023.

The FDA's top five approvals in 2022 represent an eclectic mix of cancer, cardiovascular and rare disease drugs.

2022 featured pivotal data that could change the landscape in non-alcoholic steatohepatitis, Alzheimer’s disease, sickle cell disease and many more indications.

BioSpace spoke with several leaders to gather their thoughts and predictions for the new year. Here is the first installment of this two-part series.

See inside for BioSpace's compilation of the five biggest M&A deals of 2022.

Today, the U.S. Food and Drug Administration approved Sunlenca (lenacapavir), a new type of antiretroviral medication for adult patients living with human immunodeficiency virus type 1 (HIV-1), whose HIV infections cannot be successfully treated with other available treatments due to resistance, intolerance, or safety considerations.

Eli Lilly and ProQR Therapeutics expand their RNA-editing collaboration.

Merck entered into a license and collaboration agreement with Chinese company Kelun-Biotech to develop seven antibody-drug conjugates candidates against oncology targets.

Lexicon Pharmaceuticals’ LX9211 fell short of its primary efficacy endpoint, according to topline data from the Phase II RELIEF-PHN-1 study in post-herpetic neuralgia.