At the 66th American Society of Hematology Annual Meeting, a plethora of companies presented clinical trial data highlighting their drugs targeting Bruton tyrosine kinase (BTK) in patients with blood cancers.

Agios Pharmaceuticals Inc. is learning the hard way the downside of having a pipeline in a product. When a side effect crops up, investors are likely to worry that it may affect the potential of the drug in the numerous diseases the drug could potentially treat.

Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder, according to Jun R. Huh, professor of immunology at Harvard Medical School.

At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.

Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.

Advertisements for Rezdiffra (resmetirom, Madrigal Pharmaceuticals Inc.), which was approved by the U.S. FDA in March 2024, adorned the lobby of The Liver Meeting 2024 being held at the San Diego Convention Center as well as the trolley stop across the street and other areas that doctors attending the meeting might be swayed. But inside the ballrooms of the convention center, companies were making presentations of data from clinical trials testing their drugs in patients with metabolic dysfunction-associated steatohepatitis (MASH) in hopes of potentially competing with Rezdiffra in a few years.

Patients infected with hepatitis C have had the ability to rid their livers of the virus for some time, while patients with chronic hepatitis B virus infection have been required to take medications for the rest of their lives in the hopes of just dampening damage to the liver caused by the virus. But, at The Liver Meeting 2024, Arbutus presented data from the phase IIa Im-prove study suggesting a cure might be on its way with its DNAi drug, which binds to the viral mRNA promoting its cutting, leading to loss of translation of the viral proteins.

“I think elections are like pregnancy. … Everyone puts all of the energy into D-day – the birth. We’ve had the gender reveal, but what really, really matters is what happens now and the path ahead.” That was the instant response of Emma Walmsley, CEO of GSK plc, reacting to breaking news from the U.S. that Donald Trump has won a second term in office.

Celltrion Inc. posted preclinical study results of two new antibody-drug conjugate (ADC) candidates – CT-P70 and CT-P71 – at the World ADC 2024 conference in San Diego Nov. 6, with plans to move the assets into clinical trials. Poster presentations of both ADC candidates “drew significant attention from the attendees” at the oncology meeting, Incheon, South Korea-based Celltrion said, while highlighting its efforts to transition from a biosimilar maker to a novel therapy developer.