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.

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, on Sunday April 14 presented results from the 18-month Phase Ib clinical trial for AB-1005, an investigational gene therapy for treating patients with Parkinson's disease (PD).(1,2) The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA. The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen.

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.