By exploiting the technology used in COVID-19 vaccines, a team led by UCL, King's College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology’s potential therapeutic use in people.

The research, published in Science Translational Medicine, found that messenger RNA (mRNA) could be used to correct a rare liver genetic disease known as argininosuccinic aciduria in a mouse model of the disease.

Pfizer Inc. (NYSE: PFE) and Genmab A/S (Nasdaq: GMAB) announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) seeking to convert the accelerated approval of TIVDAK® (tisotumab vedotin-tftv) to full approval, for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after first-line therapy.

International research led by the Translational Synthetic Biology Laboratory of the Department of Medicine and Life Sciences (MELIS) at Pompeu Fabra University has succeeded in efficiently engineering Cutibacterium acnes -a type of skin bacterium- to produce and secrete a therapeutic molecule suitable for treating acne symptoms. The engineered bacterium has been validated in skin cell lines and its delivery has been validated in mice.

Amino acids, such as tyrosine and tryptophan, are the fundamental building blocks that make up proteins. These biomolecules have different chemical groups on each end and side chain, and so, have the natural ability to form a chain through the formation of an amide (peptide) bond. However, such linkages are weak and easily degraded under physiological conditions.

In a scientific breakthrough that aids our understanding of the internal wiring of immune cells, researchers at Monash University in Australia have cracked the code behind IKAROS, an essential protein for immune cell development and protection against pathogens and cancer.

This disruptive research, led by the eminent Professor Nicholas Huntington of Monash University's Biomedicine Discovery Institute, is poised to reshape our comprehension of gene control networks and its impact on everything from eye colour to cancer susceptibility and design of novel therapies.

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson's disease (PD).

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Could healthy fats found in nuts and fish slow the progression of potentially deadly lung scarring known as pulmonary fibrosis and delay the need for lung transplants?

UVA pulmonary researchers looked at the association between blood-plasma levels of omega-3 fatty acids - the heart-healthy fats found in foods such as salmon and flaxseeds - and the progression of pulmonary fibrosis, as well as how long patients could go without needing a transplant.