The world’s largest and most comprehensive repository of somatic mutations found in cancer is celebrating its 20th birthday by adding 307,000 new genomic variants to its 24 million-strong database. With somatic mutations being the main drivers of tumor development, the Catalogue of Somatic Mutations in Cancer (COSMIC) is firmly established as a source of potential drug targets, for the discovery and validation of biomarkers associated with prognosis, treatment response and patient outcomes, and as a resource for characterizing the mutational landscape of individual tumors.

Cancervax Inc. has announced that its University of California, Los Angeles (UCLA) research team has achieved a critical milestone by creating a process to add any antibody to lipid nanoparticles (LNPs) using click chemistry, for targeting cancer cells.

Totus Medicines Inc. has entered into a multi-target research collaboration with Eli Lilly & Co. to discover small-molecule drug candidates against undisclosed targets in different therapeutic areas.

The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.

Neosphere Biotechnologies GmbH has entered into a collaboration with Kymera Therapeutics Inc. that seeks to unlock undrugged or poorly drugged disease-causing protein targets that can be addressed by targeted protein degradation.

Researchers from the University of Florida have developed a novel mRNA cancer vaccine that aims to reprogram the immune system to target and attack cancer cells effectively. The study led by Elias Sayour reported a new mRNA vaccine design where the mRNA payload was wrapped into a multilamellar vesicle within an onion-like hierarchical structure. The findings were published in the May 9, 2024 issue of Cell.

Avicenna Biosciences Inc. has introduced an extension to its machine learning (ML) technology platform to enhance medicinal chemistry and expedite clinical-stage drug discovery.

Nanite Inc. has been awarded a $1.8 million grant by the Bill & Melinda Gates Foundation to design and optimize polymeric delivery vehicles to deliver DNA-encoded therapeutics.

“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”

Japanese researchers have transplanted human induced pluripotent stem cells (iPSCs) in a primate model of myocardial infarction and were able to restore heart muscle and function in monkeys. Developed by Tokyo-based Heartseed Inc., the grafted iPSCs consist of clusters of purified heart muscle cells (cardiomyocyte spheroids) that are injected into the myocardial layer of the heart. Published in Circulation on April 26, 2024, the study showed that the cardiomyocyte spheroids survived long term and showed improved contractile function with low occurrence of post-transplant arrhythmias.