Programmable genome insertion of long DNA sequences, useful for both gene therapy and basic research, commonly relies on cellular responses to double-strand breaks (DSBs) using programmable nucleases, such as CRISPR-Cas9, for induction of repair pathways such as non-homologous end joining (NHEJ). To overcome the current limitations of gene integration approaches, scientists from the Massachusetts Institute of Technology and colleagues developed a new strategy based on advances in programmable CRISPR-based gene editing, such as prime editing, together with the application of precise site-specific integrases.
Researchers at the Walter and Eliza Hall Institute of Medical Research (WEHI) in Melbourne, Australia, have developed a new genome editing technique than can activate any gene, including those that have been silenced, allowing new drug targets and causes of drug resistance to be explored.
Researchers at the Walter and Eliza Hall Institute of Medical Research (WEHI) in Melbourne, Australia, have developed a new genome editing technique than can activate any gene, including those that have been silenced, allowing new drug targets and causes of drug resistance to be explored.
CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
Bearish investors dwelling on a single grade 4 liver enzyme elevation seemed to be the cause for Intellia Therapeutics Inc.’s sinking stock Sept. 16, despite the company reporting impressive, though early stage, data for its leading systemically administered CRISPR candidates targeting hereditary angioedema (HAE) and amyloid transthyretin (ATTR) amyloidosis.
Arsenal Biosciences Inc. closed on an oversubscribed $220 million series B financing so it could continue developing its programmable cell therapy research programs and its candidates for treating solid tumor malignancies. Arsenal’s lead program is AB-1015 for treating ovarian cancer.
As the resident innate immune cells of the brain, microglia are emerging as key drivers of neurological diseases, but as yet there is no systematic way of exploring their potential as drug targets.
Controlling the epigenetics of a patient, figuring out what genes are expressed and understanding their level of expression, is at the center of Epic Bio, a new company founded by Stanley Qi, co-inventor on the CRISPR patent held by the University of California.
Despite a huge amount of progress in the hot space CD47, there’s a large space beyond it to explore, according to DEM Biopharma Inc.’s CEO David Donabedian. The new company just raised $70 million to develop therapies targeting don’t eat me, hence DEM, and eat me signals on cancer cells and macrophages.
Spotlight Therapeutics Inc., a company developing cell-targeted in vivo CRISPR gene editing biologics, has raised $36.5 million in series B financing to support advancement of its first-in-class immuno-oncology program and further applications of its in-house technology platform. The financing round was co-led by new investors GordonMD Global Investments and Epiq Capital Group, with participation from Magnetic Ventures, as well as existing investors GV, Emerson Collective and others.