After long years of painstaking work, the commercialization of cell and gene therapies picked up pace in 2022, with multiple approvals. More progress is expected in 2023, with several firsts in the offing and products for larger patient populations reaching the market.
CRISPR, or clustered regularly interspaced palindromic repeats, is transforming biomedical research, and making rapid inroads into the clinic, with its ability to easily target specific DNA and RNA sequences. CRISPR itself is made of RNA. It recognizes target sequences and delivers CRISPR-associated (Cas) proteins, nucleases that cut the target sequence. In two papers published online in Nature on Jan. 4, 2023, researchers have demonstrated that a recently discovered type of Cas protein, Cas12a2, can degrade double-stranded DNA when its associated CRISPR guide RNA recognizes its target sequence.
CRISPR gene editing has been one of the important advances of the last decade, in biotechnology and increasingly in medicine. First applied to human cells in 2013, and honored with the 2020 Nobel Prize in Physiology or Medicine, its meteoric rise can make CRISPR look like the molecular equivalent of a miracle healer. But in the research and clinical trenches, CRISPR-based approaches, like any others, need to find applications where their desired effects outweigh their side effects. And finding those applications necessitates ways to identify off-target effects.
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.