It’s only been nine years since Kevan Shokat’s group at the University of California, San Francisco published the seminal paper breaking the “undruggable” spell thought to ensnare KRAS, one of the most common oncogenes. In less than a decade — breakneck speed by drug development standards — the space went from utterly barren to having one approved drug, plus a close rival ready to duke it out.

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Researchers use adeno-associated viruses (AAVs) as vehicles to deliver CRISPR/Cas systems, correct gene copies and DNA and RNA sequences into human cells as treatment modalities. But the size of the viruses themselves has been a limiting factor for cargo that can be stuffed into it. For instance, gene-editing technologies such as CRISPR/Cas and base editors must be divided into two adenoviruses.

Now, for the first time, scientists have engineered a way to shrink base editors, allowing them to be loaded into a single virus particle. David Liu, from MIT and Harvard’s Broad Institute, led the research and published a paper Thursday in Nature Biomedical Engineering.

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Just over a month after it scored an approval for a next-gen RNAi therapy for hATTR amyloidosis, Alnylam has unveiled a bit more on where it plans to go next.

The Cambridge, MA-based biopharma announced Wednesday morning that it would be putting its RNAi technology toward some much, much broader indications — cardiometabolic diseases, which implicates a broad range of conditions, such as heart disease, diabetes and stroke, that collectively are the leading cause of death in the world.

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Scientists have long poked at tumor collagen. In some cases, collagen helped keep a tumor at bay. In others, it protected a tumor from T cells. One 2013 paper even described collagen as a “double-edged sword” when it came to cancer.

In a study published in Cancer Cell today, Raghu Kalluri of the University of Texas MD Anderson Cancer Center and colleagues illustrate how pancreatic cancer cells make their own unique type of collagen — one that not only protects them from the immune system, but also changes the very microbiome of the tumor itself. By drugging this unique cancer collagen, scientists hope to improve the effectiveness of cancer therapies such as checkpoint inhibitors, which is especially important for pancreatic cancers, where checkpoint inhibitors have had middling results.

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ALS is a debilitating, universally fatal disease. As motor neurons die, patients lose their abilities to walk, cut their own food, swallow and eventually, breathe. Most patients die within three to five years of symptom onset, and there are few approved treatments that only modestly impact function and survival.

Though patients and advocates have rallied around an experimental drug from Amylyx Pharmaceuticals, currently being reviewed by the FDA, they continue to emphasize how more work needs to be done. Research into ALS pathology remains scant relative to other fields, as scientists have yet to discover a confirmed biomarker that measures patients’ progress and have only identified a handful of genetic targets implicated in the disease.

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While RNAi and Cas13 have been used as some of the early methods for gene silencing, a new preprint from top researchers says there might be something better coming down the pike.

A group of UC Berkeley researchers, comprising famed biochemist and Nobel Prize winner Jennifer Doudna and her lab members David Colognori and Marena Trinidad, has described a new method that can provide potentially safer and more efficient gene editing: CRISPR-Csm.

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