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(L-R) Peter Rohweder, Kevan Shokat, Ziyang Zhang, Daniel Green, Charly Craik

The sci­en­tist who pi­o­neered KRAS drugs has a new idea about mak­ing them more pow­er­ful

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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The Doherty Institute's Sharon Lewin (Graham Hughes/The Canadian Press via AP Images)

From Aus­tralia, new $173M re­search cen­ter strives to be at the fore­front of pan­dem­ic drug dis­cov­ery

In the battle against Covid-19, vaccines came much earlier than therapeutics. A new research institution in Melbourne wants to change that.

Geoffrey Cumming, a Canadian-born oil and gas businessman, donated AUD $250 million ($173.63 million) to establish the Cumming Global Centre for Pandemic Therapeutics — whose mandate is to fund “an ambitious research program focusing on treatments that can be rapidly adapted after a new virus is identified.”

Illustration: Shutterstock

Through the lymph nodes it goes — re­searchers de­vel­op a can­cer drug that avoids tox­i­c­i­ties by skirt­ing the liv­er

PI3K is a protein that is part of a pathway that regulates cell growth, survival and metabolism — earning it the inscription of master regulator for cancer. However, while a number of PI3K inhibitor drugs have been approved since 2014, the class as a whole has dwindled, as it has been plagued by toxicity issues in various blood cancers.

For example, the FDA hit Secura Bio’s PI3K inhibitor Copiktra, which earned accelerated approval in 2018, with an increased death warning in June following the results of its confirmatory Phase III trial. That warning came after a number of companies, including Secura, Gilead and Incyte, withdrew their accelerated approvals for their PI3K inhibitors after failing to complete confirmatory trials. The FDA now requires randomized trials to be conducted for PI3K inhibitors in blood cancers.

David Liu (Broad Institute)

David Li­u's lab de­vel­ops next-gen base ed­i­tors, small enough to fit in­to sin­gle AAV

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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RNAi for com­mon dis­eases? Al­ny­lam plans to forge a path for­ward, start­ing with new obe­si­ty gene tar­get

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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Raghu Kalluri (University of Texas MD Anderson Cancer Center)

'Like a bul­let­proof vest': Can­cer cells make their own kind of col­la­gen. Can pierc­ing through make treat­ments bet­ter?

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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Alex Zhavoronkov (L) and Jeffrey Rothstein (Insilico/Johns Hopkins)

As ALS pa­tients unite around Amy­lyx, a new pa­per hints at po­ten­tial drug­gable tar­gets

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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Jennifer Doudna (Photo by Brian Ach/Getty Images for Wired)

Doud­na re­search team finds CRISPR-Csm de­liv­ery po­ten­tial­ly out­per­forms RNAi, Cas13

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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Bonnie Berger, MIT professor of mathematics (via MIT)

MIT lab cre­ates tool to find can­cer mu­ta­tion dri­vers 'any­where in the genome'

Precision oncology has been heralded as the way forward in treating cancer, but searching for driver mutations has complicated that journey as existing methods can’t be applied to the entire genome.

But a new method out of MIT looks to change the way cancer driver mutations are found and, in doing so, speed up the process. A group out of Bonnie Berger’s lab at the institution has developed what they named “Dig,” an interactive map for finding driver elements and mutations “anywhere in the genome,” according to a paper published this week in Nature Biotechnology.

David Perlin (L) and Charles Rice

'Vac­cines alone are in­suf­fi­cien­t': Mer­ck, Ali­gos work with aca­d­e­m­ic ex­perts to find Covid an­tivi­rals as part of $65M NIH grant

Merck is throwing its weight behind a new consortium of academic, nonprofit and biotech scientists to discover oral antivirals that can fight Covid-19 — and pandemic viruses of the future.

The NIH and the NIAID will provide $65 million over three years to fund the The Metropolitan AntiViral Drug Accelerator, or MAVDA, as it works to find and test small molecule drugs to target coronaviruses. With an emphasis on SARS-CoV-2 and one or more select RNA viruses with pandemic potential, the goal is to make oral treatments that can be given in an outpatient setting.