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David Liu (Casey Atkins Photography courtesy Broad Institute)

In­tel­lia an­nounced a new tool for gene edit­ing. Aca­d­e­mics said they rewrote his­to­ry

David Liu, the Harvard biochemist, was sitting in a meeting last week when his phone started buzzing repeatedly.

Unbeknownst to him, minutes prior the CRISPR company Intellia had finished unveiling their approach to base editing at a Cold Spring Harbor Lab conference. First pioneered by Liu and his lab in 2016, the method allows you to change individual DNA bases without breaking the double helix, an advance that could prove critical for treating a long list of cancers and genetic diseases.

Wild Biotech co-founder Neta Raab

Two Church lab vets, a se­cre­tive in­sti­tute and an Is­raeli bil­lion­aire hunt for drugs in the guts of wild an­i­mals

Over the last four years, Neta Raab and Ido Bachelet have received hundreds of padded cardboard boxes containing frozen poop from wild animals on five different continents.

The packages came regularly to their lab outside Tel Aviv, sent from a squad of zoologists and specialized Israeli army veterans who tracked enough animals to fill a Rudyard Kipling novel, rushing to collect vulture and rhino and frog samples within an hour of extrusion. Raab and Bachelet carefully opened the boxes, removing the samples from plastic-wrapped tubes and then using specialized magnetic beads to fish out the bacterial DNA inside for sequencing.

Neil King (University of Washington)

Is a 'su­per-sea­son­al' flu vac­cine on the hori­zon? The NIH and UW are tak­ing a shot in­to the clin­ic this April

Several biotechs and pharma companies are looking to discover a universal flu vaccine that could protect humans from any strain of the virus, regardless of which is predicted to circulate. But given how many of these efforts are likely years away at best, are there any intermediate steps available to bridge that gap?

That’s the question University of Washington researcher Neil King and a group of NIH scientists are hoping to answer. In a new Nature paper published Wednesday, the team was able to demonstrate broad effectiveness in animal models for a “super-seasonal” flu vaccine by displaying multiple flu strains at the same time. And King says it’s starting human trials at the end of April.

Mon­tre­al AI start­up with a tout­ed ad­vi­sor li­cens­es its 'few-shot' plat­form to Re­pare for syn­thet­ic lethal­i­ty mol­e­cules

The rise of AI in drug discovery has presented a buffet of tasty options for drugmakers to identify better molecules for hard-to-hit targets. That promise is mixed, to say the least, but startups like Montreal’s Valence Discovery think they’ve cracked the code — and at least one small biotech is convinced.

Valence, a recently uncloaked AI startup specializing in deep learning in drug discovery, has licensed its tech to Canada’s Repare Therapeutics to identify effective synthetic lethality molecules, the companies said Wednesday.

Ana Moreno and Prashant Mali

Can a CRISPR start­up suc­ceed where Pfiz­er, Glax­o­SmithK­line, Bio­gen and Genen­tech failed and cure chron­ic pain?

A few years ago, when Ana Moreno was doing her PhD work at the San Diego lab of one of the early CRISPR gene editing researchers, she came across a paper that made national headlines a decade prior.

Researchers in the UK followed up on stories of a Pakistani boy who could walk on coals and pass knives through his arms and determined that rare mutations in one gene, called Nav1.7, made him and several relatives unable to feel pain. The finding, the New York Times reported, raises “hopes of developing novel drugs that would abolish pain by blocking the gene’s function.”

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Lil­ly diss­es Am­gen and Mi­rati as it de­buts new KRAS mol­e­cule

Eli Lilly is rejoining the KRAS hunt.

The Indianapolis Big Pharma revealed Thursday, in an abstract for AACR, that it has been working on a new small molecule to target the infamous oncogene and are planning to put it into Phase I later this year. It marks Lilly’s first public foray into the field in nearly a year, since it ditched its first molecule after seeing dangerous side effects in five patients.

In race for in vi­vo sick­le cell cure, In­tel­lia un­veils (very) ear­ly proof-of-con­cept

Vertex and CRISPR Therapeutics have shown they can use the gene editing tool to cure, for at least a couple years, a handful of patients with sickle cell disease and are now entering late-stage development.

It’s a major advance, but it comes with the same caveats that have plagued other gene therapies for sickle cell. You have to give patients what amounts to a bone marrow transplant: a laborious, expensive and occasionally risky procedure that involves removing stem cells from their marrow, editing them in a facility and then re-injecting them. Any company that could find a way to edit cells with a simple IV infusion could make those older approaches obsolete.

David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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Scoop: Deer­field and Dana-Far­ber forge clos­er ties to launch a new trans­la­tion­al can­cer re­search en­gine

A little over two years since first teaming up on protein degradation, Deerfield and Dana-Farber are putting their heads — and their wallets — together again.

The pair is forming a new partnership in the form of Riverway Discoveries, a translational research engine aiming to boost efforts at getting preclinical cancer work into human studies. Deerfield is putting up $130 million over the next 10 years to advance such endeavors, after funding the protein degradation effort for $80 million in late 2018.

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Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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