Sanofi's latest bet on a potential Dupixent sequel is a preclinical pill developed by a British small-cap that specializes in virtual reality chemistry.
READ MOREEli 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.
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.
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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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’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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Close to 70 years after Oxford scientists purified penicillin and confirmed its effect as an antibacterial drug, the university is establishing a new research institute at the forefront of combating antimicrobial resistance.
The Ineos Oxford Institute for AMR Research will initially be powered by a $136 million (£100 million) donation from Ineos, the UK-based chemicals giant founded by billionaire Jim Ratcliffe, that also plays a hand in manufacturing medical and pharma products.
Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.
Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.
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Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.
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JP Morgan isn’t typically a data conference. But there are always exceptions for data that could make or break a company, piquing the interest of potential investors and partners alike.
Data, for instance, like what Sekar Kathiresan is bringing to the virtual conference, showing that a one-time treatment with Verve Therapeutics’ base editor can keep PCSK9 levels low — thereby lowering LDL cholesterol, aka the bad kind — out to 6 months. In non-human primates.
Since joining British drugmaker GlaxoSmithKline in 2018, CSO Hal Barron has pushed to beef up the company’s oncology portfolio, taking a particular eye to “synthetic lethality” discovery work. Now, GSK is putting down a big chunk of change on the flip side of the coin — cell viability — for a range of potential therapies outside of cancer.
GSK will co-lead a Series A financing round with an undisclosed amount of cash for Adrestia Therapeutics, a biotech hoping to develop medicines targeting the “synthetic viability” of dysfunctional or dying cells in a range of therapeutic areas, GSK said Friday.
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