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Take­da maps out a dis­cov­ery al­liance aimed at break­ing through the safe­ty and ef­fi­ca­cy bound­aries that lim­it the first-gen gene ther­a­pies

MIT headliner Harvey Lodish and Jiahai Shi in Hong Kong leant some of their work in red blood cells to help found Rubius, a Flagship startup that has been disappointing investors with its inability to stay on track with the clinical work.

But Lodish and Shi also backed another, lesser-known startup — Carmine Therapeutics — that has now inked its first Big Pharma partnership. And this one is hoping to make it big in one of the hottest fields in drug R&D: gene therapy.

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Sekar Kathiresan (Verve)

Sekar Kathire­san show­cas­es his­toric mon­key da­ta in hunt for a once-and-done gene edit­ing ap­proach to pre­vent heart at­tacks

In a first for CRISPR — a field that’s seen quite a few historic moves in the past couple of years — scientists have slashed cholesterol and lipid levels in monkeys using a technique known as base editing, generating crucial proof-of-concept for both the biotech behind the experiment and a key partner.

Verve Therapeutics designed two similar experiments to test whether they can reproduce protective mutations in a pair of genes using an adenine base editor, which precisely alters a chosen A to a G in the genome. CEO and co-founder Sekar Kathiresan presented the results at a virtual keynote for the International Society for Stem Cell Research over the weekend.

Back to the im­munol­o­gy draw­ing board, Ab­b­Vie inks dis­cov­ery deal with old Al­ler­gan part­ner

Allergan’s neurology-focused R&D pact with Sosei Heptares hasn’t been all roses, but the BD team at AbbVie found enough to like about the G protein-coupled receptor specialist’s discovery engine to kick off a new collaboration. The goal? Finding small molecules targeting inflammatory and autoimmune diseases.

The partnership starts small, with $32 million in upfront and near-term milestones plus potential option, development and commercial payments of up to $377 million. But if AbbVie chooses to execute on all four targets, the total deal value could grow to a size “in a similar ballpark” to the billion-dollar pacts with Genentech and Takeda, a Sosei spokesperson told Reuters.

Tony Coles, Yumanity Executive Chairman (Yumanity)

Burned by Alzheimer’s fail­ures, a cau­tious Mer­ck turns to a start­up biotech for some pre­clin­i­cal neu­ro pro­grams

After playing a leading role in proving that the BACE theory was worthless in treating Alzheimer’s, Merck is going back to the drawing board for new ideas on the neurosciences. And in the spirit of its renewed focus on discovery, the pharma giant is turning to a biotech startup called Yumanity Therapeutics for some fresh, preclinical ideas.

The crux of the deal they announced today focuses on the rights to two of Yumanity’s pipeline programs for ALS and frontotemporal lobar dementia. After working on the preclinical programs together, Merck will have the right to continue on into development with a shot at commercialization — if they can get that far.

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Nim­bus picks 4 pre­clin­i­cal tar­gets for the next chap­ter of its pi­o­neer­ing com­pu­ta­tion­al drug dis­cov­ery work

Big name partnerships were critical for Nimbus Therapeutics’ first decade. With a head-turning $1.2 billion — $600 million of which were paid within months — deal from Gilead and a returning customer in Celgene, the biotech emerged as a prolific pioneer of computational chemistry and structure-based drug discovery while the industry went through a seismic shift in its thinking of the role that algorithms play in engineering new therapies.

Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”

Gary Firestein (UCSD via YouTube)

A UCSD cen­ter that's been fa­cil­i­tat­ing Covid-19 clin­i­cal re­search gets $55M from NIH to boost trans­la­tion­al work

A team at the University of California San Diego that’s been facilitating studies on the frontlines of Covid-19 has received a $54.7 million grant from the NIH to bind basic research with clinical care even more tightly together.

Every year the 1,000 faculty and staff at the Altman Clinical and Translational Research Institute partner with UCSD Health to help run 250 trials — including recent trials investigating “an antiviral drug, an arthritis drug and a medication for hypertension” as treatments for the coronavirus infection.

The newest, $86M Third Rock start­up chas­es the tiny bi­o­log­i­cal ma­chines in­side of you

It took 23 years from the isolation of the gene for cystic fibrosis to the approval of the first drug to target it, and another 7 for a drug that could treat the vast majority of CF patients. Third Rock Venture’s latest startup thinks they can build similar drugs a whole lot faster.

MOMA Therapeutics has raised $86 million to investigate and drug a class of enzymes known as molecular machines. These proteins include everything from enzymes involved in DNA repair to the transport proteins that go awry in cystic fibrosis — over 400 different types, by MOMA’s count. And yet, MOMA contends, they have been overlooked to date, with researchers both failing to understand them as a cohesive group and failing to employ systematic ways of finding and drugging them.

Join­ing No­var­tis in siR­NA space, As­traZeneca hands Si­lence $80M up­front for dis­cov­ery deal

Mene Pangalos is doubling down on RNA-based technologies for his side of the AstraZeneca R&D machine.

Just two months after bringing a small activating RNA (saRNA) program into its fold, Pangalos now has his eyes on Silence Therapeutics’ small interfering RNA (siRNA) platform. Calling it an “exciting new modality” for the pharma giant’s drug discovery toolbox, AstraZeneca has shelled out $60 million in cash and purchased $20 million worth of Silence’s stock on the London exchange.

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How to build CAR–M: Carl June pro­tégé and his biotech pub­lish first ev­i­dence in Na­ture Biotech­nol­o­gy

Michael Klichinsky joined the University of Pennsylvania for his PhD at the height of the revolution.

It was 2014, and though the CAR-T therapy that would make Penn professor Carl June famous would not be approved for four years, it had already caused headline-grabbing remissions in a small handful of patients. Klichinsky turned down offers from other elite universities to take a spot between June’s lab and colleague Saar Gill’s lab.

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