Inscripta pushes funding to $260 million as they launch genome editing platform

Inscripta presentations can begin with the advent of agriculture. Or even further back: The emergence of man.

Nandini Krishnamurthy

“We’ve come a long way, starting with natural selection,” Inscripta microbial director Nandini Krishnamurthy told a session this year at SynBioBeta, the new annual conference for the synthetic biology field.

Behind her was a slide that’s recurred in company presentations, showing from left to right across the bottom the classic evolution-of-man chart (the ‘humorous’ kind that ends on an overweight soda-drinker), a picture showing the development of corn from thin strand to bulbous Iowan, and then a squiggly protein close-up of “directed evolution.” Below that runs an arrow and a ticker of how long each takes, from 10^9 years to 1.

“The question is how do you go from precisely engineering proteins to precisely engineering pathways and genomes to harness the power of biology?” she said.

Kevin Ness

It’s the kind of grand rhetoric and ambition you’ve come to expect from a synthetic bio field that features Gingko Bioworks and a company billing itself as the “Nespresso machine for DNA synthesis.” Inscripta keynoted the 2019 conference to launch their Onyx platform for genome engineering, and today they’ve announced $125 million in Series D funding to commercialize that platform. It brings their total funding to $260 million. The pitch is simple: Do for CRISPR what laptops and phones have done for supercomputers.

“We’ve democratized access to the foundry,” CEO Kevin Ness told Endpoints News, referring to the microtiter plates often used for DNA-edited cell lines. “We’ve created the equivalent of a desktop computer.”

The foundries are groups like Gingko and Zymergen that often use their large facilities to edit organisms for a service fee. Inscripta is trying to put much of that system in the hands of researchers, a key cog they think will help unleash emergent “bioeconomy:” a world where genomes are edited as much as they are read. The price, though, is not insignificant: Around $350,000 total.

Inscripta first made a name when they unveiled MAD7, an alternative CRISPR enzyme to CAS9 and then gave it away for free to all researchers. They pitched it as an altruistic ways of breaking the licensing fees around CAS9 and helping move the entire field forward, but it was good business: In eliminating one bar to genome editing, they widened the potential market for their real product: Onyx. It was a similar strategy to the one employed Illumina – the genome reading company Inscripta specifically markets themselves around.

“We do for genome editing what Illumina did for genome reading,” Ness said.

Ness has experience on the genome-reading side, working early in his career on PCR and later co-founding 10x Genomics, and Inscripta employs multiple Illumina alumni, including their co-founder and first CEO: John Stuelpnagel.

So how does it work?

A biologist with the platform can select the genes they want to knock out from Inscripta’s computer interface. Inscripta then creates the agents to knock out those genes at their manufacturing facility and sends them to the biologist’s lab. The biologist applies those agents to the cell lines, creating thousands of different lines that are “bar-coded.” The biologist can run whatever experiment — exposing them to a drug, say, or an agent — and the Inscripta tech can see which cell lines survived.

So far the platform is only available for E. Coli and S. cerevisiae and the company’s exposure outside synthetic biology and bacteria spaces has been limited, but Inscripta has found a big proponent in MIT’s Jim Collins. Collins has used the platform to create bacteria cell lines with 5,000 different gene edits. He then used those to test which genes conferred resistance.

It allowed us “to explore the genetic dependencies of antibiotic function in unprecedented detail.” Collins told GEN in October.

Adaptive Design Methods Offer Rapid, Seamless Transition Between Study Phases in Rare Cancer Trials

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

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A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Alan Hirzel, Abcam

Drug supplier Abcam brings a longtime collaborator in house as part of $340M buyout pact

BioVision has supplied Abcam with research tools since 1999, and now the two are making it official as part of a merger unveiled Monday.

Abcam will buyout BioVision as part of a $340 million acquisition deal to bring aboard the supplier’s biochemical and cell-based assays for biological research, as well as recombinant proteins, antibodies and enzymes.

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Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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Not all mRNA vaccines are created equal. Does it matter?; Neuro is back; Private M&A affair; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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Bristol Myers pulls lymphoma indication for Istodax after confirmatory trial falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's oncology head Rick Pazdur defends the accelerated approval pathway, claiming it is 'under attack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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