Go­ing big: Biotech vets blast off with Ar­rakis on a new jour­ney of RNA ex­plo­ration


Michael Gilman and an ex­pe­ri­enced band of biotech vets are bust­ing out of stealth mode to­day with a start­up that marks the se­r­i­al en­tre­pre­neur’s third launch in 10 years. And he’s com­ing out in style, with a $38 mil­lion A round led by Canaan Part­ners.

Af­ter lit­er­al­ly stum­bling across the com­pa­ny while it was still in the white-board phase of de­vel­op­ment in 2015, Gilman says he was ripe for the chal­lenge and ad­ven­ture. And it’s a big one.

CSO and founder Russ Pet­ter has been hon­ing the bioin­for­mat­ics tools, as­says and chem­i­cal li­braries Ar­rakis Ther­a­peu­tics will need to cre­ate a plat­form tech­nol­o­gy that can be used to de­vel­op small mol­e­cules to in­hib­it dis­ease-caus­ing RNA.

This biotech has a long jour­ney ahead in pre­clin­i­cal work be­fore it can start try­ing this out in hu­mans. But if they’re right, the com­pa­ny will be on their way to work­ing on oral ther­a­pies that would be able to hunt down a host of what had been con­sid­ered un­drug­gable tar­gets — start­ing with an ini­tial aim at neu­rol­o­gy and can­cer.

Most pre­clin­i­cal star­tups like this come out of acad­e­mia. But this is a unique­ly Cam­bridge-based out­fit, bring­ing to­geth­er peo­ple with decades of ex­pe­ri­ence right in the heart of one of the world’s biggest biotech hubs.

“I’ve known Russ for a long time,” says Gilman, whose CV in­ter­sects with the founder’s stint at Bio­gen. “He ran chem­istry when I ran re­search there. He’s been work­ing on this for awhile.”

And Pet­ters isn’t the on­ly oth­er Bio­gen vet on board. Chief Busi­ness Of­fi­cer Daniel Ko­er­w­er and James Bar­soum, SVP of bi­ol­o­gy, al­so trace their ca­reers back to the Cam­bridge, MA gi­ant. Col­lec­tive­ly, they list stints at more than a dozen dif­fer­ent biotech com­pa­nies on their re­sumes.

Gilman crossed paths with Pet­ter in 2015, when the fel­low sci­en­tist was hatch­ing his plans for Ar­rakis — a com­pa­ny named af­ter the plan­et in Frank Her­bert’s Dune — in some shared of­fice space where Gilman had one of his board meet­ings. Pet­ter had had his in­ter­est whet­ted at a con­fer­ence pre­sen­ta­tion on small mol­e­cules and RNA in­ter­ac­tions.

They talked, Bris­tol-My­ers Squibb de­cid­ed it would pay a hand­some price for Gilman’s last com­pa­ny, Pad­lock, and af­ter tak­ing last sum­mer off, they got down to se­ri­ous­ly ex­plor­ing the idea to­geth­er.

The mon­ey fol­lowed the ideas.

Along with Canaan Part­ners, Ad­vent Life Sci­ences, Pfiz­er, Cel­gene, Os­age Uni­ver­si­ty Part­ners, and biotech in­dus­try leader Hen­ri Ter­meer chipped in to the A round. The UK’s Ad­vent and Ter­meer helped seed the ini­tial work. And if Ar­rakis’ team is right, there will be plen­ty of op­por­tu­ni­ties along the way to set up plat­form col­lab­o­ra­tions and part­ner­ships.

Colleen Cuf­faro

“I was very in­trigued from the minute I heard the con­cept,” says Colleen Cuf­faro, a prin­ci­pal at Canaan who’s tak­ing a board spot at Ar­rakis.  “It has enor­mous po­ten­tial, how it opens up a whole new space for un­drug­gable tar­gets on our radar for a long time. I re­al­ly like the ap­proach of us­ing small mol­e­cules that are fun­da­men­tal­ly dif­fer­ent than any oth­er RNA-tar­get­ing ap­proach I’ve seen. The oth­er piece was the team, a re­al­ly proven team, with a track record that is out­stand­ing.”

Gilman isn’t con­cen­trat­ing sole­ly on Ar­rakis. He’s been deeply in­volved with At­las Ven­ture, and they’re plan­ning an­oth­er com­pa­ny launch lat­er in the year. But Gilman isn’t stressed by the idea of helm­ing two ven­tures at once.

At Pad­lock, says Gilman, “I felt like I had ex­tra time on my hands.” Af­ter all, CEOs of star­tups of­ten “spend a lot of time wait­ing around for stuff to hap­pen.” And if you have the kind of team he’s work­ing with at Ar­rakis, it’s not a full-time job.

This is not Gilman’s first biotech rodeo. He knows first hand that a ven­ture-backed start­up of­ten lands in oth­er hands, as hap­pened with Stromedix and Pad­lock. This time around though, he would like to take it all out much fur­ther.

“You can nev­er rule it out,” he says, “but this is a com­pa­ny I would like to see built for the long haul. I tru­ly be­lieve we’ll have the ca­pa­bil­i­ty to crank out new drugs oth­er peo­ple haven’t been able to make.”

Gilman’s a long­time be­liev­er in the adage that in biotech, you should go big or go home.

Once again, he’s go­ing big.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.

Morningside continued to lead the $30 million Series B, with participation from Termeer’s widow, Belinda, and other unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.

An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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