Scoop: Google’s GV spear­heads the Spot­light syn­di­cate — back­ing an up­start biotech aimed at ‘de­moc­ra­tiz­ing’ gene edit­ing

CRISPR had no soon­er start­ed to shake the very foun­da­tions of drug de­vel­op­ment be­fore its lim­i­ta­tions be­gan to loom large. Gene edit­ing could change the world — if on­ly you could get around the hur­dles that threat­ened to trip up every pro­gram.

Blake By­ers

So it’s on­ly nat­ur­al to see CRISPR 2.0 tak­ing shape be­fore the pi­o­neers can get the lead ther­a­pies through de­vel­op­ment. And who bet­ter than Google’s GV ven­ture arm to take the lead spot in a small syn­di­cate back­ing some sci­en­tists with their own unique twist on a so­lu­tion?

GV gen­er­al part­ner Blake By­ers took point on this one, join­ing with some in­di­vid­ual in­vestors who are stay­ing in the back­ground. They were all at­tract­ed by the idea that the small team at Spot­light Ther­a­peu­tics could by­pass the de­liv­ery is­sues em­bed­ded in every gene edit­ing pro­gram by us­ing pro­gram­ma­ble CRISPR ri­bonu­cle­o­pro­teins de­signed for in vi­vo cell-tar­get­ed de­liv­ery.

And now the Spot­light team is jump­ing in­to the in­dus­try spot­light with a $30 mil­lion launch round to go af­ter lead pro­grams in he­mo­glo­binopathies and im­muno-on­col­o­gy.

Right now, de­spite all the promise of CRISPR, the pi­o­neers are un­able to ad­dress spe­cif­ic cell types in vi­vo, says CEO Mary Haak-Frend­scho. This new ap­proach of­fers the op­por­tu­ni­ty “to re­al­ly open up, de­moc­ra­tize gene edit­ing.”

Mary Haak-Frend­scho

The tech used to­day de­pends a lot on us­ing an AAV or LNP to ex­press the ed­i­tor in the tar­get cell, says By­ers. Those have bet­ter up­take in liv­er cells, so that’s where the ear­ly play­ers have start­ed. And as we’ve seen at Sol­id and oth­er places, there are lin­ger­ing con­cerns about safe­ty and ef­fi­ca­cy.

Spot­light, he says, is tak­ing a dif­fer­ent, com­plete­ly non-vi­ral ap­proach. And it starts with a ques­tion.

“Why don’t we bind the RNP (ri­bonu­cle­o­pro­tein) ed­i­tor to oth­er things?” asks By­ers. “That could be oth­er pro­teins, let’s say an an­ti­body. Get an­ti­bod­ies to a tar­get on the cell sur­face, it’s in­ter­nal­ized at some rate, then hitch­hikes in­to the cell and from there ed­it the tar­get site in the genome.”

“There’s a lot of things you can latch your gene ed­i­tors to,” says By­ers. The goal is to ex­plore the space, search­ing for 5 to 10 port­fo­lio prod­ucts bound to dif­fer­ent hitch­hik­er mol­e­cules that you can use to get your ed­i­tor of choice in­to the ex­act cell type you want, with high speci­fici­ty and low cost of goods.

Alex Mar­son

In the process, you have to es­cape de­struc­tion by the lyso­some and go on to the nu­cle­o­some to do the edit­ing, so there’s plen­ty of de­bug­ging along the way to get it to work right.

Much of the in­spi­ra­tion for this work came from 3 key sci­en­tists who are ad­vis­ing the start­up.

Alex Mar­son and Patrick Hsu thought a lot of this out. Mar­son is at UC San Fran­cis­co and Hsu is now close by at UC Berke­ley. Ja­cob Corn at ETH Zürich rounds out the line­up of sci­en­tif­ic founders with pres­ti­gious re­sumes.

Patrick Hsu

The longterm plan is to stick with the non-vi­ral ap­proach and build a pipeline.

As A rounds go these days, $30 mil­lion isn’t a lot of mon­ey, par­tic­u­lar­ly when you’re talk­ing about a tech plat­form ap­proach like this. I point­ed that out to By­ers, who wasn’t feel­ing apolo­getic about the $30 mil­lion.

“How old school of us,” he re­spond­ed, adding that there are dif­fer­ent strate­gies for dif­fer­ent play­ers. Some should be laser fo­cused on prod­uct de­vel­op­ment. Be­sides, he adds, “small mounts of cap­i­tal are very fo­cus­ing for a com­pa­ny.”

Ja­cob Corn

The mon­ey “gets us to 2022,” says the CEO. At that point they ex­pect to have their first de­vel­op­ment can­di­date, with­in a year of the clin­ic for ei­ther or both their cho­sen in­di­ca­tions.

And then they can see about start­ing their own gene edit­ing rev­o­lu­tion.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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ProFound Therapeutics founding team

Flag­ship's lat­est biotech could turn some of the thou­sands of new pro­teins it dis­cov­ered in­to ther­a­pies — and it has $75M to start

Flagship Pioneering, the incubator of Moderna and dozens of other biotechs, says it has landed upon tens of thousands of previously undiscovered human proteins. The VC shop wants to potentially turn them into therapeutics.

Like other drug developers that have turned proteins into therapeutics (think insulin for diabetes), Flagship’s latest creation, ProFound Therapeutics, wants to tap into this new trove of proteins as part of its mission to treat indications ranging from rare diseases to cancer to immunological diseases.

Richard Silverman, Akava Therapeutics founder and Northwestern professor

This time around, Lyri­ca's in­ven­tor is de­vel­op­ing his North­west­ern dis­cov­er­ies at his own biotech

Richard Silverman was left in the dark for the last five years of clinical development of the drug he discovered. The Northwestern University professor found out about the first approval of Lyrica, in the last few days of 2004, like most other people: in the newspaper.

What became one of Pfizer’s top-selling meds, at $5 billion in 2017 global sales before losing patent protection in 2019, started slipping out of his hands when Northwestern licensed it out to Parke-Davis, one of two biotechs that showed interest in developing the drug in the pre-email days, when the university’s two-person tech transfer team had to ship out letters to garner industry appetite.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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Up­dat­ed: US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Simba Gill, CEO of Evelo Biosciences

While down 87% YOY, Evelo gets Flag­ship and oth­ers to in­fuse new cap­i­tal for come­back hope

Just four years after Flagship spinout Evelo Biosciences went public in an IPO worth $85 million, the biotech has seen its share price tank from $13 a share this time last year (ultimately reaching a peak of over $17) to now under $1.50. And today, it looks like Flagship still thinks the fledging biotech, in a down market, is still worth something after initial pre-IPO backing from the likes of Google’s GV, Celgene, Mayo Clinic and Alexandria Venture.

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Peter Thompson, Terremoto Biosciences interim CEO

For­mer Prin­cip­ia team looks to shake up co­va­lent small mol­e­cules again, this time at 'earthquake' scale

Terremoto Biosciences goes back a long ways, in a sense, to about a dozen years ago when Principia Biopharma was founded by UCSF professor Jack Taunton. Peter Thompson initially helmed the biotech.

The company helped expand covalent small molecule inhibitors beyond oncology and into autoimmune disease by targeting cystine. But that amino acid is uncommon in a lot of proteins, offering fewer drug targets than, say, lysine, which is present in most proteins of interest. So, over the years, Taunton went back to the drawing board to check out that second amino acid.

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