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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for 'New Bio­haven' with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the “New Biohaven” post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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