CEO Lex Rovner (64x Bio)

A George Church spin­out fight­ing the vi­ral vec­tor bot­tle­neck in cell and gene ther­a­py lands $55M

A syn­thet­ic bi­ol­o­gy com­pa­ny spun out of George Church’s lab is set to tack­le the gene ther­a­py man­u­fac­tur­ing bot­tle­neck, and it just land­ed $55 mil­lion in a Se­ries A fi­nanc­ing round to do so.

George Church

64x Bio comes out of the Har­vard De­part­ment of Ge­net­ics. CEO Lex Rovn­er and her team — which right now, sits around 10 peo­ple — are look­ing to tack­le a key hur­dle for ma­jor com­pa­nies: man­u­fac­tur­ing cell and gene ther­a­pies.

Rovn­er met Church while get­ting her PhD at Yale, and went on to do a post­doc­tor­al re­search fel­low­ship in his lab, and, when talk­ing to folks in the in­dus­try, found a mas­sive vi­ral vec­tor man­u­fac­tur­ing bot­tle­neck that wasn’t be­ing talked about.

Af­ter a seed fund­ing round and the com­pa­ny’s launch in 2020, it made some noise in the in­dus­try, par­tic­u­lar­ly as Covid-19 made bot­tle­neck is­sues more vis­i­ble. There’s a wait­list to get a vec­tor from man­u­fac­tur­ers, and not much of a so­lu­tion to the prob­lem

“In a lot of cas­es, in-house vec­tor man­u­fac­tur­ing ca­pac­i­ty is al­ready ex­ceed­ed for even the small­est dis­ease pop­u­la­tions. And once you get in­to the more wide­spread dis­eases, man­u­fac­tur­ing ca­pac­i­ty can be off by or­ders of mag­ni­tude,” Rovn­er said in an in­ter­view with End­points News. “It’s very like­ly go­ing to be im­pos­si­ble to out­source that much man­u­fac­tur­ing…It’s re­al­ly no se­cret that this is a prob­lem, that is and will con­tin­ue to lim­it the ther­a­pies for each pa­tient if this is left un­solved.”

Life­force Cap­i­tal led the round, with North­pond Ven­tures, Fu­ture Ven­tures, the for­mer Alde­vron CEO Michael Cham­bers, First Round Cap­i­tal, CEO of Re­cur­sion Chris Gib­son and Al­ix Ven­tures all par­tic­i­pat­ing as well. The funds will go to­ward ad­vanc­ing Vec­torS­e­lect, a plat­form that us­es ma­chine learn­ing to test mil­lions of dif­fer­ent cells for how well virus­es grow in them, mak­ing for a faster, and more ef­fec­tive process.

Once it’s test­ed mil­lions of po­ten­tial cell lines — all si­mul­ta­ne­ous­ly — 64x screens for com­bi­na­tions of genes that make for high­ly pro­duc­tive cells. So far, there’s been suc­cess with ade­no-as­so­ci­at­ed virus­es, the most com­mon vec­tor used in gene ther­a­py, and Rovn­er and her team are look­ing to ex­plore oth­er mar­kets too.

“The prob­lem is more fun­da­men­tal than phys­i­cal in­fra­struc­ture,” she said. “This is a ge­net­ics prob­lem and the so­lu­tions are go­ing to be in tai­lor­ing how a cell in­ter­acts with the vi­ral com­po­nents.”

Church said in a state­ment: “Un­til now, ge­net­ic tai­lor­ing of man­u­fac­tur­ing cells has failed to de­liv­er. Over­com­ing the cel­lu­lar bar­ri­ers to vi­ral pro­duc­tion will re­quire many cel­lu­lar mu­ta­tions in com­bi­na­tion, which means test­ing po­ten­tial­ly tril­lions of mu­tant cells. Ex­ist­ing cell line en­gi­neer­ing and screen­ing tech­nolo­gies have not been up to the chal­lenge.”

64x will “dra­mat­i­cal­ly” grow its em­ploy­ee base, from 10 em­ploy­ees right now to about 50, Rovn­er said. That will start with the ex­pan­sion of its man­age­ment team. It’s al­so go­ing to ex­pand its part­ner­ships with oth­er biotechs, and while the com­pa­ny isn’t ready to an­nounce any­thing just yet, news on that front could be com­ing in the near fu­ture.

Ed­i­tor’s Note: For more news and ex­clu­sive cov­er­age from the man­u­fac­tur­ing beat, sub­scribe to the End­points Man­u­fac­tur­ing week­ly re­port in your read­er pro­file.

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.

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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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.

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.

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.

Christophe Bourdon, Leo Pharma CEO

Leo Phar­ma looks 'be­yond the skin' in atopic der­mati­tis aware­ness cam­paign

As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, the company is launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe.

The project, unveiled on Monday, spotlights four patients: Marjolaine, Laura, Julia and África from France, Italy, Germany and Spain, respectively, in short video clips on the challenges of living with AD, the most common form of eczema.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

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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