An­dré Chouli­ka on gene edit­ing: "If you mar­ry your­self to a tech­nol­o­gy, you're doomed."

An­dre Chouli­ka

Gene edit­ing plays a ma­jor role in next-gen CAR-T pro­grams. And Cel­lec­tis can now boast of a patent that touch­es on a broad range of tech­nolo­gies that will come in­to play.

The U.S. patent of­fice has is­sued patent num­ber 9,458,439, pro­tect­ing the work that Cel­lec­tis CEO (and sci­en­tist) An­dré Chouli­ka and Har­vard Pro­fes­sor Richard C. Mul­li­gan, one of the orig­i­nal pi­o­neers in the gene ther­a­py field, have ac­com­plished.

Put sim­ply, the patent cov­ers a va­ri­ety of tech­nolo­gies they used to per­form surgery on a gene in vit­ro.

‘This patent doesn’t fo­cus on one tech­nol­o­gy,” Chouli­ka tells me this morn­ing in a call from Paris, where Cel­lec­tis is based. Rather, it en­com­pass­es sev­er­al ways to in­duce gene in­ac­ti­va­tion, cit­ing roles for CRISPR/Cas9, Zinc fin­ger Nu­cle­as­es, TAL-Ef­fec­tor Nu­cle­as­es, Mega-TALEs and some Meganu­cle­as­es.

“It’s a large way to see gene edit­ing. This, for me, is the ba­sis for nu­cle­ase-based gene edit­ing,” he adds. “It po­si­tions us in a very com­pet­i­tive way to move for­ward in the space.”

Chouli­ka and his part­ners at Pfiz­er and Servi­er are now in ear­ly-stage test­ing for an al­lo­gene­ic ap­proach to CAR-T, us­ing gene edit­ing tech­nolo­gies to en­gi­neer an off-the-shelf cell ther­a­py that would rad­i­cal­ly change the land­scape now dom­i­nat­ed by late-stage ef­forts on per­son­al­ized ther­a­pies at Kite, Juno and No­var­tis that ex­tract cells and then reengi­neer them in­to can­cer treat­ments.

Don’t ex­pect gene-edit­ing tech to re­main un­changed, adds Chouli­ka, who’s Beirut-born, with a PhD from the Uni­ver­si­ty of Paris and re­search stints in ge­net­ics at Har­vard and Boston Chil­dren’s be­hind him.

“The cell phone you’re hold­ing in your hand is ob­so­lete,” he says by way of com­par­i­son. And so is the gene edit­ing tech you’re see­ing at the fore­front of the are­na now. “If you mar­ry your­self to a tech­nol­o­gy, you’re doomed.”

This is al­so a fast-mov­ing de­vel­op­ment. In just re­cent weeks No­var­tis $NVS has dis­solved it’s 400-mem­ber cell ther­a­py unit. Juno $JUNO, al­lied with Ed­i­tas on gene edit­ing, has been deal­ing with the fall­out of a brief clin­i­cal hold on its lead ther­a­py, which de­railed its ap­proval sched­ule. That leaves Kite $KITE out front with plans to hus­tle a new drug ap­pli­ca­tion to the FDA af­ter on­ly three months of re­sults to work with.

“I’m very im­pressed by what Kite has been do­ing,” says Chouli­ka. Their au­tol­o­gous R&D work has shown that there is a clear path to the mar­ket, and that will help demon­strate that there’s a path for Cel­lec­tis as well.

Says the CEO: “Kite is def­i­nite­ly lead­ing the way.”

And Cel­lec­tis is mov­ing as fast as it can to fol­low up.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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