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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”