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.

Mov­ing Out of the Clin­ic with Dig­i­tal Tools: Mo­bile Spirom­e­try Dur­ing COVID-19 & Be­yond

An important technology in assessing lung function, spirometry offers crucial data for the diagnosis and monitoring of pulmonary system diseases, as well as the ongoing measurement of treatment efficacy. But trends in the healthcare industry and new challenges introduced by the COVID-19 pandemic are causing professionals in clinical practice and research to reevaluate spirometry’s deployment methods and best practices.

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Sanofi, Glax­o­SmithK­line jump back in­to the PhI­II race for a Covid vac­cine — as the win­ners con­gre­gate be­hind the fin­ish line

Sanofi got out early in the race to develop a vaccine using more of a traditional approach, then derailed late last year as their candidate failed to work in older people. Now, after likely missing the bus for the bulk of the world’s affluent nations, they’re back from that embarrassing collapse with a second attempt using GSK’s adjuvant that may get them back on track — with a potential Q4 launch that the rest of the world will be paying close attention to.

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Tim Mayleben (L) and Sheldon Koenig (Esperion)

On the heels of a sting­ing Q1 set­back, Es­pe­ri­on's long­time cham­pi­on is ex­it­ing the helm and turn­ing the wheel over to a mar­ket­ing pro

Just days after getting stung by criticism from a badly disappointed group of analysts, there’s a big change happening today at the helm of Esperion $ESPR.

Longtime CEO Tim Mayleben, who championed the company for 9 years from early clinical through a lengthy late-stage drive to successfully get their cholesterol drug approved for a significant niche of patients in the US, is out of the C suite, effective immediately. Sheldon Koenig — hired at the end of 2020 with a resume replete with Big Pharma CV sales experience —  is stepping into his place, promising to right a badly listing commercial ship that’s been battered by market forces.

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SCO­TUS de­clines to re­view En­brel biosim­i­lar case, tee­ing up 30+ years of ex­clu­siv­i­ty and $20B more for Am­gen’s block­buster

As the House Oversight Committee is set to grill AbbVie CEO Richard Gonzalez on Tuesday over tactics to block competition for its best-selling drug of all time, another decision on Capitol Hill on Monday opened the door for billions more in Amgen profits over the next eight years.

The Supreme Court on Monday denied Novartis subsidiary Sandoz’s petition to review a Federal Circuit’s July 2020 decision concerning its biosimilar Erelzi (etanercept-szzs), which FDA approved in 2016 as a biosimilar to Amgen’s Enbrel (etanercept). Samsung’s Enbrel biosimilar Eticovo also won approval in 2019 and remains sidelined.

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How to man­u­fac­ture Covid-19 vac­cines with­out the help of J&J, Pfiz­er or Mod­er­na? Bi­ol­yse sees the dif­fi­cul­ties up close

When Biolyse, an Ontario-based manufacturer of sterile injectables, forged a deal with Bolivia last week to manufacture up to 50 million J&J Covid-19 vaccine doses, the agreement kicked off what will prove to be a test case for how difficult the system of compulsory licenses is to navigate.

The first problem: When Biolyse asked J&J, via a March letter, to license its Covid-19 vaccine, manufacture it in Canada and pay 5% royalties on shipments to needy, low-income countries, J&J rejected the offer, refusing to negotiate. J&J also did not respond to a request for comment.

No­var­tis' En­tresto takes its 2nd fail­ure of the week­end at ACC, show­ing no ben­e­fit in most dire heart fail­ure pa­tients

Novartis’ Entresto started the ACC weekend off rough with a trial flop in heart attack patients, slowing the drug’s push into earlier patients. Now, an NIH-sponsored study is casting doubt on Entresto’s use in the most severe heart failure patients, another black mark on the increasingly controversial drug’s record.

Entresto, a combination of sacubitril and valsartan, could not beat out valsartan alone in an outcomes head-to-head for severe heart failure patients with a reduced ejection fraction (HFrEF), according to data presented Monday at the virtual American College of Cardiology meeting.

Matt Gline (L) and Vivek Ramaswamy

In­sid­er ac­count of Roivan­t's SPAC deal — and that $7.3B val­u­a­tion — re­veals a few se­crets as Matt Gline po­si­tions the com­pa­ny as the new ‘Big Phar­ma’

It was Oct. 7, 2020, and Matt Gline wasn’t wasting any time.

The CEO of Roivant had word that KKR vet Jim Momtazee’s SPAC had priced late the night before, triggering a green light for anyone interested in pursuing a big check for future operations and riding the financial instrument to Nasdaq. So he wrote a quick email congratulating Momtazee, whom he knew, for the launch.

Oh, and maybe Momtazee would like to schedule something with Gline and his executive chairman, Roivant founder Vivek Ramaswamy, to chat about Roivant and its business?

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$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; 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.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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John­son & John­son do­nates Ebo­la vac­cine amid new out­break; Ji Xing promis­es more than $127M for Mile­stone's nasal spray for rapid heart rate

As Johnson & Johnson continues to roll out its Covid-19 shot, the company is also focused on another vaccine.

J&J is donating up to 200,000 doses of its Ebola vaccine regimen developed with Bavarian Nordic to help health authorities deal with a new outbreak in Sierra Leone. The regimen, Zabdeno and Mvabea, was granted prequalification by the WHO in April, which will help accelerate its registration in countries where Ebola is a threat.