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.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving.

Loew’s departure comes amid a slow-moving shakeup to the French pharma giant’s executive team. The company also announced today that Alan Main, the head of their consumer healthcare unit, is out, and they named four executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma. Thomas Triomphe will be the one promotion from inside the company, as he moves to take Loew’s position at the helm of Sanofi Pasteur, one of the world’s largest vaccine divisions.

Look­ing to move past a tri­al fi­as­co, Ipsen poach­es their new CEO from Sanofi

Ipsen has turned to another Paris-based biopharma company for its next CEO.

Sanofi Pasteur chief David Loew is making the journey to Ipsen, 5 months after David Meek jumped ship to run a startup in late-stage development.

Loew arrives as Ipsen works to get back on track with their rare bone disease drug palovarotene, picked up in the $1.3 billion Clementia buyout, which was slammed with a partial hold after researchers observed cases of “early growth plate closure” in patients under the age of 14. But they are pushing ahead with the over-14 crowd after writing down slightly more than half of its initial development.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Iron­wood kicks de­layed-re­lease Linzess for­mu­la­tion to the curb af­ter tri­al fail­ure

The delayed-release formulation of Ironwood and Allergan’s bowel drug Linzess will not see the light of day.

The experimental drug, MD-7246, failed to help patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) in a mid-stage study, prompting the partners to abandon the therapy.

First approved in 2012, Linzess (known chemically as linaclotide) enhances the activity of the intestinal enzyme guanylate cyclase-C to increase the secretion of intestinal fluid and then transit through the intestinal tract, as well as reduce visceral pain, to relieve pain and constipation associated with IBS.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Stymied by the pan­dem­ic, Im­munomedic­s' new CEO bows out, tak­ing a mil­lion bucks plus perks as he heads out the vir­tu­al ex­it

Just a little more than a month since taking over as the latest CEO to helm Immunomedics, $IMMU Harout Semerjian is exiting the company after being confronted by “logistical” obstacles thrown up by the pandemic that made it impossible for him to move from London to carry out the job. And he’s getting a little over a million dollars in cash plus perks to grease the skids on the way out.

Word of the changeup arrived right after the market closed Wednesday.

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