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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Ivana Magovčević-Liebisch, Vigil Neuroscience CEO

FDA lifts par­tial clin­i­cal hold on Vig­il Neu­ro­science's TREM2 an­ti­body, re­mov­ing dos­ing cap

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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