Kunwoo Lee, GenEdit CEO

Eli Lil­ly gets be­hind the lat­est ap­proach to solv­ing gene ther­a­py's de­liv­ery prob­lem

Kun­woo Lee was a grad­u­ate stu­dent at UC-Berke­ley when gene edit­ing pi­o­neer Jen­nifer Doud­na — who hap­pened to work in the same build­ing where he stud­ied — pub­lished a pa­per on CRISPR/Cas9. So he did what any as­pir­ing bio­engi­neer would do: He ran to her lab, and grabbed a post­doc there.

“We start­ed re­al­ly think­ing about the fu­ture com­ing (for) gene ther­a­py and gene edit­ing,” he said.

Lee’s re­search with Doud­na led him to co-found a small San Fran­cis­co-based biotech called GenEd­it in 2016, the same year he grad­u­at­ed. Af­ter five qui­et years, the team is now un­veil­ing a $26 mil­lion Se­ries A round with sup­port from some big names like Eli Lil­ly to fund their work on one of the most press­ing chal­lenges in gene ther­a­py: what Lee calls the “de­liv­ery prob­lem.”

One of the big is­sues with ade­no-as­so­ci­at­ed virus­es (AAV) — the nanome­ter-sized de­liv­ery sys­tem used in cur­rent gene ther­a­pies — is that some pa­tients have a nat­ur­al im­mu­ni­ty. Or, once they’re dosed with an AAV-based ther­a­py, they mount an im­mune re­sponse, mak­ing it dif­fi­cult to re-dose.

GenEd­it, though, is work­ing on a non-vi­ral, non-lipid poly­mer nanopar­ti­cle ap­proach, which could make it pos­si­ble to re-dose gene ther­a­pies, among oth­er ben­e­fits. Their NanoGalaxy plat­form con­sists of a large li­brary of chem­i­cal­ly made poly­mers, which are screened for their abil­i­ty to se­lec­tive­ly tar­get dif­fer­ent tis­sues and cell types. The nanopar­ti­cles can car­ry DNA, RNA, or CRISPR ri­bonu­cle­o­pro­tein, CSO Ro­muald Cor­bau told End­points News, de­pend­ing on whether sci­en­tists are look­ing to delete, ed­it or si­lence a gene.

Ro­muald Cor­bau

Be­cause the nanopar­ti­cles are chem­i­cal­ly made, pa­tients won’t have a pre-ex­ist­ing im­mu­ni­ty, or gen­er­ate an an­ti­body re­sponse, he said. Plus, the par­ti­cles can car­ry more car­go, and come with a low­er man­u­fac­tur­ing cost com­pared to tra­di­tion­al AAV.

The abil­i­ty to re-dose could al­low sci­en­tists to treat some peo­ple with a low­er dose to start, po­ten­tial­ly avoid­ing some of the safe­ty is­sues in­volved with giv­ing high­er dos­es.

“We have gen­er­at­ed the very first sets of the da­ta that the it­er­a­tion process works,” Lee said of the com­pa­ny’s in vi­vo de­liv­ery ap­proach, adding that pre­clin­i­cal da­ta sug­gest the nanopar­ti­cles can be dosed mul­ti­ple times while main­tain­ing func­tion­al ac­tiv­i­ty of the pay­load.

While Lee de­clined to pro­vide a time­line for GenEd­it’s push to­ward the clin­ic, Cor­bau said the Se­ries A will be used in part to de­vel­op an IND-ready drug. He didn’t say much about ini­tial in­di­ca­tions, on­ly giv­ing away that the com­pa­ny will be fo­cus­ing on dis­eases of the ner­vous sys­tem.

More im­por­tant­ly, the Se­ries A funds will be used to build out the rough­ly 30-per­son team. Cor­bau joined GenEd­it about a month ago — less than a year af­ter be­ing pro­mot­ed to CSO at the AAV-based gene ther­a­py com­pa­ny Free­line. Pri­or to Free­line, he served as the trans­la­tion­al lead at Spark Ther­a­peu­tics, where he worked on Lux­tur­na, the first-ap­proved gene ther­a­py. Lee al­so re­cent­ly an­nounced the ad­di­tion of Aaron Mishel, who left mol­e­c­u­lar imag­ing leader Mag­net­ic In­sight to take the CFO spot at GenEd­it.

The crowd of com­pa­nies search­ing for gene ther­a­py 2.0 is on­ly get­ting larg­er. An­jar­i­um un­cloaked ear­li­er this month with back­ing from Pfiz­er’s ven­ture arm to de­vel­op DNA-based gene vec­tors. Just a few months ago, Glax­o­SmithK­line vet Bri­an McVeigh launched Code Bio­ther­a­peu­tics to find a non­vi­ral de­liv­ery method. And Gen­er­a­tion Bio closed a $230 mil­lion IPO last June to fund its pre­clin­i­cal push for a non-vi­ral gene ther­a­py for dis­eases of the liv­er and reti­na.

Eli Lil­ly, KTB Net­work, Com­pa­ny K Part­ners, Ko­rea In­vest­ment Part­ners, DAYLI Part­ners, KB In­vest­ment, IMM In­vest­ment, TIME­FO­LIO As­set Man­age­ment, DCVC Bio, SK Hold­ings, Bow Cap­i­tal, and Se­quoia Cap­i­tal all chipped in­to GenEd­it’s lat­est round.

“There are so many oth­er in­di­ca­tions that we are cur­rent­ly not able to ap­ply (gene ther­a­pies), but if there is a de­liv­ery tech­nol­o­gy, we can un­lock all of those parts,” Lee said.

Illustration: Assistant Editor Kathy Wong for Endpoints News

How Pur­due's $272M ad­dic­tion pay­out fund­ed a new home for its dis­card­ed non-opi­oid re­search

Don Kyle spent more than 20 years working for Purdue Pharma, right through the US opioid epidemic that led to the company’s rise and eventual infamy. But contrary to Purdue’s focus on OxyContin, Kyle was researching non-opioid painkillers — that is, until the company shelved his research.

As the company’s legal troubles mounted, Kyle found an unlikely way to reboot the project. In 2019, he took his work to an Oklahoma State University center that’s slated to receive more than two-thirds of the state’s $272 million settlement with Purdue over claims that the drugmaker’s behavior ignited the epidemic of opioid use and abuse.

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President Joe Biden at the State of the Union address with Vice President Kamala Harris and House Speaker Kevin McCarthy (Patrick Semansky/AP Images)

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President Joe Biden made clear in his “finish the job” State of the Union address last night that one of those jobs to be finished is insulin prices.

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Up­dat­ed: R&D tur­bu­lence at Bris­tol My­ers now in­cludes the end of a $650M al­liance and the de­par­ture of a top re­search cham­pi­on

This morning biotech Dragonfly put out word that Bristol Myers Squibb has handed back all rights to its IL-12 clinical-stage drug after spending $650 million to advance it into the clinic.

The news arrives amid a turbulent R&D stage for the pharma giant, which late last week highlighted Rupert Vessey’s decision to depart this summer as head of early-stage R&D following a crucial three-year stretch after he jumped to Bristol Myers in the big Celgene buyout. During that time he struck a series of deals for Bristol Myers, and also shepherded a number of Celgene programs down the pipeline, playing a major role for a lineup of biotechs which depended on him to champion their drugs.

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Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

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But Bill Haney, the CEO of Dragonfly, is taking issue with that.

The early-stage drug, still in Phase I development, has passed muster with Bristol Myers’ general clinical expectations, advancing successfully while still in Phase I, he says.

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Utpal Koppikar, new Verily CFO

Ex­clu­sive: Ver­i­ly wel­comes Atara Bio­ther­a­peu­tics vet­er­an as new CFO

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Utpal Koppikar joins Verily after a nearly five-year stint as CFO and senior VP at Atara, though his résumé also boasts roles at Gilead and Amgen.

The news follows a major reshuffling at Verily, including several senior departures earlier this year and a round of layoffs.

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Singer Nick Jonas is back at work for Dexcom, this time for its new G7 glucose monitor.

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Richard Francis, newly-appointed Teva CEO (Novartis via Facebook)

New Te­va CEO Richard Fran­cis repri­or­i­tizes to 'get back to growth'

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FDA Commissioner Robert Califf on Capitol Hill, Feb. 8, 2023 (Drew Angerer/Getty Images)

FDA com­mis­sion­er floats ideas on how to bet­ter han­dle the pan­dem­ic

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Sanofi is renewing its #VaccinesForDreams campaign with more stories, such as Juan's in Argentina (Sanofi)

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The campaign ties to Sanofi’s broader umbrella initiative “Vaccine Stories” to promote the value of vaccines and drive awareness of the need for improved vaccination coverage.

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