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.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

On Thursday evening in Boston I had the great good fortune to talk about the creation of the biotech industry with Nobel Prize-winning scientist Phil Sharp. I learned quite a bit about the early days of Genentech, Biogen and Alnylam, which all helped birth this unusual drug development ecosystem. And that’s why we can do things like the Endpoints 11. Here’s my talk with Phil Sharp, which you can either watch or read below.

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Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

Neil Desai, Aadi Bioscience CEO (via YouTube)

Pre­ci­sion on­col­o­gy biotech agrees to $72M cash in­fu­sion as it seeks to in­crease rev­enue

Almost a year after the FDA gave the green light to LA-based Aadi Bioscience’s first drug, the biotech is looking to private investors to keep itself going.

The oncology player announced Thursday that it has engaged with both new and existing investors in a PIPE financing — selling 3.3 million shares at $12.50 a share, the biotech’s closing price at Nasdaq on Wednesday. The company is also selling off pre-funded warrants to purchase over 2.4 million more shares at $12.4999 per pre-funded warrant.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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