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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Yao-Chang Xu, Abbisko Therapeutics founder and CEO

Qim­ing-backed Ab­bisko makes $200M+ Hong Kong de­but, as a SPAC and Agenus spin­out al­so price on Nas­daq

Three new entities priced their public debuts late Thursday and early Friday, including a SPAC, a traditional Nasdaq IPO and a Chinese biotech joining the Hong Kong Index.

Shanghai-based Abbisko Therapeutics raised the most money of the triumvirate, garnering $226 million in its Hong Kong debut and pricing at HK$12.46, or roughly $1.60 in US dollars. The blank check company followed up with a $150 million raise, while MiNK Therapeutics priced on Nasdaq at $12 per share and a $40 million raise.

Paul Grayson, Tentarix CEO (Versant)

Phar­ma vet­er­ans re­group with $50M and a plan to dis­cov­er new mul­ti-specifics

While a horde of drugmakers develops bispecific antibodies to more directly target tumor cells — there were about 100 programs in or nearing clinical trials back in May — a new company is emerging to go one step further.

On Thursday, Tentarix Biotherapeutics unveiled a $50 million Series A round to support its next-gen multi-specifics platform. While the field has largely focused on bispecifics, which engage two targets, Tentarix believes its multifunctional programs have the potential to be even more specific, since more conditions must be met for potent activity to occur.

Tillman Gerngross, Adagio CEO

Q&A: Till­man Gern­gross ex­plains why his Covid mAb will have an edge over an al­ready crowd­ed field

If anyone knows about monoclonal antibodies, it’s serial entrepreneur, Adimab CEO, and Dartmouth professor of bioengineering Tillman Gerngross.

Even the name of Gerngross’ new antibody startup Adagio Therapeutics is meant to reflect his vision behind the development of his Covid-19 mAb: slowly, he said, explaining that “everyone else, whether it’s Regeneron, Lilly, or AstraZeneca, Vir, they all valued speed over everything.”

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