Out to pi­o­neer gene edit­ing in Chi­na, Edi­Gene bags $67M to be­gin clin­i­cal test­ing for β-tha­lassemia ther­a­py, uni­ver­sal CAR-T

A gene edit­ing play­er hail­ing from Bei­jing has clinched $67 mil­lion to steer two of its lead can­di­dates in­to the clin­ic — and to prove it­self as a CRISPR pi­o­neer in Chi­na.

Dong Wei

Edi­Gene gath­ered the Se­ries B from a slate of top-tier Chi­nese in­vestors, in­clud­ing 3H Health In­vest­ment (who led the round), Se­quoia Cap­i­tal Chi­na, Al­win Cap­i­tal and Kun­lun Cap­i­tal. Pre­vi­ous back­ers, in­clud­ing IDG Cap­i­tal, Lil­ly Asia Ven­ture, Hua­gai Cap­i­tal and Green Pine Cap­i­tal Part­ners, al­so par­tic­i­pat­ed.

With the new cash, the biotech has of­fi­cial­ly crossed the $100 mil­lion mark in fundrais­ing over the past two years, ac­cord­ing to CEO Dong Wei.

“The round en­ables us to fur­ther scale up and trans­form our pipeline in­to clin­i­cal-stage, which is al­so a big step for­ward in build­ing a glob­al­ly com­pet­i­tive gene edit­ing com­pa­ny,” Wei said in a state­ment.

CRISPR may have had a bad rep in Chi­na in the af­ter­math of a scan­dalous hu­man ex­per­i­ment in which Jiankui He — who’s since been cast as a rogue sci­en­tist and sen­tenced to three years in jail — cre­at­ed the world’s first known gene edit­ing ba­bies by tin­ker­ing with a gene of an em­bryo in an at­tempt to con­fer pro­tec­tion against HIV. But Edi­Gene, which was launched in 2015 and has set up a BD of­fice in Cam­bridge, MA in ad­di­tion to head­quar­ters in Bei­jing and a GMP & ap­pli­ca­tion cen­ter in Guangzhou, clear­ly en­joys a dif­fer­ent po­si­tion.

“Edi­Gene is lead­ing the wave in Chi­na, as they have es­tab­lished out­stand­ing re­search and de­vel­op­ment ca­pa­bil­i­ties, and more im­por­tant­ly, they have de­vel­oped an im­pres­sive port­fo­lio of pro­pri­etary gene edit­ing tools and prod­uct can­di­dates,” said Minchuan Wang, a part­ner of 3H Health In­vest­ment.

Wen­sheng Wei

An ear­ly San Fran­cis­co trans­plant, Wei be­gan his biotech ca­reer at Ch­i­ron, took a de­tour in trad­ing and con­sult­ing, then worked his way through a slew of posts at Bio­Marin, Elan and J&J. His last role in the US — be­fore de­cid­ing in 2018 to move back and spend more time with fam­i­ly in Chi­na — was Shire’s glob­al de­vel­op­ment team leader for im­munol­o­gy.

It was nat­ur­al to then join Wen­sheng Wei, a co-founder of Edi­Gene and Dong Wei’s close friend from Peking Uni­ver­si­ty who end­ed up be­ing a PhD bud­dy at Michi­gan State Uni­ver­si­ty. By that time, Wen­sheng Wei was a pro­fes­sor at Peking Uni­ver­si­ty’s School of Life Sci­ences and had tasked Dong Wei with find­ing a CEO for Edi­Gene af­ter try­ing, in vain, to re­cruit him to the job.

The com­pa­ny boasts of four dif­fer­ent plat­forms re­volv­ing around CRISPR gene edit­ing tech: ex vi­vo edit­ing for hematopoi­et­ic stem cells, ex vi­vo edit­ing to cre­ate CAR-T cells, in vi­vo RNA-based edit­ing, as well as high-through­out genome edit­ing screen­ing for drug dis­cov­ery. Right now, the key pri­or­i­ties are ET-01 for se­vere β-tha­lassemia and U CAR-T ET-02 for can­cer.

Both are in­di­ca­tions where US biotechs have done ground­break­ing work, Dong Wei ac­knowl­edged in an in­ter­view with Pharm­Cube this March. Zyn­te­glo, blue­bird bio’s ex vi­vo ther­a­py for β-tha­lassemia, has al­ready been ap­proved in the EU.

But when he asked a friend — a blue­bird ex­ec — about whether that leaves any room for Edi­Gene on the mar­ket, the an­swer he got was blunt: Even though the Chi­nese pa­tient pop­u­la­tion is large, the coun­try’s pric­ing sys­tem and the re­quired in­vest­ment for man­u­fac­tur­ing doesn’t jus­ti­fy putting mon­ey be­hind it.

“When the com­pa­ny with the best tech­nol­o­gy in the world be­lieves there is no in­vest­ment val­ue in do­ing this in Chi­na, what should we do?” Wei said. “Are we just not go­ing to do it ei­ther?”

Ul­ti­mate­ly, he added, it all comes down to the ecosys­tem. In terms of the ac­tu­al gene edit­ing tech­nol­o­gy, he reck­ons that Chi­na is on par with in­ter­na­tion­al stan­dards — thanks to a serendip­i­tous over­lap in the tim­ing for de­vel­op­ment of gene edit­ing and the Chi­nese gov­ern­ment’s em­pha­sis on the biotech in­dus­try.

But the US is still way out front in terms of trans­la­tion, he not­ed:

For ex­am­ple, when a pro­fes­sor pub­lish­es a pa­per, you’d soon see re­searchers across Boston and San Fran­cis­co Bay Area fol­low­ing up on the tech­nol­o­gy. This is the aug­men­ta­tion ef­fect; or for ex­am­ple, 90% of VC in­vest­ment re­lat­ed to gene edit­ing is in Boston and 10% is in San Fran­cis­co — this is the clus­ter­ing ef­fect; and then if you look at the whole biotech field, San Fran­cis­co and Boston are es­sen­tial­ly equal, and to­geth­er they take up 80% of all Amer­i­can ven­ture in­vest­ing. All these are ecosys­tem ef­fects.

Mean­while in the US, say Wen­sheng pub­lished a new pa­per on gene edit­ing. Six months lat­er you’d sel­dom see oth­er labs ap­ply­ing this new tech to cre­ate new­er in­no­va­tion. This is like we con­quered a city, but didn’t have time to build the city in­to a bet­ter place.

At 5 years old and 100-plus em­ploy­ees, though, the con­struc­tion crew at Edi­Gene is ready to roll up their sleeves.

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, 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.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

Paul Hudson, Getty Images

How does Paul Hud­son's $13.5M comp pack­age stack up against oth­er CEOs? He's in the 'first quar­tile'

Paul Hudson arrived at Sanofi like a hurricane, chopping off duds in the pipeline, shaking up the C-suite, striking big M&A deals and jumping into the Covid-19 vaccine race — all in an attempt to reboot a pharma giant notorious for its setbacks.

Now, we’re getting a look at what the CEO brought home in his first year on the job.

When all is said and done, Hudson will have made about $6.7 million in 2020, about $2.5 million of which has already been paid. The bigger figure includes a $2.3 million bonus that’s subject to approval at an April meeting, and another $1.8 million in variable compensation that has yet to be paid.

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Ab­b­Vie of­floads UK site for $119M in sale to Chi­nese cell and gene ther­a­py play­er Phar­maron

With its Allergan buyout now long in the books, AbbVie has been taking a hard look at its suddenly expansive global ops to find space for a deal. Now, working with a Chinese cell and gene therapy player hungry for more elbow room abroad, AbbVie has taken one UK facility off its books.

AbbVie has offloaded its Liverpool manufacturing site as part of a $118.7 million sale to Chinese cell and gene therapy player Pharmaron, which is pitching the purchase as the next step in its global expansion plans, the companies said last week.

An Ar­ray co-founder re-emerges as CEO of a small aca­d­e­m­ic spin­out, look­ing to re­make an old class of can­cer drugs

Tony Piscopio hadn’t worked as a bench scientist in years when, around 2011, he got put in touch with a team at the University of Colorado trying to revitalize an old approach to treating cancer.

Piscopio, who had co-founded Array Biopharma before heading to South Korea to launch a new company, was back in the states, unattached and intrigued. He founded a three-person company with two professors, Xuedong Liu and Gail Eckhardt, and while they worked on the biology side, he returned to his old chemist chair and began drawing up potential compounds on a computer, along with manufacturing processes to make them. Outsourcing companies synthesized or analyzed the results.

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