At­las builds its dream team and hands over $25M to launch a new ap­proach to gene ther­a­py

Robert Kotin

Robert Kotin is one of the world’s top ex­perts on the vi­ral trans­port of gene ther­a­pies. Ex-NIH, ex-Voy­ager ex­ec, he’s played a siz­able role in shap­ing the field.

So when Kotin says there’s a bet­ter way to do gene trans­fer with­out a vi­ral ve­hi­cle, he man­aged to get the at­ten­tion of quite a few peo­ple in the Boston/Cam­bridge hub.

Ja­son Rhodes, a part­ner at At­las, was one of them. At­las formed a com­pa­ny around this called Gen­er­a­tion Bio with a $25 mil­lion A round and Rhodes is chair­ing the board. Ge­off Mc­Do­nough, who stepped aside as CEO of Stock­holm-based So­bi last sum­mer af­ter the com­pa­ny de­cid­ed to go with a new chief who pre­ferred to live clos­er to home, is tak­ing the helm.

Ge­of­frey Mc­Do­nough

Like Ed Kaye, who’s hav­ing his launch par­ty to­day at Stoke, Mc­Do­nough is a high pro­file fig­ure in the Mass­a­chu­setts hub. And he’s pumped about pur­su­ing a break­through ap­proach to gene trans­fer.

Kotin’s big idea cen­ters on what he calls close-end­ed DNA (ceD­NA), which can move from the cy­to­plasm of the cell to the nu­cle­us with­out that AAV trans­port ve­hi­cle. Once in­side the nu­cle­us, the the­o­ry goes, it can cre­ate durable high lev­els of gene ex­pres­sion ca­pa­ble of ad­dress­ing a broad range of ge­net­ic dis­eases. And the biotech plans to get start­ed by us­ing a lipid nanopar­ti­cle to de­liv­er ceD­NA to the liv­er, where it will make sys­temic pro­teins to fight dis­ease.

“The first or­gan is the liv­er be­cause that’s where lipid nanopar­ti­cles go,” Mc­Do­nough told me in a pre­view to to­day’s an­nounce­ment. “So we go to rare dis­eases of the liv­er, where it can be a fac­to­ry.”

And nanopar­ti­cles have some in­her­ent qual­i­ties that make it prefer­able to an AAV ap­proach, with a big­ger pay­load that can pack a big­ger punch. It’s al­so a new way to do this with­out trig­ger­ing an im­mune re­sponse that is lim­it­ing the first gen­er­a­tion of gene ther­a­pies to a one-time use.

Notes Mc­Do­nough: “This is a com­plete­ly nov­el modal­i­ty which is tremen­dous­ly dis­rup­tive to AAV.”

Ja­son Rhodes

While much of At­las’ in­vest­ing can be high­ly as­set-cen­tric, craft­ing com­pa­nies around one or two lead pro­grams in a care­ful­ly man­aged dri­ve to the clin­ic, Gen­er­a­tion Bio is every bit a plat­form com­pa­ny with a lot of po­ten­tial di­rec­tions to move. But Rhodes says you can’t rel­e­gate a ven­ture group like At­las — which has en­joyed the abil­i­ty to raise con­sid­er­able funds — to a nar­row fo­cus. The VC, he says, is “sci­ence-first, seed-led, prag­mat­ic about the busi­ness mod­el and in­tense­ly fo­cused on cre­at­ing in­no­v­a­tive new prod­ucts. And not too wor­ried about ide­ol­o­gy be­yond that.”

In re­sponse to a query from me, Rhodes added:

We al­ways match the na­ture of the op­por­tu­ni­ty to the busi­ness mod­el and build both ma­jor plat­form com­pa­nies and more fo­cused prod­uct plays. This strate­gic match­ing is a hall­mark of At­las’ ap­proach vs a cook­ie cut­ter, whether al­ways a mon­ster plat­form or al­ways an as­set play, etc. Re­cent plat­forms in­clude NT­LA, Sur­face, Ma­gen­ta, Trans­late, Nim­bus (with an LLC struc­ture in the mix), etc. Ear­ly M&A tends to oc­cur with the prod­uct-ori­ent­ed com­pa­nies, like Delinia, Pad­lock, and IFM and so gen­er­ates ear­ly press and at­ten­tion. Pad­lock is re­al­ly a plat­form around an area of bi­ol­o­gy as is IFM, which sold the onc port­fo­lio and kept the im­munol­o­gy port­fo­lio. We do con­sis­tent­ly use a seed ap­proach, where we pres­sure test the core sci­ence, be­gin build­ing a team etc. then ramp us the re­spec­tive busi­ness mod­el. I’ve been in­volved with all of these kinds of com­pa­nies whether at Al­ny­lam (new modal­i­ty plat­form), Epizyme (new bi­ol­o­gy mul­ti-prod­uct plat­form), Replim­ine (prod­uct play, though more than one), Dis­arm (new bi­ol­o­gy prod­uct), and Gen­er­a­tion, which is ob­vi­ous­ly a ma­jor new modal­i­ty, among a few ex­am­ples.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.