A gene ther­a­py pi­o­neer moves from Bio­gen to Re­genxbio, stay­ing fo­cused on gene ther­a­py 2.0


Olivi­er Danos’ ar­rival as head of Bio­gen’s gene ther­a­py group two-and-a-half years ago sig­naled an im­por­tant shift in the big biotech’s R&D fo­cus. In swift or­der Bio­gen ex­e­cut­ed a part­ner­ship with the San Raf­faele-Telethon In­sti­tute for Gene Ther­a­py, inked a $1 bil­lion-plus deal with AGTC and fol­lowed up with a $2 bil­lion pact to work with James Wil­son and Jean Ben­nett at the Uni­ver­si­ty of Penn­syl­va­nia.

Along the way, Danos picked up some tai­lor-made vec­tor tech at Re­genxbio.

On Fri­day, Danos wrapped up his job at Bio­gen. On Mon­day, he was the new CSO at Re­genxbio. But his mis­sion stays re­mark­ably the same.

Danos has spent decades in the field, start­ing out work­ing with Wil­son in Richard Mul­li­gan’s Har­vard lab back in the ‘80s. And while he’s left Bio­gen, he’ll be work­ing more close­ly than ever with Wil­son, the sci­en­tif­ic founder of Re­genxbio and an­oth­er high pro­file ground­break­er in the field who helped cre­ate the gene ther­a­py vec­tors that Re­genxbio has been li­cens­ing out to a new wave of play­ers in gene ther­a­py.

“Bio­gen was for me a very good en­vi­ron­ment,” Danos tells me. He says he was pleased by the trans­la­tion­al sci­ence and in­no­va­tion he found at Bio­gen. “At the same time, I re­al­ized that the fo­cus of a small­er struc­ture en­tire­ly ded­i­cat­ed to gene ther­a­py is in a way a more ef­fi­cient way to go.”

“The con­cept (at Bio­gen) evolved a bit re­cent­ly,” he adds. “Ba­si­cal­ly all the fo­cus in gene ther­a­py was fo­cus­ing on clin­i­cal pro­grams we had start­ed. That was ex­clud­ing the more up­stream re­search pro­grams I be­lieve are still sore­ly need­ed.”

Re­genxbio start­ed out act­ing as a cen­tral ware­house for its vec­tor tech IP, help­ing get a num­ber of new com­pa­nies off the ground as they set out for the clin­ic. But then the com­pa­ny mor­phed, start­ed work on its own pipeline and went pub­lic as in­vestor in­ter­est in gene ther­a­py reached its peak.

To­day, Spark Ther­a­peu­tics is the most ad­vanced in the field, the clos­est to a pos­si­ble FDA ap­proval. But Spark and oth­ers are still grap­pling with im­mune re­spons­es and oth­er chal­lenges that high­lights the need for new ad­vances in the sci­ence.

“The name of the game has al­ways been about de­liv­ery,” says Danos. “We are go­ing to keep work­ing on de­liv­ery,” he adds, an­swer­ing ques­tions such as how do you de­liv­er genes to the CNS in an op­ti­mal fash­ion.

“The im­por­tant move for me oc­curred a num­ber of years ago when I de­cid­ed to shift from acad­e­mia to in­dus­try,” says Danos, who’s al­so the sci­en­tif­ic founder of Paris-based Lyso­gene. And while his em­ploy­er may have changed, his sci­en­tif­ic goals re­main in place.

Gene ther­a­py 2.0 is still in the lab.

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.

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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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.