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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

George Church (courtesy EnPlusOne BioSciences)

George Church, Wyss sci­en­tists and North­pond chal­lenge con­ven­tion­al RNA man­u­fac­tur­ing with new biotech

RNA medicine has been at the forefront for the past few years, with the first RNA silencing therapy approved in 2018, and mRNA Covid vaccines following after. But flying under the radar has been the process of actually making RNA for these treatments.

That’s what Daniel Wiegand and Jonathan Rittichier have been working on in George Church’s lab for the past six years.

Friday morning, they unveiled EnPlusOne Biosciences, a biotech built on their RNA synthesis platform. Wiegand will serve as the Watertown, MA-based biotech’s CEO, and Rittichier will be CSO. And no different from his other startups, Church will be acting as scientific advisor. Its fourth co-founder, Dan Ahlstedt, joined through a Harvard Business School program, and will be COO.

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