Joe Nolan, Jaguar

Sean Nolan gath­ers AveX­is alum­ni to launch an­oth­er biotech. Is it an­oth­er race-to-IPO?

The old AveX­is crew is launch­ing an­oth­er com­pa­ny. And they may once again look to cash in quick.

Armed with patents and a steady stream of cash from Deer­field Man­age­ment, Sean Nolan and five ex-AveX­is ex­ec­u­tives have launched Jaguar Gene Ther­a­py to de­vel­op AAV9 gene ther­a­pies for a small con­stel­la­tion of dis­parate dis­or­ders, from autism to di­a­betes. Jaguar re­mains over a year from the clin­ic, but CEO Joe Nolan hint­ed they could look to fol­low a pace set by Ta­sy­ha, the biotech Sean Nolan and oth­er AveX­is alum­ni launched last year and took from Se­ries A to IPO in less than 6 months.

Sean Nolan

“It’s hard to say, ob­vi­ous­ly we’re re­al­ly fo­cused on what we’re do­ing, but I do think we can quick­ly move to oth­er fund­ing sources,” Joe Nolan (no re­la­tion to Sean) told End­points News. “I don’t know if we’ll get to IPO that fast, but, you know, we’ll see.”

The idea for Jaguar came from Deer­field, who had as­sem­bled a col­lec­tion of in­tel­lec­tu­al prop­er­ty on gene ther­a­py and went look­ing for an ex­ec­u­tive team around the time many of the for­mer AveX­is ex­ec­u­tives were leav­ing No­var­tis. Deer­field has since pumped around $40 mil­lion in­to the com­pa­ny as Jaguar has ex­pand­ed to a team of about 30 in Lake For­est, Illi­nois and North Car­oli­na.

Their gene ther­a­py for a ge­net­ic cause of autism came out of one of the col­lab­o­ra­tions Deer­field has been sign­ing with uni­ver­si­ties across the coun­try over the last few years. And last year Deer­field li­censed a gene ther­a­py for the rare neu­ro-meta­bol­ic dis­ease Bardet-Biedl Syn­drome from the Uni­ver­si­ty of Col­lege Lon­don. That ther­a­py will be de­vel­oped by Ax­ovia Ther­a­peu­tics, a ma­jor­i­ty-owned sub­sidiary of Jaguar.

So far, though, the com­pa­ny is re­main­ing tight-lipped on specifics. Nolan wouldn’t say which of the sev­er­al po­ten­tial ge­net­ic tar­gets they would go af­ter in autism or even which uni­ver­si­ty it came from. Nor would he dis­close the tar­get they’ll go af­ter for type 1 di­a­betes — a dis­ease aca­d­e­mics have the­o­rized tar­get­ing with gene ther­a­py in a va­ri­ety of ways — or where they li­censed that ther­a­py from.

The com­pa­ny’s pitch rests large­ly on AveX­is’s rep­u­ta­tion in AAV gene ther­a­py. Nolan said they bring ex­per­tise from de­vel­op­ment to man­u­fac­tur­ing and com­mer­cial­iza­tion.

“We have a lot of ex­pe­ri­ence,” Nolan said. “We be­lieve it’s a proven and re­li­able way to min­i­mize de­vel­op­ment risk, we have a suc­cess­ful track record there and we think we know the hur­dles ahead be­cause we’ve been through it be­fore.”

The AAV field has tak­en a hit in the last year, af­ter the death of three pa­tients in one high-dose Au­dentes tri­al and pos­si­ble safe­ty sig­nal in oth­ers. Nolan said they viewed those is­sues as be­ing spe­cif­ic to those spe­cif­ic con­structs as op­posed to a broad­er is­sue with AAV gene ther­a­py, though he added that the com­pa­ny wouldn’t be push­ing the dose as high as Au­dentes had.

“There’s an art to not push­ing the dose, par­tic­u­lar­ly in IV,” he said.

Suku­mar Na­gen­dran

Nolan, an Ab­bot and Take­da vet­er­an, was hired in 2018 as a gen­er­al man­ag­er at AveX­is, where he helped de­sign the com­mer­cial launch plan for Zol­gens­ma. He says he keeps a pic­ture of the first kids they treat­ed on his night­stand, next to pic­tures of his own. Suku­mar Na­gen­dran, who was CMO at AveX­is, has joined as an R&D chief. Sean Nolan is chair­man of the board.

The first ther­a­py they plan to put in the clin­ic is a gene ther­a­py for Bardet-Biedl Syn­drome, with an IND planned for the sec­ond half of 2022.

Should we ex­pect Sean Nolan to launch more biotechs in the mean­time?

“I don’t think so,” Joe Nolan said, be­fore adding: “Sean’s ob­vi­ous­ly pop­u­lar, I can’t speak to his fu­ture, but I can say he is ex­cit­ed about this one.”

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Emma Walmsley, GlaxoSmithKline CEO (Kevin Dietsch/Pool via CNP/Alamy)

Glax­o­SmithK­line hus­tles the 7th PD-1 past the fin­ish line with Jem­per­li. But how big will up­take be?

Everything came up sevens for GlaxoSmithKline on Thursday as the pharma notched the seventh PD-1 approval seven years after the first such drugs were OK’ed in Keytruda and Opdivo. But will it bring GSK good fortune?

The FDA granted accelerated approval to dostarlimab, to be branded Jemperli, to treat recurrent or advanced endometrial cancer in a specific subset of patients following platinum-based chemo. It’s a drug that came to GSK through its buyout of Tesaro, which it snapped up for $5.1 billion back in December 2018.

From left: James Brown, Michael Chambers, John Ballantyne

Alde­vron founders back a biotech start­up that's look­ing to end the moral de­bate over cell lines once and for all

For millions of Catholics around the world, the development of new vaccines to combat Covid-19 has sparked a moral dilemma. All the approved vaccines in use relied — in some fashion — on cell lines that were derived from aborted fetal tissue.

While church leaders accepted the vaccines and recommended their use to end the pandemic, a number also highlighted their preference for the mRNA vaccines from Pfizer/BioNTech and Moderna over the J&J and AstraZeneca shots, which they noted were more heavily dependent on cell lines that they found morally objectionable.

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”