Geoff McDonough, Generation Bio

With $110M to add to the bankroll, Gen­er­a­tion Bio sets its sights on en­gi­neer­ing a rev­o­lu­tion in the gene ther­a­py field

Who­ev­er comes out on top of the cur­rent race to gain pi­o­neer­ing ap­provals for new AAV-de­liv­ered gene ther­a­pies will have to look over their shoul­ders to watch the next tech wave form­ing on the hori­zon for gene ther­a­py 2.0.

One of those next-gen play­ers, Gen­er­a­tion Bio, just brought in $110 mil­lion of ven­ture cash to cov­er the cost of the rest of their pre­clin­i­cal jour­ney to­ward some­thing com­plete­ly new in the field. The lat­est round brings the biotech — which now has about 80 staffers — up to $235 mil­lion in to­tal since its in­cep­tion about 3 years ago. That will fu­el the rest of its pre­clin­i­cal stage of de­vel­op­ment as it looks to break in­to hu­man stud­ies in the back half of 2021.

That kind of 4-plus year time­line be­fore the first hu­man dos­ing could test the en­durance lev­el of a ven­ture play­er. But Gen­er­a­tion CEO Ge­off Mc­Do­nough looks over the past 2 years ad­vanc­ing a new lipid nanopar­ti­cle de­liv­ery sys­tem for their closed-end DNA ther­a­pies — work­ing to the day when gene ther­a­pies can be pro­duced and sold for far less than the $2 mil­lion-or-so price tag to­day — and sees lots of fast-paced ad­vances.

“I think the re­al­i­ty is we didn’t have an ex­pec­ta­tion at the out­set (on time­lines),” Mc­Do­nough tells me. Rec­og­niz­ing the nov­el work need­ed to build the plat­form, the in­vestors knew it would take time and mon­ey to bring them up to a GMP lev­el.

“I would say for a 40-year prob­lem,” adds the CEO, “2 years seems pret­ty good.”

The found­ing tech at Gen­er­a­tion was de­signed to do what AAV treat­ments do in the nu­cle­us, of­fer­ing en­dur­ing ex­pres­sion, while al­low­ing man­u­fac­tur­ing at a bi­o­log­ic scale with a more eco­nom­i­cal, cap­sid-free pro­duc­tion method. Tak­ing a page from the tech hand­books at com­pa­nies like Al­ny­lam and Mod­er­na, they’re build­ing a gene ther­a­py that they be­lieve can do much bet­ter than the frag­ile, one-time-on­ly pi­o­neers. And with­out the $1 mil­lion pro­duc­tion cost that keeps whole­sale prices in the low 7-fig­ure range.

They’re look­ing for much greater econ­o­my, even­tu­al­ly tak­ing these ther­a­pies to much broad­er ail­ments and out of the realm of rare dis­eases with a new ap­proach that they be­lieve can be in­fi­nite­ly re­dos­able — on an as-need­ed ba­sis.

That’s the big pic­ture.

Gen­er­a­tion’s team is work­ing on 2 lead pro­grams for he­mo­phil­ia A and phenylke­tonuria (PKU) to go in­to IND-en­abling stud­ies. They’ve now iden­ti­fied Wil­son dis­ease and Gauch­er dis­ease as like­ly start­ing points for the next steps as they move past the liv­er to skele­tal mus­cle and the reti­na and then oth­er tis­sues. And Mc­Do­nough — the for­mer CEO at So­bi — is look­ing down the road 12 to 18 months when he’d like to turn to the pub­lic mar­kets with an IPO to fund the first clin­i­cal-stage work.

In the mean­time, he’d like to con­cen­trate on open­ing an­oth­er new chap­ter of the com­pa­ny on the deal­mak­ing side.

“It felt very im­por­tant not to part­ner” ini­tial­ly,  says Mc­Do­nough. The in­vestors want­ed to re­tain own­er­ship of plat­form. “We just had tremen­dous good for­tune we didn’t need to do that for fi­nance rea­sons.” But now that they have a bet­ter grasp of the tech­nol­o­gy and what needs to be done, it’s time to part­ner — prob­a­bly lat­er in the year.

T. Rowe Price funds and ac­counts led the round, with Far­al­lon and Welling­ton Man­age­ment Com­pa­ny jump­ing in along­side. Ex­ist­ing in­vestors At­las Ven­ture, Fi­deli­ty, In­vus, Cas­din, Deer­field, Fore­site Cap­i­tal and an en­ti­ty as­so­ci­at­ed with SVB Leerink came back to stay in the syn­di­cate. Cowen served as ex­clu­sive place­ment agent for the of­fer­ing.

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:

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Steffen Schuster, ITM CEO

Ra­dio­phar­ma re­mains hot as Ger­many's ITM rais­es $109M to ad­vance neu­roen­docrine can­cer pro­gram

The world of radiopharmaceuticals has been heating up over the last few years, and Thursday saw another company focused on the field pull in a new nine-figure raise.

Germany’s ITM, or Isotopen Technologien München, scored a $109 million round of loan financing to push forward its precision oncology pipeline and fund late-stage development for its lead program. As part of the agreement, the loan will convert to shares in the event of future financial or corporate transactions, ITM said.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 107,400+ biopharma pros reading Endpoints daily — and it's free.

Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

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.

Bay­er plots a ma­jor facelift at Berke­ley cam­pus, un­cork­ing a 30-year, $1.2B plan to dri­ve cell and gene ther­a­pies

Bayer first set roots in Berkeley back in 1974, when it was still operating as Miles Labs. The site has pumped out three hemophilia A treatments for distribution worldwide; but now, as the pharma continues its cell and gene therapy push, it has something bigger in mind.

Bayer is planning a 30-year revamp at the campus, which includes 918,000 square feet in new buildings and double the jobs, according to a report by the Bay Area Council Economic Institute.