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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

Endpoints Premium

Premium subscription required

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

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Mar­ket­ingRx roundup: Ab­b­Vie’s Hu­mi­ra TV turns fo­cus to HS skin con­di­tion; Sanofi amps par­ent­ing pol­i­cy

After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

FDA can­cels ODAC meet­ing this week to re­view two more dan­gling ac­cel­er­at­ed ap­provals — but won't ex­plain why

The FDA’s Oncologic Drugs Advisory Committee has decided to cancel a planned meeting on Thursday to discuss two cancer drugs that previously won accelerated approvals but failed to confirm clinical benefit in required follow-up trials or have taken a long time to finish those trials.

The FDA said in a statement that the meeting “is no longer needed” but did not offer further detail on why exactly it was canceled, telling Endpoints News to contact the companies. Attempts to contact both Secura Bio and Acrotech went unreturned. The companies may have decided to pull these treatments from the market, or they’ve come to new agreements with the agency on their confirmatory trials.