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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.

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

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.