Haul­ing in a $100M mega-round, gene ther­a­py start­up Gen­er­a­tion Bio builds al­ter­na­tive to po­ten­tial­ly risky vi­ral vec­tors

A gene ther­a­py com­pa­ny head­ed up by big­wigs and backed ear­ly on by At­las has pulled in a mega-round of $100 mil­lion. The 2-year-old start­up cap­tured in­vestors’ at­ten­tion by sug­gest­ing it knows a bet­ter way to do gene trans­fer than the po­ten­tial­ly tox­ic vi­ral vec­tor strate­gies of late.

Gen­er­a­tion Bio is the Boston/Cam­bridge up­start, and it’s a com­pa­ny that’s strayed from the pack when it comes to the how of gene ther­a­py. While its ri­vals in the field are buck­ling down on vi­ral ve­hi­cles like ade­no as­so­ci­at­ed virus­es (AAV), Gen­er­a­tion Bio is tak­ing a dif­fer­ent route.

Robert Kotin

Found­ed in part by ex-NIH, ex-Voy­ager ex­ec­u­tive Robert Kotin, whom you may know as one of the world’s top ex­perts on vi­ral trans­port, the new com­pa­ny cen­ters on a tech they’re call­ing close-end­ed DNA (ceD­NA). This can move from the cy­to­plasm of the cell to the nu­cle­us with­out an AAV car­ry­ing it along. Once in­side the nu­cle­us, it can cre­ate high lev­els of gene ex­pres­sion ca­pa­ble of ad­dress­ing a broad range of ge­net­ic dis­eases.

Gen­er­a­tion Bio says the hope is that this tech can ad­dress the emerg­ing con­cern re­volv­ing around vi­ral vec­tors. Well-known gene ther­a­py com­pa­nies like Spark and Sol­id Bio­sciences are us­ing AAVs as vec­tors, a method that, when used in high dos­es, has re­cent­ly been called in­to ques­tion by a pi­o­neer in the field, Penn pro­fes­sor James Wil­son.

Wil­son sur­prised the gene ther­a­py com­mu­ni­ty in Jan­u­ary when he quit the ad­vi­sor board of Sol­id and raised alarms about the po­ten­tial tox­i­c­i­ty of high-dose AAV stud­ies.

Gen­er­a­tion Bio is cap­i­tal­iz­ing on the un­cer­tain­ty cloud­ing the lead­ing tech in the field by por­tray­ing their ther­a­pies as po­ten­tial­ly safer.

Last month, the com­pa­ny’s CEO Ge­off Mc­Do­nough told End­points the com­pa­ny would start by us­ing a lipid nanopar­ti­cle to de­liv­er ceD­NA to the liv­er, where it will make sys­temic pro­teins to fight dis­eases.

“These ther­a­pies are de­signed to over­come the im­muno­genic­i­ty as­so­ci­at­ed with vi­ral-vec­tor gene ther­a­pies, which has his­tor­i­cal­ly lim­it­ed the num­ber of pa­tients that can be treat­ed and pre­vent­ed re-dos­ing,” Gen­er­a­tion Bio stat­ed in a press re­lease. “Since its found­ing in 2016, Gen­er­a­tion Bio has demon­strat­ed durable, dose-de­pen­dent ex­pres­sion and the abil­i­ty to in­crease ex­pres­sion in the liv­er with re-dos­ing in vi­vo with the Ge­neWave plat­form.”

In­vestors ap­pear to be in­trigued. The $100 mil­lion fi­nanc­ing was a Se­ries B led by Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, with par­tic­i­pa­tion from In­vus, Deer­field Man­age­ment Com­pa­ny, Cas­din Cap­i­tal, Fore­site Cap­i­tal, and Leerink Part­ners’ Af­fil­i­ates.

This round comes less than two months af­ter Gen­er­a­tion Bio an­nounced its $25 mil­lion Se­ries A backed by At­las.

The new mon­ey will be used to take two ther­a­peu­tic can­di­dates through IND-en­abling stud­ies.

“This will be a crit­i­cal­ly im­por­tant year for us as we de­vel­op the Ge­neWave plat­form and work to­wards our first de­vel­op­ment can­di­dates to ad­dress rare dis­eases of the liv­er,” Mc­Do­nough said in a state­ment. “The round will al­so sup­port the de­vel­op­ment of ap­proach­es to de­liv­er ceD­NA to oth­er tis­sues such as the eye, the cen­tral ner­vous sys­tem and the lungs.”

Im­age: Gen­er­a­tion Bio’s pres­i­dent and CEO Ge­off Mc­Do­nough. Gen­er­a­tion Bio

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Andrew Phillips, Nexo Therapeutics CEO

Scoop: Ver­sant, NEA launch new biotech helmed by ex-CEO of pro­tein de­grad­er C4 Ther­a­peu­tics

Long-time biotech venture firms Versant and New Enterprise Associates are backing a new startup run by former C4 Therapeutics chief executive Andrew Phillips.

The fledgling biotech has raised at least $30 million so far, according to paperwork filed with the SEC this week. The round could balloon to $60 million.

Phillips, who left protein degradation startup C4 in 2020 to be a managing director at Cormorant Asset Management, is running the show of the new venture as president, the SEC filing outlines. He also served as interim CEO of Cormorant-backed and Hansoh Pharmaceutical-partnered Blossom Bioscience last year.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Karen Aiach, Lysogene CEO (RE(ACT) Discovery Institute)

Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.

And now, Lysogene reported the trial has failed its primary endpoint.

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Phil L’Huillier, CatalYm CEO

Ger­man biotech CatalYm rais­es $50M to flip weight loss tar­get for can­cer

GDF15 might sound familiar. It’s a protein that Amgen, Merck and Eli Lilly built analogs for in attempts to make new weight loss drugs. But those drugs largely failed — and Amgen, the last standing of the three — quietly pulled the plug on its GDF15 program in January.

But GDF15 is not dead. The science behind the weight loss drugs goes back to the observation that some cancer patients have high levels of GDF15 and lose a lot of weight, so cancer researchers have been making antibodies that inhibit the protein instead of mimicking it.

Szabolcs Nagy, Turbine co-founder and CEO

Sim­u­la­tions for ex­per­i­ments: Bay­er-backed start­up lands $20M to test out its tech

How do you get the attention of Big Pharma when you’re a small biotech startup working out of Budapest, Hungary?

For Szabolcs Nagy and his co-founders at Turbine, the golden ticket came through Bayer’s grant program for digital health, G4A, for which the company was selected a few years back.

“We roamed around the building and sort of just knocked on a whole bunch of doors after a lot of introductions,” Nagy told Endpoints News.

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