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.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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Paul Tesar (Convelo Therapeutics)

Io­n­is, lead­ing MS re­searcher throw an­ti­sense at a new type of brain cells

No matter how many molecules he threw at them, Paul Tesar couldn’t get the brain cells to survive. Or he got them to survive, but then — to everyone’s bafflement — they still couldn’t do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.

Morningside continued to lead the $30 million Series B, with participation from Termeer’s widow, Belinda, and other unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.