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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

Phillip Gomez, Siga Technologies CEO

Siga nabs $10.7M from the US gov­ern­ment in deal for its mon­key­pox an­tivi­ral

The US government is all set to buy $10.7 million worth of Siga Technologies’ monkeypox oral antiviral, the company announced Thursday.

Of the total doses, $5.1 million worth of oral antivirals called Tpoxx (tecovirimat) will be delivered this year, with the US Department of Defense having the option of buying the rest at a later point.

The new contract follows an earlier one in which the government had purchased $7.4 million worth of Tpoxx from the company.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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