Brian McVeigh, Code Biotherapeutics CEO (Code)

No AAV? No prob­lem. Code Bio­ther­a­peu­tics joins the search for a non-vi­ral gene ther­a­py

Bri­an McVeigh’s wake-up call came one morn­ing in the form of a LinkedIn no­ti­fi­ca­tion con­grat­u­lat­ing him for 25 years at Glax­o­SmithK­line.

“I was like, ‘Oh my God, it has been a quar­ter cen­tu­ry at one com­pa­ny, what am I do­ing?’” he said.

McVeigh quit his job as VP of world­wide busi­ness de­vel­op­ment trans­ac­tions and in­vest­ment man­age­ment in No­vem­ber 2017, spent half a year as the CEO of KBP Bio­Sciences, and even­tu­al­ly end­ed up on the board of Geni­sphere, where sci­en­tists were work­ing on a drug de­liv­ery plat­form us­ing syn­thet­ic DNA.

“As this start­ed to grow and pre­clin­i­cal da­ta start­ed to get gen­er­at­ed, we re­al­ized that it re­al­ly needs to be on its own in or­der to achieve its full po­ten­tial,” he said.

Last April, he and broth­er-sis­ter duo Bob and Lori Getts found­ed Code Bio­ther­a­peu­tics to take the “next ma­jor leap for­ward” in gene ther­a­py. And on Tues­day, they of­fi­cial­ly launched with $10 mil­lion in seed fund­ing.

As CEO, McVeigh is lead­ing the de­vel­op­ment of a syn­thet­ic DNA-based vec­tor, dubbed 3DNA, to over­come the cur­rent chal­lenges of AAV. The vec­tor con­sists of a se­ries of strands of syn­thet­ic DNA linked to­geth­er to form a scaf­fold, which un­like AAV, doesn’t trig­ger an im­mune re­sponse.

“That is a big is­sue with AAV — that folks have ei­ther nat­ur­al im­mu­ni­ty to the vi­ral vec­tors, or once they’re dosed with it, they cre­ate an im­mune re­sponse to it so that you can’t re-dose,” McVeigh said.

The abil­i­ty to re-dose would al­low sci­en­tists to treat peo­ple with a low­er dose to start, po­ten­tial­ly avoid­ing some of the safe­ty is­sues in­volved with giv­ing high­er dos­es.

An­oth­er key bonus to us­ing syn­thet­ic DNA is that sci­en­tists are not lim­it­ed by the size of the gene con­struct they’re try­ing to de­liv­er. McVeigh says they have da­ta so far show­ing suc­cess­ful de­liv­ery of gene con­structs up to 10 kb in length.

“What that means is that we could ac­tu­al­ly go af­ter dis­eases that you can’t cur­rent­ly go af­ter with AAV ap­proach­es, and that we can al­so go af­ter dis­eases (for which) there are some AAV ap­proach­es but they’re not nec­es­sar­i­ly giv­ing the op­ti­mized gene con­struct be­cause it can’t fit,” he said.

Code is cur­rent­ly in the dis­cov­ery phase for ther­a­pies to treat type 1 di­a­betes and Duchenne mus­cu­lar dy­s­tro­phy (DMD), a de­bil­i­tat­ing dis­or­der caused by mu­ta­tions in the dy­s­trophin gene. McVeigh guess­es those pro­grams are still about two years away from en­ter­ing the clin­ic.

The biotech will face stiff com­pe­ti­tion for the long­time holy grail. Gen­er­a­tion Bio closed a $230 mil­lion IPO last June to fund its pre­clin­i­cal push for a non-vi­ral gene ther­a­py to treat dis­eases of the liv­er and reti­na. And Michael Ehlers is lead­ing a new ef­fort over at Ap­ple Tree Ven­tures.

4BIO Cap­i­tal and UPMC En­ter­pris­es co-led the seed fi­nanc­ing, with a lit­tle help from Cure­Duchenne Ven­tures, JDRF T1D Fund, New En­ter­prise As­so­ci­ates, and Take­da Ven­tures. The com­pa­ny re­cent­ly tapped Spark Ther­a­peu­tics alum Lau­ren Kask­iel as CBO. Kathy Rouan, for­mer SVP of R&D at GSK, is on the board.

“This is the thing that has so caught my at­ten­tion and pas­sion more than any­thing else,” McVeigh said. “It’s got the po­ten­tial here to be such a game chang­er and to move the field for­ward in a dra­mat­ic way.”

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; 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.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Jason Kelly (Photographer: Kyle Grillot/Bloomberg via Getty Images)

Gink­go nabs $DNA, biotech's most sought af­ter tick­er, for free in sweet­en­er from NYSE

When Ginkgo went comparison shopping for a financial market to list their now $15 billion company, the New York Stock Exchange had a back-pocket sweetener the Nasdaq couldn’t offer: The most sought-after ticker in biotech, $DNA.

DNA — the most famous three letters in biology and the ticker for the world’s first biotech, Genentech, from 1999 until it was bought out by Roche for $48 billion in 2009 — will now be the ticker for Ginkgo, a 12-year-old synthetic biology startup with grand ambitions to change not only how drugs, but also everyday products like meat and perfumes, are made.

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Lark­spur Health Ac­qui­si­tion files to go pub­lic as this year's SPAC flood surges over $14B

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Another day, another SPAC vying for a spot on Nasdaq.

On Wednesday, OncoSec Medical CEO Daniel O’Connor filed the S-1 paperwork for a new blank-check company he’s leading called Larkspur Health Acquisition. The former Advaxis chief penciled in a $75 million raise, with plans to offer 7.5 million shares at $10 apiece.

BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.