At­las builds its dream team and hands over $25M to launch a new ap­proach to gene ther­a­py

Robert Kotin

Robert Kotin is one of the world’s top ex­perts on the vi­ral trans­port of gene ther­a­pies. Ex-NIH, ex-Voy­ager ex­ec, he’s played a siz­able role in shap­ing the field.

So when Kotin says there’s a bet­ter way to do gene trans­fer with­out a vi­ral ve­hi­cle, he man­aged to get the at­ten­tion of quite a few peo­ple in the Boston/Cam­bridge hub.

Ja­son Rhodes, a part­ner at At­las, was one of them. At­las formed a com­pa­ny around this called Gen­er­a­tion Bio with a $25 mil­lion A round and Rhodes is chair­ing the board. Ge­off Mc­Do­nough, who stepped aside as CEO of Stock­holm-based So­bi last sum­mer af­ter the com­pa­ny de­cid­ed to go with a new chief who pre­ferred to live clos­er to home, is tak­ing the helm.

Ge­of­frey Mc­Do­nough

Like Ed Kaye, who’s hav­ing his launch par­ty to­day at Stoke, Mc­Do­nough is a high pro­file fig­ure in the Mass­a­chu­setts hub. And he’s pumped about pur­su­ing a break­through ap­proach to gene trans­fer.

Kotin’s big idea cen­ters on what he calls close-end­ed DNA (ceD­NA), which can move from the cy­to­plasm of the cell to the nu­cle­us with­out that AAV trans­port ve­hi­cle. Once in­side the nu­cle­us, the the­o­ry goes, it can cre­ate durable high lev­els of gene ex­pres­sion ca­pa­ble of ad­dress­ing a broad range of ge­net­ic dis­eases. And the biotech plans to get start­ed by us­ing a lipid nanopar­ti­cle to de­liv­er ceD­NA to the liv­er, where it will make sys­temic pro­teins to fight dis­ease.

“The first or­gan is the liv­er be­cause that’s where lipid nanopar­ti­cles go,” Mc­Do­nough told me in a pre­view to to­day’s an­nounce­ment. “So we go to rare dis­eases of the liv­er, where it can be a fac­to­ry.”

And nanopar­ti­cles have some in­her­ent qual­i­ties that make it prefer­able to an AAV ap­proach, with a big­ger pay­load that can pack a big­ger punch. It’s al­so a new way to do this with­out trig­ger­ing an im­mune re­sponse that is lim­it­ing the first gen­er­a­tion of gene ther­a­pies to a one-time use.

Notes Mc­Do­nough: “This is a com­plete­ly nov­el modal­i­ty which is tremen­dous­ly dis­rup­tive to AAV.”

Ja­son Rhodes

While much of At­las’ in­vest­ing can be high­ly as­set-cen­tric, craft­ing com­pa­nies around one or two lead pro­grams in a care­ful­ly man­aged dri­ve to the clin­ic, Gen­er­a­tion Bio is every bit a plat­form com­pa­ny with a lot of po­ten­tial di­rec­tions to move. But Rhodes says you can’t rel­e­gate a ven­ture group like At­las — which has en­joyed the abil­i­ty to raise con­sid­er­able funds — to a nar­row fo­cus. The VC, he says, is “sci­ence-first, seed-led, prag­mat­ic about the busi­ness mod­el and in­tense­ly fo­cused on cre­at­ing in­no­v­a­tive new prod­ucts. And not too wor­ried about ide­ol­o­gy be­yond that.”

In re­sponse to a query from me, Rhodes added:

We al­ways match the na­ture of the op­por­tu­ni­ty to the busi­ness mod­el and build both ma­jor plat­form com­pa­nies and more fo­cused prod­uct plays. This strate­gic match­ing is a hall­mark of At­las’ ap­proach vs a cook­ie cut­ter, whether al­ways a mon­ster plat­form or al­ways an as­set play, etc. Re­cent plat­forms in­clude NT­LA, Sur­face, Ma­gen­ta, Trans­late, Nim­bus (with an LLC struc­ture in the mix), etc. Ear­ly M&A tends to oc­cur with the prod­uct-ori­ent­ed com­pa­nies, like Delinia, Pad­lock, and IFM and so gen­er­ates ear­ly press and at­ten­tion. Pad­lock is re­al­ly a plat­form around an area of bi­ol­o­gy as is IFM, which sold the onc port­fo­lio and kept the im­munol­o­gy port­fo­lio. We do con­sis­tent­ly use a seed ap­proach, where we pres­sure test the core sci­ence, be­gin build­ing a team etc. then ramp us the re­spec­tive busi­ness mod­el. I’ve been in­volved with all of these kinds of com­pa­nies whether at Al­ny­lam (new modal­i­ty plat­form), Epizyme (new bi­ol­o­gy mul­ti-prod­uct plat­form), Replim­ine (prod­uct play, though more than one), Dis­arm (new bi­ol­o­gy prod­uct), and Gen­er­a­tion, which is ob­vi­ous­ly a ma­jor new modal­i­ty, among a few ex­am­ples.

The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Af­ter de­cou­pling from Re­gen­eron, Sanofi says it’s time to sell the $13B stake picked up in the mar­riage

With Regeneron shares going for a peak price — after doubling from last fall — Sanofi is putting a $13 billion stake in their longtime partner on the auction block. And Regeneron is taking $5 billion of that action for themselves.

Sanofi — which has been decoupling from Regeneron for more than a year now — bought in big in early 2013, back when Regeneron’s stock was going for around $165 a share. Small investors flocked to the deal, buzzing about an imminent takeover. The buyout chatter wound down long ago.

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Covid-19 roundup: Janet Wood­cock steps aside — for now — as FDA drug czar; WHO hits the brakes on hy­droxy study af­ter lat­est safe­ty alarm

The biopharma industry will soon get a look at what the FDA will look like once CDER’s powerful chief Janet Woodcock retires from her post.

Long considered one of the most influential regulators in the agency, if not its single most powerful official when it counts, Woodcock is being detached to devote herself full time to the White House’s special project to fast-forward new drugs and vaccines for the pandemic. The move comes a week after some quick reshuffling as Woodcock and CBER chief Peter Marks joined Operation Warp Speed. Initially they opted to recuse themselves from any FDA decisions on pandemic treatments and vaccines, after consumer advocates criticized the move as a clear conflict of interest in how the agency exercises oversight on new approvals.

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Eric Edwards, Phlow president and CEO (PR Newswire)

BAR­DA of­fers a tiny start­up up to $812M to cre­ate a US-based drug man­u­fac­tur­er — and the CEO comes with a price goug­ing con­tro­ver­sy on his ré­sumé

BARDA has tapped a largely unknown startup to ramp up production of a list of drugs that may be at risk of running short in the US. And the deal, which comes with up to $812 million in federal funds, was inked by a CEO who found himself in the middle of an ugly price gouging controversy a few years ago.

The feds’ new partner — called Phlow — won a 4-year “base” contract of $354 million, with another $458 million that’s on the table in potential options to sustain the outfit. That would make it one of the largest awards in BARDA’s history.

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Janet Woodcock, director of the Center for Drug Evaluation and Research (AP Images)

Covid-19 roundup: Hit with new con­flict ac­cu­sa­tions, Janet Wood­cock steps out of the agen­cy's Covid-19 chain of com­mand

Two weeks ago, FDA drug chieftain Janet Woodcock was assuring a top Wall Street analyst that any vaccine approved for combating Covid-19 would have to meet high agency standards on safety and efficacy before it’s approved. But over the weekend, after she and Peter Marks took top positions with the public-private operation meant to speed a new vaccine to lightning-fast approvals — they both recused themselves from the review process after an advocacy group argued their roles close to the White House could pose a conflict of interest.

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Re­searchers de­fine ex­act­ly what they saw in the first pos­i­tive remde­sivir study for Covid-19. But what's that worth to Gilead?

Remdesivir can work in fighting Covid-19, particularly for patients with less severe cases, but this is just a first step in the journey to finding combos that can do the job much better.

That’s the bottom line from Gilead’s randomized study published in the New England Journal of Medicine. Analysts were quick to draw conclusions about how the big biotech could turn this into a profitable advantage — with widespread expectation of considerable pricing restraint on Gilead’s part. Anyone looking for a new mountain of cash to count as the world grapples with the pandemic is likely to come away disappointed.

An­oth­er NASH de­lay for In­ter­cept frus­trates in­vestors, shares wilt

A previous FDA advisory committee delay for Intercept’s NASH drug may have dampened spirits, but investors perked up after French rival Genfit recently failed to best a placebo with its offering in a keenly anticipated pivotal study. In yet another twist on Friday, the New York drugmaker said the FDA is postponing its adcom again to accommodate the review of additional data it has asked the company to furnish.

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FDA ap­proves the first gener­ic for Amar­in's Vas­cepa — but is a fish oil price war im­mi­nent?

Late last year, enthusiasm for Amarin’s fish-oil pill Vascepa burgeoned when the FDA signed off on expanding the cholesterol fighter’s label to include the drug’s beneficial impact on cardiovascular risk, but months later the exuberance for the blockbuster-to-be took a big hit when a judge invalidated key patents protecting Vascepa.

Despite Amarin’s $AMRN pledge to appeal — a process that could take months — the ruling opened the door for generic competition. Hikma Pharmaceuticals, one of three challengers in the Nevada suit, on Friday said that its generic copy of pure EPA, the omega-3 fatty acid that constitutes Vascepa, has been approved by the FDA.

Covid-19 roundup: CanSi­no beats Mod­er­na to pub­li­ca­tion of first-in-hu­man da­ta for a coro­n­avirus vac­cine

China’s CanSino Biologics has released results from the Phase I trial of its Covid-19 vaccine — data that have propelled its entry into Phase II more than a month ago.

In a paper published in the Lancet, the biotech reported that their adenovirus-based vaccine was “tolerable and immunogenic at 28 days post-vaccination” among the 195 volunteers enrolled in the trial. In addition to antibody responses, T cell responses were also observed.

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