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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; 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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Daisy Robinton, Oviva Therapeutics co-founder and CEO

A new spin­out wades in­to ovar­i­an ag­ing, armed with a seed round and ex­per­i­men­tal drugs from Mass Gen­er­al

There aren’t many biotechs emphasizing women’s health, but a new spinout is trying to change that.

Oviva Therapeutics, a pipeline company of New York-based Cambrian Biopharma, emerged from stealth earlier this week to dive into the idea of extending women’s “healthspans,” or what it says is the part of a person’s life spent in generally good health, with a specific focus on ovaries. The emergence comes both with a seed financing worth $11.5 million from Cambrian, and an in-licensing agreement with Massachusetts General Hospital for a trio of patents.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Castle Creek Biosciences chair Jeff Aronin

Scoop: Af­ter pulling IPO am­bi­tions last De­cem­ber, Jeff Aron­in's Cas­tle Creek turns to pri­vate back­ers

Jeff Aronin’s cell and gene therapy biotech Castle Creek Biosciences has raised $112 million in equity, Endpoints News has learned.

The Exton, PA, biotech secured the financing from 54 investors, according to an SEC filing dated May 2. The late-stage startup had last year considered a $100 million Nasdaq debut, but in a sign of the bear market that has plagued hundreds of newly minted public biotechs, Castle Creek pulled those ambitions in the last few weeks of 2021.