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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Deborah Waterhouse, ViiV Healthcare CEO

MPP re­cruits three gener­ic man­u­fac­tur­ers to ex­pand use of Vi­iV's in­jectable PrEP drug

ViiV Healthcare has teamed up with the UN-backed Medicines Patent Pool and three generic manufacturers to expand access to medicine that can prevent HIV.

ViiV and the Medicines Patent Pool jointly announced Thursday that the MPP signed sub-licensing agreements with Aurobindo, Cipla and Viatris to manufacture generics of a long-acting form of cabotegravir for HIV pre-exposure prophylaxis (PrEP). As a result of the agreement, the manufacturers will be able to develop, manufacture and supply generic versions of cabotegravir LA in 90 countries — subject to regulatory approvals.