Gene edit­ing up­start backed by Broad star Feng Zhang nabs $135M, blue­print­ing a plat­form biotech for the long haul

Im­age: John Evans. BEAM

Last May, when Beam Ther­a­peu­tics had its com­ing out par­ty, com­plete with $87 mil­lion in in­vest­ment back­ing, a sol­id core of 15 staffers and a dream team of sci­en­tif­ic co-founders, CEO John Evans was sure they were set for sev­er­al years of R&D work.

But that’s not the way it played out. 

At a time when the mon­ey spig­ot is wide open for the right biotech plans and teams, Beam is an­nounc­ing that it went back to the ven­ture well to flesh out its syn­di­cate and added a whop­ping $135 mil­lion mon­ster round to the bank. That core team has swelled to 70, with more hires on the way as the ex­ec­u­tive team pre­pares to host a staff of 130. And Evans says they are well on their way to cre­at­ing a pipeline of pro­grams be­gin­ning with the 10 pre­clin­i­cal ef­forts they have un­der­way in new­ly opened labs.

“We de­cid­ed to go broad,” says Evans. “There are like­ly a lot of places for this be­ing the best op­tion for pa­tients.”

The big idea at Beam is that in­stead of us­ing a spe­cial gene edit­ing cut­ting tool to do per­ma­nent sur­gi­cal al­ter­ations of DNA, they’re pen­cilling in their ed­its with base edit­ing en­zymes that mod­i­fy nu­cle­obas­es  — cor­rect­ing or mod­i­fy­ing dis­ease caus­ing genes or writ­ing in code for ge­net­ic vari­a­tions that can pre­vent dis­ease. It’s one of a group of next-wave gene edit­ing out­fits that have come along in the wake of pi­o­neers like Ed­i­tas, In­tel­lia or CRISPR Ther­a­peu­tics. 

The Broad In­sti­tute’s Feng Zhang, who has backed Ed­i­tas and oth­er up­starts in the field, joined with Broad col­league David Liu and Har­vard Med’s Kei­th Joung to pro­vide the sci­en­tif­ic in­spi­ra­tion.


Im­age: David Liu, Kei­th Joung, Feng Zhang. BEAM

At this point in Beam’s ex­is­tence, go­ing broad means stick­ing with gen­er­al­i­ties and avoid­ing specifics about the work they’re do­ing in the lab. That’s stan­dard op­er­at­ing pro­ce­dure in start­up land. The de­tails can start to come lat­er as they be­gin to pub­lish their work and line up INDs for the first clin­i­cal pro­grams now in the works.

The plan now is to tran­si­tion from their plat­form con­cept in­to a pipeline sto­ry, says Evans. And they’re adding AAV vec­tors, lipid nanopar­ti­cles and oth­er kinds of tech that can get their ed­its wher­ev­er they need to go in the body.

“We’re not go­ing to throw this over the wall to a phar­ma com­pa­ny,” adds the CEO, adding that the team at Beam in­tends to build it all in house from end-to-end — much like they did at Agios, where he was SVP of cor­po­rate de­vel­op­ment.

Go­ing all the way means in­vest­ing heav­i­ly in man­u­fac­tur­ing ear­ly on and lay­ing the foun­da­tion for an even­tu­al shift to com­mer­cial­iza­tion. And that means plan­ning for the long haul, with lots of cap­i­tal re­quired to make it a re­al­i­ty.

That takes a syn­di­cate of back­ers ready for the long haul as well. And here’s who’s back­ing the B round: new in­vestors Red­mile Group, LLC; Cor­morant As­set Man­age­ment; GV; Al­ti­tude Life Sci­ence Ven­tures, and “ad­di­tion­al undis­closed in­vestors.” Then there’s al­so new mon­ey from ex­ist­ing in­vestors: F-Prime Cap­i­tal, ARCH Ven­ture Part­ners, Eight Roads Ven­tures, and Omega Funds.

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.

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.

Four community leaders who are living with HIV celebrate life and accomplishments in Theratechnologies' new campaign

Re­al pa­tient ‘cham­pi­ons’ liv­ing with HIV star in Ther­at­e­ch­nolo­gies cam­paign

Over the past several years, people living with HIV have been more often telling Theratechnologies that they wanted more representation. Specifically, they wanted more African American people and more focus on living and thriving versus more typical medication regimen messaging.

So Theratechnologies came up with a new campaign called “I Am A Champion,” initially launched at the US Conference on HIV last year. The initial conference, print and digital media efforts highlight the triumphs of four long-term survivors from across the US.

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