It's not de­vel­op­ing drugs that bind to RNA, or mor­ph­ing RNA in­to a ther­a­peu­tic — this Cam­bridge start­up has scored $63M to work on RNA-mod­i­fy­ing pro­teins

First came the con­cept of epi­ge­net­ics, the study of chem­i­cal mod­i­fi­ca­tions made to the blue­print of life — DNA — to switch genes on or off. Then, sci­en­tists re­al­ized DNA’s cousin RNA could al­so be sub­ject to such chem­i­cal ma­nip­u­la­tion, and the field of “epi­tran­scrip­tomics” was born in the last decade.

As re­searchers found that chem­i­cal mod­i­fi­ca­tions across a cell’s RNA were seem­ing­ly dis­tort­ed in some can­cers, a trio of biotech­nol­o­gy com­pa­nies spawned: Storm Ther­a­peu­tics, Ac­cent Ther­a­peu­tics and Gotham Ther­a­peu­tics. On Thurs­day, 2017-found­ed Ac­cent raised $63 mil­lion in a Se­ries B round, led by EcoR1 Cap­i­tal.

Chuan He, a pro­fes­sor of chem­istry at the Uni­ver­si­ty of Chica­go, first pro­posed the field of RNA epi­ge­net­ics, sug­gest­ing methyl mod­i­fi­ca­tions on RNA can be re­moved, in 2012. Five years lat­er, along with Stan­ford’s Howard Chang, and for­mer Epizyme and GSK ex­ec­u­tive Robert Copeland, Ac­cent was born.

Shak­ti Narayan

“What we’re do­ing is dif­fer­ent from a lot of oth­er com­pa­nies and oth­er re­searchers in the RNA field. There are many com­pa­nies that are us­ing RNA as a ther­a­peu­tic it­self, there are some com­pa­nies that are drug­ging the RNA di­rect­ly,” not­ed Ac­cent chief Shak­ti Narayan in an in­ter­view.

Akin to ac­cents in mu­sic that are used to mod­u­late tunes, the com­pa­ny was chris­tened Ac­cent Ther­a­peu­tics to re­flect mod­i­fi­ca­tions to chem­i­cal en­ti­ties that af­fect RNA mol­e­cules, which can ul­ti­mate­ly trans­form the func­tion of the mol­e­cule.

“What we’ve fo­cused on is tar­get­ing the en­zymes that work on RNA, and there­by, we’re ac­tu­al­ly able to take ad­van­tage of sev­er­al decades of med­i­c­i­nal chem­istry ex­pe­ri­ence and ex­per­tise that has de­vel­oped to specif­i­cal­ly tar­get en­zymes. So, we’re not hav­ing to take on a whole bunch of modal­i­ty risk in our ap­proach,” Narayan said.

The fresh in­jec­tion of fund­ing will be used to take Ac­cent’s lead ex­per­i­men­tal pro­grams — which are fo­cused on two pro­teins that are un­der­stood to mod­i­fy RNA, MET­TL3 and ADAR1 in AML and sol­id tu­mors, re­spec­tive­ly — in­to the clin­ic.

But, as with all best-laid plans, Covid-19 has put a span­ner in the works.

“We’ve been able to in­crease our work via CROs in cer­tain lo­ca­tions, ver­sus de­creas­ing it and oth­er lo­ca­tions de­pend­ing on the im­pact of the virus in par­tic­u­lar lo­ca­tions, which has been ex­treme­ly help­ful for us to be able to keep our pro­grams ad­vanc­ing to­wards the clin­ic,” Narayan said. “It’s cer­tain­ly not op­ti­mal, but it’s been a great way to mit­i­gate the im­pact of the dis­ease on our over­all op­er­a­tional mod­el.”

The new round of fi­nanc­ing in­clud­ed the par­tic­i­pa­tion by GV, Ab­b­Vie Ven­tures, The Mark Foun­da­tion for Can­cer Re­search, NS In­vest­ment and Droia Ven­tures as well as ex­ist­ing in­vestors, At­las Ven­ture and The Col­umn Group. The Lex­ing­ton, Mass­a­chu­setts-based com­pa­ny, which cur­rent­ly has 25 em­ploy­ees, raised $40 mil­lion at launch in 2018.

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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John Flavin, Portal Innovations CEO

Por­tal In­no­va­tions takes its Chica­go biotech in­vest­ing, lab space mod­el to Boston with Bob Langer start­up in tow

Life sciences investor and lab operator Portal Innovations is taking the blueprint it set in Chicago and expanding to Massachusetts, where it will deploy $25 million, and help house new startups in the former Boston Globe headquarters.

Portal CEO and founder John Flavin told Endpoints News the company will open its Boston-area operations in the coming weeks, and its first lab occupant will be Syntis Bio, an early-stage biotech out of Moderna co-founder Bob Langer, MIT professor Giovanni Traverso and Sherlock Biosciences co-founder Rahul Dhanda.

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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.