Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

Shinya Ya­mana­ka No­bel Prize

In 2006, Shinya Ya­mana­ka shook stem cell re­search with his dis­cov­ery that ma­ture cells can be con­vert­ed in­to stem cells, re­liev­ing a long­stand­ing po­lit­i­cal-eth­i­cal block­age and throw­ing open med­ical re­search on every­thing from curb­ing eye de­gen­er­a­tion to or­gan print­ing.

But that process still has pit­falls, in­clud­ing in risk and scal­a­bil­i­ty, and some re­searchers are ex­plor­ing an­oth­er way first hint­ed at years ago: new tech­nol­o­gy to con­vert ma­ture cells di­rect­ly in­to oth­er ma­ture cells with­out the com­plex and time-con­sum­ing process of first mak­ing them in­to stem cells.

One of those com­pa­nies, Mo­gri­fy, just raised $16 mil­lion in Se­ries A fi­nanc­ing to bring its over­all fund­ing to over $20 mil­lion since its Feb­ru­ary launch. Led by CEO Dar­rin Dis­ley, the fund­ing will help ex­pand their new base in Cam­bridge to a 60-strong staff and push for­ward their di­rect-con­ver­sion ap­proach to cell ther­a­py through re­search and li­cens­ing. In­vestors in­clude Park­walk Ad­vi­sors and Ahren In­no­va­tion Cap­i­tal.

They list po­ten­tial ap­pli­ca­tions as treat­ments for mus­cu­loskele­tal and au­to-im­mune dis­or­ders, can­cer im­munother­a­py, and ther­a­pies for oc­u­lar and res­pi­ra­to­ry dis­eases. For ex­am­ple, you could use it re­gen­er­ate car­ti­lage in arthri­tis pa­tients.

“If you could take a cell from one part of the body and turn it in­to any oth­er cell at any oth­er stage of de­vel­op­ment for an­oth­er part of the body, you ef­fec­tive­ly have the Holy Grail of re­gen­er­a­tive med­i­cine,” Dis­ley told Labiotech.eu in April.

Mo­gri­fy’s ad­van­tage over the Ya­mana­ka method called in­duced pluripo­tent stem cells (iPS), is that in the­o­ry it can be more scal­able and avoid the prob­lems as­so­ci­at­ed with iPS. These in­clude in­sta­bil­i­ties aris­ing from the in­duced im­ma­ture state and an in­creased risk of can­cer if any pluripo­tent cells re­main in the body.

The con­cept be­hind Mo­gri­fy ac­tu­al­ly pre­dates, by near­ly 19 years, Ya­mana­ka’s dis­cov­ery, which fast won him the 2012 No­bel Prize in Med­i­cine. A 2017 Na­ture study on “trans­d­if­fer­en­ti­a­tion,” as the process is called, of fi­brob­lasts in­to car­diac tis­sue traced the idea to a 1987 find­ing that a mas­ter gene reg­u­la­tor could con­vert mice fi­brob­lasts in­to skele­tal mus­cle.

The prob­lem though, ac­cord­ing to Mo­gri­fy, is that most cur­rent ef­forts re­ly on an ex­haust­ing guess-and-check process. With hun­dreds of cell types and an even greater num­ber of tran­scrip­tion fac­tors — the pro­gram that re­codes the cell — find­ing the right fac­tor for the right cell can be like a cus­to­di­an with a jan­gling, un­marked key ring try­ing to get in­to a build­ing with thou­sands of locks.

Mo­gri­fy’s key tech is a com­put­er mod­el they say can pre­dict the right com­bi­na­tion. The sci­en­tists be­hind the plat­form pub­lished a 2016 study in Na­ture ap­ply­ing the mod­el to 173 hu­man cell types and 134 tis­sues.

Be­fore Mo­gri­fy, Dis­ley led the Cam­bridge-based gene-edit­ing com­pa­ny Hori­zon Dis­cov­ery.

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Tillman Gerngross, Adagio Therapeutics CEO

An­ti­body leg­end Till­man Gern­gross is el­bow­ing his way in­to the Covid-19 R&D cru­sade: 'I don’t see this end­ing any­time soon'

One of the most influential — and outspoken — scientists at work in the field of antibody discovery is jumping into the frenzied race to create new therapeutics to treat and prevent Covid-19. And he’s operating with the conviction that the current outbreak now once again spreading like wildfire will create plenty of demand for what he has in mind.

Dartmouth professor and Adimab CEO Tillman Gerngross tells me he’s raised $50 million from a group of close VCs to spin out a new company — Adagio Therapeutics — with a full C-suite team assembled to hire up a staff and keep rolling toward the clinic.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

Full Bril­in­ta study re­sults show the blood thin­ner re­duces rate of sec­ondary stroke

AstraZeneca once projected its Brilinta drug to peak at $3.5 billion in sales, and though the blood thinner never reached that lofty goal, it received the latest positive signs in a string of recent good news.

The pharma released full details from its THALES study Thursday morning, which measured the effects of Brilinta and aspirin against aspirin alone in treating patients who had an acute ischemic stroke or transient ischemic attack. When taken twice daily with once-a-day aspirin for 30 days, the Brilinta combo reduced the risk of stroke and death by 17 percent, meeting the primary endpoint of the study.

Norbert Bischofberger, Kronos CEO

Gilead­'s ex-R&D chief Bischof­berg­er heads back to the biotech gi­ant to pick up a pair of late-stage drugs that had been put aside

Norbert Bischofberger knows entospletinib well.

Back during his long, blockbuster run as head of R&D at Gilead, researchers had once held some high hopes for this drug. But to make it work, he and the team felt it would need a new companion diagnostic to identify patients. There was talk of a combo approach to give it more punch. But the market was small, making them wonder if it would be worth going through a lengthy development cycle to get it through a pivotal.

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The $1B Mer­ck-Bay­er drug that di­vid­ed car­di­ol­o­gists in March gets pri­or­i­ty re­view

Three months after Merck published in the New England Journal of Medicine data that left doctors and investors divided over just how well its experimental heart drug worked, the FDA has handed that drug priority review. A decision is now due by January 20, 2021.

Merck first announced the drug, known as vericiguat, as a Phase III success last November. In 2016, Merck had paid $1 billion upfront for US rights to the Bayer-developed drug. Early projections foresaw a few hundred million a year in sales, but the unspecified late-stage success raised the possibility for far more. After all, Novartis’s flagship heart drug, Entresto, was earning $1.7 billion per year and was expected to reach up to $4 billion in annual sales.

GSK’s Shin­grix leader Guil­laume Pfe­fer has jumped on board Flag­ship to helm a biotech hy­brid as Afeyan’s lat­est CEO-part­ner

After spending 4 years in a senior post with GlaxoSmithKline’s star team positioning Shingrix for a blockbuster approval, Guillaume Pfefer is headed back to the biotech world — in style.

Pfefer has signed on to join Noubar Afeyan’s busy group of partners at Flagship, and he’s taking the helm of an upstart — which today is being merged with another Flagship startup — with some grand plans of its own. The announcement this morning notes that Pfefer will run Kintai Therapeutics, one of the grads of the Flagship labs.

Donald Trump and Anthony Fauci (AP Images)

Covid-19 roundup: Rus­sia hack­ers tar­get US, UK vac­cine and drug re­searchers; Fau­ci fires back at White House cam­paign to un­der­mine him

Russia has tried to steal a Covid-19 vaccine and therapeutics researcher from pharmaceutical and academic institutions in the US, UK and Canada, Britain’s National Cyber Security Centre said Thursday.

The NCSC said that hacking attempts came from a group known as APT129, also known as “Cozy Bear,” that “almost certainly operate as part of Russian intelligence services.” The Canadian Communication Security Establishment, US Department for Homeland Security, the Cybersecurity Infrastructure Security Agency, and the National Security Agency shared the assessment, the NCSC said.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.