(L-R) Samta Kundu (COO), Jake Becraft (CEO), Tasuku Kitada (head of R&D) [Strand Therapeutics]

A pair of for­mer MIT re­searchers think they've un­locked the next gen­er­a­tion of mR­NA us­ing syn­bio 'log­ic cir­cuit­s'

The time of mR­NA is in full swing as Mod­er­na and Pfiz­er/BioN­Tech have blown the doors off the field. But in drug de­vel­op­ers’ eyes, cur­rent-gen mR­NA vac­cines are just an ap­pe­tiz­er to the full course of ther­a­peu­tics fur­ther down the menu — at least that’s what two for­mer MIT re­searchers with syn­thet­ic bi­ol­o­gy roots are gam­bling on.

Strand Ther­a­peu­tics emerged from stealth Wednes­day with a $52 mil­lion Se­ries A to ad­vance its pipeline of pro­gram­ma­ble, self am­pli­fy­ing mR­NA ther­a­pies ini­tial­ly tar­get­ed at sol­id tu­mor im­muno-on­col­o­gy, the biotech said.

The team’s re­search stems from the work of CEO Jake Be­craft and head of R&D Tasuku Ki­ta­da at MIT’s Syn­thet­ic Bi­ol­o­gy Cen­ter and the Weiss Lab, where the pair met and fo­cused on ap­ply­ing syn­bio prin­ci­ples to mR­NA ther­a­peu­tic de­vel­op­ment. Along the way, their work formed Strand’s plat­form, which turns out long act­ing mR­NA ther­a­pies with greater speci­fici­ty and a wider ther­a­peu­tic in­dex than the cur­rent gen­er­a­tion of mR­NA vac­cines.

The biotech is us­ing self am­pli­fy­ing tech, which in­duces a sin­gle copy of the mR­NA drug to repli­cate it­self in vi­vo. That of­fers a much longer ther­a­peu­tic win­dow than cell ther­a­pies like the Covid-19 vac­cines from Mod­er­na and BioN­Tech, Be­craft told End­points News, which work on the scale of days rather than weeks.

But per­haps the biggest dif­fer­ence from its range of com­peti­tors is Strand’s use of “ge­net­i­cal­ly pro­gram­ma­ble log­ic cir­cuits,” which al­low its mR­NA drugs to en­ter a spe­cif­ic cel­lu­lar en­vi­ron­ment and turn pro­tein ex­pres­sion on or off de­pend­ing on a se­ries of bio­mark­ers. It’s a for­mu­la fa­mil­iar in syn­thet­ic bi­ol­o­gy: Use “sen­sors” to iden­ti­fy the tar­get en­vi­ron­ment, “log­ic process” or com­pute those sig­nals, and then ef­fect a tar­get out­put.

The com­pa­ny’s syn­bio plat­form serves a dual pur­pose of al­low­ing tight con­trol over RNA self-repli­ca­tion, en­sur­ing the ther­a­py is on­ly around in the cell as long as it needs to be.

“One of the ma­jor prob­lems with self repli­ca­tion mR­NA is that you ac­tu­al­ly need to con­trol their repli­ca­tion at a much tighter lev­el — and that’s ac­tu­al­ly what syn­thet­ic bi­ol­o­gy is re­al­ly per­fect for,” Be­craft said. “You go in­to the se­quence of the repli­ca­tion ma­chin­ery and you can make mod­i­fi­ca­tions to how it repli­cates and ac­tu­al­ly con­trol it.”

All that, ul­ti­mate­ly, means one drug could the­o­ret­i­cal­ly have an ef­fect on a range of dis­ease types — par­tic­u­lar­ly in a het­ero­ge­neous ther­a­peu­tic area such as can­cer. No sur­prise, then, that can­cer is ex­act­ly where Strand is aim­ing first.

The biotech is hop­ing to take its lead drug, which is fo­cused in the sol­id tu­mor I/O space in­to the clin­ic by 2022. The com­pound is the tar­get of a li­cens­ing pact with Chi­nese drug­mak­er BeiGene, which has pledged to cov­er some de­vel­op­ment and com­mer­cial­iza­tion costs in the re­gion. Can­cer is no easy area to jump head­first in­to, but it is a prof­itable one. Be­craft high­light­ed the field’s well-es­tab­lished bi­ol­o­gy as ripe with big tar­gets for Strand’s drugs.

“I think can­cer is a great first tar­get for things like the next gen­er­a­tion of RNA ther­a­peu­tics be­cause can­cer has a lot of known tar­gets and known bi­ol­o­gy for ther­a­peu­tic in­ter­ven­tion,” Be­craft said. “It just gives you a great start­ing point to de­vel­op a new plat­form. Can­cer kills peo­ple; we got­ta do some­thing.”

Be­craft said his com­pa­ny would look to sign sim­i­lar deals in the com­ing months, aim­ing to sign on “high­ly strate­gic” part­ners to ad­vance Strand’s pipeline and com­mer­cial op­por­tu­ni­ties.

It’s the first go-round as CEO for Be­craft, who made the jump straight from MIT as he worked to build Strand in stealth mode along­side Ki­ta­da start­ing in 2018. He said his first pri­or­i­ties are dri­ving the com­pa­ny’s lead pro­gram ahead while build­ing the pipeline and adding a strong team around him. The com­pa­ny is cur­rent­ly on a hir­ing spree with 25 on board and hopes to hit as many as 80 em­ploy­ees as it nears the clin­ic, Be­craft said.

The Se­ries A round was joined by a syn­di­cate of in­vestors in Red­mile Group, BeiGene and Cam­ford Cap­i­tal, as well as ex­ist­ing in­vestors Play­ground Glob­al and AN­RI.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Fu­ji­film con­tin­ues CD­MO ex­pan­sion, break­ing ground on $435M UK site

Fujifilm’s CDMO arm, Fujifilm Diosynth, has been on a roll this month as the company has recently broken ground on a major project in Europe and it appears to be keeping up the momentum.

Fujifilm Diosynth announced that it has kicked off an expansion project for its microbial manufacturing facility at its campus in the town of Billingham, UK, in the northeast of England.

The 20,000 square-foot, £400 million ($435 million) expansion will add clean rooms, purification suites and a packing area along with more space for the manufacturing itself.

Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

The following Q&A has been edited for length and clarity.

John Carroll:

We’ve had a chance to talk a little bit before here about some of the things that you’ve done. Just really remarkably, a lot of the things that you’ve done early in your career puts you in the path with some amazing science that has had an absolutely huge impact in terms of what we’re seeing now on drug development and some of the new technologies that are coming out here, and not only the new technologies, but also some of the most remarkable people ever.

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Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.