Take­da hops on­to $61M round for CRISPR play­er syn­the­siz­ing Cas9 al­ter­na­tives

Take­da Ven­tures and Or­biMed have helped pump $61 mil­lion in­to a New York biotech join­ing the rush to dra­mat­i­cal­ly ex­pand the CRISPR tool kit.

One cru­cial com­po­nent in the CRISPR gene edit­ing process is a nu­cle­ase that cuts the tar­get DNA once it’s led there by a guide RNA. Cas9 is the most well known and broad­ly used among them, al­though re­searchers have been com­ing up with a va­ri­ety of al­ter­na­tives. The one that Emen­do Bio­ther­a­peu­tics will be ad­vanc­ing with its new fund­ing is dubbed OM­NI.

Their class of “op­ti­mized” en­zymes, ac­cord­ing to Emen­do, opens a new lev­el of pre­ci­sion un­at­tain­able with old­er ap­proach­es.

“For ex­am­ple, to treat dom­i­nant in­di­ca­tions, one al­lele needs to be edit­ed while leav­ing the oth­er one in­tact. Cur­rent CRISPR nu­cle­as­es are not spe­cif­ic enough to do this,” the biotech wrote on its web­site. “Dom­i­nant in­di­ca­tions are the vast ma­jor­i­ty of ge­net­ic dis­eases, high­light­ing the need for a nov­el ap­proach.”

That plat­form tech has at­tract­ed Take­da to a part­ner­ship in which Emen­do will syn­the­size OM­NI nu­cle­as­es for two undis­closed tar­gets. In re­turn, the Japan­ese phar­ma gi­ant agreed to par­tic­i­pate in this new fi­nanc­ing along­side Or­biMed Ad­vi­sors, Or­biMed Is­rael Part­ners and AnGes.

Tokyo and Os­a­ka-based AnGes was re­spon­si­ble for $50 mil­lion of the raise, up­ping its stake in Emen­do to 32%, ac­cord­ing to a fil­ing last month.

“Mak­ing Emen­do an af­fil­i­at­ed com­pa­ny of AnGes will as­sist us in the de­vel­op­ment of bio­phar­ma­ceu­ti­cals through genome edit­ing, mak­ing it pos­si­ble to fur­ther ex­pand our­de­vel­op­ment pipeline as the fourth pil­lar of our op­er­a­tions af­ter HGF gene ther­a­py,nu­cle­ic-acid med­i­cines, and DNA vac­cines,” AnGes wrote.

Emen­do, which has an R&D op­er­a­tion in Is­rael, is col­lab­o­rat­ing with the Uni­ver­si­ty of Wash­ing­ton and Seat­tle Chil­dren’s Hos­pi­tal on its lead pro­gram se­vere con­gen­i­tal neu­trope­nia. Oth­er pro­grams in pri­ma­ry im­mun­od­e­fi­cien­cy, bone mar­row fail­ure, in­her­it­ed eye dis­ease and can­cer are al­so at­tached to pres­ti­gious aca­d­e­m­ic part­ners.

So­cial im­age: Take­da

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.