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

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

The venture tide might have subsided, the IPO window may be closing and certain listed biotechs may be having a tough time amid Neil Woodford’s well-publicized demised, but there’s still plenty to celebrate in the UK BioIndustry Association’s eyes.

Overall investment in UK biotech last year fell from the record-breaking £2.2 billion levels of 2018 to £1.3 billion — including £679 million in venture capital, a meager £64 million in IPOs plus £596 million when you add up all public financings, according to a new report from the BIA.

→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering.  Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III VOYAGER before they give a judgment.

Late last year Novartis abandoned a cardio drug from Ionis’ spinoff Akcea just after the pharma giant snapped up inclisiran, going the RNAi way in guarding against heart disease in the $9.7 billion Medco buyout.

Now the pharma goliath — which is headed down the PCSK9 road with a drug it believes can be used in a mass population — can get a clearer picture of just what they gave up.

Akcea $AKCA and the mother company $IONS put out a statement early Wednesday saying that their Phase II study of AKCEA-APOCIII-LR delivered solid efficacy data, with the high dose clearly outperforming placebo in significantly reducing triglycerides as a means to cutting the risk of cardiovascular disease. In addition, investigators concluded that the drug slashed apoC-III, very low-density lipoprotein and remnant cholesterol while boosting “good” HDL levels.

Update: Nominations open through end of day, Monday, January 27

Two years ago, when we did our first Endpoints 20-under-40, we profiled a set of up-and-comers who promised to help reshape the industry as we know it. Now we’re back and once again looking for the top 20 biopharma professionals under the age of 40. We’ll be profiling folks who have accomplished a lot at a young age but seem on the verge of accomplishing so much more.