Reck­on­ing the po­ten­tial of CRISPR/Cas9 tech, NIH launch­es $190M genome edit­ing re­search ini­tia­tive

Fran­cis Collins, the di­rec­tor of the Na­tion­al In­sti­tutes of Health CQ/Roll Call Get­ty


As the first hu­man tri­al in­volv­ing CRISPR comes in­to view in the Unit­ed States, the NIH has stepped up with a $190 mil­lion re­search ini­tia­tive to help float all the boats rid­ing on the wave of new genome edit­ing tech­nolo­gies.

Named So­mat­ic Cell Genome Edit­ing, the pro­gram will award funds over six years to re­searchers col­lab­o­rat­ing to ex­pand the toolk­it for the whole sci­en­tif­ic com­mu­ni­ty.  im­prov­ing the de­liv­ery mech­a­nisms, de­vel­op­ing bet­ter ed­i­tors, de­sign­ing as­says to eval­u­ate those edit­ing tools, and as­sem­bling a pack­age of knowl­edge and meth­ods. Ul­ti­mate­ly, the hope is to re­move bar­ri­ers to broad adop­tion of the tech in the clin­ic.

“Genome edit­ing tech­nolo­gies such as CRISPR/Cas9 are rev­o­lu­tion­iz­ing bio­med­ical re­search,” said di­rec­tor Fran­cis Collins in a state­ment. “The fo­cus of the So­mat­ic Cell Genome Edit­ing pro­gram is to dra­mat­i­cal­ly ac­cel­er­ate the trans­la­tion of these tech­nolo­gies to the clin­ic for treat­ment of as many ge­net­ic dis­eases as pos­si­ble.”

As its name sug­gests, the pro­gram fo­cus­es on so­mat­ic cells — non-re­pro­duc­tive cells that do not pass DNA down to the next gen­er­a­tion. In oth­er words, any changes to the DNA in­tro­duced by these kinds of ther­a­peu­tics would not be in­her­it­ed. That avoids the stick­i­est eth­i­cal con­cerns tied to the use of US tax­pay­er funds.

This new in­vest­ment by the NIH comes at a piv­otal mo­ment for gene edit­ing.

The pi­o­neers have had their patent fight, and star-stud­ded new play­ers such as Ex­on­ics and Tmu­ni­ty are start­ing to come on the scene. With Chi­na rac­ing ahead in the clin­ic — 86 pa­tients have had their genes edit­ed, per the Wall Street Jour­nal — de­vel­op­ing the in­fra­struc­ture need­ed to ad­vance next-gen tech ben­e­fits all play­ers.

Mary Ellen Per­ry

In fact, the spe­cif­ic di­rec­tions of the pro­gram are the prod­uct of a brain­storm­ing work­shop with in­dus­try ex­perts, pa­tient ad­vo­cates and aca­d­e­m­ic re­searchers. That’s ac­cord­ing to Mary Ellen Per­ry, who first pre­sent­ed the con­cept to a group of NIH in­sid­ers in Sep­tem­ber. What comes out of this pro­gram, she said, could im­pact every­thing from com­mon dis­or­ders to rare dis­eases.

“It’s al­most dif­fi­cult to think of a con­di­tion that this might not be ap­plic­a­ble to,” Per­ry said at the meet­ing.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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