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.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a plan to near­ly dou­ble its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

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Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.