Take that Ed­i­tas: CRISPR Ther­a­peu­tics and col­leagues win a Eu­ro­pean round in the IP tug-of-war over gene edit­ing tech

Jen­nifer Doud­na

Still boil­ing over the lat­est patent de­ci­sion on CRISPR/Cas 9 IP in the US, CRISPR Ther­a­peu­tics $CR­SP says its sci­en­tif­ic founders have won a broad patent cov­er­ing their work in Eu­rope.

The Eu­ro­pean Patent Of­fice has come through with patent no. 13793997, says the biotech. And the UK’s patent of­fice fol­lowed through, tak­ing the same po­si­tion.

Em­manuelle Char­p­en­tier

“We and the li­cen­sors of these foun­da­tion­al cas­es for CRISPR/Cas9 gene edit­ing have long ap­pre­ci­at­ed that Em­manuelle Char­p­en­tier, Jen­nifer Doud­na, and their doc­tor­al and post-doc­tor­al col­leagues pro­vid­ed es­sen­tial teach­ings that ef­fec­tive­ly en­abled and fa­cil­i­tat­ed the CRISPR field, which has al­lowed both re­searchers and gene edit­ing com­pa­nies to eas­i­ly and pre­cise­ly mod­u­late tar­get DNA se­quences in any cell type and set­ting, in­clud­ing in hu­man cells for po­ten­tial ther­a­peu­tic ap­pli­ca­tions,” said Dr. Tyler Dy­lan-Hyde, the chief le­gal of­fi­cer of CRISPR Ther­a­peu­tics. “We al­so ap­pre­ci­ate that, de­spite at­tempts by third par­ties try­ing to de­lay or pre­vent these grants through TPOs or po­ten­tial op­po­si­tions, patent of­fices are be­gin­ning to rec­og­nize both the fun­da­men­tal sig­nif­i­cance of the teach­ings by the Char­p­en­tier-Doud­na team and their wide ap­plic­a­bil­i­ty to the CRISPR field. We look for­ward to pur­su­ing sim­i­lar find­ings in the U.S. un­der the first-to-in­vent sys­tem, and through­out the ap­prox­i­mate­ly 80 oth­er coun­tries our fil­ings cov­er world­wide, all of which are on a first-to-file pri­or­i­ty sys­tem.”

Feng Zhang

Ed­i­tas Med­i­cines $ED­IT came out on top re­cent­ly in the lat­est US matchup. The ri­val biotech re­lies in part on patents held by The Broad, which orig­i­nat­ed in work done by Feng Zhang, a one-time col­lab­o­ra­tor with Doud­na and Char­p­en­tier.

Back in De­cem­ber, just days af­ter CRISPR Ther­a­peu­tics and a group of ri­vals formed an IP al­liance around CRISPR/Cas9 gene edit­ing tech, Ed­i­tas fired back in the patent war by bag­ging an ex­clu­sive li­cense to what was be­ing billed as an up­grade to im­prove re­searchers’ abil­i­ty to ed­it genes and fight dis­ease.

How does this new patent af­fect Ed­i­tas?

Sam Kulka­rni, Crispr

Sam Kulka­rni, the chief busi­ness of­fi­cer at CRISPR Ther­a­peu­tics, tells me in an email that the Eu­ro­pean patent sets up a key IP road­block for any com­pa­ny look­ing to com­mer­cial­ize CRISPR/Cas9 on the con­ti­nent.

The de­ci­sion of the EPO to grant the patents with broad claims for CRISPR/Cas9 genome edit­ing across all cell types means that ac­cess to the CRISPR/UC Foun­da­tion­al IP will like­ly be nec­es­sary for any par­ty try­ing to com­mer­cial­ize CRISPR-based ther­a­peu­tics in Eu­rope.  The patents grant­ed to the Broad In­sti­tute in Eu­rope are lim­it­ed in na­ture and al­so the sub­ject of pro­ceed­ings chal­leng­ing their va­lid­i­ty and is­suance at the Op­po­si­tion Di­vi­sion of the EPO.

A spokesper­son for Ed­i­tas, though, doesn’t sound too wor­ried.

Our strat­e­gy from day one has been to build a strong IP po­si­tion that is fun­da­men­tal, broad, and mul­ti­fac­eted. It’s an im­por­tant in­vest­ment for Ed­i­tas and is part of build­ing the lead­ing ge­nom­ic med­i­cine com­pa­ny for the long-term. Ed­i­tas’ foun­da­tion­al IP in­cludes patents is­sued by both the US and Eu­rope Patent Of­fices cov­er­ing the use of CRISPR/Cas9 in eu­kary­ot­ic cells.  Con­se­quent­ly, we are con­fi­dent in our IP po­si­tion, which we be­lieve is a nec­es­sary fac­tor for mak­ing CRISPR med­i­cines for peo­ple.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

Top Har­vard chemist caught up in FBI’s 'T­hou­sand Tal­ents' drag­net, ac­cused of ly­ing about Chi­nese con­nec­tions, pay

The FBI’s probe into the alleged theft of R&D secrets by Chinese authorities has drawn Harvard’s top chemist into its net.

The agency accused Charles M. Lieber, who chairs the university’s chemistry and chemical biology department, with lying about his involvement in China’s Thousand Talents campaign, which was established as a way of drawing in innovators from around the world. And the scientist, 60, was charged with making false statements about his ties to China.

The US attorneys office in Boston also announced charges against two Chinese nationals for helping the Chinese government.

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Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Passage Bio — Jim Wilson’s self-described “legacy company” — has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.

UP­DAT­ED: Ac­celeron res­ur­rects block­buster hopes for so­tater­cept with pos­i­tive PhII — and shares rock­et up

Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

“We’re thrilled to report such positive topline results from the PULSAR trial,” Acceleron CEO Habib Dable said in a statement. The company said in a conference call they plan on discussing a Phase III trial design with regulators.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”