Paul Wotton, Obsidian CEO

Ver­tex adds a new gene edit­ing part­ner, pay­ing Ob­sid­i­an $75M for con­trol­lable CRISPR sys­tem

When Paul Wot­ton be­came CEO of Ob­sid­i­an Ther­a­peu­tics two years ago, he asked his team to ex­plore where they could ap­ply the com­pa­ny’s tech­nol­o­gy be­yond can­cer.

Launched by Michael Gilman in 2017, the GV-backed com­pa­ny had been work­ing on a way of con­trol­ling cell ther­a­pies af­ter they’re ad­min­is­tered to a pa­tient, al­low­ing doc­tors to mod­u­late the po­ten­cy of CAR-T and oth­er drugs as you might vol­ume on a tele­vi­sion set. Wot­ton want­ed to them look be­yond can­cer, in­to ar­eas such as gene edit­ing.

“Gene edit­ing was par­tic­u­lar­ly at­trac­tive be­cause it’s a plat­form ap­proach, where we can re­al­ly make a dif­fer­ence,” Wot­ton told End­points News. “And it gave us a lot of flex­i­bil­i­ty with re­spect to grow­ing the com­pa­ny through busi­ness de­vel­op­ment, while we fo­cused on our own pro­grams.”

That search has now paid off, to the tune of up to $75 mil­lion over the next 4 years and $1.3 bil­lion in long-term mile­stones. On Thurs­day, Ob­sid­i­an an­nounced that they struck a deal with Ver­tex to use the biotech’s tech­nol­o­gy’s to de­vel­op con­trol­lable gene edit­ing ther­a­pies. Al­though the part­ners didn’t dis­close what dis­eases they’re go­ing af­ter, Wot­ton said they hoped to have a can­di­date in the clin­ic in 3 to 4 years.

For Ver­tex, the deal rep­re­sents an­oth­er in­vest­ment in gene edit­ing. The Cam­bridge drug­mak­er, best known for its cys­tic fi­bro­sis pills, first in­vest­ed in the field in 2015 when they struck a long-term col­lab­o­ra­tion with CRISPR Ther­a­peu­tics, but it’s be­come a greater fo­cus as they looked to push in­to new dis­ease ar­eas.

Two years ago, they bought out the Duchenne mus­cu­lar dy­s­tro­phy gene edit­ing biotech Ex­on­ics, and they are now build­ing a new fa­cil­i­ty ded­i­cat­ed sole­ly to cell and gene ther­a­py. Ear­li­er this week, they spent $900 mil­lion to take an ad­di­tion­al 10% stake and op­er­a­tional con­trol of the sick­le cell ther­a­py they de­vel­oped with CRISPR.

David Alt­shuler

In a state­ment, Ver­tex CSO David Alt­shuler not­ed the deal al­so plays in­to the com­pa­ny’s ex­pe­ri­ence with small mol­e­cules, like their CF drugs. Ob­sid­i­an’s tech­nol­o­gy us­es ap­proved small mol­e­cule drugs as the “on/off” switch for cell or gene ther­a­pies in the body.

“This col­lab­o­ra­tion with Ob­sid­i­an builds up­on and will ex­pand Ver­tex’s lead­er­ship in small mol­e­cule and ge­net­ic ther­a­pies, and we’re ex­cit­ed to part­ner with the team at Ob­sid­i­an to ex­plore the ca­pa­bil­i­ties of their tech­nol­o­gy,”  Alt­shuler said. “The abil­i­ty to tune gene-edit­ing ac­tiv­i­ty to a spe­cif­ic lev­el is an im­por­tant in­no­va­tion that has the po­ten­tial to ad­dress sev­er­al se­ri­ous dis­eases.”

For Ob­sid­i­an, the deal marks their third part­ner­ship, adding to a CAR-T deal with Bris­tol My­ers Squibb and a deal with MD An­der­son to de­vel­op tu­mor in­fil­trat­ing lym­pho­cytes.

Wot­ton said Ob­sid­i­an has fig­ured out how to con­trol the Cas9 pro­tein that CRISPR sys­tems use to cut DNA. Es­sen­tial­ly, they add a pep­tide tag on top of Cas9. The pro­tein is on­ly ac­tive when doc­tors give a par­tic­u­lar small mol­e­cule that binds to that tag. With­draw the small mol­e­cule, and Cas9 stops cut­ting.

“It’s like a rheo­stat,” Wot­ton said of Ob­sid­i­an’s tech. “It al­lows you to di­al up or di­al down the ac­tiv­i­ty of a spe­cif­ic pro­tein.”

In can­cer, Ob­sid­i­an is try­ing to give doc­tors a tool to turn down im­mune-boost­ing ther­a­pies that can some­times at­tack healthy tis­sue in ad­di­tion to tu­mors.

With Ver­tex, the com­pa­nies will try to de­vel­op a sys­tem that boosts Cas9’s DNA-cut­ting abil­i­ties when they’re first ad­min­is­tered, but then turn down its ac­tiv­i­ty once the ed­it is made.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

Prahlad Singh, PerkinElmer

PerkinElmer hits the check­book again, this time dol­ing out $260M for next-gen ther­a­py bioser­vices firm

When PerkinElmer iced a deal to pick up UK gene editing firm Horizon Discovery, it trumpeted its big move into next-gen therapeutics. Now, not content to sit on its laurels, PerkinElmer is dipping into the war chest again, this time for a firm specializing in cutting-edge bioservices.

Life sciences services giant PerkinElmer will shell out $260 million to acquire Lawrence, MA-based Nexcelom Bioscience, which offers clinical services for next-gen cell and gene therapies, immuno-oncology drugs and vaccines, the companies said Thursday.

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UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).