Ge­nomics meets blockchain: Har­vard team fea­tur­ing George Church launch­es peer-to-peer genome se­quenc­ing start­up

George Church

George Church has long ad­vo­cat­ed for big da­ta in ge­nomics; his lab at Har­vard pi­lot­ed a project to col­lect and share vol­un­teers’ ge­nom­ic da­ta for col­lab­o­ra­tive re­search. Now, with two oth­er Har­vard-con­nect­ed founders, he’s tak­ing the shar­ing one step fur­ther by in­cor­po­rat­ing blockchain, the ledger sys­tem most fa­mous for pow­er­ing Bit­coin.

Neb­u­la Ge­nomics emerged from stealth mode Wednes­day with a whitepa­per — not a press re­lease — de­tail­ing its vi­sion. In that world, any­one can se­quence their genome at a low cost, glean in­sights from the re­sults, and have to­tal con­trol on what they want to do with them.

Cur­rent per­son­al ge­nomics play­ers like He­lix and 23andMe adopt a mod­el where the com­pa­nies func­tion as a mid­dle­man be­tween in­di­vid­u­als who want da­ta about their genome and phar­ma com­pa­nies will­ing to pay for that da­ta to use in drug de­vel­op­ment. Neb­u­la says it doesn’t want to be that mid­dle­man. In­stead, the in­di­vid­ual would be in the mid­dle in­ter­act­ing with both Neb­u­la and phar­ma com­pa­nies on a Neb­u­la-built plat­form.

To be sure, Neb­u­la is not the first com­pa­ny to mar­ry blockchain to ge­nomics. Lu­na DNA, a San Diego start­up found­ed by for­mer ex­ec­u­tives of genome se­quenc­ing gi­ant Il­lu­mi­na, is al­so build­ing a DNA data­base with blockchain. The key dif­fer­ence is that Lu­na would pay cus­tomers (in Lu­na coins) for the da­ta that it even­tu­al­ly sells to phar­mas, while Neb­u­la would stay out of the di­rect buy­ing and sell­ing, on­ly of­fer­ing an in­fra­struc­ture in which genome se­quenc­ing would be es­sen­tial­ly sub­si­dized.

Ac­cord­ing to the white pa­per, this ap­proach will re­sult in low­er se­quenc­ing costs (as com­pa­nies would es­sen­tial­ly sub­si­dize in­di­vid­u­als for the tests); more se­cure da­ta pro­tec­tion (blockchain is en­crypt­ed and anony­mous); and more ef­fi­cient da­ta ag­gre­ga­tion.

“It’s a new ap­proach to chal­lenges of ge­nomics, in­clud­ing se­quenc­ing costs, ge­net­ic da­ta pro­tec­tion, da­ta man­age­ment, and ge­nomics big da­ta,” Church told STAT.

Den­nis Gr­ishin

So what would the process look like? First off, the ac­tu­al se­quenc­ing will be done by Ver­i­tas Ge­net­ics, an­oth­er Church start­up. The pay­ment is where it gets fuzzy. The idea is cus­tomers would pay for the se­quenc­ing da­ta with Neb­u­la to­kens, its in-house cryp­tocur­ren­cy, which would be sold to phar­ma com­pa­nies for cash. The to­kens then cir­cu­late back to the cus­tomers as phar­ma com­pa­nies use them to pay for per­son­al da­ta. It’s un­clear how the cus­tomers would get the to­kens in the first place; whether to­kens would be of any val­ue to some­one who’s al­ready had their genome se­quenced al­so re­mains a ques­tion.

“We are not an­nounc­ing any to­ken sale at the mo­ment,” co-founder Den­nis Gr­ishin told STAT. “A to­ken is nec­es­sary for the func­tion­al­i­ty of our pro­to­col, but we have not de­cid­ed yet how they will be dis­trib­uted.”

Neb­u­la is now op­er­at­ing on an ini­tial $600,000 in­vest­ment from an an­gel in­vestor, Gr­ishin al­so said, and they ex­pect to have raised an ad­di­tion­al $1 mil­lion by the end of the week.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.