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.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.
The oral TKI, designed to zero in on HER2 with a bonus for penetrating the blood brain barrier, delivered a 46% hazard ratio — reduction in risk of disease progression or death — when combined with Herceptin and Xeloda compared to the branded combo alone. That was the primary endpoint. And then two key secondaries followed, starting with a 34% reduction in risk of death alone with a p value for overall survival that hit an approval worthy p=0.0048. There was a more dramatic 52% HR among almost half of the patients who had a brain metastasis at the time of the trial, which some of the analysts see as a key to market success.
We’ll have to wait until the San Antonio Breast Cancer Symposium for the breakdown on just how many months of PFS and OS Seattle Genetics is talking about.
While the researchers were after drug resistant third-line cases in the study, where regulators are generous in allowing high levels of toxicity for dying patients, the safety profile is still key here to the drug’s competitive position on the market. By steering clear of EGFR-linked toxicity, the company is looking for a better safety profile. And while the triplet led to a 5.7% drop out rate due to adverse events, compared to 3% for the double, analysts have been scoring this one much better than Nerlynx or Tykerb.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

FDA ap­proval lets Foamix set its maid­en ac­ne ther­a­py on course for US mar­ket launch

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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