Feng Zhang, heavy­weight col­lab­o­ra­tors un­veil lat­est CRISPR up­start — a di­ag­nos­tic com­pa­ny

Im­age: David Walt, Todd Gol­ub, Rahul Dhan­da, Feng Zhang, Deb­o­rah Hung. SHER­LOCK

One of the more dra­mat­ic dy­nam­ics in the aca­d­e­m­ic CRISPR world in­volves the Broad In­sti­tute and the Uni­ver­si­ty of Cal­i­for­nia, where Feng Zhang and Jen­nifer Doud­na, two pi­o­neers of the field, have their labs re­spec­tive­ly. As the dust starts to set­tle on their patent dis­pute re­gard­ing ther­a­peu­tic us­es of the gene edit­ing tech, a new storm is brew­ing in the di­ag­nos­tic ap­pli­ca­tions of CRISPR.

Zhang and eight oth­er lead­ing ex­perts in CRISPR, syn­thet­ic bi­ol­o­gy, di­ag­nos­tics and in­fec­tious dis­eases have pulled back the cur­tain on Sher­lock Bio­sciences, a start­up aimed at pro­vid­ing “bet­ter, faster and more af­ford­able” ways to de­tect ge­net­ic sig­na­tures of dis­ease.

The com­pa­ny stands on two foun­da­tion­al tech­nolo­gies: SHER­LOCK, or Spe­cif­ic High-sen­si­tiv­i­ty En­zy­mat­ic Re­porter un­LOCK­ing, was de­vel­oped by Zhang and col­lab­o­ra­tors in­clud­ing co-founders Jonathan Gooten­berg and Omar Abu­dayyeh. Pair­ing CRISPR with the Cas13 en­zyme, the tech­nol­o­gy uti­lizes a guide RNA to find a ge­net­ic se­quence, where Cas13 not on­ly cuts the tar­get but be­gin shriv­el­ing oth­er RNA near­by, cre­at­ing a sig­nal that can then be pre­sent­ed in ob­serv­able forms — such as a line on a pa­per strip.

“Any one tech­nol­o­gy out there had some re­al­ly valu­able at­trib­ut­es in mol­e­c­u­lar di­ag­nos­tics but SHER­LOCK brought all of those to­geth­er,” Rahul Dhan­da, a co-founder and di­ag­nos­tics vet­er­an who took on the CEO role, told me. “It had speed, so you didn’t have to wait days for an in­fec­tious dis­ease re­sult; it had af­ford­abil­i­ty, which meant that you can do on­col­o­gy test­ing in a way that let every­body par­tic­i­pate in those re­sults; and it had sim­plic­i­ty, which means that you can reach peo­ple to do test­ing that couldn’t do it be­fore in de­cen­tral­ized set­tings.”

First in­vent­ed in 2017, an en­hanced ver­sion of SHER­LOCK was de­scribed in a pa­per pub­lished last Feb­ru­ary along­side an­oth­er ex­plain­ing DE­TEC­TR, a sim­i­lar tool that de­ploys Cas12 and a flu­o­res­cent mol­e­cule for de­tec­tion.

Doud­na of UC Berke­ley, who led the work on DE­TEC­TR, de­buted Mam­moth Bio­sciences short­ly there­after with a group of grad stu­dents, some of them from her lab. When we last heard from them, Mam­moth was work­ing with a $23 mil­lion round and back­ing from May­field, NFX, 8VC as well as Ap­ple’s Tim Cook and Grail found­ing CEO Jeff Hu­ber.

Im­age: Omar Abu­dayyeh, Jonathan Gooten­berg, Rahul Dhan­da, James Collins, Par­dis Sa­beti. SHER­LOCK

While com­par­isons with Mam­moth might be in­evitable, Dhan­da says he’s “very con­fi­dent” on the patent side and has faith in Sher­lock’s mul­ti-plat­form ap­proach to en­gi­neer­ing bi­ol­o­gy.

“Now I’ve been in the in­dus­try 20 years, I know how to nav­i­gate this field; it’s a nu­anced enough field that ex­pe­ri­ence mat­ters,” he added. “I would say that when I think about who we should be fo­cused on as com­pe­ti­tion, those are the large play­ers, the es­tab­lished mul­ti-bil­lion dol­lar di­ag­nos­tic com­pa­nies out there, and those are the com­pa­nies that we in­tend to build strong part­ner­ships with if we can find the right com­ple­ment to our goals and theirs.”

Sher­lock now has $35 mil­lion in the bank — half of it in the form of a grant from the Open Phil­an­thropy Pro­ject, which al­so pro­vid­ed an ad­di­tion­al in­vest­ment. As it ex­plores ap­pli­ca­tions in in­fec­tious dis­eases, pre­ci­sion on­col­o­gy and food in­spec­tion, it is on the look­out for “se­lec­tive part­ner­ing” while ad­vanc­ing de­vel­op­ment pro­grams and de­sign­ing new as­says on its own, the com­pa­ny said. So far, that has in­volved every­thing from phar­ma­ceu­ti­cal com­pa­nies look­ing for com­pan­ion di­ag­nos­tics to an­timi­cro­bial de­vel­op­ers seek­ing help with clin­i­cal tri­als to con­sumer health play­ers in search of at-home test­ing. Agri­cul­ture and bio­process test­ing are among the pos­si­bil­i­ties, too.

Dhan­da is still rais­ing cash for Sher­lock’s Se­ries A, which will al­so help fund the sec­ond plat­form in Sher­lock’s ar­se­nal: IN­SPEC­TR (IN­ter­nal Splint-Pair­ing Ex­pres­sion Cas­sette Trans­la­tion Re­ac­tion). James Collins at the Wyss In­sti­tute was cred­it­ed for the tech, which com­pris­es two halves of a cod­ing re­gion that fuse to cre­ate a sig­nalling pro­tein when the tar­get is present, ba­si­cal­ly repli­cat­ing how a cell would gen­er­ate a pro­tein.

In the com­ing year, Dhan­da is al­so plan­ning to hire 6 to 10 peo­ple to ramp up the R&D work cur­rent­ly han­dled by 10 staffers.

Oth­er co-founders in­clude Todd Gol­ub, Deb­o­rah Hung, Par­dis Sa­beti and David Walt — il­lus­tri­ous aca­d­e­mics who make up a team “like none oth­er.”

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.
Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.
JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.
As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.
Here’s what physicians and patients are likely to be confronted with in the not too distant future, as Pfizer goes about the tricky business of getting a JAK inhibitor past regulators at the FDA and EMA.
Lined up side by side we see:
IGA response rate (clear or nearly clear skin)

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Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

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A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

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Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

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