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.”

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.

Morningside continued to lead the $30 million Series B, with participation from unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.

Days af­ter In­ter­cept re­jec­tion, Akero surges on ‘un­prece­dent­ed‘ NASH da­ta

A year and a half after scoring a $70 million Series B and a top Gilead executive as CEO, Akero Therapeutics has announced new data on their NASH drug. And with the field still reeling from a surprise FDA rejection this week, the news was enough to send their stock surging.

Akero had already said in March that its lead drug had beaten placebo in its Phase II trial, reducing liver fat by 14% in the highest dose group compared to 0.3% in placebo, according to MRI scans. But although NASH is an obesity-related condition and results from fatty buildup in the liver, the real immediate question for any therapy is whether it can resolve the fibrosis and inflammation that results from that buildup. Those data require biopsying the patients, a longer and more invasive process that was further complicated by a pandemic.