WHO is­sues new guide­lines for gene edit­ing; Doud­na-backed Cari­bou launch­es its first al­lo­gene­ic CAR-T tri­al

Three years af­ter news of the birth of two gene-edit­ed ba­bies shocked ethi­cists, reg­u­la­tors and bi­ol­o­gists around the world, World Health Or­ga­ni­za­tion ex­perts called for the es­tab­lish­ment of a new whis­tle-blow­ing mech­a­nism to help pre­vent fu­ture un­safe or un­eth­i­cal us­es of CRISPR and oth­er gene edit­ing tech­nolo­gies.

The rec­om­men­da­tion was one of a se­ries the WHO pan­el is­sued Mon­day in a pair of re­ports de­signed to guide glob­al gov­er­nance of gene edit­ing. The pan­el was con­vened in De­cem­ber 2018, weeks af­ter Chi­nese sci­en­tist He Jiankui an­nounced two ba­bies were born from em­bryos he edit­ed, and fol­lows a sim­i­lar ef­fort from the In­ter­na­tion­al Com­mis­sion on the Clin­i­cal Use of Hu­man Germline Genome Edit­ing, which re­leased a se­ries of rec­om­men­da­tions last year.

Both pan­els have broad­ly con­demned germline edit­ing — i.e. mak­ing her­i­ta­ble changes to DNA, by edit­ing em­bryos, sperm or eggs — not­ing the tech­nol­o­gy is not so­phis­ti­cat­ed enough for re­searchers to make such ed­its safe­ly and ar­gu­ing that, even if it be­comes pos­si­ble to do so, it should on­ly be done for se­ri­ous dis­or­ders.

In ad­di­tion to the whistle­blow­ing mech­a­nism, the new re­ports al­so call for an in­ter­na­tion­al reg­istry to track all gene edit­ing ex­per­i­ments, al­though they ac­knowl­edge that the or­ga­ni­za­tion can’t com­pel coun­tries or com­pa­nies to co­op­er­ate. Ac­cord­ing­ly, they call for new meth­ods to spot bad ac­tors, par­tic­u­lar­ly as such ex­per­i­ments be­come cheap­er and more wide­ly ac­ces­si­ble. — Ja­son Mast

Doud­na-backed Cari­bou launch­es its first al­lo­gene­ic CAR-T tri­al

Less than two weeks af­ter Cari­bou Bio­sciences, launched out of No­bel Prize win­ner Jen­nifer Doud­na’s lab, filed its IPO pa­per­work to go pub­lic, the biotech is mak­ing more waves in the cell ther­a­py world.

Rachel Hau­r­witz

Cari­bou of­fi­cial­ly launched its first in-hu­man study of an al­lo­gene­ic CAR-T with an eye on liq­uid tu­mors Mon­day morn­ing, an­nounc­ing the dos­ing of its first pa­tient in a Phase I tri­al. The ther­a­py, known as CB-010, is be­ing eval­u­at­ed to re­duce the risk of graft-ver­sus-host dis­ease in or­der to boost CAR-T an­ti­tu­mor ac­tiv­i­ty.

“We be­lieve that im­prov­ing cell per­sis­tence is the key to un­lock­ing the full po­ten­tial of these ther­a­pies,” CEO Rachel Hau­r­witz said in a state­ment. “Us­ing our tech­nolo­gies, we ed­it the genome of healthy donor-de­rived T cells to en­able high­ly spe­cif­ic and ef­fi­cient in­ser­tion or dele­tion of genes at mul­ti­ple sites.”

Cari­bou’s ap­proach in­volved a hy­brid DNA/RNA edit­ing plat­form, which Hau­r­witz calls chRD­NA (pro­nounced “chardon­nay,” like the wine). The biotech be­lieves the hy­brid guides can pre­vent off-tar­get edit­ing com­mon­ly seen in cur­rent CRISPR ap­pli­ca­tions.

The ther­a­py tar­gets CD-19 us­ing a CRISPR-based ap­proach, and in ad­di­tion al­so take out PD-1. In the­o­ry, the sec­ondary step amps up the body’s T cells even more than they would be oth­er­wise.

The study will en­roll about 50 adults with re­lapsed or re­frac­to­ry B cell non-Hodgkin lym­phoma, ac­cord­ing to clin­i­cal­tri­als.gov. In the dose-es­ca­la­tion por­tion, adults who have failed at least two lines of chemo or im­munother­a­py will be ad­min­is­tered CB-010 af­ter go­ing through a process that de­pletes their nat­ur­al T cells.

Cari­bou will fol­low a “stan­dard 3+3 dose es­ca­la­tion de­sign,” the com­pa­ny said. Those who pre­vi­ous­ly re­ceived a CD-19 ther­a­py will be ex­clud­ed. — Max Gel­man

Rayze­Bio nabs new dis­cov­ery part­ner

Rayze­Bio has seen a quick rise since launch­ing back in Au­gust 2020, and now they have a new part­ner to fur­ther their ef­forts in ra­dio­phar­ma­ceu­ti­cals.

Ken Song

The San Diego biotech is team­ing up with Cana­di­an out­fit 48Hour Dis­cov­ery to de­vel­op new pep­tide-based treat­ments for can­cer us­ing the ra­dio­phar­ma plat­form, the com­pa­nies an­nounced Mon­day. Fi­nan­cial terms of the deal were not dis­closed.

“For this col­lab­o­ra­tion project, 48Hour Dis­cov­ery will iden­ti­fy po­tent lig­ands for a se­ries of tar­gets se­lect­ed by Rayze­Bio,” Rat­mir Der­da, CEO of 48Hour Dis­cov­ery, said in a state­ment. “This project will help us fur­ther val­i­date the po­ten­tial of our bil­lion-scale mol­e­c­u­lar li­braries and cloud-based dis­cov­ery pipeline.”

Rayze­Bio CEO Ken Song hint­ed at a pos­si­ble IPO last month when the biotech com­plet­ed its Se­ries C raise, not­ing the round might be its last pri­vate fundraise. Song has al­ready led the com­pa­ny to more than $250 mil­lion across pri­vate fund­ing rounds. — Max Gel­man

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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Mod­er­na es­tab­lish­es pub­lic health-fo­cused char­i­ty; FDA ap­proves As­traZeneca di­a­betes drug for pe­di­atric use

To help promote public health and healthcare in underserved areas of the world, Moderna will establish a charity with a $50 million endowment.

The Cambridge, MA-based company announced the board of directors’ approval Thursday. The foundation will focus on “charitable, scientific and educational endeavors” with an emphasis on promoting public health and the access to healthcare, the press release said. The foundation will start operations once its status as a 501(c)(3) is approved.

EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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Mol­e­c­u­lar Di­ag­nos­tics Can Trans­form Can­cer Care. Let’s Make It Hap­pen.

Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States.