Stan­ley and Rosanne Crooke stay fo­cused on an­ti-sense, steal­ing back pa­tients from dis­ease with N-of-1 tech

A lit­tle over a year af­ter a Boston Chil­dren’s Hos­pi­tal neu­rol­o­gist used an­ti-sense tech­nol­o­gy to cre­ate what may be the world’s first ful­ly be­spoke ther­a­py, two of the old­est and biggest names in an­ti-sense have launched a char­i­ty to make those treat­ments pos­si­ble to far more pa­tients – and free of charge.

Stan­ley Crooke

The hus­band-and-wife team of long­time Io­n­is CEO Stan­ley Crooke and long­time Io­n­is re­searcher Rosanne Crooke are launch­ing the n-Lorem foun­da­tion. The cou­ple will per­son­al­ly con­tribute $1.5 mil­lion to the project, with Io­n­is adding an­oth­er $1.5 mil­lion and Bio­gen $1 mil­lion. Stan­ley Crooke stepped down from Io­n­is’ helm last year.

Found­ed by Crooke in 1989, Io­n­is was the first com­pa­ny to launch a clin­i­cal tri­al for an­ti-sense tech­nol­o­gy, sin­gle strands of RNA that can be used to reg­u­late gene ex­pres­sion. The tech­nol­o­gy has since be­come a ver­i­fied ap­proach, lead­ing to drugs such as Spin­raza and Waylivra.

The ad­vance­ments crys­tal­lized in 2018, when, in un­der 10 months, Tim­o­thy Yu used an­ti-sense tech­nol­o­gy to cus­tomize a treat­ment for a 6-year-old girl who came in­to Boston’s Chil­dren’s Hos­pi­tal with a nev­er-be­fore-seen ge­net­ic mu­ta­tion. The news sparked hope and prompt­ed pleas for their own ther­a­pies from fam­i­lies af­fect­ed by rare mu­ta­tions.

Rosanne Crooke

The Crookes launched the new foun­da­tion to make these “N-of-1” ther­a­pies pos­si­ble for more of those fam­i­lies. Stan­ley Crooke told Chem­i­cal & En­gi­neer­ing News that he sees this n-Lo­ram as a mid­dle man who can co­or­di­nate be­tween all the parts re­quired to scale be­spoke treat­ments: Pa­tients, a clin­i­cian who can man­age a one-pa­tient tri­al, and an in­sti­tu­tion who can sub­mit an IND-en­abling ap­pli­ca­tion, among oth­er things.

“I con­ceived of n-Lorem as sort of the mid­dle man—the bro­ker of the ge­net­ics, the pa­tients, the needs, and one so­lu­tion to bring ther­a­peu­tic op­tions for these pa­tients who have ul­tra, ul­tra-rare mu­ta­tions,” Crooke told C&EN.

The foun­da­tion will con­vene an “ac­cess to treat­ment” pan­el of re­searchers, pa­tients and Bio­gen and Io­n­is ex­ec­u­tives. They will re­view US-based pa­tients who come through the Un­di­ag­nosed Dis­ease Net­work, a group of NIH-fund­ed sites with ex­per­tise in ul­tra-rare dis­eases.

There will be sig­nif­i­cant road­blocks, though. In ad­di­tion to the sci­en­tif­ic hur­dles of de­vel­op­ing in­di­vid­u­al­ized ther­a­pies, it’s still un­clear how the FDA will re­spond. When the New Eng­land Jour­nal of Med­i­cine pub­lished Yu’s work in Oc­to­ber, they ac­com­pa­nied it with an op-ed from the FDA’s Janet Wood­cock and Pe­ter Marks. The pair de­tailed 9 dif­fer­ent ques­tions reg­u­la­tors will face go­ing for­ward and pro­vid­ed few an­swers.

“In these ‘N-of-one’ sit­u­a­tions, what type of ev­i­dence is need­ed be­fore ex­pos­ing a hu­man to a new drug?” they wrote. “Even in rapid­ly pro­gress­ing, fa­tal ill­ness­es, pre­cip­i­tat­ing se­vere com­pli­ca­tions or death is not ac­cept­able, so what is the min­i­mum as­sur­ance of safe­ty that is need­ed? How per­sua­sive should the mech­a­nis­tic or func­tion­al da­ta be? How should the dose and reg­i­men be se­lect­ed? How much char­ac­ter­i­za­tion of the prod­uct should be un­der­tak­en? How should the ur­gency of the pa­tient’s sit­u­a­tion or the num­ber of peo­ple who could ul­ti­mate­ly be treat­ed af­fect the de­ci­sion-mak­ing process?”

So­cial im­age cred­it: Oligonu­cleotide Ther­a­peu­tics So­ci­ety

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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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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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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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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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

Jan van de Winkel, Genmab CEO

Seat­tle Ge­net­ics, Gen­mab turn on TV for a high­light reel in cer­vi­cal can­cer — but a ri­val biotech promis­es a bet­ter show

Seattle Genetics $SGEN and their partners at Genmab $GMAB polished up some positive Phase II numbers for their antibody drug conjugate tisotumab vedotin — you can call it TV — for recurrent cervical cancer. And while they mapped out a shortcut to a potential quick approval, the big challenge for this team is being presented by a rival biotech which muscled its way into the spotlight for the same indication a year ago.

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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.

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.