#ASH17: Blue­bird touts ear­ly signs that its new-and-im­proved sick­le cell gene ther­a­py is work­ing

Blue­bird bio $BLUE says they’ve been able to gath­er ev­i­dence to help prove that their new-and-im­proved ap­proach to mak­ing a gene ther­a­py for sick­le cell dis­ease has the po­ten­tial to be a con­sis­tent­ly ef­fec­tive once-and-done ther­a­py.

Fol­low­ing up on some dis­ap­point­ing ev­i­dence of in­con­sis­ten­cy among the first small group of pa­tients treat­ed with Lenti­Glo­bin, blue­bird’s team went back to the draw­ing board to whip up a new ap­proach to man­u­fac­tur­ing that they be­lieved would over­come their ini­tial set­back.

Two pa­tients — 1312 and 1313 — were treat­ed us­ing the re­fined process as least once. (The first was treat­ed twice, first us­ing the old and then the new ap­proach.) Their ex­pe­ri­ences will be spot­light­ed at the up­com­ing ASH meet­ing in ear­ly De­cem­ber, with up­dates com­ing on their re­sponse.

Mo­hammed As­mal

It takes a step in a di­rec­tion we feel is quite pos­i­tive,” says Mo­hammed As­mal, blue­bird’s VP of clin­i­cal de­vel­op­ment.

That mes­sage helped stoke a 15% in­crease in the biotech’s share price.

The re­al proof will come when re­searchers have a chance to see if bet­ter cell en­graft­ment al­lows these pa­tients to pro­duce nor­mal red blood cells, po­ten­tial­ly cur­ing the ail­ment.

It was clear ear­ly on among the first group of pa­tients that there was an en­graft­ment prob­lem, says blue­bird CEO Nick Leschly. So they de­cid­ed to take a new course. First, they took ac­tion to bat back the in­flam­ma­tion and oth­er prob­lems that oc­curred in pa­tients’ bone mar­row, giv­ing them bet­ter stem cells to work with. Then they used a new man­u­fac­tur­ing process — to be high­light­ed at ASH — that gave them a much high­er vec­tor copy num­ber to work with, im­prov­ing the odds of suc­cess.

That was clear­ly ap­par­ent in the first pa­tient, who was first treat­ed with a ther­a­py made from the first ap­proach, fol­lowed by more ef­fec­tive treat­ment in the sec­ond.

Nick Leschly

“The copy num­ber was (ini­tial­ly) in a range of .3, .5 to 1,” says the CEO. “Now you look across, and we’re now in the range of 2 to 3.” And he adds: “The num­ber of cells mod­i­fied is dra­mat­i­cal­ly dif­fer­ent.”

To be sure, it’s a small num­ber of pa­tients to re­view, but in sick­le cell dis­ease small num­bers can be com­pelling. And blue­bird has lots of rea­sons to tout a new, more ef­fec­tive ther­a­peu­tic ap­proach af­ter stum­bling ear­ly on, rais­ing doubts about their abil­i­ty to beat out oth­er ther­a­pies now in the clin­ic.

That’s what they will be dis­cussing at ASH as they prep for more work with a new batch of pa­tients who will be in­volved in the piv­otal work ahead.

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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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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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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George Yancopoulos (Regeneron)

UP­DAT­ED: 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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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

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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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.

Paul Tesar (Convelo Therapeutics)

Io­n­is, lead­ing MS re­searcher throw an­ti­sense at a new type of brain cells

No matter how many molecules he threw at them, Paul Tesar couldn’t get the brain cells to survive. Or he got them to survive, but then — to everyone’s bafflement — they still couldn’t do what they were supposed to.

Tesar, a professor of innovative therapeutics at Case Western University, had spent years building stem cell models for multiple sclerosis, growing brain organoids in dishes and then seeing what small molecules restored myelin production. Now he was trying to do the same for other myelin diseases, particularly an ultra-rare genetic condition called Pelizaeus-Merzbacher disease, where a single mutation leads to the death of the myelin-producing neurons, called oligodendrocytes, and can kill patients in infancy.