In the glare of an in­tense gene ther­a­py spot­light, blue­bird con­tin­ues to tout each small step for­ward for its pi­o­neer­ing Lenti­Glo­bin

blue­bird bio CEO Nick Leschly. GET­TY

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As one of the pi­o­neers in the new wave of gene ther­a­py de­vel­op­ers, blue­bird bio $blue has spent con­sid­er­able time pro­vid­ing in­cre­men­tal ev­i­dence that it can step up re­sults for Lenti­Glo­bin, im­prov­ing the man­u­fac­tur­ing process and beef­ing up pos­i­tive out­comes for a ther­a­py that’s had its prob­lems along the way.

To­day, the re­search team has two new up­dates to of­fer on a hand­ful of pa­tients af­flict­ed by an in­abil­i­ty to pro­duce suf­fi­cient quan­ti­ties of healthy he­mo­glo­bin, couched in the re­lent­less­ly op­ti­mistic terms that has helped CEO Nick Leschly build their mar­ket cap to close to $10 bil­lion. Prepar­ing for an up­date this week­end at the 23rd Con­gress of the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion, they need to im­press an­a­lysts in the lead-up to a cru­cial mar­ket­ing ap­pli­ca­tion.

Blue­bird’s shares inched up to the $200 mark in ear­ly trad­ing Fri­day, with a gen­er­al­ly pos­i­tive spin on the lat­est da­ta. But they couldn’t hold on in the short run. By mid-morn­ing the stock was down 4% as the EHA gen­er­at­ed a touch crowd of ob­servers.

David David­son

In sick­le cell dis­ease, their fo­cus is on Group C, where 4 pa­tients past the 3-month fol­lowup stage are mak­ing more than 30% of an­ti-sick­ling HbAT87Q  — their de­f­i­n­i­tion of suc­cess. And one of those pa­tients who’s gone past 6 months has a nor­mal to­tal he­mo­glo­bin lev­el of 14.2 g/dL with 60%-plus pro­duc­tion.

These pa­tients are get­ting the new and im­proved ver­sion of the gene ther­a­py af­ter the orig­i­nal fell well short of a cure, and in­ves­ti­ga­tors are stay­ing on each pa­tient as blue­bird builds its case for the steep price they have in mind for Lenti­Glo­bin.

Dura­bil­i­ty is key here.

Blue­bird has made enough progress with its two stud­ies for trans­fu­sion-de­pen­dent β-tha­lassemia that CMO David David­son says to­day that they are on track to file for Eu­ro­pean ap­proval lat­er this year — even though it isn’t per­fect.

The key da­ta point here: Sev­en of 8 pa­tients are pro­duc­ing ≥ 7.6 g/dL of HbAT87Q and are main­tain­ing to­tal he­mo­glo­bin lev­els of 11.1 – 13.3 g/dL by 6 months. 

Blue­bird has be­come a fa­vorite tar­get for some ob­servers, ready to catch them out for hyp­ing less-than-per­fect num­bers in a field try­ing to achieve a near-per­fect record for a once-and-done ther­a­py. Their lat­est round of da­ta on the lead­ing BC­MA-tar­get­ing CAR-T caught some ear­ly flak at AS­CO a few days ago, ding­ing the stock. But the un­der­ly­ing pos­i­tives turned it around with in­vestors who re­main be­liev­ers.

“Con­sis­tent­ly high­er in vi­vo vec­tor copy num­bers and HbAT87Q he­mo­glo­bin lev­els in pa­tients in­di­cate that Lenti­Glo­bin man­u­fac­tur­ing re­fine­ments have re­sult­ed in im­proved gene ther­a­py char­ac­ter­is­tics and may en­able sus­tained trans­fu­sion in­de­pen­dence for a great ma­jor­i­ty of pa­tients,” said Pro­fes­sor Fran­co Lo­catel­li, the lead in­ves­ti­ga­tor of the North­star-2 study. 

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.