Blue­bird’s gene ther­a­py for be­ta-tha­lassemia falls short of a cure, but still wows in 22 pa­tients

Af­ter go­ing back to the draw­ing board to come up with a bet­ter gene ther­a­py for be­ta-tha­lassemia, a dis­ease that in­ter­feres with the body’s abil­i­ty to pro­duce he­mo­glo­bin, in­ves­ti­ga­tors work­ing for blue­bird bio $BLUE have pro­duced some com­pelling da­ta to demon­strate that their ther­a­py can elim­i­nate or re­duce the need for blood trans­fu­sions.

But they don’t have a cure — yet.

Pub­lish­ing in the New Eng­land Jour­nal of Med­i­cine, the re­searchers re­port that they test­ed the treat­ment in 22 pa­tients. In the 9 pa­tients with the most se­vere form of the dis­ease, they re­port they were able to dra­mat­i­cal­ly re­duce the need for blood trans­fu­sions by 73% and elim­i­nate it for now in 3. Six of those pa­tients, though, have need­ed to con­tin­ue trans­fu­sions. And 12 of 13 pa­tients with a less se­vere form of the dis­ease cur­rent­ly no longer re­quire blood trans­fu­sions af­ter a sin­gle treat­ment — with a me­di­an of 26 months trans­fu­sion-free.

The in­ter­im da­ta great­ly ex­pands on the pa­tient ex­pe­ri­ences they can now bank on to demon­strate the ef­fi­ca­cy of the at­tempt at a once-and-done ther­a­py which af­fects hun­dreds of thou­sands of pa­tients. And the in­ves­ti­ga­tors have not aban­doned an at­tempt to find a com­plete cure for all.

“There is room for im­prove­ment, as we’d like to see the elim­i­na­tion of de­pen­den­cy on trans­fu­sion even for pa­tients with the most se­vere form of the dis­ease,” Har­vard Med­ical’s Philippe Leboulch told their in-house pub. “But there is al­so hope with pro­to­col mod­i­fi­ca­tions we have in­tro­duced in our Phase III tri­als.”

Blue­bird — run by CEO Nick Leschly — was forced to go back and change the man­u­fac­tur­ing process on Lenti­Glo­bin af­ter some un­even re­spons­es ear­ly on. Last sum­mer, the com­pa­ny came up with some en­cour­ag­ing da­ta on the first three pa­tients.

The gene ther­a­py is made by ex­tract­ing blood stem cells from pa­tients and in­sert­ing a func­tion­al be­ta-glo­bin gene in­to them, trans­plant­i­ng them af­ter a round of chemo to clear their bone mar­row.

Jerome Groop­man in The New York­er writes:

The chal­lenge now is find­ing ways to im­ple­ment a com­plex, po­ten­tial­ly life-sav­ing treat­ment in parts of the world where med­ical care is lim­it­ed. Es­tab­lished fa­cil­i­ties for au­tol­o­gous mar­row trans­plan­ta­tion al­ready ex­ist in many de­vel­oped na­tions, as do lab­o­ra­to­ries that can in­tro­duce a healthy glo­bin gene in­to stem cells. But, in some parts of the world where be­ta tha­lassemia is most com­mon, these fa­cil­i­ties do not yet ex­ist.

Con­trary to what the en­thu­si­asts may be pre­sent­ing to­day, there’s clear­ly room for im­prove­ment. And blue­bird is clear­ly not alone in the field try­ing to pro­duce a cure. CRISPR Ther­a­peu­tics, for ex­am­ple, has been us­ing its gene edit­ing tech to see if it can find a last­ing cure for the dis­ease. And those oth­er pro­grams, in­clud­ing ef­forts at Bel­licum, will like­ly con­tin­ue to see if they can leapfrog blue­bird with some­thing bet­ter.


blue­bird bio CEO Nick Leschly Get­ty

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Lynn Baxter, Viiv Healthcare's head of North America

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The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

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