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. 

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Ivana Magovčević-Liebisch, Vigil Neuroscience CEO

FDA lifts par­tial clin­i­cal hold on Vig­il Neu­ro­science's TREM2 an­ti­body, re­mov­ing dos­ing cap

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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