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Bristol Myers Squibb's facility in Devens, MA

Bris­tol My­ers scales up cell ther­a­py pro­duc­tion with liso-cel nod in hand and an­oth­er ap­proval maybe on the way

After a long-awaited approval for its CD19 targeting CAR-T liso-cel, Bristol Myers Squibb is gearing up for the looming review of another cell therapy in its pipeline, ide-cel. Taking not one but two CAR-Ts to market — and potentially more — means Bristol will have to step on the gas to get its manufacturing in order.

Looking to address that potential roadblock, Bristol will add a 244,000 square-foot cell therapy manufacturing facility at its Devens, MA site as it looks to grow its production capacity around liso-cel and, potentially, ide-cel in the coming weeks, the drugmaker said Tuesday.

Derek Jantz, Precision Biosciences

Jim Wilson's team hands Pre­ci­sion Bio a big proxy win for its PC­SK9 gene edit­ing tech with 3-year mon­key da­ta

James Wilson and his gene editing team at UPenn have published a new paper into a one-time PCSK9 therapy, one that Precision Biosciences hopes can turn up the heat on its competitors at Verve.

Using Precision’s proprietary ARCUS gene editing platform, Wilson was able to demonstrate that PCSK9 protein and LDL cholesterol reductions could be sustained in monkeys for at least three years after treatment, Precision announced Friday. Researchers administered the therapy to 10 monkeys back in 2017 and reported reductions of up to 85% in PCSK9 protein levels and a 56% reduction of LDL cholesterol levels.

Cen­tu­ry Ther­a­peu­tics boosts iP­SC man­u­fac­tur­ing space in New Jer­sey as part of com­pa­ny wide ex­pan­sion

It’s been a busy few years for Pennsylvania-based Century Therapeutics since it burst onto the scene with a $250M funding haul in summer 2019. Now, the biotech is scaling up its manufacturing capacity as part of a companywide footprint buildout.

Century has leased 53,000 square feet of space in Branchburg, New Jersey, for a new manufacturing facility for its induced pluripotent stem cell therapies. That buildout is part of a larger expansion that will include expanded lab and corporate space in Philadelphia.

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Nick Leschly (Credit: Jeff Rumans at Endpoints News JPM20)

UP­DAT­ED: Blue­bird hits the brakes on Lenti­Glo­bin stud­ies, Zyn­te­glo mar­ket­ing af­ter AML, myelodys­plas­tic syn­drome cas­es spur fresh safe­ty con­cerns

Bluebird bio $BLUE has run into a fresh set of troubles for its gene therapy LentiGlobin.

The biotech, which has seen its gene therapy work mired at the FDA due to CMC issues, revealed Tuesday morning it had suspended work on its early-stage study as well as the Phase III sickle cell trial for LentiGlobin in order to investigate two more serious side effects among patients in the program.

In addition, bluebird CEO Nick Leschly hit the brakes on marketing Zynteglo (betibeglogene autotemcel; beti-cel) in Europe, where it’s approved for beta thalassemia, because it uses the same BB305 lentiviral vector used in the gene therapy for sickle cell disease.

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George Church (Mary Altaffer/AP Images)

George Church and an en­tre­pre­neur­ial post­doc join the hunt for AAV 2.0 with a new vec­tor-cloak­ing tech­nol­o­gy

About five years ago, George Church and his new postdoc, Ying Kai Chan, sat hunched over a laptop in the genetics pioneer’s Harvard office and stared in bewilderment at an old paper.

The paper documented early trials for Glybera, the first and at the time only gene therapy approved anywhere on the planet. Less than three dozen patients ever received it, but in the years before Luxturna and Zolgensma, it gave researchers an example they could point to for gene therapy actually working.

Dyno sketch­es tens of thou­sands of vi­able next-gen AAV cap­sids with the help of ma­chine learn­ing — pa­per

Since its launch in May 2020, Dyno Therapeutics has touted its platform’s potential to discover viable variations of current-gen AAVs with noticeable limitations in terms of patient safety and efficacy. Now, according to a new study, Dyno has used its neural network to outline tens of thousands of variants that could add weight to its mission to build a better capsid.

In a study meant to determine how many viable variants of the AAV2 capsid it could design with the aid of machine learning, Dyno sketched out more than 100,000 viruses that could be used to carry gene therapies, according to a new paper published in Nature Biotechnology.

Rachel Haurwitz (Caribou)

Ab­b­Vie taps Jen­nifer Doud­na start­up for li­cens­ing deal worth up to $300M for 2 'off-the-shelf' CRISPR-edit­ed CAR-Ts

“Off-the-shelf” CAR-Ts have emerged as a hot-ticket target in next-gen oncology R&D, with Big Pharmas placing big down payments on novel tech. Now, a California biotech player founded by one of the CRISPR movement’s most prominent mouthpieces has added major drugmaker AbbVie as a teammate.

AbbVie will pay Caribou Biosciences $40 million in upfront cash and $300 million in biobucks to develop two of the biotech’s allogeneic CAR-T therapies, the partners said Wednesday. The biotech will also be due royalty payments for any future commercial assets.

Marco Boorsma, Forbion

With a ros­ter of gene ther­a­py vets, For­bion makes a bet on 'rel­a­tive­ly emp­ty space' of vec­tor­ized an­ti­bod­ies

The future waves of gene therapy could look quite different from the gene replacement offerings currently on the market, Forbion reckons.

It’s the key idea behind VectorY, a startup seeded by the European VC firm that’s out to develop vectorized antibodies for muscular and neurodegenerative disorders.

Voyager Therapeutics, which briefly pushed the technology to the limelight with its short-lived AbbVie collaboration, is among the “several other companies working on it too, but definitely it’s a pretty relatively empty space,” said Marco Boorsma — a general partner at Forbion who’s stepping in as interim CEO.

CEO David Main (Notch)

'In­dus­tri­al­iz­ing the pro­duc­tion of cell­s': An­oth­er iP­SC play­er joins the quest for off-the-shelf cell ther­a­pies

Scientists have been after the “Holy Grail” of cell therapy — an off-the-shelf product — for years. Notch Therapeutics is now joining the quest with a tech platform it says can industrialize the production of cells. And on Wednesday, it unveiled a $85 million Series A to get going.

Notch was founded in 2018 by Juan Carlos Zúñiga-Pflücker and Peter Zandstra, but “really kicked off” in late 2019 when it signed a deal with Allogene to research allogeneic cell therapy candidates, CEO David Main said. The nascent biotech got $10 million upfront, and stands to earn up to another $294.2 million in milestones.

FDA lifts clin­i­cal hold on Mus­tang Bio's lentivi­ral gene ther­a­py, paving the way for quick jump in­to piv­otal tri­al

Things were looking good for Mustang Bio’s MB-107 gene therapy program. By early September, it had nabbed both rare pediatric disease and orphan drug designations, adding to the regenerative medicine advanced therapy designation it already scored at the FDA. It all boded well for the pivotal trial they were looking to start in newly diagnosed infants with X-linked severe combined immunodeficiency.