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Steven Kelly, Carisma CEO and president

Caris­ma's CAR-M steals spot­light among 42 bid­ders to re­verse merge with Sesen Bio

Sesen Bio’s decade-plus journey is over after failing to secure a bladder cancer drug approval and carve its own path for IL-6 antibodies. The Cambridge, MA company’s spot on Nasdaq will serve as the new home for Carisma Therapeutics, which is attempting to usher in a new CAR cell therapy in the world of macrophages and monocytes.

The two are executing an all-stock reverse merger, with the future resting solely on Carisma’s lineup of therapies, including a Phase I asset that had some limited data presented at ASCO in June. The Philadelphia startup will begin trading under the aptly symboled ticker $CARM in three to four months, the companies said Wednesday morning.

David Liu (Broad Institute)

New patent on the prime edit­ing of DNA award­ed to Broad In­sti­tute's David Liu

The genome editing revolution will be patented. Or at least that’s the case for this latest US patent, awarded Tuesday to the Broad Institute’s David Liu and team for the compositions and methods behind the prime editing of a target DNA molecule (e.g., a genome) that enables a nucleotide change and/or targeted gene mutation, according to the abstract.

Developed in Liu’s lab at the Broad Institute, which famously defeated Jennifer Doudna and UC Berkeley in court over CRISPR-related patents, the idea behind prime editing — dubbed CRISPR 3.0 — was published in Nature in December 2019, and that spawned a startup known as Prime Medicine.

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Choon-Peng Ng, ImmunoScape CEO

TCR up­start gets fresh fund­ing as it looks to charge to­ward the clin­ic

While several companies have been finding success in the TCR-T cell therapy space — with Intellia having its treatment accepted by the FDA last year — another company is using machine learning to get in on the action and find a candidate to bring toward the clinic.

ImmunoScape, a biotech based in Singapore and California, has raised $14 million in a fresh round of financing. The company itself was founded in 2016 as a spinout from the Singapore-based Agency for Science, Technology and Research, setting up its US operations in San Diego in 2020.

Gaurav Shah, Rocket Pharmaceuticals CEO

Ren­o­va­cor ships its car­diac gene ther­a­py to Rock­et in $53M all-stock deal

Renovacor wanted to be in the clinic already. The biotech expected an $11 million Series A in summer 2019 would bankroll an IND filing for its cardiac gene therapy, but as it goes with drug R&D, the path took some twists.

Fifty-four weeks after shipping up to New York City for a blank check merger, Renovacor is turning off the lights on $RCOR and going the way of $RCKT, or Rocket Pharmaceuticals, in an all-stock deal that adds to the latter’s gene therapy ambitions, which includes two likely BLA filings next year.

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Andrew Obenshain, bluebird bio CEO

Blue­bird lands an­oth­er rare dis­ease gene ther­a­py OK — but there are a few caveats to note

Relative to the protracted journey of developing a gene therapy delivered by a lentiviral vector, the speed at which bluebird bio moved from having one approved product to two was extremely fast.

Exactly a month after the FDA gave its green light to bluebird’s Zynteglo — to treat beta thalassemia — the agency came through with an approval for a second gene therapy for early, active cerebral adrenoleukodystrophy (CALD). The drug, previously known as eli-cel and now branded Skysona, will have a wholesale acquisition price of $3 million and should become available by the end of this year.

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Fu­ji­film Diosynth breaks ground on a $1.6B ex­pan­sion project in Den­mark

As Fujifilm has been investing heavily in its CDMO arm for over a year, one of its major projects is now getting off the ground.

The Japanese manufacturer announced that it has broken ground on an expansion project at its site in the city of Hillerød, Denmark. The expansion, which was first announced in June, is part of a $1.6 billion project to expand the cell culture production capabilities in Denmark and at another facility in Texas.

Laura Shawver, Capstan Therapeutics CEO

Backed by five phar­ma gi­ants and a crew of Penn su­per­stars, a start­up us­es mR­NA to cre­ate in vi­vo CAR-T — with a pro at the helm

Earlier this year, Jonathan Epstein and other scientists at Penn published a paper in Science that spelled out the intriguing results of a mouse study. Using CD-5 targeted lipid nanoparticles, the tiny vehicles used to carry messenger RNA, they were able to reprogram T lymphocytes into CAR-Ts in vivo, in the body. Trained to recognize fibrotic cells specifically in the heart, the scientists involved were able to restore cardiac function in mice.

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Stéphane Boissel, SparingVision CEO

‘Dif­fi­cult to go pub­lic with’: Spar­ingVi­sion turns to ex­ist­ing back­ers to fund its first two oc­u­lar gene ther­a­py tri­als

On the hunt to improve upon the foundations of Luxturna by creating a gene therapy that it hopes can be given to a broader swath of patients with a rare retinal disease than its Big Pharma rival, SparingVision has secured another round of financing. But it didn’t come easy.

The Paris and Philadelphia biotech’s leadership began the Series B process in January, and at that time, they agreed that if by the end of spring it proved to be “too challenging, impossible or that the value that we were looking for was just impossible to obtain, we were going to focus on an insider-only round,” CEO and president Stéphane Boissel told Endpoints News.

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Pablo Cagnoni, Rubius Therapeutics CEO

Ru­bius re­sorts to lay­offs and aban­dons its lead pro­grams as its sur­vival strat­e­gy

Four years ago, Cambridge-based Rubius Therapeutics was having the time of its life. The company, which makes engineered red cells, was raking in ample cash and had enjoyed a $1.8 billion market valuation registered at the IPO.

Today it is a whole different story.

Rubius has decided to scrap two of its leading ongoing trials, let go of 75% of its workforce and explore the sale of its manufacturing facility in Rhode Island, the biotech announced Tuesday.

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Bio­Marin says PhI­II gene ther­a­py study par­tic­i­pan­t's can­cer is un­re­lat­ed to ther­a­py

As the FDA has delayed any hope of BioMarin launching its hemophilia A gene therapy until next year at the earliest, the company said in an SEC filing Monday that a participant in one of its Phase III studies for the therapy was diagnosed with a type of cancer known as B-cell acute lymphoblastic leukemia.

After submitting the safety report to FDA, BioMarin said it “believes at this time that this cancer is unrelated” to the treatment, known as valoctocogene roxaparvovec. None of the gene therapy’s ongoing trials have been halted as a result, the company said. On the status of its investigation into this case of B-ALL, BioMarin told investors: