Cell/Gene Tx channel feed

Siddhartha Mukherjee, AP Images

Sid Mukher­jee's Vor taps a CAR-T de­vel­op­ment part­ner to strap to its at­tempt at bet­ter stem cell trans­plants

In the buzzy world of oncology, few names shine brighter than Columbia oncologist and bestselling author Siddhartha Mukherjee, whose Vor Biopharma is looking to rewrite the rules of stem cell transplants. If his team’s tech works as planned, it would also rewrite the rules on how CAR-Ts can be deployed in blood cancer — and now it’s partnering up with a little-known biotech to test that hypothesis.

Michael Yang, ViaCyte CEO

Vi­a­Cyte adds to the war chest in what looks like an IPO-bound ef­fort to bring re­gen­er­a­tive med­i­cine to di­a­betes

Stem cell therapies have long held promise if providing curative answers to chronic disease, but the field has taken decades to bear fruit despite some big hopes. A long gestating biotech out of San Diego thinks it has the secret sauce to cure type 1 diabetes, and now it’s raking in piles of cash with the potential for a major IPO in the offing.

ViaCyte has closed an additional $45 million tranche for its Series D, bringing its total fundraise for the round to $115 million with the backing of a slate of crossover investors that appear to spell a looming IPO for the long-lived regenerative medicine player, the company said Wednesday.

Trail­ing the Big Phar­ma play­ers, a less­er known BC­MA CAR-T can­di­date comes in­to the spot­light

Bristol Myers Squibb and bluebird cleared the first BCMA CAR-T therapy for multiple myeloma patients back in March, and J&J and their partners at Legend Biotech aren’t far behind. Now, a lesser known candidate developed in China is coming into the spotlight at EHA.

IBI326, a BCMA CAR-T therapy developed by Innovent Biologics and IASO Biotherapeutics, had a 97.1% overall response rate in 35 patients with relapsed or refractory multiple myeloma, according to updated Phase I data the companies are taking to EHA. IASO CEO Maxwell Wang says it’s the only oral presentation on a Chinese BCMA CAR-T at this year’s conference.

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Armon Sharei, SQZ

#AS­CO21: SQZ of­fers first clin­i­cal glimpse of un­ortho­dox cell ther­a­py man­u­fac­tur­ing tech — but check­point com­bo is still the way to go

More than seven years after Armon Sharei, then a PhD student at Bob Langer’s lab, first published a way to reengineer cells without vectors, the biotech they founded together has the first human data in its hands.

SQZ Biotech — which gets its name from the cell-squeezing device they created to open up a temporary window for delivery of cargoes into cells — has two take-home messages for the virtual ASCO crowd: Its HPV-targeted cell therapy looks safe among 12 patients with HPV-positive solid tumors, and could be manufactured reliably within 24 hours.

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Or­chard dis­creet­ly ax­es li­cense for 'bub­ble boy' gene ther­a­py, de­spite re­cent pos­i­tive long-term da­ta up­date

Less than a month after publishing positive long-term data for an experimental gene therapy, Orchard Therapeutics has quietly terminated its licensing agreement over the program.

In an 8-K filing to the SEC last Friday, Orchard reported that it is ending the license related to its OTL-101 candidate, in development to treat adenosine deaminase severe combined immunodeficiency or ADA-SCID, known colloquially as “bubble boy syndrome.” It’s a rare genetic disease where patients have almost no immune system whatsoever.

Ying Huang, Legend Biotech CEO

FDA steers J&J/Leg­end CAR-T to a quick de­ci­sion as part­ners prep a loom­ing show­down with Bris­tol My­ers, blue­bird

Just days after investigators posted a jaw-dropping set of positive updated data for the BCMA CAR-T cilta-cel — or JNJ-4528 — J&J’s partners at Legend are back with the news that the FDA is putting their application for the multiple myeloma treatment in the fast lane and speeding it to a fall regulatory decision deadline. And that would put them right behind the newly launched BCMA drug Abecma from Bristol Myers Squibb and bluebird, widely considered now as a runner-up on efficacy.

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Emile Nuwaysir, BlueRock CEO

Look­ing to build new smart cells, Blue­Rock teams with Tim Lu's Sen­ti Bio in 'sec­ond-or­der' en­gi­neer­ing al­liance

BlueRock, the Versant-backed and Bayer-owned biotech best known for its Parkinson’s cell therapy, has been quietly laying out much grander plans for their technology.

The company, CEO Emile Nuwaysir explains, doesn’t just want to recreate the healthy human cell — as they’ve done in Parkinson’s — but also to manipulate it or add whole new features: a so-called kill-switch that allows doctors to control cells after they’ve been implanted, for example, or molecular “sensors” that prevent the cell from functioning anywhere but in the precise tissue it’s needed.

Image credit: Shutterstock

Re­searchers in biotech, acad­e­mia and Big Phar­ma are of­fer­ing puz­zle pieces to crack CAR-T for sol­id tu­mors. Will they ever snap to­geth­er?

The idea seemed promising enough.

By the time a group of scientists reported an attempt to treat colon cancer with engineered T cells drawn from patients, expanded and armed with artificial surface proteins in 2010, the experimental therapy — known as CAR-T, short for chimeric antigen receptor transduced T cells — had turned from an eccentric therapeutic proposal to a somewhat viable approach being tested in the clinic. The target, HER2, was one of the most extensively studied targets in cancer, implicated in cancers of breast, bladder, ovary, pancreas, and more. A “next-generation” construct containing a fragment of a well-known antibody, deployed alongside additional tried and true tweaks, looked like a promising next step.

That’s not to mention the study was being conducted by Steve Rosenberg’s lab — the famed National Cancer Institute group that had produced some of the earliest data that convinced scientists of T cells’ ability to attack solid tumors.

Yet the trial took a shocking and tragic turn. Within 15 minutes of receiving the infusion, the first patient had serious trouble breathing. She was sent to the hospital, intubated, and died five days later.

“I think it shut every CAR-T cell therapy study down which targeted HER2 at the time,” recalled Stephen Gottschalk, who was working on a different HER2 program himself at Baylor College of Medicine. “We spent quite a lot of time with the FDA coming up with an approach that would allow us to do it safely.”

While CAR-Ts for hematological malignancies have largely sailed through those early safety scares — a group at Memorial Sloan Kettering had disclosed another death in a hematology trial that same year — and impressed the biopharma world with astonishing response rates, the same can’t be said about solid tumor efforts. Early clinical trials that didn’t turn up serious side effects also failed to demonstrate nearly the same level of efficacy seen in acute lymphoblastic leukemia and non-Hodgkin lymphoma.

Rather than giving up, both academic and industry researchers are doubling down, thanks in part to the big success in the hematologic space and the large market that awaits anyone who can finally unlock any kind of solid tumors with CAR-T.

The few skeptics haven’t stopped a torrent of venture dollars from propelling these efforts, most still preclinical, that promise to tear down the well-documented roadblocks now shielding many cancers from these potent killing cells. The question boils down to how you choose among them — and who will be the last ones standing.

Defining the challenge

Martin Pule remembers the early days when everything was harder in CAR-T.

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Blue­bird’s new gene ther­a­py among three new EMA OKs ahead of FDA

Ahead of a usually speedy FDA, the European Medicines Agency on Friday recommended marketing authorizations for bluebird bio’s new gene therapy for children with a rare, inherited neurological disease, GlaxoSmithKline and Vir’s monoclonal antibody treatment for Covid-19, and a new treatment for a rare liver disease.

In the case of bluebird’s Skysona (elivaldogene autotemcel), the gene therapy is for those younger than 18 who have what’s known as early Cerebral Adrenoleukodystrophy (CALD). If untreated, nearly half of those with CALD die within 5 years of symptom onset, EMA said.

#AS­CO21: Watch out Bris­tol My­ers, J&J is back at AS­CO with some com­pet­i­tive BC­MA CAR-T da­ta

Bristol Myers Squibb and bluebird beat Johnson & Johnson to market back in March with their BCMA CAR-T drug, Abecma, for heavily pre-treated multiple myeloma patients. But watch out: J&J and their partners at Legend Biotech are now heading to ASCO with updated data that could give Abecma a run for its money.

A longer-term follow-up from the Phase Ib portion of a Phase Ib/II trial shows that all patients responded to J&J’s JNJ-4528, 86% of whom achieved a stringent complete response at a median of 11.5 months. In Phase II, there was a 95% overall response rate as of the February data cutoff, with a 75% stringent complete response, the company said Friday.  The data are part of the package J&J is submitting in rolling applications for approvals in Europe and the US.