Cell/Gene Tx channel feed

Amy DuRoss, Vineti CEO (Vineti)

Ex­clu­sive: An in­dus­try con­sor­tium wants to stan­dard­ize cell track­ing for re­gen­er­a­tive meds. Could that avert a dis­as­ter?

In the fast-moving world of regenerative medicine, the open secret among industry experts is that manufacturing is exceedingly difficult and will take a Herculean effort to scale. Left behind in that shuffle have been new advances in how drugmakers actually keep track of where a patients’ cells are going — and that could leave the field ripe for a disaster.

As part of a broad consortium of biopharma and tech companies working to drive new standards for advanced therapies, San Francisco’s Vineti plans to create what it calls a “modern patient ID” that can track human cells from apheresis centers through a complicated manufacturing supply chain and back into those patients.

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Step Pharma CEO Andrew Parker

Step Phar­ma clos­es $41M round to take en­zyme tar­get­ing lead pro­gram in­to ear­ly-stage tri­al for T cell ma­lig­nan­cies

The search for better T and B cell therapies have absorbed billions in R&D funds from some of the biggest drugmakers in the world. A French biotech thinks it could have a hyper-focused approach to T and B cell malignancies, and it’s taking home a decent-sized check to race to the clinic.

Step Pharma has bagged $41.5 million in funding to take its lead CTPS1 inhibitor into clinical trials for T cell malignancies by the first quarter of 2022, the French biotech said Wednesday.

In­no­force gets $96M to build up Chi­na cam­pus, in­clud­ing a new Ther­mo Fish­er-part­nered mon­o­clon­al an­ti­body man­u­fac­tur­ing fa­cil­i­ty

Several months after completing what it calls a “Pre-A” financing round, Chinese contract manufacturer Innoforce Pharmaceuticals is back at the venture well, drawing $96 million to build up its Hangzhou campus. This time, it’s bring a big-name partner along.

The money will go straight to construction, Innoforce said, with blueprints for a new monoclonal antibody manufacturing facility through a partnership with Thermo Fisher Scientific.

Xy­lo­Cor finds new back­ing to push re­gen­er­a­tive heart dis­ease gene ther­a­py deep­er in­to the clin­ic

XyloCor Therapeutics is one step closer to accomplishing the tough task of delivering gene therapies straight into the heart for coronary artery disease.

Three years after its first fundraise, XyloCor has raised an additional $22.6 million in a Series A extension, with the goal of expanding its lead program into further trials. Called XC001, the program is currently being studied in patients who have refractory angina, and XyloCor wants to study its use as a potential adjunctive for those undergoing bypass surgery.

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CAR-M era be­gins as Caris­ma dos­es first pa­tient

Last month, doctors at the Abramson Cancer Center in Philadelphia inserted a needle into a patient with an untreatable tumor and began drawing blood into a machine.

The machine filtered out everything but a specific set of immune cells that were then packaged, put on a plane and shipped, still warm, to a facility in Sunnyvale, CA. Over 24 days, technicians expanded the cells, armed them with a new kind of receptor and sent them back, now cryogenically frozen, on a plane to Philadelphia to be infused back into the patient.

An­oth­er blue­bird ex­ec jumps ship as ques­tions swirl over gene ther­a­pies and im­mi­nent split

On the same day that their former COO found a new job steering the ship at a biotech focused on engineering B cells, bluebird bio said goodbye to another C-suite executive.

David Davidson, bluebird’s longtime chief medical officer, is jumping ship as questions continue to swirl about the safety of their gene therapy programs, the company revealed in an SEC filing Tuesday. Davidson will leave effective April 16, and will work as a consultant for six months as bluebird transitions to a new CMO. He’ll also receive 12 months of severance starting at the end of April.

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Fresh out of the blue­bird bio on­col­o­gy split, Joanne Smith-Far­rell flies the coop to Be Bio and its B cell plat­form

Stung by setbacks and a stagnant pipeline, bluebird bio, the once-darling of gene therapy biotech, made the tough call to sever its rare disease and oncology units earlier this year. One of the business leads on bluebird’s oncology side has now popped up at a newly launched B cell player — and she’s taking her former employer’s “pioneering” example to heart.

Joanne Smith-Farrell, formerly bluebird’s chief operating officer and head of the oncology business unit, is now CEO at Be Biopharma, an early-stage biotech targeting engineered B cells for therapeutic use. Smith-Farrell is joined on the team by CSO Rick Morgan, another bluebird vet who was most recently senior VP of immunogenetics at Editas.

Aravind Asokan and Sapan Shah (StrideBio)

Take­da, Sarep­ta dou­ble down on a biotech part­ner's plat­form to en­gi­neer bet­ter AAV cap­sids by map­ping out their struc­ture

When Aravind Asokan and Mavis Agbandje-McKenna first pulled together their respective research at the University of North Carolina-Chapel Hill and the University of Florida on engineering adeno-associated virus vectors and started StrideBio in 2015, the FDA was still two years away from an eventual groundbreaking first approval for an AAV gene therapy.

But even then — and moreso since — preclinical and clinical studies had exposed the glaring room for improvement in the way genes are delivered.

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CEO Sonny Hsiao (Acepodia)

Tai­wan's Ace­po­dia clos­es Se­ries B with the promise of con­ju­gat­ing an­ti­bod­ies and off-the-shelf NK cells

What if researchers could take the antibody-drug conjugate model and apply it to off-the-shelf NK cell therapy? For the Taiwan-based biotech Acepodia, that’s the $47 million question.

Acepodia closed its Series B round Tuesday morning, hoping to build upon CEO Sonny Hsiao’s work from when he was a biologist at UC Berkeley. The biotech plans to use the funds to advance its lead program through the end of a Phase I trial and launch a Phase II study, as well as to get two other candidates into the clinic.

Ilan Ganot, Solid Bio CEO (Solid Bio)

UP­DAT­ED: Sol­id Bio’s long, trou­bled slog through the clin­ic yields 'en­cour­ag­ing' ear­ly da­ta and no new safe­ty con­cerns. It's not win­ning over in­vestors yet

Those who have been following the gene therapy space would be familiar with Solid Bio’s IGNITE-DMD trial. Beset with safety issues, the Phase I/II study was held down by clinical holds for the better part of the last three years, while the Duchenne-focused biotech watched bigger rivals dash by toward pivotal work.

There was just one thing that we’ve never seen: data from the study on how patients are doing.

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