Cell/Gene Tx channel feed

Mikael Dolsten, Pfizer CSO

FDA re­leas­es sec­ond Pfiz­er gene ther­a­py from clin­i­cal hold, as he­mo­phil­ia pro­gram gets OK to restart

The FDA has released a clinical hold on Pfizer and Sangamo’s hemophilia A gene therapy after ordering the companies to stop the trial last year due to concerns about blood clots.

Just a week after Pfizer announced the FDA had unleashed its gene therapy for Duchenne muscular dystrophy from a clinical hold — which was paused as one patient died and others experienced severe side effects  — another one of its gene therapy programs has been given the green light to resume, Pfizer announced during its Q1 earnings call yesterday.

Still-in-stealth biotech part­ners with Sloan Ket­ter­ing sci­en­tists to make per­son­al­ized can­cer ther­a­py more ‘pub­lic’

One of the obstacles of personalized medicine is that it is, well, personalized. With cancer therapies especially, personalized treatments take money, time, resources and access — all of which are finite.

But some scientists instead want to take a more “public” approach to what has traditionally been a therapy tailored to individual tumors. The vast majority of neoantigens — mutated protein bits exclusively made by cancers — are “private,” meaning that they are unique to a single tumor and person. So while their specificity makes them a good target, private neoantigens may be too specific.

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FDA puts a hold on Ver­tex’s at­tempt­ed di­a­betes cure

Vertex thinks it has enough data to progress its experimental stem cell therapy for type 1 diabetes. The FDA has other thoughts.

The large biotech announced Monday that the agency put a hold on the clinical trial for the program, which grabbed “cure” headlines last year after Vertex released promising data from the first patient dosed in the study. The experimental treatment is designed to replace the insulin-producing cells T1D patients lose with lab-grown insulin-producing cells.

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Tom Anderson, SwanBio Therapeutics CEO

Scoop: Af­ter fail­ing to se­cure more funds, a pri­vate gene ther­a­py de­vel­op­er lays off staff

Last Friday, SwanBio Therapeutics laid off about one-quarter of its 60-person workforce, a person with knowledge of the matter told Endpoints News.

The gene therapy biotech was unable to secure an additional round of financing in the first quarter after investors backed out of the round, according to the source, who spoke on condition of anonymity. It’s been 24 months since the Philadelphia-area biotech disclosed it had expanded its Series A to bring total financing to $77 million.

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Pfizer CEO Albert Bourla (AP Images)

UP­DAT­ED: Pfiz­er can restart PhI­II tri­al of Duchenne gene ther­a­py, now on track for late-2023 FDA ap­proval re­quest 

Pfizer’s path at getting a gene therapy to market for patients with Duchenne muscular dystrophy just got a little clearer after a hazy 2021 that saw a clinical hold, an amended late-stage trial and a patient death in an early-stage study.

US regulators will allow the Big Pharma to resume its Phase III study dubbed CIFFREO, Pfizer said Thursday morning, noting it has amended the protocol to assess patients in the hospital for seven days post-administration. The clinical hold lift follows reviews of data and protocol amendments, Pfizer added.

Jean-Jacques Bienaimé, BioMarin CEO (BioMarin via YouTube)

Bio­Marin plots come­back year, with plans to turn around prof­its and head back to the FDA with re­ject­ed gene ther­a­py

Could this be a comeback year for BioMarin? CEO Jean-Jacques Bienaimé certainly hopes so, as he plans to finally turn the corner on profitability and send the company’s hemophilia gene therapy back to the FDA.

But there are still a few things analysts want to know. Like, what’s going on with the clinical hold on its phenylketonuria gene therapy? And when will the company seek full approval for Voxzogo, its costly achondroplasia drug that got the FDA’s accelerated approval treatment late last year?

Sio Gene Therapies founder Vivek Ramaswamy (JPM20)

Ax­o­vant-turned-Sio ditch­es two re­main­ing pro­grams, lays off most em­ploy­ees and will seek a buy­er

A year and a half ago, Sio Gene Therapies attempted to steer into a new history, with then-CEO Pavan Cheruvu telling Endpoints News it was “not a vant any longer” after breaking away from the Axovant name, of the Roivant descent.

The company might be getting a rebrand again soon. That is: Sio is looking for a new owner, or another company to merge with as the biotech is shuttering work on its two remaining programs, for GM1 and GM2 gangliosidosis, or Tay-Sachs and Sandhoff disease. SVB Securities will help the company find a new path forward.

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Dave Greenwald (Deerfield)

Deer­field-backed biotech emerges from stealth with gene ther­a­py de­liv­ery plat­forms from Har­vard and MIT

As a PhD student at Tufts, Dave Greenwald studied the use of adeno-associated virus (AAV) vectors to deliver gene therapies to people with retinitis pigmentosa, a group of rare genetic eye diseases that causes the retina to break down over time.

While he later left lab life, he now sits at the helm of Apertura Gene Therapy, a biotech that hopes to customize AAV vectors that are used widely in delivering most gene therapies. Apertura announced its launch from stealth with $67 million in Series A financing from Deerfield, where Greenwald is also a vice president of business development.

Paul Hastings, Nkarta CEO

Nkar­ta re­veals its first off-the-shelf NK cell da­ta, show­ing mul­ti­ple com­plete re­spons­es — shares soar

Nearly two years after pricing its IPO, Nkarta is finally reporting its first clinical data Monday.

The South San Francisco biotech revealed the first human results from its two lead off-the-shelf NK cell therapies, with the high-dose regimens registering multiple complete responses in small groups of late-line blood cancer patients. Nkarta also touted a relative lack of side effects compared to CAR-T treatments, as well as no dose-limiting toxicities.

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Oz Azam (L) and Carl June

New pa­per sheds fresh light on Tmu­ni­ty's high-pro­file CAR-T deaths

The industry-wide effort to push CAR-T therapies — wildly effective in several blood cancers — into solid tumors took a hit last year when Tmunity, a biotech founded by CAR-T pioneer Carl June and backed by several blue-chip VCs, announced it shut down its lead program for prostate cancer after two patients died.

Since then, Tmunity has gone virtually dark, losing or letting go all but one member of its 6-person C-suite, including CEO Oz Azam, and not releasing a single press release. The company has also yet to publish a detailed analysis of the prostate cancer trial.

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