Cell/Gene Tx channel feed

All of the news, delivered with full-text to your inbox. For professionals discovering, developing, and marketing biopharmaceutical drugs.

SIGN UP NOW
Subscribe to Endpoints News

Biotech pain con­tin­ues as Ver­sant, MPM gene ther­a­py start­up calls it quits

A gene therapy startup backed by two key biopharmas and two premier biotech VC firms has ceased operations.

California biotech Coda Biotherapeutics shut down recently, investors Versant Ventures and MPM Capital confirmed to Endpoints News Tuesday night, declining to provide further details. The upstart had presented preclinical data on its program for focal epilepsy last summer but had not provided an update since then. MPM removed Coda from its portfolio page in recent weeks, per internet archives, and the biotech’s LinkedIn page has been wiped.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FDA de­lays re­view of Bio­Mar­in's he­mo­phil­ia gene ther­a­py af­ter da­ta up­date

The FDA has extended its review of BioMarin’s gene therapy for hemophilia A by three months, formalizing an expected delay that will now push out a decision to the end of June.

BioMarin told investors in November, after submitting three-year data analysis from a Phase III trial, that the FDA said it may constitute a major amendment that warrants extra time for review.

And that’s exactly what happened, with the agency citing “substantial amount of additional data,”

French CAR-T biotech Mnemo Ther­a­peu­tics shut­ters US op­er­a­tions

A Sofinnova-backed CAR-T maker has shut down its New York and Princeton, NJ sites and will continue operating out of its Paris hub.

Mnemo Therapeutics turned the lights off at its US operations effective immediately on Feb. 23, according to employees’ posts on LinkedIn and confirmation from co-founder and operating chief Alain Maiore.

“When Sebastian [Amigorena] and I started Mnemo Tx, we were looking to build a company where the human value of ‘standing shoulder to shoulder’ in difficult time would typify the DNA of our teams across the ocean,” Maiore wrote on LinkedIn in recent days. “The decision we had to make about shutting down our US operations, to move back our CT effort to Europe, next to our target discovery platform was a very difficult one.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Adaptimmune CEO Adrian Rawcliffe at EUBIO22 (Rachel Kiki for Endpoints News)

Months af­ter re­org, Adap­ti­m­mune merges with fel­low cell ther­a­py de­vel­op­er

Adaptimmune is merging with TCR² Therapeutics in a bid to create a T cell therapy developer aimed at solid tumors.

The all-stock deal brings together both companies’ lead candidates — Adaptimmune’s afami-cel, which targets MAGE-A4, and TCR²’s mesothelin-targeting gavo-cel — as well as a pipeline of candidates engineered with T cell receptors, or TCRs. Unlike CAR-T therapies, TCR-T is not limited to surface antigens but can target antigens inside cancer cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Al Sandrock, Voyager CEO

Voy­ager nabs $25M cash as No­var­tis runs with two AAV cap­sids for neu­ro gene ther­a­py

After spending a year looking at Voyager’s next-gen AAV capsids, Novartis has picked two of them to be the delivery vector for its gene therapy programs targeting two neurologic diseases.

The license triggers a $25 million payment — $12.5 million for each target — and opens up a road to $600 million in total milestones. But it also leaves some money off the table as Novartis, which had the option to license capsids against a total of three targets, decided not to act on the third.

Ex-AveX­is crew trims staff at Chica­go-area Jaguar Gene Ther­a­py

After Novartis acquired AveXis, executives from the leading gene therapy biotech shuffled off to form new startups looking to repeat their Zolgensma story for other diseases. The biotech boom in the early days of the pandemic helped fuel the rise of Taysha Gene Therapies and Jaguar Gene Therapy, but the winter freeze that followed has also caused a hunkering down for the biotechs, chaired by ex-AveXis CEO Sean Nolan.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Gina Chapman, CARGO Therapeutics CEO

Cell ther­a­py biotech from Stan­ford CAR-T leader Crys­tal Mack­all nabs $200M for PhII 

A biotech born out of the work of Stanford CAR-T leader Crystal Mackall is publicly unveiling its $200 million Series A Wednesday, after already going through a Phase I clinical trial, rebranding and setting up plans for a potential registrational study.

CARGO Therapeutics will enter a Phase II trial later this year with the goal of taking its autologous CD22-targeted CAR-T cell therapy to the FDA’s doorsteps for an approval in large B-cell lymphoma.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Doug Ingram, Sarepta CEO

Sarep­ta won't need FDA ad­comm, boost­ing Duchenne gene ther­a­py's odds

On its quest to get the first gene therapy for Duchenne muscular dystrophy approved by the FDA, Sarepta won’t have to face an FDA advisory committee after all.

The biotech disclosed in its quarterly update Tuesday afternoon that after a mid-cycle review of SRP-9001 with the FDA, in which it answered questions about chemistry, manufacturing and controls, the agency said it does not plan to hold an adcomm. A decision on the therapy is due by May 29.

Catherine Stehman-Breen, Chroma Medicine CEO

A gene edit­ing 3.0 play­er goes deep on cash, fron­tier sci­ence to put pre­ci­sion epi­ge­net­ics to the big test

Issi Rozen is as deeply embedded into the gene editing field as anyone in the buzzy Cambridge, MA biotech hub. As chief business officer and strategy chief for the Broad over a decade-long stretch, he’d worked with all the big names in a scientific discipline that promises to upend the therapeutics field — eventually. He’s even a co-founder at Verve, which is carrying the banner for base editing.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Stan Wang, Thymmune CEO

Re­gen­er­a­tive cell biotech Thym­mune launch­es with back­ing from George Church, John Maraganore

Thymmune Therapeutics is going after “the most important organ you’ve never heard of,” according to Pillar VC’s Thomas de Vlaam.

The Cambridge, MA-based biotech has raised $7 million in seed funding to push its thymic cell therapy through preclinical studies, it announced Wednesday. Thymmune was founded in 2019 by famed Harvard geneticist George Church’s lab alumni Stan Wang, who is also the biotech’s CEO.