Cell and Gene Therapy

A push to reshore some drug production and progress in advanced manufacturing technologies have been prominent trends this year, industry leaders say.

Of all the stories we published this year, these deep dives by BioSpace editors stand out as relevant re-reads going into the New Year.

Pfizer and Metsera, Sarepta and uniQure made the list with dramatic tales. The other two spots went to the regulatory challenges facing biopharma under the new administration, especially in the vaccines sector.

A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 million.

Addition joins a growing list of launches this year, following in the footsteps of startups like Crystalys Therapeutics and Ollin Biosciences.

Eli Lilly’s retatrutide exceeds expectations in Phase III, capping off a sparkling 2025 for the obesity titan; an internal FDA safety review finds no confirmed pediatric deaths caused by COVID-19 vaccines, and Commissioner Marty Makary says no black box warning will be attached to the shots; and BioSpace looks at six biotechs that could be pharma’s next buy.

These deals radically reshaped the biopharma world, either by one vaccine rival absorbing another, a Big Pharma doubling down after another failed acquisition or, in the case of Pfizer and Novo, two heavyweights duking it out over a hot obesity biotech.

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval.

For traditional approval, CAR T therapies will need to establish superiority over current standard treatments, including already-approved CAR T products.

As big pharmas including Takeda and Novo Nordisk flee the cell therapy space and smaller biotechs shutter their operations, these players are sticking around to take the modality as far as it can go.