Cell and Gene Therapy
THE LATEST
Lexeo Therapeutics’ investigational gene therapy reduces left ventricular volume and wall thickness in patients with Friedreich’s ataxia, according to a small study.
Interius BioTherapeutics has received approval from Australia’s Human Research Ethics Committee to begin the first-in-human trial of an investigational in vivo CAR-T therapy designed to treat B-cell malignancies.
Following a disappointing readout last year, uniQure on Tuesday posted promising Phase I/II data for its investigational gene therapy AMT-130 and nabbed the first-ever Regenerative Medicine Advanced Therapy designation from the FDA in Huntington’s disease.
The groundwork being done in 2024 is building the foundation for global collaboration in the future.
Johnson & Johnson and Legend Biotech’s Carvykti cell therapy significantly improved survival in patients with multiple myeloma when used in the second-line setting, the companies announced on Tuesday.
Rocket Pharmaceuticals’ gene therapy Kresladi has been hit with an FDA Complete Response Letter requesting additional chemistry, manufacturing and controls information to complete its review.
One patient died of respiratory failure in a Phase I study of Lyell Immunopharma’s investigational CAR-T therapy. The company on Wednesday said it has not definitively linked the fatality to the treatment.
After winning expanded approval for its gene therapy for Duchenne muscular dystrophy, Sarepta’s leadership and analysts see a sizeable commercial opportunity on the horizon.
Exsilio Therapeutics emerged from stealth on Tuesday with a platform that leverages mRNA technology to develop redosable genomic medicines for a range of complex diseases.
While Thursday’s label expansion and traditional approval for the gene therapy is an important milestone, many challenges still face the Duchenne muscular dystrophy community.