Cell and Gene Therapy
THE LATEST
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio—have products in the pipeline that could improve treatment options for this rare disease.
CAR T–focused biotech Cargo Therapeutics surprised and disappointed analysts when it announced that it would discontinue a mid-stage trial of its lead program, firi-cel.
In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis talks to Tom Whitehead, co-founder of the Emily Whitehead Foundation, about how standard care, cell and gene therapies and their impact on patients.
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
At J.P. Morgan, most biopharma executives expressed a neutral stance on the incoming administration, but just days later, President Trump issued multiple executive orders that concern the industry.
Five years ago, Gilead signed a massive deal with Galapagos. After a restructuring, the pharma is still hunting for the potential it saw at the original signing.
As the year gets underway, analysts and biotech executives highlight cell therapy’s pivot from oncology to autoimmune diseases, a continued appetite for next-generation obesity drugs and an increased focus on neuromuscular, kidney and cardiovascular diseases.
Traditionally carrying a dire prognosis, the treatment paradigm for multiple myeloma is changing, with CAR T therapies, bispecifics and more contributing to multifaceted regimens unique to each patient’s needs.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis talks to Dr. Peter Marks, Director, CBER about his thoughts on the future of cell and gene therapies.