Cell and Gene Therapy

In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis and Tom Whitehead continue to discuss the patient and caregiver experience, where Tom gives his insights to the future of CGTs.

Gilead beat consensus estimates in Q4 with $7.6 billion in revenue, driven largely by its HIV drug Biktarvy and CAR T therapies Trodelvy and Yescarta.

Vertex expects to make the newly approved non-opioid pain medicine Journavx available by the end of February.

Adding a new indication for the CAR T cell therapy could help BMS offset the loss-of-exclusivity headwinds it faces in the coming years.

Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies.

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.