Gene Therapy

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While the findings suggest that switching to onasemnogene abeparvovec after non–gene therapy treatments could be beneficial to patients with spinal muscular atrophy, it is not clear which patients may benefit the most from switching therapies. | Image credit: Rochu_2008 - stock.adobe.com

Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

By Rose McNulty

August 15th 2025

Children with spinal muscular atrophy (SMA) showed motor function improvements after switching to onasemnogene abeparvovec following prior treatment with nusinersen or risdiplam in a real-world study.

Gene therapy appears to be a promising approach for a subset of genetic deafness. | Image credit: NathanDeverycom - stock.adobe.com

Progress and Challenges With Gene Therapy in Hereditary Hearing Loss

By Rose McNulty

July 31st 2025

The combination of novel vectors with tissue- and cell-specific regulatory elements to construct a toolbox for targeted delivery is a promising strategy. | Image credit: Dr_Microbe - stock.adobe.com

The State and Future of Viral, Nonviral Vectors for Gene Therapy

By AJMC Contributor

July 25th 2025

OneOncology Appoints Michael Byrne, DO, as Medical Director for Cellular Therapy

OneOncology Appoints Michael Byrne, DO, as Medical Director for Cellular Therapy

July 22nd 2025

The first gene therapies for sickle cell disease were FDA approved in 2023, but high upfront costs have been barriers to patient access to these life-changing treatments. | Image credit: RFBSIP - stock.adobe.com

CGT Access Model Aims to Expand Access to Sickle Cell Treatments

By Rose McNulty

July 18th 2025

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