In vivo applications and toxicities of AAV-based gene therapies in rare diseases

Zhao, Q., Peng, H., Ma, Y. et al. In vivo applications and toxicities of AAV-based gene therapies in rare diseases. Orphanet J Rare Dis 20, 368 (2025). doi:10.1186/s13023-025-03893-z 

As of early 2024, only eight AAV-based gene therapy drugs have been approved.AAV-based gene therapies have revolutionized treatment for rare diseases; however, addressing toxicity and improving long-term efficacy remain key challenges.

 

First treatment option SKYCLARYS™ (omaveloxolone) for Australians diagnosed with ‘neurogenerative disease’ Friedreich ataxia1

Sydney, Australia – 21st July 2025 – Biogen Australia welcomes the TGA (Therapeutic Goods Administration) approval of SKYCLARYS™ (omaveloxolone) as the first treatment for adults and adolescents aged 16 years and older with the rare, genetic, progressive disease Friedreich ataxia (FA).

SKYCLARYS is not listed on the Pharmaceutical Benefits Scheme (PBS).