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Gene Therapy: What To Expect Now And In The Future

A Look At The State Of The Industry

 
• By Alaric DeArment

The US FDA could approve as many new gene therapies in 2023 as it has in the previous five years. Development in the field has rapidly accelerated, but still faces hurdles.

• Source: Shutterstock

A trickle becoming a stream might describe the state of gene therapy development in the US, where in the year 2023 regulators anticipate approving as many gene therapies as they had from 2017-2022. In the process, the US Food and Drug Administration will have unleashed not only viral vector-based gene therapies for ultra-rare diseases, but treatments using newer modalities, like gene editing, to treat more common conditions like sickle cell disease (SCD).

The most recent addition to the armamentarium is BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec), which the FDA approved for...

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