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Sidestepping Gene Therapy ‘Land Grab’ Mistakes, Rocket Is Set For First Filings In 2023

Two Rare Disease Filings Expected This Year

 
• By Andrew McConaghie

While other gene therapy companies faced major clinical and financing problems in 2022, Rocket Pharma sidestepped some of these pitfalls and is gearing up for two US filings this year.

The adeno-associated viral vector (AAV) based gene therapy field bore the brunt of the biotech sector slump in 2022, but New Jersey-based Rocket Pharmaceuticals emerged relatively unscathed – a fact its CEO attributed to ignoring an ill-advised “land grab” for the biggest targets in genetic diseases.

Many other pre-market companies had to slash headcount and budgets last year due to limited R&D progress and dwindling cash...

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