FDA Leader: We Need To Remove Surprises From Gene Therapy Development
Embattled gene therapy companies will take comfort from desire of FDA and EMA help smooth the process of bringing the novel treatments to market.
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The Israeli firm’s gene therapy asset has disappointed in an advanced ovarian cancer study, leaving experts skeptical of its potential in other tumors and the company turning to an earlier program.
Sanofi/Sobi’s efanesoctocog is in line for US FDA action three months after CSL/uniQure’s gene therapy user fee goal, while BioMarin’s Roctavian resubmission and Novo Nordisk’s concizumab are waiting in the wings.
Utilizing the genetic overlap between frontotemporal dementia and amyotrophic lateral sclerosis, AviadoBio is seeking to develop gene therapies which can stop both these devastating diseases in their tracks using novel delivery techniques.