Micro-Dosing Gene Therapy: A Recipe For Success?
King’s College Spinout AviadoBio Is Targeting FTD And ALS
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.
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Embattled gene therapy companies will take comfort from desire of FDA and EMA help smooth the process of bringing the novel treatments to market.
AviadoBio believes it can overcome barriers to making gene therapy work in neurodegenerative diseases, and its new CEO knows what it takes to make a commercial success in the field.
The head of Novartis Gene Therapies in Europe tells Scrip that innovative payment models and involving all stakeholders have helped payers see beyond a simple price tag and see the benefits of a one-time gene therapy for spinal muscular atrophy.