Different approaches reflect optimism and doubts around gene therapies
Proving their commercial viability is arguably the biggest barrier that gene therapies have to overcome, with the growing body of evidence and regulatory approvals showing that they are winning the battle to prove efficacy and safety.
On Tuesday, there was another reminder of their therapeutic potential with some groundbreaking results for Sarepta Therapeutics’ (Nasdaq: SRPT) lead gene therapy, targeting Duchenne muscular dystrophy.
"It is currently unclear whether gene therapy pipelines will produce sustainable revenue in the long term"
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