Gene Therapy: AAV Doses Should Not Be Subject To Fixed Upper Limit, US FDA Panel Says
Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.
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Regenerative Medicines: Development Slowed By Lack of Standardization, Regulatory And Manufacturing Challenges
A Government Accountability Office report identifies 10 policy options aimed at speeding development and boosting the use of regenerative therapies, but breaks little new ground, highlighting the continuing difficulties in the space.
Product Quality ‘Wild Card’? Sarepta’s Gene Therapy Manufacturing Process Change Means More Empty Capsids
SRP-9001 intended for commercialization is not analytically comparable to the product used in earlier clinical studies; FDA CMC reviewer said agency does not mandate the extent of empty or full capsids but, instead, looks at the totality of data to assess safety and efficacy.
The upcoming review of two novel gene therapies from bluebird bio marks will be the first time the US FDA’s CTGTAC has met for a genetic product review in five years. That is a surprisingly long gap – and one that probably will not be repeated.