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BIO Comments on Draft Guidance on Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)

November 14, 2018

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs).

BIO says the comprehensive draft guidance effectively covers a range of chemistry, manufacturing, and control (CMC) topics relevant to gene therapy products.

However, BIO notes the draft guidance is quite prescriptive and does not necessarily match modern manufacturing. BIO would like to see a better balance between the information FDA needs to see in order to allow an IND to move forward, and over-reporting of information that may be unnecessary. On the whole, it seems much of the information required by the draft guidance is more appropriate for a BLA filing, an approved product, or postapproval changes to a product rather than an IND. BIO suggests specific changes to avoid misinterpretation and clarify the scope, the stage of development the guidelines apply to, and testing expectations, among other topics.

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2018-12-10 BIO Comments On CMC Information For Human Gene Therapy INDs FINAL
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