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Gene Therapy: BIO Comments on Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up

November 14, 2018

Re: Docket No. FDA-1999-D-0081: Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding the draft guidance titled “Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up”.

BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial, and environmental biotechnology products.

BIO applauds the FDA on the work done to develop this Draft Guidance along with its companion documents on the topic of Gene Therapies. We find the Agency’s Draft Guidance regarding product testing for replication competent retrovirus (RCR) to be well-reasoned and we concur with most recommendations. Particularly, BIO commends the Agency for inviting the Gene Therapy community to publish findings of relevant studies. BIO believes such an effort can potentially benefit and advance the field. In addition, FDA should consider conveying a public workshop with industry, academic experts, and other key stakeholders to discuss current scientific data regarding RCR in gene therapy applications that would be of tremendous benefit for all parties involved. BIO would be happy to work with the Agency on such effort. Lastly, there are a number of passages in the Draft Guidance that BIO believes would benefit from further clarity. We have provided more detailed information on the table below.

BIO appreciates this opportunity to submit comments regarding FDA’s draft guidance titled “Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up”. We would be pleased to provide further input or clarification of our comments, as needed.