BIO Comments on FDA Draft Guidance on Human Gene Therapy for Retinal Disorder and Hemophilia
November 14, 2018
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance on Human Gene Therapy for Retinal Disorder and Human Gene Therapy for Hemophilia.
BIO says the guidance will assist stakeholders developing human gene therapy (GT) products to treat rare diseases, including hemophilia and retinal disorders, and provided specific suggestions for the FDA to clarify both guidances. The topics discussed in BIO's comments include preclinical in vitro and in vivo proof-of-concept studies and disease-specific animal models, science-based regulatory flexibility when evaluating appropriateness of animal models, the use of the word "pharmacokinetics," biodistribution studies, reproductive/developmental toxicity studies, fit-for-purpose clinical trial designs, and patient experience.
Download Full Comments Below
FINAL BIO Letter GT For Hemophilia And GT For Retinal Disorder 12-10-18
Dear Colleagues,This month marks my one-year anniversary as BIO’s CEO. Like most of us in biotech, this post has been a labor of love. At no time in my 25-year career in this industry have I ever witnessed such awe-inspiring science. Today, we…
Re: BIO Comments on Office of Management and Budget (OMB) Statistical Policy Directive No. 8 North American Industry Classification System (NAICS)-Request forComments on Possible Revisions for 2027 (USBC–2024–0032)Dear Ms. Orvis,The Biotechnology…
Under the 340B program, participating manufacturers must offer 340B pricing on their covered outpatient drugs by covered entities, as a condition of having those drugs federally payable under Medicare Part B and Medicaid. Critically, Congress…
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance on Human Gene Therapy for Retinal Disorder and Human Gene Therapy for Hemophilia.
BIO says the guidance will assist stakeholders developing human gene therapy (GT) products to treat rare diseases, including hemophilia and retinal disorders, and provided specific suggestions for the FDA to clarify both guidances. The topics discussed in BIO's comments include preclinical in vitro and in vivo proof-of-concept studies and disease-specific animal models, science-based regulatory flexibility when evaluating appropriateness of animal models, the use of the word "pharmacokinetics," biodistribution studies, reproductive/developmental toxicity studies, fit-for-purpose clinical trial designs, and patient experience.