BIO Comments on FDA Draft Guidance on Human Gene Therapy for Retinal Disorder and Hemophilia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BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance on Human Gene Therapy for Retinal Disorder and Human Gene Therapy for Hemophilia.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
On behalf of its member organizations, Biotechnology Innovation Organization (“BIO”) respectfully submits this Comment in response to the United States Patent and Trademark Office’s (the “Patent Office” or “PTO”) February 13, 2024 Request for…
BIO submits comments on the MDH’s proposed regulations regarding Drugs of Substantial Public Interest: Draft Methodology for Public Comment as required in statute by the Prescription Drug Price Transparency Act.
BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance on Human Gene Therapy for Retinal Disorder and Human Gene Therapy for Hemophilia.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.