Our team currently provides regulatory affairs support and consulting for numerous academic, private, and publically traded programs working towards clinical development of AAV-based gene and cell-based therapies. Since 2012, we have launched eighteen investigator-initiated first-in-human clinical biologic programs at Nationwide Children’s (14 AAV-based gene vectors and four cell-based products), have received fast-track designations for two programs, one breakthrough designation, eight orphan designations, and two rare pediatric disease designations. With this experience, we provide clients expert advice on regulatory pathways and submission requirements during the course of program development. This support may include consulting on pre-clinical studies and product manufacturing as well as review of various regulatory applications such as INTERACT, pre-IND, and IND submission packages.
Regulatory Document Writing and Publishing
The team also has the capacity to support full writing, editing, formatting and publishing of a variety of regulatory documents including
For those clients of The Clinical Manufacturing Facility at Nationwide Children’s, we have extensive CMC experience and offer CMC writing services for clients utilizing our cGMP facility to manufacture AAV-based gene therapy products for Phase I/II studies. In particular, we can provide all Module 3 CMC documents and supportive appendices (as phase appropriate) in eCTD format for both pre-IND and IND applications as well as the Quality Overall Summary of Module 2 and responses to CMC reviewer’s pre-IND meeting comments required as part of Module 1 of the initial IND application.