Table of Contents
terminated earlier, the AskBio License automatically expires on the date on which we no longer have any payment obligation under the AskBio License. The AskBio License may be
terminated prior to its expiration:
- by us upon six months advance written notice to AskBio;
- by either party upon the other party's breach of the AskBio License that is not cured within the specified cure period based on the
nature of such breach;
- by either party in the event of either party's bankruptcy, insolvency or certain similar occurrences;
- by AskBio if, during a specified period of time and subject to certain conditions being met, we research, develop or commercialize an
AAV-based treatment for hemophilia or we undergo a change of control with, or are otherwise acquired by, a third party that conducts such activities;
- by AskBio if we bring any action or proceeding challenging the validity or enforceability of any of the licensed patents.
of September 30, 2015, we have paid AskBio the $1.0 million upfront license fee and accrued $16,941 in annual maintenance fees.
Government Regulation and Product Approval
In the United States, the FDA regulates biologic products including gene therapy products under the Federal Food, Drug, and Cosmetic
Act, or the FDCA, the Public Health Service Act, or the PHSA, and regulations and guidance implementing these laws. The FDCA, PHSA and their corresponding regulations govern, among other things, the
testing, manufacturing, safety, efficacy, labeling, packaging, storage, record keeping, distribution, reporting, advertising and other promotional practices involving biologic products. Applications
to the FDA are required before conducting human clinical testing of biologic products. Additionally, each clinical trial protocol for a gene therapy product candidate is reviewed by the FDA and, in
limited instances the National Institutes of Health, or the NIH, through its Recombinant DNA Advisory Committee, or RAC. FDA approval also must be obtained before marketing of biologic products. The
process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local and foreign statutes and regulations require the expenditure of substantial time and
financial resources and we may not be able to obtain the required regulatory approvals.
the FDA, the Center for Biologics Evaluation and Research, or CBER, regulates gene therapy products. Within CBER, the review of gene therapy and related products is consolidated
Office of Cellular, Tissue and Gene Therapies, or the OCTGT, and the FDA has established the Cellular, Tissue and Gene Therapies Advisory Committee, or the CTGTAC, to advise CBER on its reviews. CBER
works closely with the NIH and the RAC, which makes recommendations to the NIH on gene therapy issues and engages in a public discussion of scientific, safety, ethical and societal issues related to
proposed and ongoing gene therapy protocols. Although the FDA has not yet approved any human gene therapy product for sale, it has provided guidance for the development of gene therapy products. This
guidance includes a growing body of guidance documents on chemistry, manufacturing and control, or CMC, clinical investigations and other areas of gene therapy development, all of which are intended
to facilitate the industry's development of gene therapy products.
social and legal concerns about gene therapy, genetic testing and genetic research could result in additional regulations restricting or prohibiting the processes we may use.