The field of genetics is moving rapidly with new techniques that focus on DNA manipulation in vivo resulting in the alteration of genes to correct mutations, introduce new genetic information, and remove specific DNA sequences. Zinc-Finger Nucleases, TALENs and CRISPR/Cas9 are a few of the genome editing tools that are now available to the scientific research community. To address the societal issues surrounding genome editing, the International Summit on Human Gene Editing, hosted by the scientific academies of China, the United Kingdom and the U.S. was held in December 2015 in Washington DC. The summit concluded that somatic therapies based on genome editing should proceed under the existing FDA regulatory framework, but editing the human germline would be irresponsible to pursue at this time.
FDA has regulatory authority over genetically manipulated cells and/or their derivatives and requires submission of an Investigational New Drug application (IND) before a clinical study can proceed. FDA's regulations on investigational new drugs, including those for the submission and review of an IND are described in Title 21 of the Code of Federal Regulations (CFR), Parts 50, 56, and 312. A Federal Register (FR) notice describing FDA's authority over cell and gene therapy products ("Application of Current Statutory Authorities to Human Somatic Cell Therapy Products and Gene Therapy Products") was published on October 14, 1993, (58 FR 53248) and a final rule establishing criteria for regulation of human cellular and tissue- based products (HCT/P) based on a tiered approach, including reproductive cells and tissues was published on January 19, 2001.
Cellular & Gene Therapy Products
The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight.
Cellular therapy products include cellular immunotherapies, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications, including hematopoetic stem cells and adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a person's DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition. CBER has approved both cellular and gene therapy products – a list of these products may be found here.
Cellular and gene therapy-related research and development in the United States continue to grow at a fast rate, with a number of products advancing in clinical development. In addition to regulatory oversight of clinical studies, CBER provides proactive scientific and regulatory advice to medical researchers and manufacturers in the area of novel product development.
Related Information
- What is Gene Therapy?
- Approved Cellular and Gene Therapy Products
- Cellular, Tissue, and Gene Therapies Advisory Committee
- Therapeutic Cloning and Genome Modification
- Compliance Actions (Biologics)
- Safety & Availability (Biologics)
- Advisory on Legal Restrictions on the Use of Mitochondrial Replacement Techniques to Introduce Donor Mitochondria into Reproductive Cells Intended for Transfer into a Human Recipient
Regulatory Information
- Cellular & Gene Therapy Guidances
- Application of Current Statuatory Authorities to Human Somatic Cell Therapy Products and Gene Therapy Products (PDF - 489KB)
- Consolidation of Wound Care Products Containing Live Cells
- Proposed Approach to Regulation of Cellular and Tissue-Based Products (PDF - 452KB)
- References for the Regulatory Process for the Office of Cellular, Tissue and Gene Therapies
- Regenerative Medicine Advanced Therapy Designation
- Reinventing the Regulation of Human Tissue
- References for the Regulatory Process for the Office of Cellular, Tissue and Gene Therapies
Spotlight
Contact FDA
Division of Communication and Consumer Affairs
Office of Communication, Outreach and Development
Food and Drug Administration
10903 New Hampshire Avenue
Building 71 Room 3103
Silver Spring, MD 20993-0002Page Last Updated: 08/30/2017
-- You received this message because you are subscribed to the Google Groups DIYbio group. To post to this group, send email to diybio@googlegroups.com. To unsubscribe from this group, send email to diybio+unsubscribe@googlegroups.com. For more options, visit this group at https://groups.google.com/d/forum/diybio?hl=en
Learn more at www.diybio.org
---
You received this message because you are subscribed to the Google Groups "DIYbio" group.
To unsubscribe from this group and stop receiving emails from it, send an email to diybio+unsubscribe@googlegroups.com.
To post to this group, send email to diybio@googlegroups.com.
Visit this group at https://groups.google.com/group/diybio.
To view this discussion on the web visit https://groups.google.com/d/msgid/diybio/CAAhtNQtuUSxQhTrdGoH0QvuQFtiRZzVeTzKVXUkwdnAFEEJ_YA%40mail.gmail.com.
For more options, visit https://groups.google.com/d/optout.
0 comments:
Post a Comment