INSIGHTS ON CELL & GENE REGULATORY ISSUES

• Surveying The U.S. Regulatory Landscape For Cell And Gene Therapies

  The FDA has been issuing guidance documents addressing gene therapy development issues for approximately 20 years — a remarkable dedication of resources to an area that did not have a licensed product until 2017. Of the six gene therapy-related draft guidances the agency issued last month, two represent the first of the “suite of disease-specific guidance documents on the development of specific gene therapy products” Commissioner Scott Gottlieb promised in Dec. 2017. 

• 4 Future Scenarios For Cell And Gene Therapy Adoption

  Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?

• Summarizing FDA’s New Guidance On Gene Therapy For Hemophilia

  The FDA recently issued six draft guidance documents. This article discusses the guidance Human Gene Therapy for Hemophilia.

• FDA’s New Guidance On Gene Therapy For Retinal Disorders — A Primer

  This is the fifth article in a six-part series summarizing each of the FDA's gene therapy draft guidance documents. It will discuss the draft guidance Human Gene Therapy for Retinal Disorders.

• Gene Therapy For Rare Disease — Examining FDA's New Draft Guidance

  The FDA recently issued for public comment six draft guidance documents. This is the fourth article in a series and will discuss the guidance Human Gene Therapy for Rare Disease.

• An Overview Of FDA's New Guidance On Long-Term Follow-Up After Administration Of Gene Therapies

  The FDA recently issued for public comment six draft guidance documents intended to serve as part of a modern, comprehensive framework for how CBER will help advance the field of gene therapy. This is the third article in a six-part series.

• FDA’s New Guidance On Replication Competent Retrovirus Testing During Gene Therapy Manufacture

  The draft guidance document provides recommendations regarding the testing for RCR during the manufacture of retroviral vector-based gene therapy products, and for follow-up monitoring of patients who have received retroviral vector-based gene therapy products.

• FDA’s New Guidance On CMC For Gene Therapy INDs: What You Need To Know

  The FDA recently issued for public comment six draft guidance documents intended to serve as part of a modern, comprehensive framework for how CBER will help advance the field of gene therapy. 

• Regulators, Standards Groups Address Emerging Tech In Biopharma

  Manufacturers, suppliers, and regulators have recognized that introducing new technologies also introduces a degree of unfamiliarity and uncertainty.

• New Microbiological Methods In Bioprocessing

  Testing for evidence of microbial contamination in bioprocessing has a long history — and can be expensive, slow, and burdensome. So suppliers, testing facilities, and regulators have been seeking improvements.