Guest Columns & From The Editor

  1. FDA Takes Aim At Regenerative Medicine Compliance With New Rapid Inquiry Program

    Regenerative medicine holds a lot of promise for patients, while many current therapies being used have an underwhelming amount of evidence to back claims being made. A June 2019 FDA announcement for a time-limited program, the Tissue Reference Group (TRG) Rapid Inquiry Program (TRIP), focuses attention once again on the FDA’s comprehensive regenerative medicine policy framework. Through Dec. 31, 2019, the TRIP aims to provide a three-day turnaround on responses to manufacturers who wish to obtain a “gut check” on the regulatory pathway of their product.

  2. Does Blockchain Make Sense For The Cell & Gene Therapy Supply Chain?

    Understanding the impact and requirements for a clearly defined supply chain strategy and implementing this into the complex environment of cell and gene therapies is not a simple task. It requires dedication and participation from all the stakeholders across the development-to-delivery life cycle. All the details need to be defined, simulation models developed, and risk assessed — and remediation plans put in place. 

  3. Inside Adaptimmune’s Road to ODD

    Adaptimmune’s SPEAR (Specific Peptide Enhanced Affinity Receptor) T-cell platform enables the engineering of T-cells to target and destroy cancer across multiple solid tumors. The FDA has granted Orphan Drug Designation (ODD) to SPEAR T-cells targeting MAGE-A4 (Adaptimmune’s ADP-A2M4 program) for the treatment of soft tissue sarcomas. Orphan Designation by FDA was created to encourage the development of drugs for rare diseases, such as sarcomas. I caught up with Dr. Elliot Norry, interim Chief Medical Officer at Adaptimmune, to learn more about the designation’s impact on the treatment of soft tissue sarcomas.

  4. Patient Group Advances Clinical Trials, Diverts Patients From Unregulated Therapies

    For a patient with a debilitating disease, such as pulmonary fibrosis (PF), moments of hope can be few and far between. Many of these patients search desperately for new therapies to improve their quality of life, or possibly cure their condition, sometimes outside of the approved treatments prescribed by their healthcare providers.

  5. The Three Pillars Of Patient Advocacy In Industry

    Patient advocacy plays a tremendously crucial role in the cell and gene sector. Jamie Ring, Head of Patient Advocacy at Spark Therapeutics and Cell & Gene Editorial Advisory Board member shared with me valuable information about role of patient advocacy in industry (i.e. what it is; what it’s not; the difference between patient advocacy in healthcare overall versus patient advocacy in gene therapy), which we will cover in detail in the coming weeks. To start, Ring lists and explains the three pillars of the role of patient advocacy in industry.   

  6. Developing And Manufacturing Cell & Gene Therapies: Do Biopharma Methods Apply?

    Are the methods used for developing the current generation of biopharmaceuticals, monoclonal antibodies, hormone replacements, etc., applicable to the next generation of Cellular and gene therapies?

  7. What’s A Cure Worth? ICER Publishes Value Assessment Framework For Curative Therapies

    The Institute for Clinical and Economic Review (ICER) recently conducted a study of value assessment methods considered for “cures” or “potential cures” and solicited input from international HTA bodies. The research will form the basis of a value framework that will be applied in the assessment of potential cures and other treatments that qualify as what ICER refers to as “single or short-term transformative therapies.”

  8. Progress On First Gene Therapy For DCM

    Dilated cardiomyopathy (DCM) is a condition that affects more than 3 million patients in the U.S. Recently subpopulations have been identified that develop DCM due to mutations in specific genes that have been shown to result in the development of DCM. One of these specific genes is the Bcl2-associated athanogene 3 (BAG3) gene.

  9. Gene Delivery Technologies To Create Tomorrow’s Cell Therapies

    As more cell-based therapies transition towards the market, hurdles surrounding safety, efficacy, and manufacturing are becoming increasingly apparent. The advancement of gene delivery technologies is fundamental to driving more clinical prospects of cell therapies.

  10. AskBio Talks AAV Gene Therapy Success

    I reached out to Dr. Jude Samulski, co-founder and chief scientific officer of AskBio, to learn more AskBio's recent acquisition of Synpromics and to better understand just how the end-to-end AAV gene therapy platform will affect and improve gene therapy in the United States.