Cell and Gene Regulatory

1. Strategies To Reduce Regulatory Risks In Gene Therapy 5/15/2020

   Learn how to mitigate regulatory risk for your gene therapy process.

2. Reducing The Risk During Cell And Gene Therapy Development And Manufacturing 5/13/2020

   While these new modalities are generating significant excitement among patients and caregivers, cell and gene therapy manufacturers are experiencing growing pains. The rapid growth and continued momentum of the industry has led to the issuance of new guidance. In this article we share insights for navigating within a novel and complex regulatory environment, mitigating risks posed by raw materials and implementing proper risk assessments during manufacturing.

3. Snapshot: A New Guidance Document Program From The FDA 4/21/2020

   In a progressive effort to mitigate these long-standing concerns and bring a level of clarity to its guidance documents, the FDA has created a pilot program called “Guidance Snapshots.” As experts, we are always debating about what the guidance is asking and how the contents are to be applied.

4. Understanding New FDA Guidance For Pediatric Oncology Studies 4/21/2020

   The Research to Accelerate Cures and Equity (RACE) for Children Act requires that any original new drug or biologics license application submitted on or after August 18, 2020, for a new active ingredient must contain reports of molecularly targeted pediatric cancer investigations if the drug is directed at a molecular target that the FDA determines to be substantially relevant to the growth or progression of a pediatric cancer. Learn more about the changes in this article.

5. Regulatory CDMO Services 4/15/2020

   Regulatory services from Bench to Bedside
   CCRM’s extensive knowledge in cell and gene regulatory affairs can help you de-risk your projects at any stage of development, from early stage preclinical planning through to global commercialization. We have experience negotiating clinical trials and new product commercialization with regulators around the world and can leverage this to help you design a go-to-market regulatory strategy appropriate to your regenerative medicine technology, saving you time and money and improving predictability.

6. Real World Evidence (RWE) In Regulatory Decision Making: Establishing Study Credibility And Totality Of Evidence 4/2/2020

   This blog shares some of the learnings from the recent Duke-Margolis webinar entitled, "Bolstering RWE Study Credibility and Its Role in a Totality of Evidence Approach".

7. INTERACT Meetings: Streamlining Early Clinical Development 3/9/2020

   Read how the INTERACT meeting can be an invaluable engagement with the agency to inform the successful planning and execution of a novel product development program.

8. Strategies To Address The Viral Vector Manufacturing Shortage 3/5/2020

   Accelerating the production of your viral-vector based therapy requires understanding the strategies and cost-efficiencies available to address the viral vector manufacturing shortage.

9. CCDS Or Local Labeling, A Chicken-And-Egg Situation? 2/25/2020

   In this blog, UBC’s Dr. Luca Cariolato, Sr. Safety Scientist, speaks to the importance of developing a Company Core Data Sheet (CCDS) before local safety information.

10. An Analysis Of FDA’s Recently Issued Gene Therapy Guidances 2/20/2020

    In this article, we'll examine key changes to FDA's six finalized draft guidance documents on its framework to advance gene therapy. We'll also review a new draft guidance on determining “sameness” of gene therapy products for orphan drug designation/exclusivity.