INSIGHTS ON CELL & GENE REGULATORY ISSUES

  • Clinical Strategy For Successful Cell And Gene Therapy — How Did I Get Here?

    There are major challenges to the widespread adoption of cell therapies, primarily related to the high manufacturing costs of what is largely a manual process with complex logistics and supply chain. Experts share their experiences and discuss emerging trends in the field to accelerate life-saving therapies from the bench to the bedside.

  • Strategies For Startups — How To Structure Milestones For Successful Outcomes

    Experts from the cell and gene therapy field share their experiences and discuss trends to accelerate therapies from the bench to the bedside, with specific focus on strategies for start-ups, how to look at robust scale-up, and reducing CMC risk.

  • Attracting The Right Capital — Forming Partnerships For Long-Term Success

    Experts share their experiences on investing in the cell and gene therapy field and how to form long-term partnerships for companies on the path towards bringing a potentially life-changing therapy to patients.

  • Key Changes In The Revised EMA Guidance On Clinical Trials During COVID

    In February, the European Medicines Agency (EMA) released the fourth version of its Guidance on the Management of Clinical Trials During the COVID-19 (Coronavirus) Pandemic. As the pandemic continues to impact clinical research worldwide, these updated guidelines reflect the EMA’s evolving stance, clarifying questions raised by previous versions and taking into account the most recent changes in the global health landscape. Here’s an overview of what’s new.

  • Regulatory Requirements Featuring bluebird bio

    Just recently, I had the chance to sit down with Matt Pillar and bluebird bio’s VP Regulatory Operations and Compliance, Scott Cleve, for a candid discussion on the regulatory trends shaping the advance of bluebird’s pipeline of gene therapies for the treatment of serious, life-altering diseases including Cerebral Adrenoleukodystrophy, Multiple Myeloma, Transfusion-Dependent β-Thalassemia, and Sickle Cell Disease.

  • Reducing Human Error In Gmp Pharma/Biopharma Operations

    CAI clients often seek help in closing open investigations, but ignore the source of these investigations, even while product sits quarantined. Where clients really need help is in cutting human error off at the source. This blog explores the factors within an organization’s control that impact ongoing human performance.

  • How To Attract Investors – Funding Advice For Biotechs From VC Experts

    How do you find the initial capital and attract new investors to hit your milestones and stay solvent? Experts share their experiences and offer advice for companies on the path towards bringing a potentially life-changing therapy to patients.

  • Reducing CMC Risk From IND To Commerical Manufacturing Of Cell And Gene Therapies

    Now that the industry’s got some cell and gene therapy approvals under its belt, how do we rapidly but safely move new candidates through the clinic? Experts in the cell and gene therapy field share their experiences and discuss emerging trends to accelerate life-saving therapies from the bench to the bedside.

  • Bench To Bedside And FDA Submission Strategies For Cell And Gene Therapies

    Cell therapies have the potential to transform global healthcare by providing curative therapies for once incurable diseases, but there are major challenges to their widespread adoption. Industry experts share their experiences and discuss emerging trends to accelerate life-saving therapies from the bench to the bedside.

  • FDA’s 2021 Focus Areas Of Regulatory Science: 5 Trends To Watch

    The FDA recognized that a new approach was required to keep pace with the rapid evolution of science and technology driving drug development, and it released the report Advancing Regulatory Science at FDA: Focus Areas of Regulatory Science (FARS) early in 2021 to identify and communicate areas requiring continued targeted investment. These five key elements deserve our attention.