Industry Insights

  1. New Reference Standards To Support Quality of AAV Raw And Starting Materials 1/31/2025

    The United States Pharmacopeia aims to help gene therapy manufacturers address the challenges that come with complex starting materials like endonuclease and plasmids.

  2. These Were FDA's Top Citation Issues For Data Quality In 2024 1/31/2025

    Pharma, biopharma, and medical device makers should watch out for these perennial problems, which include inadequate procedural controls and poor record-keeping.

  3. 2024 Trends In FDA Observations For Sterile Drug Manufacturers 1/30/2025

    This data-driven analysis of the 2024 trends in FDA observations for sterile drug manufacturers includes a comparison to the 2023 trends. Which 21 CFR 211 sections surprise you most?

  4. Tessera Therapeutics Advances In Vivo Gene Therapy For SCD 1/30/2025

    Tessera Therapeutics' CSO, Michael Holmes, Ph.D., shares information on the company's Gene Writers for sickle cell disease (SCD) and how their work has garnered investment from of the Bill & Melinda Gates Foundation to support their efforts.

  5. The Evolution Of Cell And Gene Therapy: Pioneering Breakthroughs For A New Era In Medicine 1/30/2025

    This article explores the rapid evolution of cell and gene therapy (CGT), highlighting its expansion beyond hematology and oncology into autoimmune and metabolic diseases, emphasizing the need for safety, patient access, and a collaborative, knowledge-sharing approach to accelerate innovation and ensure broader therapeutic impact via the "Knowledge Research Organization."

  6. The Genetic Link Between Gaucher Disease And Parkinson's: How GBA1 Could Play A Role In The Next Generation Of Gene Therapy 1/30/2025

    GBA1 mutations, which cause Gaucher disease and are the most common genetic risk factor for Parkinson's disease.

  7. Catalysts of Change: How the Cell and Gene Therapy Market Has Evolved 1/28/2025

    Since 2021, the CGT market has continued to grow, but challenges remain, especially with regard to regulatory compliance.

  8. Computer Systems Validation Pitfalls, Part 4: Inattention To Details 1/24/2025

    The authors conclude their 4-part article series on computer systems validation (CSV) pitfalls using real case studies to illustrate points related to inattention to details.

  9. Digital Certificates Of Analysis: A Vision For The Transfer Of Quality Data 1/23/2025

    The pharma/biotech industry must ramp up adoption of digital certificates of analysis, which are transforming the way quality data is transferred between organizations in the supply chain.

  10. Easing Autologous Cell Collection, Automating Manufacturing 1/22/2025

    To circumvent the challenges of leukapheresis, CellProthera developed a novel method for collecting therapeutic stem cells from whole blood. CSO Ibon Garitaonandia, Ph.D., discusses the company's mission to streamline manufacturing.