Industry Insights

51. Inside AviadoBio And Astellas's Shared Mission To Bring Gene Therapy To Patients With FTD-GRN 7/2/2025

    AviadoBio and Astellas have joined forces to advance an investigational gene therapy designed to treat frontotemporal dementia with progranulin mutations (FTD-GRN), aiming to address a critical unmet need in neurodegenerative disease.

52. Cleaning Process Capability: Risk-Based Cleaning Process Performance Qualification 7/1/2025

    A cleaning validation professional must know how many process performance qualification runs are necessary. Here's how to go about demonstrating this with a high degree of assurance while minimizing the number of qualification runs.

53. How CGT Leaders Are Reducing Failures, Scaling Processes, And Overcoming Regulatory Friction 6/30/2025

     Industry leaders share their philosophy on reducing product failure, embracing AI, and building dynamic teams to stay ahead of the curve to bring curative CGT therapies to patients.

54. Why 'Which Is Best?' Is The Wrong Question In CGT Development 6/27/2025

    In cell therapy development, the instinct to seek a single “best” option is natural — but in reality, there’s rarely a universal winner.

55. Understanding FDA's New National Priority Voucher Pilot Program 6/25/2025

    The FDA's vouchers will be issued to companies addressing health issues in the U.S. and increasing domestic manufacturing, among other criteria.

56. The Latest Techniques And Technology For Differentiating Capsids 6/24/2025

    Since AAV gene therapy’s origins, scientists and regulators have sought optimized ways to measure empty/full capsids. This expert reviews pros and cons of modern methods.

57. Advancing Gene Delivery: LNPs, Adenovirus, Lentivirus, And More 6/24/2025

    During this month’s Cell and Gene Live, Advancing Gene Delivery: LNPs, Adenovirus, Lentivirus, And More, expert panelists Francesca Barone, M.D., Ph.D., CSO at Candel Therapeutics and Karen Kozarsky, Ph.D., President of Vector BioPartners shared their perspectives on current challenges, technological innovations, and future directions in gene therapy vector development and application.

58. Hope For People With Spinocerebellar Ataxia 6/24/2025

    Spinocerebellar ataxia (SCA) is a rare, progressive, and fatal neurodegenerative disease with no approved treatments, but Biohaven Pharmaceuticals’ new drug troriluzole — designed to modulate glutamate and slow disease progression — has shown promise in clinical trials and is currently under FDA review, offering hope to patients and families facing this devastating disorder.

59. AI-Assisted QMS: Bridging ISO/IEC 42001:2023 And ICH Q10 For Pharmaceutical Excellence 6/23/2025

    Integrating AI into quality management systems (QMS) can be a transformative opportunity to enhance quality assurance, improve compliance, and enable proactive risk management. 

60. Navigating FDA Regulatory Changes: Policy Shifts, Deregulation, Restructuring, And Future Oversight 6/23/2025

    Recent shifts within the U.S. FDA reflect an increasingly complex tension between scientific rigor and accelerating political and economic pressures. Here are the newest developments and emerging focal points.