INSIGHTS ON CELL & GENE REGULATORY ISSUES

  • Navigating Regulatory And Development Milestones

    The biotherapeutics market is evolving, and emerging companies are advancing cutting-edge therapies to keep pace with innovation. The potential for life-changing, curative solutions where limited options exist makes this an exciting time to be in biotech. Although patient focus is constant, regulatory concerns and investment capital needs evolve as biopharma startups move their products from development to approval. This article addresses how early-stage biotherapeutic companies can avoid common pitfalls and access support as they navigate the funding and regulatory processes to bring novel therapeutics from research to market.

  • Gap Analysis Fundamentals To Maintain Forward Progress

    Gap analysis—defined as the process of reviewing all the available information for a candidate product to assess current development status, identify potential gaps in information, and the subsequent steps required to develop a strategy to fill those gaps a fundamental and ongoing exercise. In a recent The Business Of Biotech podcast Dr. Valenzuela,  CEO at Andes Biotechnologies, was joined on the podcast by Cristian Hernández-Cuevas, COO at Andes. Their collective reflections and advice on gap analysis execution provides real-world guidance for the leaders of emerging biopharma organizations.

  • CMC Considerations For First In-Human Studies

    As you gear up for your first-in-human studies, your CMC (chemistry, manufacturing, and controls) and medical affairs leaders will come together to play principal—and interdependent—roles in the success of those initiatives.  On a recent episode of The Business of Biotech podcast, Dr. Perez, Chief Medical Officer and Dr. Ihle, VP of CMC and Quality at Bolt Biotherapeutics, Inc. shared their collective advice for emerging biotech leaders who are new to first-in-human studies. Here’s a sample of what they shared.

  • The Relationship Between GMP And CMC - And The Team That Ties It Together

    While GMP adherence might seem like a prescriptive series of boxes that biopharmaceutical producers are responsible for checking off, there’s a bit more strategy involved as it relates to a company’s overarching CMC (chemistry, manufacturing, and controls) strategy.

  • The Data Quality & Master Files Challenge For FDA Submissions

    From its beginnings in 2016, Celularity Inc.’s Dr. Robert Hariri knew his company would face a particularly acute challenge with the FDA. The company’s therapeutic approach—the development of medicine from allogeneic placental-derived cells—is entirely dependent on its ability to do something that had never been done before: accomplish the at-scale harvest and preservation of healthy, postpartum fetal stem cells from maternity wards far-and-wide.

  • Particle Size Analysis Overview

    This paper reviews different analytical techniques for particle size analysis, including types of distributions, laboratory versus online systems, and sample result comparisons. It is intended to help those new to particle size analysis understand each technique’s various strengths and weaknesses in order to select the proper technique for their samples.

  • Subvisible Particulate Matter Testing

    Particulate matter is both unwanted contamination and a potential health risk to the patient. Combining the highest specification technology with advanced software and automation options make these instruments the most advanced instruments available for USP particle testing.

  • 4 Critical Processes Impacting Cost Of Goods For Gene Therapy

    Many gene therapies rely on plasmids and viral vectors. There is an urgent need to drive down the costs of these critical components. Learn about the critical steps that impact cost and process innovations for viral vector manufacturing.

  • Long-Term Follow-Up In Gene Therapy Trials: Ensuring Patient Engagement & Regulatory Compliance

    To understand and mitigate the risk of these delayed adverse events, participants in gene therapy trials may be monitored for a long-term follow-up (LTFU) period, which may be as long as 15 years. During this period, sponsors are challenged with navigating complex regulatory requirements as well as finding innovative ways to keep patients engaged for a decade or more.

  • Creating A Compliant eCTD: Avoiding Common Regulatory And Technical Pitfalls On The Path To Submission

    Failure to comply with the regulatory and technical requirements when creating your eCTD could jeopardize the success of your submission and your product development plan as a whole, especially for early-stage biotechs working with limited resources. Here are strategies for avoiding some of the most common electronic publishing pitfalls on the path to submission.