Industry Insights

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

    The FDA has finalized six draft guidance documents intended to serve as part of a modern, comprehensive framework for how the agency will help advance the field of gene therapy. At the same time, it issued a new draft guidance on determining “sameness” of gene therapy products for the purposes of orphan drug designation/exclusivity. This article highlights key changes made in each of the final guidances and also reviews the new draft guidance.

  2. Part 2: Cell And Gene Therapy Is Transforming Healthcare 2/13/2020

    Healthcare sector players are neither adequately equipped nor strategically placed near patients to accommodate the anticipated volume of clinical trials to evaluate cell and gene therapies (CGT) in development. While the industry is adjusting and deploying the required capabilities, the transformation is evolving and incomplete. Real change requires rethinking how current sector players are adopting and how they will operate in the future.

  3. Cell & Gene’s 2020 Outlook: Enzyvant, Spark Therapeutics, And Orchard Therapeutics 2/10/2020

    Hopefully you’ve been following Cell & Gene’s 2020 Outlook series, in which many of our Editorial Advisory Board members have provided their data-driven insights for the coming year — and beyond — for the cell and gene sector based on their professional role. Here, Rachelle Jaques, CEO of Enzyvant, Jamie Ring, Head of Patient Advocacy at Spark Therapeutics, and Mark Rothera, President and CEO of Orchard Therapeutics discuss continued momentum in regenerative medicines, demystifying gene therapies, and treatment of disorders of the central nervous system (CNS) respectively.

  4. Cell & Gene Therapy Deals: Emerging Trends To Watch 2/6/2020

    The pace of deal-making in cell and gene therapy is faster and occurring much earlier in the drug development process compared to what has historically been seen for other innovative therapies. To understand how the landscape is evolving, we reviewed more than 30 deals in the sector and compared them to deals that were executed for mAbs between 1999 and 2013.

  5. Cell & Gene’s 2020 Outlook: Stem Cell Research 2/5/2020

    Bruce Bunnell, Ph.D., Director, Center for Stem Cell Research and Regenerative Medicine, Tulane University School of Medicine shares valuable data on three intense areas of stem cell research in 2020 and beyond. Read on as Bunnell explains what’s to come for extracellular vesicles, tissue-on-a-chip and microphysiological systems, and stem cell clinical trials.

  6. Reflections On An Unprecedented Decade In The Cell And Gene Therapy Field 2/4/2020

    Having launched near the beginning of the last decade, Toronto-based CCRM has had a ringside seat to the noteworthy advances in our industry — from 2011 to today. And there have been many.

  7. How Cell And Gene Therapy Is Transforming Healthcare 2/4/2020

    But overcoming challenges is key to meeting CGT promise.

  8. Cell And Gene Therapy Foresight In 2020: 7 Trends To Watch 1/28/2020

    In the 1966 science fiction film Fantastic Voyage, a submarine and crew are miniaturized and injected into the body of a scientist to remove an inoperable blood clot in his brain. We now no longer need to miniaturize ourselves; we can use cells as medicines to treat diseases including cancer. In 2020, we will see clinical evidence of exquisite regulating mechanisms designed in to cells, and the use of cells to deliver drugs themselves in a controlled and directed fashion.

  9. A Harvard Assist For Cell And Gene Bottlenecks 1/27/2020

    Bottlenecks and backlogs in development and production of cell and gene therapies. Harvard to the rescue. Actually, Harvard with partners, such as companies familiar to Outsourced Pharma readers. But what will  go into the the $50 million Center for Advanced Biological Innovation and Manufacturing? We’ve got exclusive details.  

  10. Straight Talk From BeiGene On Quality, Compliance, And Outsourcing 1/26/2020

    It was time within our ruminations on China to speak directly with a quality executive at a drug developer and manufacturer based there. That’s now accomplished via a discussion with Diana Francis, Vice President, Quality & Compliance, BeiGene, Ltd., a biopharma some have dubbed as a potential “Genentech of China.” Francis spoke openly about her role, BeiGene’s global strategy, and outsourcing within and from China.