INSIGHTS ON CELL & GENE CLINICAL TRIALS

  • Designing Gene Therapy Trials For Access: What Payers & Health Systems Want To See
    Designing Gene Therapy Trials For Access: What Payers & Health Systems Want To See

    Gene therapy may be the next revolution in healthcare. The opportunity to cure diseases through the modification of the genome was once considered science fiction, but no longer. The United States has seen a few approvals in this area, including the approval of voretigene neparvovec for a rare form of blindness. In addition, CAR-T agents, two of which are available, have demonstrated alternative ways to treat disease by manipulating genes of different cells.

  • Digital Platforms For Patient Engagement In Clinical Trials — Trends & Outlook
    Digital Platforms For Patient Engagement In Clinical Trials — Trends & Outlook

    Pharmaceutical companies have three targeted users: physicians, pharmacists, and patients. Various digital platforms are used to create a good customer experience for educating these end users. The core focus is on patients driving patient care, education, and adherence.

  • Take Cell And Gene Therapy Supply Challenges By The Horn
    Take Cell And Gene Therapy Supply Challenges By The Horn

    While traditional biotechnology focuses on material inventory, cell therapy products must account for the entirety of a patient’s journey in order to develop a robust supply strategy. 

  • Managing Medicine’s Most Complex Patient Journey
    Managing Medicine’s Most Complex Patient Journey

    In today’s breakthrough medicines, personalization changes everything. Patients are used to receiving medications. Now, in the era of cell and gene therapies, patients are actually providing the key components to create them.

  • Beyond The IND: Regulatory And Clinical Planning For Cell And Gene Therapies
    Beyond The IND: Regulatory And Clinical Planning For Cell And Gene Therapies

    A quick and nimble approach is often sought after in the cell and gene therapy product space due to the likely one-time or limited patient dosing and/or rare disease conditions limiting the number of available patients, which reduces the number of clinical studies.

  • A Patient’s Journey To Join A CAR-T Cell Therapy Trial
    A Patient’s Journey To Join A CAR-T Cell Therapy Trial

    Nicole Gularte is currently battling cancer for the 8th time. The last time she had to do so, it seemed a CAR-T cell therapy trial might be the solution. This article is about her long battle with cancer, the struggle to get into a CAR-T cell trial, and the ups and downs she has faced along the way. Her story is an inspiration to others.

  • State Of The Cell And Gene Therapy Sector — Clinical, Financial, And Regulatory Perspectives
    State Of The Cell And Gene Therapy Sector — Clinical, Financial, And Regulatory Perspectives

    The regenerative medicine sector is at a remarkable moment. Transformative products are now on the market and accessible to greater numbers of patients every day. Dozens of additional therapies are in late stage studies. The regulatory and policy environment has evolved rapidly alongside the science, enabling a surge of incoming innovation.

  • 4 Barriers To Cell And Gene Therapy Development For Rare Diseases — And How To Overcome Them
    4 Barriers To Cell And Gene Therapy Development For Rare Diseases — And How To Overcome Them

    A number of challenges are hindering efforts to develop cell and gene therapies to treat rare diseases. While these challenges may seem common across other drug markets, in the case of rare diseases, they are exacerbated by limited patient populations

  • Clinical Trial Networks: Overcoming The Complexities Of Regenerative Medicine Studies
    Clinical Trial Networks: Overcoming The Complexities Of Regenerative Medicine Studies

    The FDA’s RMAT and breakthrough designations have created a streamlined environment for product sponsors. To effectively leverage this regulatory policy environment, sponsors must conduct high-quality clinical trials that are often operationally complex. Clinical trial networks, capable of managing the array of regenerative medicine technologies, are well suited to manage this complexity.

  • 4 Future Scenarios For Cell And Gene Therapy Adoption
    4 Future Scenarios For Cell And Gene Therapy Adoption

    Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?

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