Skin contains essential stem cell populations and other cell types that are critical for renewing and maintaining its structural integrity and function.1 Of the many types of skin stem cells that have been identified, epidermal stem cells — primarily keratinocyte...
When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. The success...
Background With their potential for long-term or even curative efficacy, gene therapies are of great interest to researchers, clinicians, patients and caregivers alike. But coordinating and conducting a global, multi-center gene therapy trial is a complex, high-risk undertaking. Beyond the...
Gene therapy holds the potential of long-term efficacy or even a cure for diseases with an underlying genetic basis, offering hope to patients with currently incurable diseases. The use of gene therapy for dermatologic conditions is attractive for many reasons:1...
At Premier Research, we attend a lot of events throughout the year – and through these events, we have the opportunity to meet individuals from all sides of the clinical development process. At the recent Global Genes RARE Patient Advocacy...
Risk-Based Monitoring (RBM) makes an impact. The US Food and Drug Administration (FDA) requires that clinical trial sponsors “provide oversight to ensure adequate protection of the rights, welfare, and safety of human subjects and the quality of the data submitted...
Patient advocacy groups have exploded in number and scope in recent years and in many ways are reshaping the drug development landscape, from trial design to recruitment support to participation in the regulatory approval process. Thousands of organizations now advocate...
In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development,[1] addresses...
Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. The approvals of voretigene neparvovec-rzyl (Luxturna) for inherited vision loss, tisagenlecleucel (Kymriah) for lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta) for...
Careful planning is important for all early drug development programs, but it is particularly critical in rare diseases where study populations are limited and precedents for drug development are lacking. Pre-IND meetings with the FDA give sponsors the opportunity to...