Expertise: Cell & Gene Therapy 38 results
Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the science of precision medicine. In our previous blog post, we discussed site selection, manufacturing, long-term…
Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. In this blog post, we introduce a case study as a framework for exploring critical study design considerations of…
As scientific knowledge, clinical experience, and acceptance of gene therapy products have evolved, so have the regulatory frameworks for ensuring the safety of these novel treatments. To date, there is no harmonized international standard for regulating gene therapy products; however, the U.S., EU, and Japan have established regulatory frameworks with subtle variations. Understanding how gene…
Rare Disease
The State of Cell Therapies in Dermatology
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 stem cells — are recognized to play the key role in tissue repair and skin…
Rare Disease
The State of Gene Therapy in Dermatology
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 of gene-based therapies relies on three critical elements:1 In our first article, we reviewed gene…
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 Despite factors that make the skin a desirable target for gene therapy, there are still…
The field of gene medicine has a history filled with hope and tragedy, successes and cautionary tales. The world’s first gene therapy was approved in Europe in 2012, only to be taken off the market five years later due to regulatory and commercial barriers. Now, more than six years after the first approval, gene therapy…