Tag: Rare Disease

Clinical Research: Phase 1 - Phase 4

Achieving the Promise of Gene Therapy: New Pathways to Overcome Patient Enrollment and Safety Challenges, Upcoming Webinar

TORONTO (PRWEB) SEPTEMBER 15, 2020 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. This webinar will examine the evolving...

Clinical Research: Phase 1 - Phase 4

Novel Trial Approaches for Rare Cancer Patients, Upcoming Webinar

TORONTO (PRWEB) AUGUST 4, 2020 Join Rupa Doshi, Ph.D., Executive Director –Program Strategy, Oncology, Premier Research and Sameena Sharif, Ph.D., Senior Vice President, Product Strategy, Premier Research in a live webinar on Monday, August 24, 2020 at 11am EDT (4pm BST/UK). Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no...

Consulting

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 A well-defined disease geneA therapeutic geneAn efficient...

Clinical Research: Phase 1 - Phase 4

Premier Insight 272: Delivering the Global Expertise Required to Coordinate a Pediatric Gene Therapy Trial

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 usual protocols and procedures required to ensure patient safety and data quality, gene therapy studies must...

Consulting

BIO International: Discover the Latest in Innovative Rare Disease Trial Design

DURHAM, N.C., June 5, 2019 — Premier Research is sponsoring the Orphan & Rare Disease session track at this year’s BIO International Convention, June 3-6 in Philadelphia. Our own Angi Robinson, vice president for clinical development services in rare diseases and pediatrics, will participate in the panel discussion Rare Finds: Innovative Clinical Trial Design for...

Medical and Regulatory Affairs

The FDA’s New Guidance on Natural History Studies in Rare Diseases: What You Need to Know

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 one of the major challenges sponsors encounter when developing therapies for rare disease: the lack...

Consulting

Regulatory Oversight on Gene Therapy in the U.S. and EU

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 lymphoma have ushered in a new era of therapeutics. With the rapid evolution of the...

Clinical Research: Phase 1 - Phase 4

From Psoriasis to Behçet’s: The Evolution of Biologics in Dermatology

Over the last two decades, biologics have improved the management of patients with psoriasis and advanced melanoma, many of whom either did not respond to traditional treatments or experienced severe side effects from them. With their success in deadly forms of melanoma and more common chronic inflammatory conditions such as psoriasis, drug developers are now targeting rarer...

Consulting

Ashanthi DeSilva’s Story: A Look Back at the First Gene Therapy Trial

In 1976, pop culture icon John Travolta starred in the made-for-television movie The Boy in the Plastic Bubble about a young man with a compromised immune system. The premise of the film is that Travolta’s character has to decide between living in isolation behind a protective bubble or dying engaged with the physical world. Hollywood...