With hundreds of companies and clinical trials investigating CAR T-cell therapies, there exist opportunities to expand the applications of—and broaden access to—these novel therapeutics to address unmet medical needs.
Expertise: Cell & Gene Therapy 62 results
Cell & Gene Therapy
Bringing Patient Centricity to the Forefront in CAR-T Trials
There is growing recognition, among both industry and regulators, that patients are experts in their own illnesses, that they are increasingly well-educated about treatments, and that they want to have a say in how investigative therapies are studied. The concept...
Cell and Gene Therapy Expert Dr. Kenneth Ndugga-Kabuye to Lead Clinical Strategy With Collaboration From Operations, CMC and Regulatory Experts MORRISVILLE, N.C., April 17, 2023-- Premier Research announced today the addition of Kenneth Ndugga-Kabuye, M.D., FACMG, as VP, Cell &...
Cell & Gene Therapy
5 Tips for Operationalizing Gene Therapy Studies
Gene therapy clinical trials are logistically complex studies that require highly qualified sites and engaged patients for success. The ability to select the right sites and patients can be the difference between product failure and market approval. In this final...
Cell & Gene Therapy
Designing Robust Gene Therapy Studies
To develop a successful gene therapy, sponsors must conduct research studies that balance the need for safety oversight and robust clinical evidence with the challenges of finding the right patients, pairing them with qualified sites, providing informed consent, and confirming...
Cell & Gene Therapy
Pursuing the Promise of Gene Therapy: Pathways to Successful Clinical Trials
The promise of gene therapy is to provide transformative treatments that meaningfully improve quality of life for patients, many of whom are currently living with debilitating diseases. To bring these treatments to market, sponsors are tasked with conducting clinical trials...
Insights into the human genome have created myriad therapeutic opportunities for previously untreatable diseases. Gene therapy offers promise for addressing unmet medical needs across therapeutic areas and even the potential for curing certain genetically based conditions. As research tools and...
Cell & Gene Therapy
PDUFA VII: Implications for Developers of Cell and Gene Therapies
One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of...
The gene therapy era can be said to have begun in 1990, when the first gene therapy clinical trial took place. Some 3,000 clinical trials have followed that first study, a resounding affirmation of innovators’ increasing recognition of gene therapy’s...
Immunotherapy has led to substantial advances in cancer therapy in recent years. Still, unpredictable response rates and immune-related adverse events have hampered the widespread use of immune checkpoint therapy to treat cancers. To tackle these challenges, sponsors are increasingly looking...