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 part of a three-part blog series on gene therapy development, we provide tips on operationalizing...
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 they are capable of — and committed to — fulfilling all protocol requirements. In...
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 that generate robust evidence with appropriate safety oversight. In addition, gene therapy trials are faced...
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 technology become more sophisticated, gene therapy development is accelerating at an unprecedented pace. According to...
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 advancements included in PDUFA VII are driven in large part by an increase in...
Advanced therapy medicinal product (ATMP) development is on the rise. According to the American Society of Gene + Cell Therapy, there were 1,745 gene therapies in development in May 2021, 70% of which were in preclinical studies, and more than 1,300 of these candidates were in development for oncology, the most active therapeutic area. Given...
Advanced therapies – including gene-, cell-, and tissue-based products – offer groundbreaking new opportunities for the treatment of disease. As of the end of 2020, there were 1,085 active developers of these therapies and 152 ongoing Phase 3 trials worldwide. According to the Alliance for Regenerative Medicine, regulatory decisions are expected on a record eight...
According to the Alliance for Regenerative Medicine, there were 1,085 active advanced therapy medicinal product (ATMP) developers and more than 150 phase III trials underway at the end of 2020. These numbers are not small, and they make it clear that ATMPs have much to offer in helping us overcome as-yet undefeated diseases.ATMPs are different...
Since the first gene therapy trial in 1990, the regulatory landscape has shifted from broad, loosely defined guidance with little variation between different cell and gene therapies to a more robust collection of guidelines and directives focused on testing and manufacturing considerations for these products in specific disease states and therapeutic settings. This article will...
In recent years, the gene therapy regulatory landscape has evolved from a broad, general guidance to a narrower focus on testing and manufacturing considerations in specific disease states. This article explores how factors such as environmental risk assessment and biosafety can impact gene therapy clinical study start-up as well as the continuing evolution of guidances...