I recently had an informative conversation with Dr. Jiwen Zhang, VP, Regulatory Affairs at Passage Bio to gain a better understanding of just how rapidly regulators must move to keep pace with the sector’s ever-evolving regulation landscape and what industry professionals should do to meet requirements. She shared with me the critical action items cell and gene therapy professionals should heed to keep pace with the sector’s ever-evolving regulations, which will be detailed in an upcoming article.
Part one of our conversation covered how regulators in the cell and gene sector are actively developing guidance documents that are intended to help product development. Much work is being done at Passage Bio, a Philadelphia-based a fully integrated genetic medicines company that is developing a portfolio of six life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. The company has a research, collaboration, and license agreement with the University of Pennsylvania and its Gene Therapy Program as well as the Penn Orphan Disease Center. Currently at Passage Bio, two out of three development programs are for pediatric patients,” explains Zhang.
“One program that is targeted to initiate clinical trials in 2020 is for infantile type of GM1 gangliosidosis, which is the most common and severe form of the disease, typically presenting with gait abnormalities by four months of age and developmental regression by six months. Many infantile GM1 patients experience rapid regression with mortality by approximately two years of age. However, it is important to keep in mind that even for approved products, long-term data are still being collected in terms of durability of treatment effect and potential risks. Therefore, there are regulatory requirements on long-term follow-up. Time and data will tell.”
In order to keep pace with the evolving regulation landscape, Zhang suggests sector professionals utilize other innovative approaches for efficient product development such as the use of real-world data (RWD) and real-world evidence (RWE). As cell and gene therapies are geared towards personalized medicines, other enabling technologies such as genomic sequencing, new diagnostic technologies should be carefully evaluated, appropriately incorporated into cell and gene product development to increase the likely success of the treatment and reduce risks. Development and adoption of standards will also help advancement. She also explains that stakeholder engagement and collaborations are critical for the success of the sector. “Alliance groups, public-private partnerships, and consortia on various initiatives are all making progress and contributing to the sector’s growth,” states Zhang. “One stakeholder segment for active industry engagement are patient advocacy groups that are integral to enable therapy development, patient care and management, and ultimately patient access to innovative treatment for unmet needs.”
Zhang stresses there are non-negotiable key concepts regarding the regulation landscape that sector professionals should adopt and practice. She explains that the key focus areas include gene editing; new manufacturing and analytical technologies; new use of data based on RWE; and personalized medicine that other sectors such as diagnostics and AI are progressing and impacting cell and gene therapy development and adoption.”
Stay tuned for the full-length article on the challenges and short-term outlook facing the cell and gene sector’s regulation landscape. In the meantime, email me with your insight about global regulatory policies for cell and gene therapies.