Clarion Blog

Last week, Bobby Moy, managing director of Clarion, a Lumanity business, attended the 26^th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, California. Here, he shares four key takeaways from the conference:

1. FDA willing to take a flexible approach in rare, severe disorders – The recent 8-6 Sarepta adcomm  was a hot topic, with attendees mixed on whether the FDA would override the adcomm vote. The FDA noted that they are looking to build flexibility into the system, but also hinted that the recent Makena withdrawal may increase the FDA’s willingness to allow products on market if alternatives do not exist/ are not forthcoming.
2. Significant expansion of new approaches to improve delivery of genetic information to cells –Multiple groups are developing innovative methods to improve delivery, target more organs, and are making progress in redosing of gene therapies.
3. The ability to quickly port nucleic acid-based approaches is allowing a diverse set of groups to target smaller population sets – Companies, academics, and not-for-profit organizations are focused on targeting smaller sets of patients down to and including = 1 disorders.      
4. Proactive efforts to enhance diversity in trials – Companies and physicians are actively working to diversify trials for cell and gene therapies by sharing best practices and practical approaches for making trials easier for patients and more welcoming to diverse populations.

Are you a company that is looking for guidance in the rare disorder and/or gene therapy space? Clarion is a life sciences strategy and organizational consultancy that works together with its clients to envision, craft, and enable growth through scientific and commercial innovation and leadership. Connect with us to learn how we can help you today. For more information, visit www.clarionhealthcare.com.