ACT Brief: Asia Pacific Site Perceptions, Community Access Gap, and VeonGen Gene Therapy Alignment

This is the Applied Clinical Trials Brief—your fast track to the latest insights shaping clinical operations and drug development.

• In part one of a video interview, James Cheong, senior vice president of Asia Pacific at Precision for Medicine, explains how regulatory reform and inspection data contradict the view that APAC sites are slower or lower quality. He says sponsors need to update their assumptions about these sites.
• In a new Q&A, Kent Thoelke, CEO of Paradigm Health, discussed why participation gaps in community and rural settings come down to both patient cost barriers and site infrastructure. He described the barriers as running in two directions at once, with patients who cannot absorb the financial cost of participating and sites that cannot absorb the administrative weight of legacy workflows. He also covered what policy and technology changes would make clinical research a real care option for more patients.
• VeonGen reached FDA alignment on the pivotal Phase III design and manufacturing strategy for its gene therapy for an inherited eye disease that causes progressive vision loss. In a Phase I/II study of 15 patients, all 13 evaluable patients improved, and 11 gained at least 5 letters on a standard vision chart through month 12. The company did not disclose the planned Phase III primary endpoint, study duration, or enrollment.

That’s all for today’s ACT Brief. Join us next week for more updates shaping clinical operations and drug development. Thanks for listening.