ASO-focused biotech SynaptixBio is pushing the UK’s MHRA to strengthen incentives inside its draft rare disease therapy framework. Dan Williams, the company’s CEO, argues that the current structure—while allowing routes such as investigational marketing authorization—does not replicate the early financial pull that tools like the FDA’s Priority Review Voucher create for developers and investors. Williams points to the lack of direct financial benefits for holding investigational status, warning that without an incentive element, companies may still prioritize the US authorization pathway. The remarks also highlight how capital availability remains the critical bottleneck for earlier-stage rare disease programs. The MHRA’s effort is designed to support therapies for very small patient populations, but the industry call is for mechanisms that reduce economic risk during pre-registration development.