With no approved therapies globally for Stargardt's, competition in the indication is increasing in Japan, where Kubota Vision is seeking for conditional approval for emixustat, while Belite Bio’s contender tinlarebant has received Sakigake designation as a local Phase III program continues.

The recent reimbursement and launch of Zolgensma showed Japan willing and able to approve and pay for gene therapies, and others including from Otsuka/Takara Bio are moving forward under a supportive regulatory framework.

French biotech Horama has built-up its executive team and has two potential gene therapies for retinitis pigmentosa nearing the clinic.

Pipeline Watch has been updated to bring greater depth and detail to a potential therapeutic product’s progress through the R&D pipeline. The table brings you a snapshot of selected late-stage clinical trial events in the pharma and biotech industries.