Pathogenic variants of fused in sarcoma (FUS) cause amyotrophic lateral sclerosis (FUS-ALS), with evidence of gain of function. Jacifusen is an antisense oligonucleotide targeting FUS pre-mRNA, previously shown to delay neurodegeneration in a mouse model and potentially slow functional decline in a first-in-human study. Here, we sought to further evaluate use of jacifusen as a treatment for FUS-ALS.

Trachoma, the leading infectious cause of blindness worldwide, is one of several neglected tropical diseases targeted by WHO for elimination by 2030. The disease starts in childhood with repeated episodes of conjunctival Chlamydia trachomatis infection. This infection is associated with recurrent conjunctivitis (active trachoma), which, if left untreated, leads to cicatricial trachoma characterised by scarring of the conjunctiva, and potentially in-turned eyelashes (trachomatous trichiasis) in later life. Trachoma mainly affects the poorest and most rural communities; these populations typically have limited access to water and hygiene facilities. Blinding complications are most common in women who, in many cultures, act as caregivers from a young age for infected children. To eliminate trachoma as a public health problem, programmes implement a package of interventions known as SAFE; namely, surgery to treat trachomatous trichiasis, antibiotic mass drug administration to treat infection, facial cleanliness, and environmental improvement to limit transmission. The SAFE strategy has brought considerable success in the last two decades. As of December, 2024, 21 countries have eliminated the disease, and several others are on track to eliminate it soon. However, persistent and recrudescent active trachoma in some populations might challenge the success of the 2030 global elimination target. In such settings, novel, or more intensive, approaches must be promptly developed, tested, and scaled up to accelerate elimination.