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RO7021707
- Development stage
- Preclinical
- Lead developer
- Roche
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
RO7021707 (also referred to as RIS-L or a risdiplam-like compound) is a small-molecule splicing modifier developed by Roche that targets survival of motor neuron-2 (SMN2) pre-mRNA. It is designed to promote the inclusion of exon 7 during alternative splicing, thereby increasing the production of full-length, functional SMN protein. RO7021707 is investigated preclinically as a therapeutic candidate for Spinal Muscular Atrophy (SMA), with research focusing on its potential in early neurodevelopmental windows and prenatal intervention strategies.
- Other names
- RIS-Lrisdiplam analoguerisdiplam-like compound
02
Targets
SMN2 (Survival motor neuron 2)
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