Ractigen Garners Orphan Drug Designation for saRNA Therapy RAG-18 - CGTLive®
Ractigen Therapeutics' RAG-18, an investigational saRNA therapy, received FDA orphan drug designation for treating DMD and BMD by activating the UTRN gene to increase utrophin production. This marks the first saRNA therapy to receive both orphan drug and rare pediatric disease designations from the FDA. RAG-18 aims to provide clinical benefit by upregulating utrophin, structurally similar to dystrophin, via RNA activation. Ractigen's pipeline includes RAG-01, which is in a phase 1 trial for NMIBC, targeting a tumor suppressor gene.
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Ractigen Therapeutics' RAG-18, an investigational saRNA therapy, received FDA orphan drug designation for treating DMD and BMD by activating the UTRN gene to increase utrophin production. This marks the first saRNA therapy to receive both orphan drug and rare pediatric disease designations from the FDA. RAG-18 aims to provide clinical benefit by upregulating utrophin, structurally similar to dystrophin, via RNA activation. Ractigen's pipeline includes RAG-01, which is in a phase 1 trial for NMIBC, targeting a tumor suppressor gene.
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