Eluforsen

33-nucleotide antisense oligonucleotide designed to restore CFTR function

Phase of research

Phase One Clinical Trial

How it helps

Restore CFTR Function

General information

Eluforsen is an experimental substance used in studies and not yet available as a human pharmaceutical. It is an RNA-based therapeutic approach to cystic fibrosis. Previously known as QR-010, it is an antisense 33-mer RNA oligonucleotide designed to bind specifically to the mRNA region around the F508-encoding deletion and to restore CFTR protein function in the airway epithelium. In relation to cystic fibrosis, our AIM tool found the data that eluforsen can help improve CFTR function in patients with F508del-CFTR mutation.

Synonyms

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Marketed as

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Dietary sources

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Structure image not available

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Drug-Mutation Relation

results for D1152H / F508del

See all data on Eluforsen

Treats

Mutation	Link	Tested on	Impact factor	Notes
F508del/F508del	Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis.	humans	4.29

Does not treat

Mutation	Link	Tested on	Impact factor	Notes