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Rapirosiran · AD-288996, ALN-288996

Oligonucleotide (siRNA) · Gene silencing: HSD17B13 · developed by ALNYLAM PHARMACEUTICALS

Phase 1 Most advanced trial (none active)

ALNY Developer

1 Registered trials

What is Rapirosiran?

Rapirosiran is a GalNAc-conjugated small-interfering RNA that targets liver-expressed HSD17B13 mRNA. The GalNAc moiety binds asialoglycoprotein receptors on hepatocytes, and the siRNA silences HSD17B13 mRNA to reduce HSD17B13 protein expression. It is studied in nonalcoholic steatohepatitis.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: AD-288996, ALN-288996, ALN-HSD
Drug type: Oligonucleotide (siRNA)
Targets: HSD17B13
Mechanism: Gene silencing: HSD17B13
Pathways: liver inflammation, progression from steatosis to steatohepatitis
Therapy area: Gastroenterology
Developer: ALNYLAM PHARMACEUTICALS (ALNY)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1
UNII: F3NU7CDG3E

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT04565717	Phase 1	Nonalcoholic Steatohepatitis	Terminated	ALNY

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

NASHNonalcoholic Steatohepatitis

More from ALNYLAM PHARMACEUTICALS

Givosiran FDA approved Lumasiran FDA approved Patisiran FDA approved Vutrisiran FDA approved ALN-2232 Phase 1/2 ALN-4285 Phase 1 ALN-4324 Phase 2 ALN-4915 Phase 1 ALN-5288 Phase 1 ALN-6222 Phase 1 ALN-6400 Phase 1/2 ALN-AGT01 RVR Phase 2

Sources

Updated Oct 11, 2026. Something wrong or missing? Report it on the ALNY page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z