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Endpoints·1h ago·1 min read
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Silence Therapeutics moves siRNA drug for polycythemia vera to Phase 3 after Phase 2 success

Aug 10, 2026·read at Endpoints ↗Positive readoutPositive

Silence Therapeutics announced that its siRNA candidate divesiran achieved its primary endpoint in a Phase 2 study in patients with polycythemia vera, a rare blood cancer.

The trial results showed the drug reduced key disease markers and was well tolerated, meeting the pre-specified efficacy criteria.

Based on the data, the company plans to initiate a pivotal Phase 3 program later this year, aiming for broader enrollment and regulatory filing.

The advancement highlights the potential of gene-silencing approaches for hematologic disorders and may attract further interest from investors and partners.

source

This writeup was produced by pharmadog from original reporting by Endpoints.

Original headline: “Silence spells out Phase 2 success for gene-silencing drug in rare blood cancer”

read at Endpoints ↗
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companies & drugs in this story

companies
Ultragenyx Pharmaceutical Inc.4Silence Therapeutics plcVera Therapeutics, Inc.
drugs
divesiran
topics
Hematology4Rare Disease4OncologysiRNA

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