Silence Therapeutics (SLN) Morgan Stanley 24th Annual Global Healthcare Conference summary

Company overview and technology

• Focuses on small interfering RNA (siRNA) compounds using a proprietary GalNAc oligonucleotide delivery platform targeting the liver.

• Holds a robust intellectual property portfolio covering sequences, chemistry modifications, and delivery linkers.

• Transitioned from a research to a clinical-stage company with three products in the market, currently prioritizing divesiran.

Lead program: divesiran for polycythemia vera

• Divesiran targets polycythemia vera, a rare blood disorder driven by JAK2 mutations, aiming to maintain hematocrit below 45% to reduce cardiovascular risks.

• Current treatments include phlebotomy and cytoreductive agents, but many patients fail to maintain target hematocrit, highlighting unmet needs.

• Market opportunity estimated at $1–2 billion, with potential for expansion due to broad labeling of similar therapies.

Mechanism of action and differentiation

• Divesiran targets TMPRSS6, elevating endogenous hepcidin to restrict iron availability, reducing red blood cell production.

• Offers durable, infrequent dosing (every 12 weeks) compared to weekly or monthly injections for competitors.

• Consistent hepcidin elevation and quarterly dosing may improve compliance and fit standard patient management routines.