Drug intelligence / Profile preview

Muc5b siRNA

Development stage
Preclinical
Lead developer
Alnylam Pharmaceuticals
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Inhalation
01

Overview

Muc5b siRNA is a small interfering RNA (siRNA) therapeutic candidate designed to silence the expression of the MUC5B gene, which encodes Mucin 5B, a key gel-forming mucin in the respiratory tract. In conditions such as Alpha-1-antitrypsin deficiency (AATD), the overproduction of Muc5b—driven by neutrophil elastase activity and environmental factors like cigarette smoke—leads to impaired mucociliary transport and small airway obstruction. By utilizing the RNA interference (RNAi) pathway to degrade MUC5B mRNA, this siRNA reduces the synthesis of Mucin 5B protein, thereby restoring mucociliary clearance and potentially mitigating airway disease. Research presented by the University of Alabama at Birmingham highlights its potential as a targeted therapy for AATD-associated lung dysfunction.

Other names
MUC5B siRNAMUC-5B siRNAMUC 5B siRNAsiRNA Muc5b
02

Targets

MUC5B (Mucin-5B)

Beyond the preview

Go deeper on Muc5b siRNA.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Full profile access

Follow clinical development from study design and recruitment through results.

  • Trial phase
  • Status
  • Readouts

Indications & development

Full profile access

Explore development by indication, patient population, and geography.

  • Indications
  • Development status
  • Countries

Licensing & deals

Full profile access

Trace asset ownership, licensing agreements, and commercial partnerships.

  • Partners
  • Deal terms
  • Milestones

Patents & exclusivity

Full profile access

Explore the patent landscape and regulatory exclusivity around an asset.

  • Patents
  • Expiration dates
  • Exclusivity

Competitive landscape

Full profile access

Compare development programs by target, modality, and indication.

  • Competing assets
  • Targets
  • Development stage

Research & analysis

Full profile access

Connect source evidence and development news to your research questions.

  • Publications
  • News
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Muc5b siRNA.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call