Drug intelligence / Profile preview

SRP-1003

Development stage: Phase 2
Lead developer: Sarepta Therapeutics
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: Unknown

Overview

SRP-1003 (formerly known as ARO-DM1) is an investigational RNA interference (RNAi) therapeutic being developed for the treatment of myotonic dystrophy type 1 (DM1). The drug is designed to target and suppress the expression of myotonic dystrophy protein kinase (DMPK) in skeletal muscle. By reducing DMPK levels, SRP-1003 aims to address the underlying molecular cause of DM1, a genetic disorder characterized by progressive muscle wasting and weakness. The therapy utilizes RNA-based technology to specifically silence mutant DMPK mRNA transcripts associated with disease pathology[2][7].

Targets

CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)

Beyond the preview

Go deeper on SRP-1003.

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

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

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 SRP-1003.

Explore the full profile