Drug intelligence / Profile preview

EXC 001

Development stage
Phase 2
Lead developer
Pfizer
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intradermal
01

Overview

EXC 001 is a second-generation antisense oligonucleotide drug designed to inhibit the expression of connective tissue growth factor (CTGF), a protein activated during skin scarring and fibrosis. By binding to CTGF mRNA, EXC 001 suppresses CTGF protein production, thereby reducing fibrotic activity and scar formation. The drug was initially developed by Excaliard Pharmaceuticals in collaboration with Isis Pharmaceuticals (now Ionis Pharmaceuticals) and later acquired by Pfizer. Its primary indication was for the reduction of hypertrophic skin scars following surgery or injury. Clinical studies demonstrated that intradermal injection of EXC 001 significantly reduced scar severity compared to placebo, supporting its antifibrotic mechanism targeting CTGF[1][3][5][6].

Other names
EXC-001EXC001EXC 001PF-6473871PF6473871PF 6473871
02

Targets

CTGF (Connective tissue growth factor)

Beyond the preview

Go deeper on EXC 001.

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 EXC 001.

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