ACE-031
ACE-031

A single-component research material supplied for controlled research environments. ACE-031 (1 mg) is a recombinant fusion protein designed for research focused on activin receptor signaling pathways, muscle growth regulation, and myostatin inhibition in model systems.

• ACE-031
• Appearance: Lyophilized powder in a sealed research vial

$180.00

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These compounds are intended for in-vitro research use only and are not approved for human or veterinary use, consumption, or therapeutic application.

Research Focus
Research Focus
  • Characterization of ACE-031 activity in activin receptor type IIB signaling studies
  • Investigation of myostatin pathway inhibition in skeletal muscle research models
  • Development of in vitro and in vivo assays for muscle mass regulation studies
  • Stability and solubility profiling under controlled laboratory storage conditions
ACE-031 is a myostatin inhibitor a potential treatment for muscle-wasting diseases such as Duchenne muscular dystrophy (DMD) and other conditions involving muscle loss. Myostatin (also known as growth differentiation factor 8 or GDF-8) is a protein that regulates muscle growth by limiting it. By inhibiting myostatin, ACE-031 promotes muscle growth and strength.
Mechanism of Action
  • Myostatin Inhibition: Myostatin acts as a negative regulator of muscle growth. It binds to receptors (activin receptor type IIB, or ActRIIB) on muscle cells, signaling them to limit growth.
    • ACE-031 is a recombinant fusion protein that binds to myostatin and related proteins, preventing them from interacting with their receptors.
    • This blocks the inhibitory signals, allowing muscles to grow larger and stronger.
  • Additional Benefits: ACE-031 also neutralizes other proteins in the TGF-β (transforming growth factor-beta) family that limit muscle growth, further amplifying its effects on increasing muscle mass.
Potential Benefits of ACE-031
  1. Increased Muscle Mass:
    • In preclinical and early clinical studies, ACE-031 significantly increased lean muscle mass by promoting muscle fiber growth. (Study)
  2. Improved Muscle Strength:
    • By blocking myostatin, ACE-031 enhanced muscle strength in animal models and showed potential for improving functional outcomes in patients with muscle-wasting diseases. (ClinicalTrials.gov NCT01099761)
  3. Therapeutic Potential for Muscle-Wasting Diseases:
    • Conditions such as Duchenne muscular dystrophy (DMD), sarcopenia, and cachexia could benefit from ACE-031 by slowing or reversing muscle loss.
Research and Clinical Development

Preclinical Studies

  • Preclinical animal models demonstrated robust increases in muscle mass and strength with ACE-031.
  • Myostatin inhibition also improved metabolic health markers, including reduced fat mass and improved insulin sensitivity. (Journal of Muscle Research)
Phase 1 Clinical Trial
  • Study Design: A randomized, placebo-controlled trial tested ACE-031 in healthy volunteers.
  • Findings:
    • Significant increases in lean body mass were observed after short-term treatment.
    • No serious adverse events were reported at lower doses.
Phase 2 Clinical Trial (NCT01099761)
  • Population: Boys with Duchenne muscular dystrophy (DMD).
  • Primary Goals: Assess safety, tolerability, and efficacy in improving muscle mass and function.
  • Results:
    • Positive Effects: Increased lean muscle mass and functional improvements.
    • Safety Concerns: Side effects included nosebleeds, gum bleeding, and blood vessel changes, possibly linked to the drug’s impact on the vascular system.
    • The trial was terminated early due to these safety concerns and the need for further investigation.
Discontinuation
  • In 2011, Acceleron Pharma stopped the development of ACE-031 due to safety concerns related to vascular side effects. (Official Statement)
Side Effects and Limitations
  1. Observed Side Effects:
    • Nosebleeds (epistaxis).
    • Gum bleeding.
    • Telangiectasia (small dilated blood vessels).
    • Potential concerns about long-term off-target effects, such as vascular abnormalities.
  2. Limitations:
    • Safety issues prevented further clinical development.
    • Lack of data on long-term efficacy and safety in humans.
Comparison to Other Myostatin Inhibitors
Several other myostatin inhibitors are in development or have been studied, with ACE-031 being one of the most well-known early candidates.
  • Follistatin-based Therapies:
    • Follistatin binds to and neutralizes myostatin. Therapies like RK-35 have similar mechanisms but may avoid some of the safety concerns seen with ACE-031.
  • Other Myostatin-targeting Antibodies:
    • Stamulumab (MYO-029): Another myostatin inhibitor studied for muscle-wasting diseases, but it also showed limited efficacy in trials. (Study)
  • Gene Therapies:
    • Emerging approaches focus on gene editing or gene silencing to inhibit myostatin expression directly, potentially offering more precise results with fewer side effects.

Current Status and Future Potential

Despite its discontinuation, ACE-031 paved the way for research into myostatin inhibition as a therapeutic approach. Lessons learned from ACE-031 are helping to design safer and more effective myostatin blockers.
  • Future Directions:
    • Improved specificity for myostatin (avoiding off-target effects).
    • Combination therapies to address muscle-wasting diseases more comprehensively.
    • Gene-editing techniques like CRISPR-Cas9 to target myostatin directly.
  • Ongoing Research: Other companies continue to explore similar mechanisms with newer, safer candidates in both preclinical and clinical trials.

References and Studies

  1. ACE-031 Preclinical and Clinical Overview:
  2. Clinical Trial in DMD (NCT01099761):
  3. Myostatin Biology and Therapeutic Potential:
  4. Official Statement on ACE-031 Discontinuation:
  5. Comparison to MYO-029:

All research peptides offered are manufactured using a lyophilization (freeze-drying) process,
which is designed to enhance stability during storage and transport. When stored appropriately,
lyophilized materials are generally stable at room temperature for extended periods prior to reconstitution.

Lyophilized (Unreconstituted) Material

  • Store vials away from heat, moisture, and direct light.
  • For short-term storage, refrigeration at approximately 4°C (39°F) is suitable.
  • For long-term storage (months to years), vials may be stored in a freezer at approximately −80°C (−112°F).
  • Freezing is the preferred method for preserving stability over extended storage durations.

Reconstituted Material

  • Once reconstituted using appropriate laboratory-grade solvents, materials should be stored refrigerated.
  • Under refrigerated conditions, reconstituted material is commonly considered stable for up to 30 days,
    depending on solvent choice, concentration, handling, and experimental conditions.
  • Avoid repeated freeze–thaw cycles, as these may impact material integrity.

About Lyophilization

Lyophilization is a dehydration process in which compounds are frozen and exposed to reduced pressure,
allowing ice to sublimate directly from solid to vapor. This process results in a dry, crystalline structure
that enhances stability prior to reconstitution.

⚠️ Important Notice: These materials are intended for research and laboratory use only.
Not for human or veterinary use, consumption, or application.

99%+ Purity

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Essential information about our research-grade peptides and quality standards.

What are research peptides?

Peptides are short chains of amino acids linked by peptide bonds, typically consisting of 2-50 amino acids. They serve as the building blocks of proteins and play crucial roles in biological processes.

Research peptides are synthesized versions of naturally occurring peptides, manufactured to precise specifications for use in scientific studies.

Key characteristics: High purity (≥99%), lyophilized form for stability, accompanied by Certificates of Analysis (COA), and manufactured under strict quality control protocols.

Peptide purity refers to the percentage of the target peptide present in a sample, measured via High-Performance Liquid Chromatography (HPLC).

Our Standard: All our research peptides maintain ≥99% purity, verified by third-party laboratory testing and documented in our Certificates of Analysis.

Higher purity is critical for research because impurities can interfere with experimental results and compromise the reproducibility of studies.

We maintain rigorous quality control through a multi-step verification process:

  • HPLC Analysis: Every batch tested to verify ≥99% purity
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