This entry is not a peptide · It is included because this compound is commonly discussed alongside peptide research.
AAV1-FS344 gene therapy
Also known as: AAV1.CMV.huFollistatin344, AAV1.CMV.huFS344
At a glance
PeptiGuide dataset · reviewed Aug 2026
What it is
Myostatin-binding protein studied in muscle and body-composition research
Status
Clinical Trials
Typical dose
2.4 × 10^12 vector genomes/kg total dose in NCT02354781; investigational protocol, not a dosing recommendation
Researched for
Muscle growth, myostatin inhibition, body composition
Trial progress
- PrePreclinical
- IPhase I
- IIPhase II
- IIIPhase III
- IVPhase IV
- FDAFDA approved
Phase II - Emerging human evidence with important limitations
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In plain language
- Investigational AAV1 gene-transfer modality encoding human follistatin-344.
- Administered intramuscularly in the registered DMD study.
- The protocol records a total dose of 2.4 × 10^12 vector genomes/kg and follow-up through 24 months.
- Not FDA approved; the study is safety-focused and remains investigational.
How it's thought to work
The AAV1 vector carries a human FS344 expression cassette into skeletal muscle, where the transgene is intended to produce follistatin and reduce activin/myostatin-family signaling. This is gene transfer, not direct administration of a scalar .
What the research shows
Where the research stands
NCT02354781 is a small safety-focused study of intramuscular AAV1.CMV.huFollistatin344 gene transfer in Duchenne muscular dystrophy. The protocol distributes a total dose of 2.4 × 10^12 vector genomes/kg across selected muscles and follows participants for 24 months. Efficacy measures are secondary; the record does not establish approved treatment efficacy.
What it shows
✓ Human studies
Emerging human evidence with important limitations
△ Limitations
Study protocols describe research and are not dosing recommendations.
Bottom line: Investigational intramuscular AAV1 follistatin-344 gene-transfer program Not FDA Approved
Research dosing
Study-specific protocolsHuman trials under way - protocols are study-specific
Possible side effects
Not everyone experiences these. Individual responses vary with dose, duration, and personal factors.
- Investigational gene-therapy safety risks require protocol monitoring
Not everyone experiences side effects, and severity can vary by context, purity, route, and individual health factors.
Sources
Frequently asked questions
One open dataset
Every fact on this page - doses, side effects, references - comes from the same PeptiGuide dataset that powers the Tracker, Compare, and the Lab.
Browse the datasetEducational resource, not medical advice. Talk to your clinician before acting on anything here.
Community discussion
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