Meta Information

ID:follistatin-gene-therapy

Name:Follistatin Gene Therapy

Schema Version:1.5

Alternate Names

• FST Gene Therapy

Active Compound

Follistatin

Intervention Type

procedure

Delivery

Route:

intramuscular_injection

Scope:

• local
• systemic

Regulatory Status

• us:investigational_new_drug

Indication

Focus:

• disease_treatment
• healthspan

Description:

Aims to increase muscle mass and strength by inhibiting myostatin. Investigated for muscle-wasting diseases (e.g., muscular dystrophy) and potential healthspan applications like sarcopenia.

Typical Protocol

• A single administration of an adeno-associated virus (AAV) vector carrying the follistatin gene.
• Dosing is highly experimental, measured in vector genomes per kilogram (vg/kg), and only performed in clinical trials.

Expected Cost Mean

Low Cost:

0

High Cost:

0

Cost Type:

unquantifiable

Description:

Not commercially available; costs are associated with clinical trials or unregulated black market sources.

Summary

Follistatin gene therapy introduces a gene that causes the body to produce excess follistatin, a protein that binds to and inhibits myostatin, a key negative regulator of muscle growth. This leads to significant muscle hypertrophy and is being explored to combat age-related sarcopenia, a primary driver of frailty. While effective in animal models, its application for human healthspan is purely experimental.

Purpose

Genetically increases muscle mass and strength by inhibiting myostatin.

Card Summary

An experimental gene therapy that increases muscle mass by inhibiting myostatin, a key negative regulator of muscle growth. It is being investigated for muscle-wasting diseases and age-related sarcopenia.

Drug Class

myostatin-inhibitors

Drugs In Class

Empty Array

Impact

Score:

3.8

Justification:

Theoretically high impact on sarcopenia, but lacks human healthspan data and long-term safety evidence.

Evidence

Grade:

C

Score:

3.5

Justification:

Human trials exist for specific muscular dystrophies, but only animal data for healthspan/sarcopenia.

Safety

Score:

9.2

Justification:

Irreversible genetic modification with unknown long-term risks, including immune response and off-target effects.

Flags

• This is an irreversible, experimental genetic modification.
• Potential for severe immune response to the AAV viral vector.
• Long-term consequences of systemic myostatin inhibition are unknown.
• Risk of contamination or incorrect dosing from unregulated, non-clinical sources is extremely high.
• Not approved by any major regulatory agency for any indication.

Verdict

Extremely high-risk, irreversible, and unproven for healthspan; avoid outside clinical trials.

Created

2026-05-06T22:00:47.794384Z

Ai Model

Gemini 2.5 Pro (High)