In-vivo base and prime editing

In-vivo CRISPR base & prime editing. Genetic & blood disorders. Reviewed Jul 2026.

Single doses durably knock down target proteins in early trials, but the platform is young and long-term data do not exist.

Stage

Phase 1/2

Rung: In human trials. Phase 1 to 3. Being tested in people now. Most will not make it.

Evidence grade

early trials

Small or uncontrolled human data, or strong animal data. How grades work.

What it is

Editing genes directly inside the body via a single infusion (no cells removed), rewriting single DNA letters without cutting both strands.

The evidence

Intellia's in-vivo editor for hereditary ATTR amyloidosis and HAE shows durable target knockdown after one dose; a custom base-editing therapy saved an infant with a rare metabolic disease in 2025. Platform still young.

What you can do today

Outside a registered clinical trial, this is not something you can responsibly get. If it is relevant to you, the route is a trial: ask your specialist, or search a trial registry such as ClinicalTrials.gov for studies you may qualify for.

Sources

Coverage, not a sourcefbae.org

No primary paper or registry record is linked for this entry yet. The evidence summary names the studies it relies on.

Change history

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