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CTX310 (ANGPTL3)CRISPR Therapeutics—CTX310 (ANGPTL3)CRISPR Therapeutics—

CRISPR Therapeutics

CRISPR, Casgevy, in vivo liver editing

CH/USpublicest. 2013crisprtx.com (opens crisprtx.com)Checked 25 Sep

The brief

Analyst view
  • Casgevy became the world's first approved CRISPR therapy (UK Nov 2023, US Dec 2023), sold with Vertex, which leads it.
  • CTX310, a one-time CRISPR edit in the liver, lowered blood fats and LDL cholesterol in an early trial (NEJM, Nov 2025).
  • Casgevy uptake is slow: each patient needs stem-cell collection, a factory slot and harsh chemotherapy at a special center.

Technical approach

As reported
Nuclease

Cutting DNA with Cas9

A guide RNA leads the Cas9 protein to one spot in the DNA, where it cuts. The cell's sloppy repair usually breaks the gene, switching it off.

Ex vivo

Edit cells outside the body

For Casgevy, a patient's blood stem cells are taken out, edited, checked, and put back after chemo clears room in the bone marrow.

In vivo

Inside the body, via the liver

For CTX310, the editing tools ride in tiny fat bubbles (lipid nanoparticles) given by drip. Liver cells absorb them and a gene is switched off for good.

Allogeneic

Off-the-shelf immune cells

Donor immune cells are edited to attack a target and to not be rejected, so one batch can treat many unrelated patients.

Programs

CRISPR Therapeutics, two programs

Updated 28 Aug 2026
Casgevy (exa-cel)A one-time treatment: a patient's own stem cells are edited to switch fetal hemoglobin back on. That healthy baby form of hemoglobin makes up for the faulty adult form in sickle cell and beta thalassemia.ScalingNextMore treatment centers, more patientsUpdated 1 Feb 2024View program →CTX310 (ANGPTL3)A drip that delivers CRISPR to the liver to switch off the ANGPTL3 gene. People born without it have low blood fats and LDL, so one permanent edit aims to lower both for life.ProtoNextNext trial in very high blood fats or cholesterolUpdated 28 Aug 2026View program →
Research · Proto · Pilot · Deploy · Scaling

Research & demos

4 items

Open technical problems

Open · Active · Partial
  • Harsh chemo before treatmentActive research

    Edited-cell therapies still need strong chemo (busulfan), which risks infertility and long hospital stays. Gentler options are a big goal.

  • Permanent edits need long safety checksOpen

    Liver edits can't be undone, so years of follow-up for off-target edits and immune reactions matter more than for normal drugs.

  • Access and costOpen

    Casgevy costs about $2.2M in the US and takes months at special centers, so few eligible patients get it.

Peers

Gene editing · 3

Company & funding

HQ
CH/US
Founded
2013

Partners & customers

1 partner

People

Key people
Primer