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PM359 (CGD)Prime Medicine—PM359 (CGD)Prime Medicine—PM577a (Wilson disease)Prime Medicine—

PM359 (CGD)

by Prime MedicineUS

ProtoStage 2 of 5

Two patients treated; FDA gave RMAT status in Jun 2026 and Prime is discussing a path to filing.

Updated 22 Jun 2026Checked 25 Sep0 updates this week

Milestones

No announced next step
  1. FDA trial approval2024Complete.
  2. First patient data for prime editingMay 2025Complete.
  3. Drug shelved2025Complete.
  4. FDA grants RMAT designation22 Jun 2026Complete.

Most important updates

  • 22 Jun 2026
  • 7 Dec 2025
  • May 2025
  • 17 Oct 2024

Physics limits

  • Edited stem cells need room: chemo clears the marrowBlood stem cells live in limited niches in the bone marrow. For edited cells to take over, the patient's own must be wiped out with chemotherapy like busulfan, which risks infections and infertility.
  • Prime editors are big and edit less efficientlyThe editor joins a DNA-cutting enzyme with a reverse transcriptase, making a long mRNA (over 6,000 letters) plus a long guide. Efficiency varies by site and is often lower than simpler editors.
  • Only so many stem cells can be collected and editedA patient gives a limited number of blood stem cells, and some are lost in editing and freezing. If too few survive, collection must be repeated, which stretches treatment to months.

How it works

3 parts
A neutrophil (yellow) grabbing bacteria: the germ-killing white cells that PM359's edited stem cells should restore
A neutrophil (yellow) grabbing bacteria: the germ-killing white cells that PM359's edited stem cells should restorePhoto: Volker Brinkmann · CC BY 2.5 (opens commons.wikimedia.org)
Collect

Take blood stem cells

A patient's blood stem cells are collected and taken to the lab.

Write

Fix the NCF1 typo

A prime editor restores two missing DNA letters in NCF1, a gene the white cells need to build their germ-killing enzyme.

Return

Chemo, then infusion

After chemotherapy clears the marrow, edited cells are infused and grow into white cells that can make bleach-like chemicals to kill germs.

Update log

4 updates

Mon 22 Jun

  • Minor: PressRegulatory

Sun 7 Dec 2025

  • Minor: PressResearch

May 2025

  • Major: PressTest

Thu 17 Oct 2024

  • Major: RegulatorTest

About Prime Medicine

The team behind PM359 (CGD)

Prime Medicine

Prime editing, Wilson disease

Company built on prime editing, invented in David Liu's lab. A prime editor nicks one DNA strand and writes in a new sequence, like find-and-replace, without fully cutting DNA. After first patient data in 2025 it refocused on liver diseases.

  • May 2025: first-ever prime-editing patient data (PM359) restored immune-cell function; the drug was then shelved for business reasons.
  • FDA cleared a trial of PM577a for Wilson disease in Jul 2026; dosing expected in H2 2026.
Founded
20197 yrs
Headquarters
United States
Status
Public
Valuation
public
Works in
BiotechGene editing
Coverage
2 programs · 6 updateslatest 23 Jul 2026checked 25 Sep
Primer