CRISPR gene editing
CRISPR is now an approved treatment. The US Food and Drug Administration lists Casgevy for eligible patients aged two and older with recurrent sickle-cell crises or transfusion-dependent beta-thalassaemia. Other uses each still need their own evidence that delivery is safe and the benefit lasts.
Research snapshot · updated Nov 12, 2026
Core thesis
CRISPR has crossed from research tool to approved therapy. Casgevy (CRISPR Therapeutics, 2023) proved clinical viability for sickle cell. The frontier is now in vivo delivery — editing genes inside the body. Intellia leads with liver-targeted NTLA-2001 (hATTR), but a patient death from liver toxicity and subsequent FDA hold revealed the delivery challenge. ~250 CRISPR clinical trials active globally. The winner of the next decade is whoever solves delivery for non-liver tissues.
State of the art (2026)
CRISPR is now an approved treatment. Casgevy, from Vertex and CRISPR Therapeutics, treats sickle cell disease and transfusion-dependent beta thalassaemia by editing a patient's own cells outside the body. Patients need chemotherapy before the edited cells are returned, which limits how quickly it can be given. The next step is editing inside the body. Intellia is developing nex-z for transthyretin amyloidosis. Lilly completed its purchase of Verve Therapeutics, which develops base editors to lower cholesterol, on 25 July 2025. Each new use will need its own evidence that it is safe and lasts.
Beyond Cas9: precision editing without breaks
Base editing (Beam Therapeutics) and prime editing (Prime Medicine) modify DNA without double-strand breaks — dramatically reducing off-target risk. Epigenetic editing can silence genes without changing DNA sequence at all. These 'CRISPR 2.0' approaches are earlier-stage but potentially safer for widespread use.
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Signal stack
Evidence stacked leading → lagging
11 signals
Signal stack
Evidence stacked leading → lagging
Technology-native KPIs
Metrics that predict trajectory, tracked over time
1 tracked
Technology-native KPIs
Metrics that predict trajectory, tracked over time
Landscape map
Who builds what, and who depends on whom
185 players · 7 layers
Landscape map
Who builds what, and who depends on whom
Catalyst calendar
Dated events that will move the position
6 ahead
Catalyst calendar
Dated events that will move the position
Technology roadmap
Milestones on the path to maturity
7 milestones
Technology roadmap
Milestones on the path to maturity
Watchlists
Companies, people and papers, each with a condition for removing it
20 · 20
Watchlists
Companies, people and papers, each with a condition for removing it
Decision frameworks
What our view means for your job
Locked
Decision frameworks
What our view means for your job
Thesis changelog
When our view changed, and why
9 updates
Thesis changelog
When our view changed, and why
Change our mind
3 disconfirming conditions
Comparable wave
The historical analogue on the S-curve
Common mistakes
What the market gets wrong right now
You've read the verdict. The file is much deeper.
The full signal stack, technology-native KPIs tracked over time, the landscape of who depends on whom, the dated catalyst calendar, decision frameworks for every desk, live watchlists and a record of every time our view on CRISPR gene editing has changed. All of it is in CanaryIQ.