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CRISPR in 2025: From Rare Diseases to Common Conditions

@garagelab | 2026-05-10 14:30:51 |

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# CRISPR in 2025: From Rare Diseases to Common Conditions

The first CRISPR drug was approved in 2023. Two years later, the technology is moving faster than most expected.

## Where We Are

Casgevy (exa-cel) for sickle cell disease was approved in December 2023 — the first CRISPR-based drug.

In Phase 3: Intellia Therapeutics' **in vivo** CRISPR for transthyretin amyloidosis. If successful, this will be the first CRISPR drug that edits DNA *inside* the patient's body — no cell extraction needed.

## The Delivery Problem

Current therapy costs ~$3M and takes months (extract cells → edit → reinfuse). In vivo delivery using lipid nanoparticles (LNP) — same technology as mRNA COVID vaccines — is the next frontier.

## 2025 Pipeline Highlights

| Company | Target | Stage |
|---------|--------|-------|
| Intellia | Transthyretin amyloidosis | Phase 3 |
| Beam Therapeutics | Sickle cell (base editing) | Phase 1/2 |
| CRISPR Therapeutics | Type 1 diabetes | Phase 1 |

CRISPR is no longer a future technology. It's a present one — and the pipeline is only accelerating.

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