VERTICAL REPORT // THERAPEUTICS
Cell & Gene Therapy Landscape
Tracking the convergence of CRISPR gene editing, base editing, prime editing, epigenetic editing, mRNA therapeutics, and engineered cell therapies. From Casgevy (the first FDA-approved CRISPR therapy) to next-gen programmable cell therapies and AI-designed biologics.
SECTION 01 // KEY COMPANIES
Key Companies & Clinical Pipeline
| Company | Approach | Lead Program | Stage | Ticker |
|---|---|---|---|---|
| CRISPR Therapeutics | CRISPR gene editing | Casgevy (FDA approved) | Commercial | CRSP |
| Beam Therapeutics | Base editing | BEAM-101 (Phase 2) | Clinical | BEAM |
| Intellia Therapeutics | In vivo CRISPR | NTLA-2001 (Phase 3) | Clinical | NTLA |
| Caribou Biosciences | chRDNA editing | CB-010 (Phase 1) | Clinical | CRBU |
| Verve Therapeutics | Base editing (cardio) | VERVE-102 (Phase 1) | Clinical | VERV |
| ArsenalBio | Programmable cell therapy | AB-1015 (Phase 1) | Clinical | — |
| Mammoth Biosciences | Ultra-small Cas enzymes | Diagnostics + editing | Platform | — |
| Chroma Medicine | Epigenetic editing | Preclinical programs | Preclinical | — |
| Generate Biomedicines | Generative biology | AI-designed proteins | Clinical | — |
SECTION 02 // EDITING APPROACHES
Gene Editing Tool Landscape
CRISPR-Cas9
Double-strand break + HDR/NHEJ
CRISPR Therapeutics, Intellia, Caribou
FDA Approved (Casgevy)
Base Editing
C-to-T or A-to-G without DSB
Beam Therapeutics, Verve Therapeutics
Phase 1-2 Trials
Prime Editing
Search-and-replace via pegRNA
Prime Medicine
Phase 1 Trials
Epigenetic Editing
Gene silencing/activation without DNA changes
Chroma Medicine, Tune Therapeutics
Preclinical
RNA Editing
ADAR-mediated A-to-I conversion
Wave Life Sciences, Korro Bio
Phase 1 Trials