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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

CompanyApproachLead ProgramStageTicker
CRISPR TherapeuticsCRISPR gene editingCasgevy (FDA approved)CommercialCRSP
Beam TherapeuticsBase editingBEAM-101 (Phase 2)ClinicalBEAM
Intellia TherapeuticsIn vivo CRISPRNTLA-2001 (Phase 3)ClinicalNTLA
Caribou BioscienceschRDNA editingCB-010 (Phase 1)ClinicalCRBU
Verve TherapeuticsBase editing (cardio)VERVE-102 (Phase 1)ClinicalVERV
ArsenalBioProgrammable cell therapyAB-1015 (Phase 1)Clinical
Mammoth BiosciencesUltra-small Cas enzymesDiagnostics + editingPlatform
Chroma MedicineEpigenetic editingPreclinical programsPreclinical
Generate BiomedicinesGenerative biologyAI-designed proteinsClinical
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
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