Drug intelligence / Profile preview

AVO-100

Development stage
Preclinical
Lead developer
AAVivo
Modality
Cell Therapies, AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
01

Overview

AVO-100 is an **AAV-based gene therapy** developed by AAVivo, Inc., designed as an off-the-shelf therapy that generates **CD19-specific chimeric antigen receptor (CAR)-T cells in vivo**. It targets **B-cell malignant neoplasms** by leveraging adeno-associated virus (AAV) vectors to deliver genetic material enabling CAR-T cell production directly within the patient, potentially offering a more accessible alternative to ex vivo CAR-T therapies. Currently in **preclinical development** for immune system and hemic/lymphatic diseases, with highest R&D status as preclinical in the United States as of September 2025.[1][5]

02

Targets

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