# Gene & Cell Therapy: State of the Field (October 2026)

Research dossier · v1 · updated 2026-10-30 · 3,785 words · 57 sources · 34 claims · verification: verified

Covers adeno-associated virus (AAV) and lentiviral gene therapy, in vivo CAR-T and stem-cell-derived replacement cells such as islets and dopaminergic neurons, with the evidence as of October 2026. The main takeaway: viral gene addition has clinical precedent in several monogenic diseases, but immune responses, dose-linked toxicity and integration questions still bound the field, while in vivo CAR-T and stem-cell replacement remain at early clinical or preclinical stages.

## Contents

- Executive summary
- Background and core concepts
- Timeline of key milestones
- State of the art
  - Adeno-associated virus (AAV) in vivo gene therapy
  - Capsid engineering and immune evasion
  - Lentiviral and ex vivo HSPC gene therapy
  - In vivo CAR-T and CAR engineering
  - Stem-cell-derived islets
  - Dopaminergic cell replacement and gene correction
  - Quantitative benchmarks
- Open problems and active debates
- Major programs and players
- Outlook: what to watch (near term)
- Key papers
- Glossary

## Sample findings

- In a 2022 meta-analysis of 255 rAAV clinical trials, AAV2 was the most frequently used serotype (reported as 29.8%, n = 72 trials); the abstract does not state the denominator for this percentage.
- The same meta-analysis reports 20 trials (reported as 8.3%) using engineered capsids; the abstract does not state the denominator.

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