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Gene therapy
Gene therapy is a Gene Therapy Small molecule drug developed by AVROBIO. It is currently in Phase 2 development. Also known as: AVR-RD-02, therapy, gene, Intracerebral infusion of AAV2-hAADC viral vector, CD68-ET3-LV CD34+.
Introduces functional genetic material into patient cells to correct underlying genetic defects or provide therapeutic benefit.
Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. It has been studied for various conditions, including Adenosine Deaminase Severe Combined ImmunoDeficiency (ADA-SCID), Diabetic Macular Edema, Diabetic Retinopathy, Acute Promyelocytic Leukemia, and Pompe Disease, using gene-based interventions such as lentiviral vectors.
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Gene therapy |
|---|---|
| Also known as | AVR-RD-02, therapy, gene, Intracerebral infusion of AAV2-hAADC viral vector, CD68-ET3-LV CD34+ |
| Sponsor | AVROBIO |
| Drug class | Gene Therapy |
| Modality | Small molecule |
| Phase | Phase 2 |
Mechanism of action
Gene therapy delivers DNA or RNA sequences into target cells to replace defective genes, introduce new therapeutic genes, or modify gene expression. The approach aims to address the root cause of genetic diseases rather than treating symptoms.
Approved indications
Common side effects
Key clinical trials
- A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) (PHASE1)
- Testing the Combination of Two Anti-cancer Drugs, DS-8201a and AZD6738, for The Treatment of Advanced Solid Tumors Expressing the HER2 Protein or Gene, The DASH Trial (PHASE1)
- Measuring the Effects of Talazoparib in Patients With Advanced Cancer and DNA Repair Variations (PHASE2)
- A Clinical Study of Sacituzumab Tirumotecan (Sac-TMT, MK-2870) in People With Breast Cancer (MK-2870-032) (PHASE3)
- Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease (PHASE1, PHASE2)
- A Long Term Follow-up Study of Subjects Who Received ICM-203 or Matching Placebo
- Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) (PHASE1, PHASE2)
- Talimogene Laherparepvec and Radiation Therapy in Treating Patients With Newly Diagnosed Soft Tissue Sarcoma That Can Be Removed by Surgery (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Gene therapy CI brief — competitive landscape report
- Gene therapy updates RSS · CI watch RSS
- AVROBIO portfolio CI
Frequently asked questions about Gene therapy
What is Gene therapy?
How does Gene therapy work?
Who makes Gene therapy?
Is Gene therapy also known as anything else?
What drug class is Gene therapy in?
What development phase is Gene therapy in?
Related
- Drug class: All Gene Therapy drugs
- Manufacturer: AVROBIO — full pipeline
- Also known as: AVR-RD-02, therapy, gene, Intracerebral infusion of AAV2-hAADC viral vector, CD68-ET3-LV CD34+
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing