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AAV9 Gene therapy
AAV9 Gene therapy is a Small molecule drug developed by Celosia Therapeutics Pty Ltd. It is currently in Phase 1 development.
AAV9 gene therapy is a type of gene therapy that uses adeno-associated virus 9 (AAV9) as a vector to deliver genetic material. It has been studied in clinical trials for various conditions, including Rett Syndrome, CLN6, and Batten Disease, with specific interventions such as TSHA-102 and scAAV9.CB.CLN6.
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Baseline phase 1 → approval rate
+9.6pp
Industry-wide phase 1 drugs reach approval ~9.6% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2033–2036 | — |
| EMA | EU | 2034–2037 | +0.7 yr |
| MHRA | GB | 2034–2037 | +0.7 yr |
| Health Canada | CA | 2034–2038 | +0.9 yr |
| TGA | AU | 2034–2038 | +1.2 yr |
| PMDA | JP | 2034–2038 | +1.5 yr |
| NMPA | CN | 2035–2039 | +2.3 yr |
| MFDS | KR | 2034–2038 | +1.4 yr |
| CDSCO | IN | 2034–2039 | +1.8 yr |
| ANVISA | BR | 2035–2039 | +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 | AAV9 Gene therapy |
|---|---|
| Sponsor | Celosia Therapeutics Pty Ltd |
| Modality | Small molecule |
| Phase | Phase 1 |
Approved indications
Common side effects
- Pyrexia
- Upper respiratory tract infection
- Constipation
- Vomiting
- Aspartate aminotransferase increased
- Alanine aminotransferase increased
- Cough
- Diarrhoea
- Rash
- Scoliosis
- Teething
- Gastrooesophageal reflux disease
Key clinical trials
- Gene Replacement Therapy for Treatment of Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome (PHASE1, PHASE2)
- Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy (PHASE1)
- Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)
- Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (PHASE1, PHASE2)
- Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome (PHASE3)
- A Phase I Study of Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis (PHASE1)
- A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome (PHASE3)
- Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (PHASE1, 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:
- AAV9 Gene therapy CI brief — competitive landscape report
- AAV9 Gene therapy updates RSS · CI watch RSS
- Celosia Therapeutics Pty Ltd portfolio CI
Frequently asked questions about AAV9 Gene therapy
What is AAV9 Gene therapy?
Who makes AAV9 Gene therapy?
What development phase is AAV9 Gene therapy in?
What are the side effects of AAV9 Gene therapy?
Related
- Manufacturer: Celosia Therapeutics Pty Ltd — full pipeline
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing