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AAV2-hCHM · 1 trial · 2 indications
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were AEs that occurred on or after the day of study drug administration. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: AAV2-hCHM Dose 1 | EXPERIMENTAL | Single, unilateral subretinal administration of a single low dose range of AAV2-hCHM. |
| Cohort 2: AAV2-hCHM Dose 2 | EXPERIMENTAL | Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM. |
| Cohort 3 (Expansion Cohort): AAV2-hCHM Dose 2 | EXPERIMENTAL | Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM. |
| Name | Type | Description |
|---|---|---|
| AAV2-hCHM | BIOLOGICAL | Comparison of different dosages of AAV2-hCHM |
Inclusion Criteria: * Male at least 18 years of age diagnosed with CHM gene mutation * Central visual field (VF) \<30° in any of the 24 meridians (using Goldmann perimetry III4e isopter) in the eye to be injected * Any evidence of functioning outer retinal cells within the central 10° Exclusion Cr...
AAV2-hCHM is an investigational gene therapy being studied for the treatment of choroideremia, a rare inherited eye disease that causes progressive vision loss. It is designed to deliver a functional copy of the CHM gene to retinal cells. The drug is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
AAV2-hCHM targets the CHM gene, which encodes the Rab escort protein 1 (REP1). Mutations in this gene cause choroideremia. The therapy uses an adeno-associated virus vector to deliver a working copy of the CHM gene to the retina, aiming to restore normal protein function and slow or halt disease progression.
AAV2-hCHM is being developed by Roche Holding AG, which trades on the OTC market under the ticker RHHBY. The company is conducting clinical research on this gene therapy for choroideremia. As of the latest data, the drug is in Phase 1 development.
AAV2-hCHM is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory body. A Phase 1 trial has been completed, and the drug remains in early-stage clinical testing for choroideremia.
AAV2-hCHM has been studied in one completed Phase 1 clinical trial, identified as NCT02341807. This trial was a safety and dose-escalation study in 15 male participants with choroideremia gene mutations. The study was conducted in the United States and enrolled adults aged 18 years and older.