Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as AAV5-hRKp.RPGR
Botaretigene sparoparvovec · 4 trials · 2 indications
An AE is any untoward medical occurrence in a clinical study participant administered a investigational or non-investigational medicinal product. An AE does not necessarily have a causal relationship with the treatment.
Number of participants with abnormalities in clinical laboratory assessment (hematology and serum chemistry) will be reported.
Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.
Number of participants with ocular and non-ocular adverse events will be assessed.
Number of participants with abnormalities in laboratory assessments will be assessed.
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.
An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non investigational) product, whether or not related to that medicinal (investigational or non investigational) product.
Change from baseline in BCVA by ETDRS chart letter scores in monocular assessment of the second treated eye will be assessed. BCVA was measured using the ETDRS Scale (ranging from 0 to 100 letters) in the study eye. An increase in the number of letters read correctly means that vision has improved and vice-versa.
Change in LLVA by ETDRS chart letter scores from baseline in monocular assessment of second treated eye will be assessed. Visual function assessments included LLVA assessment in each eye by ETDRS letters. The letter score ranges from 0 (worse score) to 100 (best score), and a gain in LLVA from baseline indicates an improvement in visual acuity.
| Arm | Type | Description |
|---|---|---|
| Group 1: AAV5-hRKp.RPGR Low Dose | EXPERIMENTAL | Participants will receive bilateral subretinal administration of AAV5-hRKp.RPGR low dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart. |
| Group 2: AAV5-hRKp.RPGR High Dose | EXPERIMENTAL | Participants will receive bilateral subretinal administration of AAV5 hRKp.RPGR high dose, with surgical delivery to the 2 eyes performed within 7 to 21 days apart. |
| Experimental - Immediate Treatment | EXPERIMENTAL | Intermediate dose. |
| Deferred Treatment | OTHER | Deferred Treatment |
| Experimental Immediate Treatment | EXPERIMENTAL | Low dose. |
| Deferred Treatment From MGT-RPGR-021 of Intermediate Dose | EXPERIMENTAL | Deferred treatment |
| Already Treated in MGT-RPGR-021 | EXPERIMENTAL | Already treated |
| Deferred Treatment From MGT-RPGR-021 Low Dose | EXPERIMENTAL | Deferred treatment |
| Cohort 1 | EXPERIMENTAL | Participants will receive a dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 depending on the dosage administered in study MGT009 (NCT03252847) in the past. After receiving the treatment, participants will be assessed for safety. |
| Cohort 2 | EXPERIMENTAL | Participants will receive the treatment dose of AAV5 hRKp.RPGR under the retina (low-dose or intermediate-dose) on Day 1 in the second eye once the safety will be determined in Cohort 1. |
| Cohort 3 | OTHER | The participants who are not willing to undergo surgery or are not eligible for surgery will be assessed in this cohort. They will be assessed yearly until 5 years after their initial eye surgery in previous study MGT009. |
| Name | Type | Description |
|---|---|---|
| AAV5-hRKp.RPGR | GENETIC | AAV5-hRKp.RPGR will be administered by subretinal injection using a standardized surgical procedure. |
| Genetic: AAV5-hRKp.RPGR | BIOLOGICAL | Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group |
| Genetic: AAV5-hRKp.RPGR Intermediate Dose | BIOLOGICAL | Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group |
| Genetic: AAV5-hRKp.RPGR Low Dose | BIOLOGICAL | Bilateral, subretinal administration of AAV5-hRKp.RPGR - deferred treatment group |
| No intervention (Follow-Up assessment) | OTHER | Participants will not receive any intervention and will undergo follow-up assessment. |
Inclusion Criteria: * Participants who are Japanese male or female aged 5 years or older * Participants diagnosed as X-linked retinitis pigmentosa (XLRP) (generalized rod-cone dystrophy) associated with pathogenic or likely pathogenic variants in the retinitis pigmentosa guanosine triphosphatase re...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Ocugen Inc | OCGN | 3 | PHASE3 | OCU400, OCU410ST |
| Johnson & Johnson | JNJ | 1 | PHASE3 | Botaretigene sparoparvovec |
| Belite Bio, Inc. ADR | BLTE | 1 | PHASE2 | Tinlarebant |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE2 | SAR422459 |
| Kiora Pharmaceuticals, Inc. | KPRX | 1 | PHASE2 | KIO-301 |
| ProQR Therapeutics N.V. | PRQR | 1 | PHASE1 | QR-1123 |
Botaretigene sparoparvovec is an investigational gene therapy being developed for X-linked retinitis pigmentosa, a genetic eye condition that causes progressive vision loss. It is also being studied in retinitis pigmentosa more broadly. The therapy is designed for people with disease-causing variants in the RPGR gene.
Botaretigene sparoparvovec is a gene therapy that targets the RPGR gene. It uses an AAV5 vector to deliver a functional copy of the RPGR gene to retinal cells, aiming to address the underlying genetic cause of X-linked retinitis pigmentosa. Its target class is gene therapy.
Botaretigene sparoparvovec is being developed by Johnson & Johnson, which trades under the ticker JNJ. The company is running the clinical development program for this gene therapy in X-linked retinitis pigmentosa and related retinitis pigmentosa.
Botaretigene sparoparvovec is in Phase 3 clinical development. It is an investigational gene therapy and has not been approved by the FDA. The program includes three trials, with two currently active and one completed, enrolling a total of 226 participants.
Botaretigene sparoparvovec has been studied in four registered trials. These include NCT04671433, a completed Phase 3 trial, and NCT04794101, an active Phase 3 follow-up trial. Additional studies are NCT05926583, a Phase 3 trial in Japan, and NCT06646289, a Phase 2 second-eye treatment study.
Yes, botaretigene sparoparvovec is also known as AAV5-hRKp.RPGR. The alternative name reflects the AAV5 vector and the RPGR gene it delivers. It has also been referred to as AAV5-hRKp.RPGR intermediate dose and AAV5-hRKp.RPGR low dose in trial listings.