Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AGTC-402 · 1 trial · 1 indication
Proportion of participants experiencing grade 3 or greater adverse events
| Arm | Type | Description |
|---|---|---|
| Group 1: 4.0 x 10^10 vg/mL of AGTC-402 | EXPERIMENTAL | Subjects at least 18 y/o treated with 4.0 x 10\^10 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug. |
| Group 2: 1.2 x 10^11 vg/mL of AGTC-402 | EXPERIMENTAL | Subjects at least 18 y/o treated with 1.2 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug. |
| Group 3: 3.6 x 10^11 vg/mL of AGTC-402 | EXPERIMENTAL | Subjects at least 18 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug. |
| Group 3a: 3.6 x 10^11 vg/mL of AGTC-402 | EXPERIMENTAL | Subjects 6 to 17 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug. |
| Group 4: 1.1 x 10^12 vg/mL of AGTC-402 | EXPERIMENTAL | Subjects at least 18 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug. |
| Group 4a: 1.1 x 10^12 vg/mL of AGTC-402 | EXPERIMENTAL | Subjects 4 to 8 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug. |
| Group 5: 3.2 x 10^12 vg/mL of AGTC-402 | EXPERIMENTAL | Subjects at least 18 y/o treated with 3.2 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug. |
| Group 6: MTD of AGTC-402 | EXPERIMENTAL | Subjects 4 to 8 y/o treated with a maximum tolerated dose of rAAV2tYF-PR1/7-hCNGA3 study drug determined by Groups 1-5. |
| Name | Type | Description |
|---|---|---|
| AGTC-402 | BIOLOGICAL | AGTC-402 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGA3 gene. |
Inclusion Criteria: 1. Male or female subjects with documented mutations in both alleles of the CNGA3 gene; 2. Retinal disease consistent with a clinical diagnosis of achromatopsia; 3. At least 18 years of age for Groups 1, 2, 3, 4, and 5. At least 6 years of age for Group 3a, and and 4-8 years of ...
AGTC-402 is an investigational gene therapy being developed for the treatment of achromatopsia, a rare inherited retinal disorder characterized by color blindness, light sensitivity, and poor visual acuity. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
AGTC-402 is a gene therapy designed to deliver a functional copy of the CNGA3 gene to retinal cells. This gene is essential for normal cone photoreceptor function, and its mutation causes achromatopsia. By restoring CNGA3 expression, the therapy aims to improve cone cell function and vision.
AGTC-402 is being developed by Applied Genetic Technologies Corporation (AGTC), a biopharmaceutical company focused on gene therapies for rare diseases. The company is conducting clinical trials to evaluate the safety and efficacy of AGTC-402 in patients with achromatopsia.
AGTC-402 is currently in Phase 1 clinical development. It is an investigational therapy, meaning it has not yet received regulatory approval and is still being studied in clinical trials to assess its safety and effectiveness in patients.
AGTC-402 is being evaluated in a Phase 1 clinical trial with the identifier NCT02935517. This trial is titled 'Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia' and is actively recruiting participants in the United States and Israel.
AGTC-402 is a gene therapy that targets the CNGA3 gene, which is mutated in a form of achromatopsia. It is specifically designed to deliver a functional CNGA3 gene to retinal cells, making it a targeted treatment for this genetic cause of the condition.