Drugs / BRINRETIGENE VESGEDPARVOVEC
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | Leber Congenital Amaurosis type 10 (LCA10) | — | “Filed EDIT-101 Investigational New Drug (IND) application in October with U.S. Food and Drug Administration ( FDA ) for treatment of Leber Congenital Amaurosis type 10 (LCA10).” editasmedicine.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03872479 | Sep 2019 → May 2025 | Leber congenital amaurosis 10, vision disorder | Editas Medicine, Inc. | Unknown | No outcome recorded |
News releases announcing trial results or a regulatory action · 12
| Date | Issuer | Release |
|---|---|---|
| 2022-11-17 | Editas Medicine, Inc. | Results Editas Medicine Announces Clinical Data Demonstrating Proof of Concept of EDIT-101 from Phase 1/2 BRILLIANCE Trial editasmedicine.com ↗
Three out of 14 treated subjects met a responder threshold having experienced clinically meaningful improvements in best corrected visual acuity (BCVA) (LogMAR >0.3) and demonstrated consistent improvements in two of the following three additional endpoints: full field sensitivity test (FST), visual function navigation course (VFN), or the visual function quality of life (VFQ). |
| 2022-05-18 | Editas Medicine, Inc. | Results Editas Medicine Presents Data on SLEEK Gene Editing Technology at the American Society of Gene and Cell Therapy Annual Meeting editasmedicine.com ↗
In an oral presentation at the ASGCT Annual Meeting on Monday, May 16, 2022 , Editas presented data on its EDIT-101 program for the treatment for Leber Congenital Amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder, demonstrating a favorable immunogenicity profile. |
| 2022-05-02 | Editas Medicine, Inc. | Results Editas Medicine to Present Pipeline and Platform Data Demonstrating Progress Towards Developing Transformative Gene Editing Medicines at the American Society of Gene and Cell Therapy Annual Meeting editasmedicine.com ↗
Safety data from the BRILLIANCE trial of EDIT-101 demonstrating a favorable immunogenic profile. |
| 2021-10-20 | Editas Medicine, Inc. | Results Clinical Data from Editas Medicine’s Ongoing Phase 1/2 BRILLIANCE Clinical Trial of EDIT-101 for LCA10 to be Presented at the European Society of Gene and Cell Therapy Annual Congress editasmedicine.com ↗
I am highly encouraged by the early efficacy signals in the mid-dose cohort, which suggest positive biological activity and potential early clinical benefits. |
| 2021-09-29 | Editas Medicine, Inc. | Results Editas Medicine Announces Positive Initial Clinical Data from Ongoing Phase 1/2 BRILLIANCE Clinical Trial of EDIT-101 for LCA10 editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced positive initial clinical data from the ongoing, open label Phase 1/2 BRILLIANCE clinical trial of EDIT-101. |
| 2019-04-29 | Editas Medicine, Inc. | Results Allergan and Editas Medicine Announce Initial Data from Natural History Study to Evaluate Patients with Leber Congenital Amaurosis 10 (LCA10) editasmedicine.com ↗
Allergan plc (NYSE: AGN), a leading global pharmaceutical company, and Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, announced initial data from the ongoing natural history study to evaluate patients with Leber congenital amaurosis 10 (LCA10), a rare form of blindness caused by mutations in the CEP290 gene. |
| 2019-01-21 | Editas Medicine, Inc. | Results Editas Medicine Announces Publication in Nature Medicine of Data Supporting the Development of EDIT-101 to Treat Leber Congenital Amaurosis 10 (LCA10) editasmedicine.com ↗
Published results detail the development of EDIT-101, an experimental genome editing medicine designed to remove the abnormal splice donor created by the IVS26 mutation in the CEP290 gene found in LCA10 patients and restore normal CEP290 expression. |
| 2018-11-30 | Editas Medicine, Inc. | Regulatory Editas Medicine Announces FDA Acceptance of IND Application for EDIT-101 editasmedicine.com ↗
Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration ( FDA ) has accepted the Company’s Investigational New Drug (IND) application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of Leber Congenital Amaurosis type 10 (LCA10). |
| 2018-08-16 | Editas Medicine, Inc. | Regulatory Editas Medicine Announces the Completion of the Recombinant DNA Advisory Committee (RAC) Registration Process editasmedicine.com ↗
The NIH determined a public meeting is not necessary, and the RAC will not be convened to review this clinical trial. |
| 2018-05-18 | Editas Medicine, Inc. | Results Editas Medicine Reports Data Demonstrating Subretinal Injection of EDIT-101 Well-tolerated in Non-human Primates editasmedicine.com ↗ |
| 2018-04-30 | Editas Medicine, Inc. | Results Editas Medicine to Present New Data Demonstrating Progress Towards Creating Genome Editing Medicines at the American Society of Gene & Cell Therapy Annual Meeting editasmedicine.com ↗
EDIT-101, the Company’s experimental medicine for the treatment of Leber Congenital Amaurosis type 10, administered to non-human primates via subretinal injection was well-tolerated |
| 2017-10-19 | Editas Medicine, Inc. | Results Editas Medicine Demonstrates Dose-Dependent, In Vivo Editing with EDIT-101 in CEP290 Transgenic Mice editasmedicine.com ↗
Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today announced results from a pre-clinical study in transgenic mice demonstrating dose-dependent, in vivo editing using EDIT-101, Editas Medicine’s pre-clinical product candidate for the treatment of Leber Congenital Amaurosis type 10 (LCA10). |
All press releases naming this drug 19 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BRINRETIGENE VESGEDPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314813 ↗ |
| Known as | Brinretigena vesgedparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314813 ↗ |
| Known as | EDIT-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03872479 ↗ |
| Modality | CRISPR / gene editing | “EDIT-101 is a clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) gene-editing complex designed to treat inherited retinal...” PMID 38709228 ↗ May 2024 |
| Route | Other | “administered via subretinal injection” NCT03872479 ↗ |
| Target | CEP290 | “EDIT-101 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.” NCT03872479 ↗ |