Drugs / GS010
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | EU (EMA) | LUMEVOQ | hereditary optic neuropathy | 2020-09-21 | “has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON)” gensight-biologics.com ↗ |
Trials 6
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02064569 | Feb 2014 → Jun 2020 | Leber hereditary optic neuropathy | GenSight Biologics | Completed | No outcome recorded |
| Phase 34 trials · 3 missed | ||||||
| Phase 3 | NCT03293524 | Mar 2018 → Jul 2024 | Leber hereditary optic neuropathy | GenSight Biologics | Completed | Missed primary |
| Phase 3 | NCT03406104 | Jan 2018 → Jul 2022 | Leber hereditary optic neuropathy | GenSight Biologics | Completed | No outcome recorded |
| Phase 3 | NCT02652767 | Feb 2016 → Aug 2018 | Leber hereditary optic neuropathy | GenSight Biologics | Completed | Missed primary |
| Phase 3 | NCT02652780 | Jan 2016 → Jan 2018 | Leber hereditary optic neuropathy | GenSight Biologics | Completed | Missed primary |
| Phase not stated1 trial | ||||||
| — | NCT03672968 | — | Leber hereditary optic neuropathy | GenSight Biologics | No longer available | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT07303296 Comparator | Jan 2026 → May 2028 expected | Leber hereditary optic neuropathy | GenSight Biologics | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 46
| Date | Issuer | Release |
|---|---|---|
| 2026-03-09 | GenSight Biologics | Regulatory GenSight Biologics Provides Updates about GS010/LUMEVOQ® Early Access Programs and the Ongoing REVISE Study gensight-biologics.com ↗
Following the authorization of the AAC program by the national medicines safety agency ANSM in December 2025, individual applications that were submitted in February were approved today. |
| 2025-12-22 | GenSight Biologics | Regulatory GenSight Biologics Announces the Granting of Compassionate Use Authorization (CUA/AAC) for GS010/LUMEVOQ® in France gensight-biologics.com ↗ |
| 2025-12-22 | GenSight Biologics | Regulatory GenSight Biologics Announces Regulatory Authorization for Early Access Treatment with GS010/LUMEVOQ® in Israel gensight-biologics.com ↗ |
| 2025-12-02 | GenSight Biologics | Regulatory GenSight Biologics Announces Regulatory Approval for GS010/LUMEVOQ® REVISE Dose-Ranging Study in France gensight-biologics.com ↗ |
| 2025-10-30 | GenSight Biologics | Regulatory GenSight Biologics announces regulatory authorizations for Individual Patient Expanded Access treatment with GS010/LUMEVOQ® in the US gensight-biologics.com ↗
today announced that the Company’s gene therapy GS010/LUMEVOQ ® has been granted regulatory authorizations for an Individual Patient Expanded Access in the United States. |
| 2025-02-12 | GenSight Biologics | Results GenSight Biologics Announces Five-Year Efficacy and Safety Results for LUMEVOQ® Gene Therapy at the Conclusion of the REFLECT Study gensight-biologics.com ↗ |
| 2024-03-20 | GenSight Biologics | Results GenSight Biologics Confirms Sustained Efficacy and Safety of Bilateral LUMEVOQ® Injections Four Years After One-Time Administration gensight-biologics.com ↗ |
| 2024-03-12 | GenSight Biologics | Results GenSight Biologics Announces Initial Results from New Meta-Analyses on Visual Outcomes with LUMEVOQ® Gene Therapy at NANOS 2024 gensight-biologics.com ↗
The meta-analyses depict a gradient of efficacy of visual recovery with LUMEVOQ ® intravitreal gene therapy resulting in greater recovery rates than that of idebenone treatment, and both greater than that in the natural history of the disease. |
| 2024-03-06 | GenSight Biologics | Results GenSight Biologics Announces Update on Real-World Data from Early Access Programs of LUMEVOQ® Gene Therapy at NANOS 2024 gensight-biologics.com ↗ |
| 2023-03-15 | GenSight Biologics | Results GenSight Biologics Announces Presentation of LUMEVOQ® Efficacy and Safety Data from Early Access Programs for ND4-LHON Patients at NANOS 2023 gensight-biologics.com ↗
And I was delighted to share these data with the wider scientific and medical community at the prestigious NANOS Conference. |
| 2022-07-20 | GenSight Biologics | Results GenSight Biologics reports 5 years’ data showing sustained efficacy and safety following one-time treatment with LUMEVOQ® gensight-biologics.com ↗ |
| 2021-11-22 | GenSight Biologics | Results GenSight Biologics to Host Key Opinion Leader Webcast on Outcomes Among Compassionate Use Patients Treated Bilaterally with LUMEVOQ® in the US gensight-biologics.com ↗
Dr. Donahue presented his findings at the 2021 Annual Meeting of the American Academy of Ophthalmology (AAO), held in New Orleans, Louisiana, USA, from November 12-15. |
| 2021-11-15 | GenSight Biologics | Regulatory GenSight Biologics Announces Extension of Day 120 Clock Stop of LUMEVOQ® EMA Regulatory Filing; MAA Review to Resume in April 2022 gensight-biologics.com ↗
The procedure will resume in April 2022 when the Company files its D120 responses, which will include batch data for three new validation batches. |
| 2021-10-06 | GenSight Biologics | Results GenSight Biologics Announces the Publication of a Review of Gene Therapy Trials for LHON in International Ophthalmology Clinics gensight-biologics.com ↗
Bilateral treatment effect following unilateral injection of gene therapy for ND4 -LHON was independently reported by three research groups |
| 2021-09-06 | GenSight Biologics | Regulatory UK MHRA grants GenSight Biologics’ LUMEVOQ® ophthalmic gene therapy Promising Innovative Medicine designation gensight-biologics.com ↗ |
| 2021-08-31 | GenSight Biologics | Results GenSight Biologics Announces Publication of RESTORE Study Data Demonstrating Sustained Efficacy 3 Years After Unilateral Injection of LUMEVOQ® gensight-biologics.com ↗ |
| 2021-07-05 | GenSight Biologics | Regulatory GenSight Biologics Announces Approval of the LUMEVOQ® Cohort Temporary Authorization for Use (ATUc) in France gensight-biologics.com ↗
The Cohort ATU greatly simplifies the process by which patients gain access to LUMEVOQ ® prior to EU marketing authorization expected in H1 2022. |
| 2021-06-30 | GenSight Biologics | Results GenSight Biologics Reports Topline Results from REFLECT Phase III Clinical Trial, Confirming LUMEVOQ® Efficacy Including Better Efficacy with Bilateral Treatment gensight-biologics.com ↗
The results show better visual acuity improvements from bilateral intravitreal injections of the gene therapy compared to a unilateral injection. |
| 2021-02-15 | GenSight Biologics | Results GenSight Biologics Announces Publication of Results from LUMEVOQ® Phase I/IIa Clinical Trial REVEAL in BioDrugs gensight-biologics.com ↗ |
| 2020-12-10 | GenSight Biologics | Results GenSight Biologics Announces Publication of Results from LUMEVOQ® REVERSE Pivotal Phase III Trial and Non-Human Primate Study in Science Translational Medicine gensight-biologics.com ↗
“The treatment has been shown to be safe and the outcomes can be life changing,” said Dr. Patrick Yu |
| 2020-11-03 | GenSight Biologics | Regulatory GenSight Biologics reports validation of LUMEVOQ® Marketing Authorisation Application (MAA) by European Medicines Agency gensight-biologics.com ↗
The application for use of LUMEVOQ ® gene therapy to treat vision loss in patients with Leber Hereditary Optic Neuropathy (LHON) due to a mutated ND4 mitochondrial gene was submitted in September, and the procedure was formally initiated on October 29. |
| 2020-09-21 | GenSight Biologics | Results GenSight Biologics reports new analysis demonstrating statistically significant and clinically meaningful difference between visual outcomes in LUMEVOQ®-treated patients and natural history of Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗ |
| 2020-07-21 | GenSight Biologics | Results GenSight Biologics reports final REALITY natural history study results confirming poor spontaneous recovery for LHON patients with ND4 mutation gensight-biologics.com ↗
Statistical analysis of the visual acuity in 23 REALITY subjects aged 15 or older with a mutated ND4 gene shows that on average, vision failed to recover from an initial sudden decline, even several years after vision loss. |
| 2020-07-06 | GenSight Biologics | Results GenSight Biologics reports sustained efficacy and safety among LHON patients three years after LUMEVOQ® treatment gensight-biologics.com ↗
One year later, or three years after the one-time injection, the bilateral benefit was maintained, with LUMEVOQ ® -treated eyes recording a mean improvement against nadir of +20.5 letters equivalent and sham-treated eyes demonstrating a mean improvement of +19.4 letters equivalent. |
| 2020-04-15 | GenSight Biologics | Regulatory GenSight Biologics on Track to Submit LUMEVOQ® for European Approval in September 2020 Following Pre-Submission Meeting with EMA gensight-biologics.com ↗ |
| 2019-12-11 | GenSight Biologics | Results GenSight Biologics reports findings from REALITY study and REVERSE-RESCUE analysis highlighting positive benefits from LUMEVOQ™ (GS010) compared to LHON natural history gensight-biologics.com ↗
The results confirm LHON experts’ observations from their clinical practice and contrast sharply against the bilateral improvement observed in LUMEVOQ™ (GS010)’s Phase III studies. |
| 2019-12-09 | GenSight Biologics | Regulatory The Quinze-Vingts Hospital and GenSight Biologics announce a first Temporary Authorization for Use (ATU) for LUMEVOQ™ (GS010) in France gensight-biologics.com ↗ |
| 2019-10-09 | GenSight Biologics | Results GenSight Biologics reports evidence of GS010 DNA transfer to contralateral eye of primates unilaterally injected with GS010 Gene Therapy gensight-biologics.com ↗
GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported positive proof of GS010 DNA transfer from one eye to the other eye following unilateral intravitreal injection of primates. |
| 2019-09-23 | GenSight Biologics | Results GenSight Biologics reports sustained efficacy and safety at 96 weeks in RESCUE Phase III clinical trial of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported the first set of results from Week 96 of the RESCUE Phase III clinical trial. |
| 2019-05-29 | GenSight Biologics | Results Sustained bilateral improvement from GenSight Biologics’ REVERSE Phase III clinical trial represents transformative therapeutic benefit for patients, Key Opinion Leaders conclude gensight-biologics.com ↗
The event was dedicated to updates on clinical results for GS010, in particular findings at Week 96 of the REVERSE Phase III clinical trial in the treatment of Leber Hereditary Optic Neuropathy (LHON). |
| 2019-05-15 | GenSight Biologics | Results GenSight Biologics reports positive 96-week data from REVERSE Phase III clinical trial of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
The results point to continued efficacy of GS010 two years past injection, with best-corrected visual acuity (BCVA) sustaining a clinically meaningful improvement over baseline. |
| 2019-04-17 | GenSight Biologics | Results GenSight Biologics reports positive follow-up results at Week 72 of the RESCUE Phase III clinical trial of GS010 in Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
The key measure of visual function – best-corrected visual acuity (BCVA) – continued to improve at Week 72 compared to Week 48, demonstrating sustained recovery from the lowest point, or nadir, experienced in the acute phase of the disease. |
| 2019-02-12 | GenSight Biologics | Results GenSight Biologics announces publication of positive safety data from Phase I/II trial of GS010 in the JAMA Ophthalmology gensight-biologics.com ↗
This study demonstrates that GS010 (rAAV2/2- ND4 ) is both safe and well tolerated 2 years after a single unilateral intravitreal administration. |
| 2019-02-04 | GenSight Biologics | Results GenSight Biologics reports topline results at Week 48 of the RESCUE Phase III clinical trial of GS010 in subjects within six months of visual loss onset due to Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗ |
| 2018-12-12 | GenSight Biologics | Results GenSight Biologics reports sustained quality of life improvements at Week 72 of Phase III REVERSE clinical trial of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗ |
| 2018-10-18 | GenSight Biologics | Results GenSight Biologics reports positive 72-week data from REVERSE Phase III clinical trial of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗ |
| 2018-06-20 | GenSight Biologics | Results Key Opinion Leaders highlight GS010 efficacy and patient benefits in discussion of findings from REVERSE Phase III clinical trial gensight-biologics.com ↗
The topline findings on Spectral-Domain Optical Coherence Tomography (SD-OCT) parameters are “ the first demonstrations of neuro-protection of neurons and of central nervous system axons in a human genetic disease,” according to Dr. Sergott. |
| 2018-06-12 | GenSight Biologics | Results GenSight Biologics reports positive additional data from REVERSE Phase III clinical trial of GS010 for treatment of Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
Statistically significant preservation of both retinal ganglion cells and temporal retinal nerve fiber layer in GS010-treated eyes vs. sham-treated eyes objectively demonstrate protective effect of GS010 |
| 2018-04-03 | GenSight Biologics | Results GenSight Biologics announces topline results from REVERSE Phase III clinical trial of GS010 in patients with Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
Topline results further highlight the favorable safety and tolerability profile of GS010, and demonstrate a clinically meaningful improvement of +11 ETDRS letters (-0.218 LogMAR) in treated eyes at 48 weeks as compared to baseline in all 37 patients. |
| 2018-02-20 | GenSight Biologics | Results GenSight Biologics announces publication of positive data from Phase I/II trial and long-term follow-up of GS010 in Ophthalmology, the journal of the American Academy of Ophthalmology gensight-biologics.com ↗
GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced publication of detailed results from the Phase I/II clinical trial and long-term follow-up of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients in Ophthalmology, the journal of the American Academy of Ophthalmology. |
| 2017-12-05 | GenSight Biologics | Results GenSight Biologics reports positive long-term visual acuity gains and safety results from Phase I/II Study of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
These results confirm long- term sustained gains in visual acuity 2.5 years after a single intravitreal injection of GS010, especially in subjects with less than 2 years of onset of vision loss. |
| 2017-06-14 | GenSight Biologics | Results GenSight Biologics reports long-term positive safety and visual acuity results at Week 96 in Phase I/II Study of GS010 for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
These results confirm the long-term positive sustained visual acuity gain after 2 years with a single intravitreal injection of GS010 in patients with Leber’s Hereditary Optic Neuropathy (LHON), especially in those with less than 2 years of disease onset. |
| 2016-12-20 | GenSight Biologics | Results GenSight Biologics reports sustained visual acuity gain at 78 weeks in its Phase I/II Study with GS010 for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
These results confirm the favorable safety and tolerability profile of GS010, while demonstrating sustainable visual acuity improvement in patients with Leber’s Hereditary Optic Neuropathy (LHON). |
| 2016-09-08 | GenSight Biologics | Regulatory GenSight Biologics receives approval to include teenage patients in RESCUE and REVERSE Phase III trials with GS010 in Leber’s Hereditary Optic Neuropathy gensight-biologics.com ↗
The initial protocols were designed to enroll adult patients, whereas this amendment now allows inclusion of patients from the age of 15. |
| 2016-06-08 | GenSight Biologics | Results GenSight Biologics announces additional promising results of its Phase I/II study with GS010 for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) gensight-biologics.com ↗
At 48 weeks post-injection, in patients with an onset of disease of less than 2 years, a gain of +30 letters (-0.59 LogMAR) was observed in the treated eye and +13 letters (-0.25 LogMAR) in the untreated eye, a difference of 17 letters in favor of the treated eye. |
| 2015-09-10 | GenSight Biologics | Regulatory GenSight Biologics Receives IND Acceptance from FDA to Enter Phase III with GS010 gensight-biologics.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) Application for two Phase III clinical trials of GS010 in the treatment of Leber’s Hereditary Optic Neuropathy (LHON). |
All press releases naming this drug 68 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | GS010 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07303296 ↗ |
| Known as | rAAV2/2-ND4 | “An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to...” NCT02064569 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2)” NCT02652767 ↗ |
| Route | Intravitreal | “via intravitreal injection” NCT02652767 ↗ |
| Target | MT-ND4 | “GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4).” NCT02652767 ↗ |