Leber Hereditary Optic Neuropathy (LHON)

GenSight Biologics Reports Clinically Meaningful Vision Improvement is Maintained 4 Years After One-time Treatment with LUMEVOQ® Gene Therapy

Retrieved on: 
Monday, January 24, 2022
Biotechnology, Pharmaceutical, Genetics, Health, Sequence, Logarithm, Research, Eye, Severe cognitive impairment, EMA, European Medicines Agency, Science Translational Medicine, LHON, Pain, Mitochondrion, Year, Mitochondrial disease, Incidence, Union, ND4, MTS, Ophthalmology, Field line, Safety, Quality of life, Visual acuity, Institute, AAV, Leber's hereditary optic neuropathy, DNA, Review, Retina, Color, Vision, Systematic review, CRR, Optogenetics, Retinal ganglion cell, CHMP, Natural history, Medical device, Pharmaceutical industry, Medical imaging, Patient, N, Optic neuropathy, LogMAR chart, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), RESCUE, REVERSE, and RESTORE, GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC), RESCUE, REVERSE, AND RESTORE

Year 4 values were the LogMAR readings nearest to 1461 days post treatment recorded between 1461 +/- 273 days post- treatment.

Key Points: 
  • Year 4 values were the LogMAR readings nearest to 1461 days post treatment recorded between 1461 +/- 273 days post- treatment.
  • 71.0% of RESTORE subjects achieved Clinically Relevant Recovery (CRR)4 against nadir four years after treatment, and 80.7% of them had on-chart vision (BCVA 1.6 LogMAR) in one or both eyes.
  • Viewed against the trend in vision typically seen in untreated patients5, the findings represent a significant departure from the natural progression of LHON.
  • Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset.

GenSight Biologics Confirms Sustained Efficacy and Safety of Bilateral LUMEVOQ® Injections at 2-Year Follow-Up of REFLECT Phase III Trial

Retrieved on: 
Tuesday, December 14, 2021
Science, Biotechnology, Research, Pharmaceutical, Optical, Health, Genetics, Clinical Trials, Sequence, Leber's hereditary optic neuropathy, Vision, LogMAR chart, Injection, Optogenetics, Severe cognitive impairment, European Medicines Agency, NADH, Incidence, AAV, LHON, EMA, Science Translational Medicine, Institute, Relevant, OCT, Mitochondrial disease, Marketing, MTS, Inflammation, Patient, Safety, Research, Pain, Mitochondrion, ND4, Retinal ganglion cell, Eye, MD, Union, Ophthalmology, Retina, Telescopic sight, Pharmaceutical industry, Medical device, Vaccine, Optic neuropathy, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), REFLECT, GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC), REFLECT

There was no study discontinuation related to systemic or ocular adverse events, and there were no serious ocular adverse events.

Key Points: 
  • There was no study discontinuation related to systemic or ocular adverse events, and there were no serious ocular adverse events.
  • REFLECT patients have been invited to participate in a long-term follow-up that will monitor the safety and efficacy of LUMEVOQ up to 5 years post-injection.
  • Newman NJ, Carelli V, Taiel M, Yu-Wai-Man P. Visual outcomes in Leber hereditary optic neuropathy subjects with the m.11778G>A (MTND4) mitochondrial dna mutation.
  • Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset.

GenSight Biologics Announces Publication of Non-Human Primate Study Reporting Presence of LUMEVOQ® Vector DNA in Contralateral Eyes After Unilateral Injection

Retrieved on: 
Wednesday, December 1, 2021
Health, Genetics, Clinical Trials, Pharmaceutical, Optical, Biotechnology, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC)

In the LUMEVOQ clinical studies, BCVA improved in both eyes of patients who received a unilateral injection of the gene therapy.

Key Points: 
  • In the LUMEVOQ clinical studies, BCVA improved in both eyes of patients who received a unilateral injection of the gene therapy.
  • The mechanism behind the contralateral therapeutic effect of LUMEVOQ on untreated eyes was explored in a non-human primate (NHP) study that analyzed the biodistribution of LUMEVOQ vector DNA (i.e., the therapeutic ND4 gene) in the visual system following a single unilateral IVT.
  • As expected, LUMEVOQ vector DNA was detected in all the eyes that were injected with the gene therapy.
  • But vector DNA was also detected or quantified in the contralateral non-injected eyes for 5 of the 6 animals in the treatment group.

GenSight Biologics to Host Key Opinion Leader Webcast on Outcomes Among Compassionate Use Patients Treated Bilaterally with LUMEVOQ® in the US

Retrieved on: 
Tuesday, November 30, 2021
Health, Genetics, Clinical Trials, Pharmaceutical, Optical, Biotechnology, Sequence, Patient, Institute, European Medicines Agency, Reflection phase change, Retinal ganglion cell, AAO, European Union, MTS, Mitochondrial disease, Retina, MD, Optogenetics, LHON, Doctor of Philosophy, Severe cognitive impairment, Eye, ND4, Pain, Research, Mitochondrion, Coleman, Incidence, EMA, Marketing, ISIN, Vanderbilt, Union, FDA, AAV, Webcast, Vanderbilt University, American Academy, Medical device, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC)

For those unable to attend the live broadcast, a recording will be accessible using the same links.

Key Points: 
  • For those unable to attend the live broadcast, a recording will be accessible using the same links.
  • GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.
  • GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases.
  • LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

GenSight Biologics to Host Key Opinion Leader Webcast on Outcomes Among Compassionate Use Patients Treated Bilaterally with LUMEVOQ® in the US

Retrieved on: 
Monday, November 22, 2021
Health, Genetics, General Health, Clinical Trials, Pharmaceutical, Optical, Biotechnology, Sequence, Patient, Institute, Safety, European Medicines Agency, Reflection phase change, Retinal ganglion cell, AAO, European Union, MTS, Mitochondrial disease, Retina, MD, Optogenetics, Severe cognitive impairment, Injection, LHON, Doctor of Philosophy, Ageing, Eye, EST, ND4, Pain, Research, Mitochondrion, Coleman, Incidence, EMA, Marketing, ISIN, Vanderbilt, Union, FDA, AAV, Webcast, Vanderbilt University, American Academy, Pharmaceutical industry, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC)

The REFLECT trial investigated the efficacy and safety of bilateral injections of LUMEVOQ among ND4-LHON patients aged 15 years or older treated within one year from the onset of visual loss.

Key Points: 
  • The REFLECT trial investigated the efficacy and safety of bilateral injections of LUMEVOQ among ND4-LHON patients aged 15 years or older treated within one year from the onset of visual loss.
  • The webcast will be held on Thursday, December 2, 2021, at 8:00 am EST (2:00 pm CET).
  • GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.
  • LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

GenSight Biologics Announces the Publication of a Review of Gene Therapy Trials for LHON in International Ophthalmology Clinics

Retrieved on: 
Wednesday, October 6, 2021
Science, Biotechnology, Research, Pharmaceutical, Optical, Health, Genetics, Clinical Trials, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC)

The paper*, published in the October issue of the International Ophthalmology Clinics under the title Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy, discusses the trials and the outcomes available to date.

Key Points: 
  • The paper*, published in the October issue of the International Ophthalmology Clinics under the title Gene Therapies for the Treatment of Leber Hereditary Optic Neuropathy, discusses the trials and the outcomes available to date.
  • Only GenSights gene therapy, LUMEVOQ, has completed Phase III trials and reached the registration phase.
  • All gene therapies investigated provide excellent systemic tolerability and mostly mild ocular side effects, responsive to conventional ophthalmologic treatments.
  • Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.

GenSight Biologics Announces Publication Analyzing Visual Parameters of ND4-LHON Subjects Before LUMEVOQ® Treatment in Phase III Trials

Retrieved on: 
Thursday, September 9, 2021
Science, Biotechnology, Research, Pharmaceutical, Optical, Health, Genetics, Clinical Trials, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), RESCUE and REVERSE, GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC), RESCUE AND REVERSE

Individual BCVA values were collected at screening and inclusion, then grouped and averaged by time since onset of vision loss.

Key Points: 
  • Individual BCVA values were collected at screening and inclusion, then grouped and averaged by time since onset of vision loss.
  • Due to a very low number of observations available before onset of vision loss, no standard error was calculated.
  • The baseline data were collected on two pre-injection visits among the 76 subjects in the RESCUE and REVERSE clinical trials.
  • The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy.

UK MHRA Grants GenSight Biologics’ LUMEVOQ® Ophthalmic Gene Therapy Promising Innovative Medicine Designation

Retrieved on: 
Monday, September 6, 2021
Health, Genetics, General Health, Pharmaceutical, Optical, Biotechnology, Branches of biology, Leber's hereditary optic neuropathy, Proteins, Optic neuropathy, Mitochondrial disease, MT-ND6, Mitochondrial optic neuropathies, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), the Early Access to Medicines Scheme (EAMS), GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC), THE EARLY ACCESS TO MEDICINES SCHEME (EAMS)

This marks another important milestone in our preparations to make LUMEVOQ available to LHON patients as soon as possible.

Key Points: 
  • This marks another important milestone in our preparations to make LUMEVOQ available to LHON patients as soon as possible.
  • GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders.
  • A Promising Innovative Medicine (PIM) designation is an early indication that a medicine is a potential candidate for the EAMS scheme.
  • A PIM designation should not be regarded as a future commitment by the MHRA to license such a medicine.

GenSight Biologics Announces Publication of RESTORE Study Data Demonstrating Sustained Efficacy 3 Years After Unilateral Injection of LUMEVOQ®

Retrieved on: 
Tuesday, August 31, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), RESCUE, REVERSE, and RESTORE, GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC), RESCUE, REVERSE, AND RESTORE

Figure 1. Evolution of best-corrected visual acuity (BCVA) in ND4-LHON subjects who received unilateral injection of LUMEVOQ® gene therapy --- BCVA: best-corrected visual acuity, CI: confidence interval; LOESS: locally estimated scatterplot smoothing, LogMAR: logarithm of the minimal angle resolution. The LOESS curve shows the evolution of BCVA in all eyes (LUMEVOQ®- and sham-treated) from 12 months to 51.5 months (last available data point) after onset of vision loss. The curve starts at 12 months after onset when 92.7% of eyes in RESCUE and REVERSE had received treatment, either with LUMEVOQ® or a sham injection. (Photo: Business Wire)

Key Points: 
  • The curve starts at 12 months after onset when 92.7% of eyes in RESCUE and REVERSE had received treatment, either with LUMEVOQ or a sham injection.
  • Mean BCVA steadily improved to 1.26 LogMAR at 48 months after onset (3 year-post injection), remaining onchart (i.e., better than 1.6 LogMAR) throughout the follow-up period.
  • A locally-estimated scatterplot smoothing (LOESS) regression analysis illustrates the progressive and sustained improvement of BCVA in RESTORE subjects (Figure 1) since treatment with LUMEVOQ.
  • The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment.

GenSight Biologics Announces Approval of the LUMEVOQ® Cohort Temporary Authorization for Use (ATUc) in France

Retrieved on: 
Monday, July 5, 2021
Biotechnology, Pharmaceutical, Health, Life sciences, Biotechnology, Health sciences, European Medicines Agency, European Union, Biopharmaceutical, Quinze-Vingts National Ophthalmology Hospital, Medicine in China, Medicine, GenSight Biologics, Leber Hereditary Optic Neuropathy (LHON), LUMEVOQ® (GS010; lenadogene nolparvovec), GENSIGHT BIOLOGICS, LEBER HEREDITARY OPTIC NEUROPATHY (LHON), LUMEVOQ® (GS010; LENADOGENE NOLPARVOVEC)

The Cohort ATU greatly simplifies the process by which patients gain access to LUMEVOQ prior to EU marketing authorization expected in H1 2022.

Key Points: 
  • The Cohort ATU greatly simplifies the process by which patients gain access to LUMEVOQ prior to EU marketing authorization expected in H1 2022.
  • French hospital-based physicians, including those practicing outside the Quinze-Vingts Hospital in Paris, will now be able to request treatment for eligible patients directly from GenSight Biologics.
  • Under the ATUc, GenSight Biologics will provide LUMEVOQ to hospitals at a price similar to that in the current ATUn.
  • LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.