ND4

GenSight Biologics Announces Update on Real-World Data from Early Access Programs of LUMEVOQ® Gene Therapy at NANOS 2024

Retrieved on: 
Wednesday, March 6, 2024

In both groups, the clinical improvement exceeded the conventional definition of clinical meaningfulness, in which a 15-letter gain is considered meaningful.

Key Points: 
  • In both groups, the clinical improvement exceeded the conventional definition of clinical meaningfulness, in which a 15-letter gain is considered meaningful.
  • The safety profile of the gene therapy continues to be favorable and comparable between bilaterally treated patients and unilaterally treated patients.
  • “Witnessing results like these energizes the GenSight team’s drive to restore early access availability for eligible patients by Q3 this year,” said Laurence Rodriguez, Chief Executive Officer of GenSight.
  • The Company aims to resume early access in France under the Autorisation de l’accès compassionnel (AAC) program in Q3 2024.

GenSight Biologics Announces Presentation of LUMEVOQ® Efficacy and Safety Data from Early Access Programs for ND4-LHON Patients at NANOS 2023

Retrieved on: 
Wednesday, March 15, 2023

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20230314006021/en/
    Figure 1: Global evolution of mean BCVA over two years in patients who received LUMEVOQ® in early access programs.
  • Individual data from 45 out of the 63 patients who had passed the one-year post-treatment point and performed their one-year visit were pooled and analyzed1.
  • The data confirmed the efficacy and safety profile of LUMEVOQ® in a real-world setting.
  • Figure 1: Global evolution of mean BCVA over two years in patients who received LUMEVOQ® in early access programs.

GenSight Biologics Confirms Sustained Efficacy and Safety of Bilateral LUMEVOQ® Injections at 3-Year Follow-Up of REFLECT Phase III Trial

Retrieved on: 
Monday, March 13, 2023

The results show sustained efficacy and favorable safety for bilateral intravitreal injection of the gene therapy with a statistically significant visual acuity improvement from baseline in both treated eyes, showing an additional benefit of a bilateral injection compared to a unilateral injection.

Key Points: 
  • The results show sustained efficacy and favorable safety for bilateral intravitreal injection of the gene therapy with a statistically significant visual acuity improvement from baseline in both treated eyes, showing an additional benefit of a bilateral injection compared to a unilateral injection.
  • "The REFLECT trial has delivered a further set of results demonstrating the sustained benefit of lenadogene nolparvovec, while reconfirming its safety profile " saidNancy J Newman, MD, Professor of Ophthalmology and Neurology at Emory University School of Medicine (Atlanta, USA) and International Principal Investigator of REFLECT. "
  • Vision typically deteriorates to a nadir and stabilises to a level below the threshold for legal blindness.
  • 48 subjects were randomized to LUMEVOQ® bilateral treatment, and 50 to lenadogene nolparvovec unilateral treatment (first-affected eye treated with lenadogene nolparvovec, second-affected eye treated with placebo).

Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON

Retrieved on: 
Monday, December 19, 2022

Professor Bin Li, Founder, Chairman and CEO of Neurophth, said, "We are thrilled to have received IND clearance for NFS-02, marking our second program to reach clinical development.

Key Points: 
  • Professor Bin Li, Founder, Chairman and CEO of Neurophth, said, "We are thrilled to have received IND clearance for NFS-02, marking our second program to reach clinical development.
  • Based on encouraging preclinical data, we believe that this gene therapy drug candidate holds promise to become a safe and effective treatment for patients."
  • Our most advanced investigational gene therapy drug candidate, NR082 (rAAV2-ND4), in development for the treatment of mtND4-mediated LHON, has been granted orphan drug designation (ODD) by the U.S. FDA and EMA.
  • Recently, our second gene therapy drug candidate NFS-02, has been granted IND clearance from U.S. FDA.

AAV Vectors in Gene Therapy - Pipeline Insight, 2022 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, November 22, 2022

The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases.
  • AAV vectors in gene therapy: Therapeutic Assessment
    This segment of the report provides insights about the different AAV vectors in gene therapy drugs segregated based on following parameters that define the scope of the report, such as:
    There are approx.
  • The companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e.

GenSight Biologics reports 5 years’ data showing sustained efficacy and safety following one-time treatment with LUMEVOQ®

Retrieved on: 
Wednesday, July 20, 2022

Year 5 values were the LogMAR readings nearest to 1,825 days post treatment recorded between 1,825 +/- 28 days post-treatment.

Key Points: 
  • Year 5 values were the LogMAR readings nearest to 1,825 days post treatment recorded between 1,825 +/- 28 days post-treatment.
  • Responder analyses at Year 5 indicate that improved BCVA was a benefit for a substantial proportion of the study participants.
  • Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset.
  • The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment.

The 3rd BIO International Convention 2022 is Approaching

Retrieved on: 
Tuesday, June 7, 2022

SAN DIEGO, June7, 2022 /PRNewswire/ --With the 3rd BIO International Convention 2022 soon approaching, Neurophth Therapeutics is looking forward to discussing collaboration opportunities in gene therapy for both rare and common ocular diseases.

Key Points: 
  • SAN DIEGO, June7, 2022 /PRNewswire/ --With the 3rd BIO International Convention 2022 soon approaching, Neurophth Therapeutics is looking forward to discussing collaboration opportunities in gene therapy for both rare and common ocular diseases.
  • Neurophth Therapeutics is a leading clinical-stage gene therapy company with robust ophthalmic pipeline.
  • With subsidiaries in China and USA, Neurophth, a fully integrated company, is striving to discover and develop AAV-delivered gene therapies for patients suffering from vision loss globally.
  • The part 1 of its Chinese Phase 1/2/3 registrational clinical trial has reached positive results and the part 2 (Phase 3) will be initiated in Q3 2022.

Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by EMA

Retrieved on: 
Tuesday, January 25, 2022

Food and Drug Administration (FDA) for this same gene therapy drug candidateas a potential treatment for mtND4-LHON," said Xin Zhang, M.D., M.Sc., Chief Medical Officer and Chief Operations Officer at Neurophth.

Key Points: 
  • Food and Drug Administration (FDA) for this same gene therapy drug candidateas a potential treatment for mtND4-LHON," said Xin Zhang, M.D., M.Sc., Chief Medical Officer and Chief Operations Officer at Neurophth.
  • Applications for orphan designation are examined by the EMA's Committee for Orphan Medicinal Products (COMP), using the Committee's network of experts.
  • Our most advanced investigational gene therapy drug candidate, NR082 (rAAV2-ND4), in development for the treatment ofmtND4-mediated LHON, has been granted orphan drug designation (ODD) by theU.S.
  • Long-term outcomes of gene therapy for the treatment of Leber's hereditary optic neuropathy.

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

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.

Neurophth Therapeutics Receives IND Clearance to Initiate Clinical Trial for China AAV-ND4 Gene Therapy NR082 in Leber Hereditary Optic Neuropathy

Retrieved on: 
Wednesday, January 19, 2022

"We are eager to initiate the enrollment of our US clinical trial this year, which will be a historic milestone as the first ever China originated AAV gene therapy clinical trial in the US.

Key Points: 
  • "We are eager to initiate the enrollment of our US clinical trial this year, which will be a historic milestone as the first ever China originated AAV gene therapy clinical trial in the US.
  • With this IND clearance by the FDA, we are embarking on a new pathway of care through this innovative gene therapy."
  • "NR082 is the first China AAV gene therapy for the LHON to receive ODD from the U.S. FDA.
  • In addition, this is the first of all China AAV gene therapy products to get an IND approval from the U.S.