AGTC 
Welcome,         Profile    Billing    Logout  
 7 Products   10 Diseases   7 Products   7 Trials   120 News 


12»
  • ||||||||||  laruparetigene zovaparvovec (AGTC-501) / AGTC
    Clinical, Journal:  Addressing Multiplicity in Retinal Sensitivity Analysis: An Alternative Approach to Assessing Gene Therapy Efficacy in Inherited Retinal Diseases. (Pubmed Central) -  Mar 27, 2025   
    We analyzed microperimetry data from a phase II trial of AGTC-501 in patients with X-linked retinitis pigmentosa (XLRP)...Using ?7 unspecified loci as the criterion for assessing sensitivity changes, offers a comprehensive assessment that can detect genuine treatment effects without being overly conservative. This alternative statistical method has the potential to improve the evaluation of retinal sensitivity changes in gene therapy trials for IRDs, providing a more accurate measure of therapeutic efficacy and enhancing clinical decision making.
  • ||||||||||  rAAV2tYF-CB-hRS1 / AGTC
    Trial completion:  Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) -  Feb 14, 2025   
    P1/2,  N=12, Completed, 
    Further studies are warranted to assess long-term safety and efficacy of AGTC-501 for XLRP treatment. Active, not recruiting --> Completed
  • ||||||||||  Luxturna (voretigene neparvovec-rzyl) / Novartis, Roche, laruparetigene zovaparvovec (AGTC-501) / AGTC
    Preclinical, Review, Journal:  Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the Horizon. (Pubmed Central) -  Feb 13, 2025   
    While early successes like LUXTURNA have set the precedent for gene therapy in retinal diseases, adapting these strategies to XLRP presents unique challenges due to the complexity of RPGR mutations and the need for efficient photoreceptor targeting...Despite setbacks in some studies, ongoing research and clinical trials continue to refine these therapies, offering hope for patients affected by XLRP. This review explores the etiology and pathophysiology of XLRP, evaluates current treatment challenges, highlights recent clinical advances in gene therapy, and discusses future perspectives for bringing these therapies into clinical practice.
  • ||||||||||  laruparetigene zovaparvovec (AGTC-501) / AGTC
    Enrollment change, Trial completion date, Trial primary completion date:  AGTC-RPGR-001: A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN) (clinicaltrials.gov) -  Oct 29, 2024   
    P2,  N=24, Enrolling by invitation, 
    This review explores the etiology and pathophysiology of XLRP, evaluates current treatment challenges, highlights recent clinical advances in gene therapy, and discusses future perspectives for bringing these therapies into clinical practice. N=18 --> 24 | Trial completion date: Aug 2029 --> Dec 2029 | Trial primary completion date: Jul 2025 --> Nov 2025
  • ||||||||||  laruparetigene zovaparvovec (AGTC-501) / AGTC
    Enrollment open, Trial completion date, Trial primary completion date:  A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP (clinicaltrials.gov) -  Mar 29, 2024   
    P2/3,  N=75, Recruiting, 
    In conclusion, this study will offer a foundation for exploring the role of the HMAs gene family in dealing with heavy metal stress conditions in A. catechu. Not yet recruiting --> Recruiting | Trial completion date: Mar 2029 --> Oct 2029 | Trial primary completion date: Jan 2024 --> Aug 2025
  • ||||||||||  laruparetigene zovaparvovec (AGTC-501) / AGTC
    Enrollment closed, Enrollment change, Trial completion date, Trial primary completion date:  HORIZON: Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (clinicaltrials.gov) -  Feb 19, 2024   
    P1/2,  N=29, Active, not recruiting, 
    These findings offer a promising path for clinical translation to human trials. Recruiting --> Active, not recruiting | N=42 --> 29 | Trial completion date: Aug 2026 --> Mar 2025 | Trial primary completion date: Aug 2022 --> Nov 2023
  • ||||||||||  rAAV2tYF-CB-hRS1 / AGTC
    Trial primary completion date:  Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) -  Nov 18, 2023   
    P1/2,  N=12, Active, not recruiting, 
    Recruiting --> Active, not recruiting | N=42 --> 29 | Trial completion date: Aug 2026 --> Mar 2025 | Trial primary completion date: Aug 2022 --> Nov 2023 Trial primary completion date: Jun 2021 --> Jul 2025
  • ||||||||||  rAAV2tYF-CB-hRS1 / AGTC
    Trial completion, Trial completion date:  Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS) (clinicaltrials.gov) -  Jun 12, 2023   
    P1/2,  N=27, Completed, 
    The ability of the novel AAV.SPR capsid to effectively deliver the RS1 to the central retina of primates following extrafoveal SRI represents a safer and potentially more effective treatment option for XLRS patients. Active, not recruiting --> Completed | Trial completion date: Oct 2023 --> May 2023
  • ||||||||||  BS01 / Bionic Sight, AGTC
    Trial completion date, Trial primary completion date:  BS01 in Patients With Retinitis Pigmentosa (clinicaltrials.gov) -  May 3, 2023   
    P1/2,  N=20, Recruiting, 
    Active, not recruiting --> Completed | Trial completion date: Oct 2023 --> May 2023 Trial completion date: Dec 2027 --> Dec 2029 | Trial primary completion date: Dec 2022 --> Dec 2024
  • ||||||||||  AGTC-402 / AGTC
    Enrollment closed, Trial completion date, Trial primary completion date, Gene therapy:  Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial) (clinicaltrials.gov) -  Jul 23, 2022   
    P1/2,  N=24, Active, not recruiting, 
    The htsFLT01 gene construct is a novel anti-angiogenic tool with promising improvements compared to existing treatments. Recruiting --> Active, not recruiting | Trial completion date: Sep 2025 --> Aug 2026 | Trial primary completion date: Sep 2021 --> Aug 2022
  • ||||||||||  rAAV2tYF-CB-hRS1 / AGTC
    Trial primary completion date:  Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) -  May 26, 2022   
    P1/2,  N=12, Active, not recruiting, 
    The clinical trial is ongoing through 5-year follow-up to assess long-term safety. Trial primary completion date: Jul 2025 --> Jun 2021
  • ||||||||||  rAAV2tYF-CB-hRS1 / AGTC, human Rhodopsin / Kubota
    Laterally Spreading AAV.SPR-hRS1 Vector for Treatment of XLRS (Poster Board Number: M-177; Hall D) -  May 6, 2022 - Abstract #ASGCT2022ASGCT_1556;    
    AAV.SPR-hGRK1-hRS1 leads to therapeutic levels of RS1 in subretinally injected RS1KO mice, resulting in improvements in both retinal structure and function. In addition, AAV.SPR safely and efficiently mediated RS1 expression in the central retina of primate eyes following peripheral SRI.
  • ||||||||||  rAAV2tYF-CB-hRS1 / AGTC
    Laterally Spreading AAV.SPR-hRS1 Vector for Treatment of XLRS (A0341) -  Apr 29, 2022 - Abstract #ARVO2022ARVO_1531;    
    AAV.SPR delivered therapeutic levels of hRS1 to subretinally injected RS1KO mice, resulting in improvements in both retinal structure and function. In addition, AAV.SPR safely and efficiently mediated hRS1 expression in the central retina of primate eyes following peripheral subretinal injection.
  • ||||||||||  rAAV2tYF-CB-hRS1 / AGTC
    Enrollment closed, Enrollment change:  Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis (clinicaltrials.gov) -  Apr 22, 2022   
    P1/2,  N=12, Active, not recruiting, 
    Further study is needed to understand the bilateral nature of the LDT improvement. N=24 --> 12 | Recruiting --> Active, not recruiting
  • ||||||||||  BS01 / Bionic Sight, AGTC
    Trial completion date, Trial primary completion date:  BS01 in Patients With Retinitis Pigmentosa (clinicaltrials.gov) -  Aug 18, 2021   
    P1/2,  N=20, Recruiting, 
    These results support the selection and use of rAAV2tYF-GRK1-hRPGRco (AGTC-501) and guided the initial doses in clinical studies in patients with XLRP caused by RPGR mutations. Trial completion date: Jun 2026 --> Dec 2027 | Trial primary completion date: Jun 2022 --> Dec 2022
  • ||||||||||  AGTC-402 / AGTC
    Clinical, Journal:  Paternal Uniparental Isodisomy of Chromosome 2 in a Patient with CNGA3-Associated Autosomal Recessive Achromatopsia. (Pubmed Central) -  Aug 18, 2021   
    This is one of few examples proving UPD as the underlying mechanism for the clinical manifestation of a recessive mutation in a patient with inherited retinal disease. It also highlights the importance of segregation analysis in both parents of a given patient or especially in cases of homozygous recessive mutations, as UPD has significant implications for genetic counseling with a very low recurrence risk assessment in such families.
  • ||||||||||  LX101 / Innostellar Biotherap
    New trial, Gene therapy:  RPE65 Gene Therapy ?LX101?for Leber's Congenital Amaurosis (clinicaltrials.gov) -  Feb 1, 2021   
    P=N/A,  N=9, Not yet recruiting, 
  • ||||||||||  laruparetigene zovaparvovec (AGTC-501) / AGTC
    Enrollment change, Trial completion date, Trial primary completion date:  HORIZON: Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (clinicaltrials.gov) -  Jan 29, 2021   
    P1/2,  N=42, Recruiting, 
    These preclinical results with AAV-mediated GJB2 gene therapy support the use of OTO-825 as a clinical candidate to treat congenital hearing loss caused by GJB2 deficiency. N=30 --> 42 | Trial completion date: Mar 2025 --> Aug 2026 | Trial primary completion date: Mar 2023 --> Aug 2022
  • ||||||||||  rAAV2-CB-hRPE65 / AGTC
    Journal:  Optimal Inhibition of Choroidal Neovascularization by scAAV2 with VMD2 Promoter-driven Active Rap1a in the RPE. (Pubmed Central) -  Nov 7, 2020   
    scAAV2-VMD2, but not scAAV2-RPE65, specifically and efficiently transduced the RPE to increase active Rap1a protein in the RPE...Increased active Rap1a in the RPE in vivo or in vitro inhibited inflammatory and angiogenic signaling determined by decreased activation of NF-κB and expression of VEGF without causing increased cell death or autophagy measured by increased LCA3/B. Our study provides a potential future strategy to deliver active Rap1a to the RPE in order to protect against both atrophic and neovascular AMD.