botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J 
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  • ||||||||||  botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J
    Journal, Gene therapy:  AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis Pigmentosa. (Pubmed Central) -  Feb 10, 2024   
    In addition, treatment with AAV-RPGR restored rhodopsin localisation within RPGR-KO ROs, reducing mislocalisation to the photoreceptor outer nuclear layer. These data provide mechanistic insights into RPGRORF15 gene supplementation functional potency in human photoreceptor cells and support the previously reported Phase I/II trial positive results using this vector construct in patients with RPGR-associated XLRP, which is currently being tested in a Phase III clinical trial.
  • ||||||||||  botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J
    Enrollment closed, Gene therapy:  MGT-RPGR-022: Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) -  Jan 24, 2024   
    P3,  N=97, Active, not recruiting, 
    These data provide mechanistic insights into RPGRORF15 gene supplementation functional potency in human photoreceptor cells and support the previously reported Phase I/II trial positive results using this vector construct in patients with RPGR-associated XLRP, which is currently being tested in a Phase III clinical trial. Recruiting --> Active, not recruiting
  • ||||||||||  botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J
    Enrollment change, Gene therapy:  Long-term Follow-up Gene Therapy Study for RPGR- XLRP (clinicaltrials.gov) -  Nov 21, 2023   
    P=N/A,  N=55, Active, not recruiting, 
    Trial primary completion date: Sep 2024 --> Sep 2025 N=42 --> 55
  • ||||||||||  botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J
    Enrollment closed, Trial completion date, Trial primary completion date, Gene therapy:  Long-term Follow-up Gene Therapy Study for RPGR- XLRP (clinicaltrials.gov) -  Sep 28, 2023   
    P1/2,  N=42, Active, not recruiting, 
    Active, not recruiting --> Recruiting | Trial completion date: Sep 2024 --> Feb 2025 Recruiting --> Active, not recruiting | Trial completion date: Jun 2023 --> Nov 2026 | Trial primary completion date: Jun 2023 --> Nov 2026
  • ||||||||||  botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J
    Enrollment closed, Trial completion date, Trial primary completion date, Gene therapy:  MGT-RPGR-022: Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) -  Sep 22, 2023   
    P3,  N=97, Active, not recruiting, 
    Recruiting --> Active, not recruiting | Trial completion date: Jun 2023 --> Nov 2026 | Trial primary completion date: Jun 2023 --> Nov 2026 Recruiting --> Active, not recruiting | Trial completion date: May 2029 --> Sep 2029 | Trial primary completion date: May 2029 --> Sep 2029
  • ||||||||||  botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J
    Enrollment closed, Trial completion date, Trial primary completion date, Gene therapy:  Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) -  Sep 22, 2023   
    P3,  N=97, Active, not recruiting, 
    Recruiting --> Active, not recruiting | Trial completion date: May 2029 --> Sep 2029 | Trial primary completion date: May 2029 --> Sep 2029 Recruiting --> Active, not recruiting | Trial completion date: Mar 2024 --> Sep 2024 | Trial primary completion date: Mar 2024 --> Sep 2024
  • ||||||||||  botaretigene sparoparvovec (AAV-RPGR) / MeiraGTx, J&J
    Enrollment change, Trial completion date, Trial primary completion date, Gene therapy:  Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene (clinicaltrials.gov) -  Dec 7, 2022   
    P3,  N=96, Recruiting, 
    N=66 --> 96 | Trial completion date: Oct 2028 --> May 2029 | Trial primary completion date: Oct 2024 --> May 2029 N=66 --> 96 | Trial completion date: Oct 2023 --> Mar 2024 | Trial primary completion date: Oct 2023 --> Mar 2024
  • ||||||||||  AAV-RPGR / MeiraGTx, J&J
    Journal:  RPGR gene therapy presents challenges in cloning the coding sequence. (Pubmed Central) -  Oct 16, 2020   
    Introduction: Currently, there are three Phase I/II clinical trials based on gene therapy ongoing to test different AAV.RPGR or deleted RPGR vectors on patients affected by X-linked retinitis pigmentosa...The instability of its sequence and the fact that its function is not well understood, can lead to the production of a non-functional or deleterious protein for the human retina. Since the RPGR protein undergoes post-translational glutamylation in the protein domain that may be particularly affected by gene instability, a functional assay of glutamylation is essential to verify the correct coding sequence.