- |||||||||| 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.
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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
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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
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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
- |||||||||| 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.
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