Core Insights - MeiraGTx Holdings plc has made significant progress in its clinical programs, including alignment with the FDA on the Phase 2 study of AAV-hAQP1 for radiation-induced xerostomia, which is expected to support a Biologics License Application (BLA) filing by late 2026 [1][2] - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD, a gene therapy for Parkinson's disease, indicating its potential to address serious conditions [1][5] - The company is preparing to file for Marketing Authorization Approval (MAA) in the UK and BLA in the US for AAV-AIPL1, targeting LCA4, with submissions expected in Q4 2025 [1][2] Regulatory Developments - The company has had productive interactions with the FDA and MHRA regarding multiple late-stage clinical programs and manufacturing processes [2] - AAV-GAD has shown significant disease-modifying effects in clinical trials, with plans to initiate a Phase 3 study later this year [1][6] - The FDA has completed a Good Clinical Practice inspection of the AAV-GAD bridging study, resulting in a clean inspection with no observations [6][8] Financial Performance - As of June 30, 2025, the company reported cash and cash equivalents of approximately $32.2 million, a decrease from $101.0 million a year earlier [18][19] - Service revenue increased to $3.7 million for Q2 2025, up from $0.3 million in Q2 2024, attributed to progress in process performance qualification services [19][22] - The net loss attributable to ordinary shareholders for Q2 2025 was $38.8 million, compared to a net loss of $48.6 million in Q2 2024, indicating an improvement in financial performance [28][32] Clinical Programs and Collaborations - The company is advancing its late-stage clinical programs, including AAV-hAQP1 for xerostomia and AAV-GAD for Parkinson's disease, with pivotal data readouts expected in late 2026 [1][2] - MeiraGTx is collaborating with Hologen to develop AAV-GAD, with a joint venture established to finance the program and utilize AI for data analysis [6][8] - The company is also preparing to initiate first-in-human studies using its riboswitch platform by the end of 2025 [2][20] Manufacturing and Technology - MeiraGTx has developed comprehensive in-house manufacturing capabilities, with facilities in the UK and Ireland holding necessary regulatory authorizations [15][17] - The company is focusing on optimizing its riboswitch gene regulation technology for various therapeutic applications, including metabolic diseases and chronic pain [29][30] - The manufacturing process for AAV2-hAQP1 will be in-house, ensuring consistency across different indications [5][8]