Financial Data and Key Metrics Changes - The company is targeting three Biologics License Applications (BLAs) in the next three years, starting with OCU 400 in 2026 for retinitis pigmentosa [2][4] - The company has a cash runway of approximately $50 million, with a recent $20 million injection into the balance sheet and a potential additional $30 million option [52][53] Business Line Data and Key Metrics Changes - OCU 400 is in Phase 3 trials, with a primary endpoint focused on baseline changes in lux levels and secondary endpoints including low luminance visual acuity [9][17] - OCU 410 for Stargardt disease is in a Phase 2/3 registration trial with an adaptive design, aiming for a reduction in lesion size and improvement in visual acuity [31][35] Market Data and Key Metrics Changes - The company is the only one targeting a broad indication for retinitis pigmentosa, potentially addressing 98-99% of patients with unmet medical needs [6][10] - The geographic atrophy (GA) program is positioned against existing approved drugs, with a focus on both structural and functional outcomes [40][41] Company Strategy and Development Direction - The company aims to become a global leader in gene therapies for blindness diseases, focusing on three key programs [46][47] - The divestment of the neocard asset is intended to allow the company to concentrate on gene therapies, with a valuation of over $100 million for the divested assets [48][50] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming BLA submissions and the potential for significant patient impact globally [4][28] - The company is preparing for regulatory submissions in both the US and Europe, with EMA agreeing to the US trial design [27][28] Other Important Information - The company has established safety and efficacy in prior trials, with no serious adverse events reported [16][35] - The company is efficiently run compared to many biotechs, with a focus on maximizing resources for its gene therapy programs [52] Q&A Session Summary Question: What is the progress on the OCU 400 study and when will top-line data be available? - The data will be available next year, and the company is on track to file for the BLA in 2026 [23][24] Question: What is Stargardt disease and what is the status of the OCU 410 program? - Stargardt disease is an inherited retinal disease primarily affecting the pediatric population, and the OCU 410 program is in a Phase 2/3 trial with promising early data [30][31] Question: How does the company plan to address the competitive landscape for geographic atrophy? - The company is focusing on both structural and functional outcomes, with promising data from Phase 1 and Phase 2 trials [40][41] Question: What is the company's strategy regarding the divestment of the neocard asset? - The divestment allows the company to focus on gene therapies, with a significant valuation for the divested assets that could enhance shareholder value [48][50] Question: What is the current strength of the company's balance sheet? - The company has a cash runway of approximately $50 million, bolstered by recent funding [52][53]