Core Insights - Bayer's subsidiary, BlueRock Therapeutics, has initiated the first patient treatment in the phase I/IIa CLARICO study with OpCT-001, an investigational iPSC-derived cell therapy for primary photoreceptor diseases [1][9] - OpCT-001 is the first iPSC-derived cell therapy tested in humans for inherited eye diseases, including retinitis pigmentosa and cone-rod dystrophy, which can cause vision loss [2][9] - The CLARICO study aims to evaluate the safety, tolerability, and clinical outcomes of OpCT-001, with a focus on safety in the phase I portion and additional safety and visual function data in the phase II part [4][9] Bayer's Stock Performance - Year-to-date, Bayer's shares have increased by 56.6%, contrasting with a 0.7% decline in the industry [7] Pipeline Developments - Bayer has expanded its pipeline to include cell therapy through the acquisition of BlueRock and gene therapy through AskBio, targeting various diseases including retinal disorders and Parkinson's disease [10] - The FDA has granted Fast Track designation to OpCT-001 for treating primary photoreceptor diseases, indicating potential for significant therapeutic advancements [11] Other Developments - Bayer is also developing bemdaneprocel (BRT-DA01) in a phase III study for Parkinson's disease, which has received Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA [12]

Core Insights - Rocket Pharmaceuticals (RCKT) has received FDA clearance for its investigational new drug application to initiate clinical studies for its gene therapy candidate RP-A701, aimed at treating BAG3-associated dilated cardiomyopathy [1][6] - The planned phase I study will assess the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM, a rare genetic heart condition [2][6] Company Pipeline - RCKT is also developing another gene therapy candidate, RP-A601, which is in early-stage development for arrhythmogenic cardiomyopathy [3] - Recent setbacks in the company's pipeline, including a voluntary pause in the phase II study of RP-A501 due to a patient death, have raised concerns [4][7] - The FDA has issued a complete response letter regarding the biologics license application for Kresladi, requesting additional information [8] Stock Performance - Year to date, RCKT's shares have declined by 80.5%, contrasting with a 3.6% decline in the industry [3] - The company currently holds a Zacks Rank of 3 (Hold), while other biotech stocks like Exelixis, Spero Therapeutics, and Puma Biotechnology have better rankings [9]

Core Insights - Klotho Neurosciences, Inc. is advancing the manufacturing and process development for KLTO-202, a gene therapy aimed at treating amyotrophic lateral sclerosis (ALS) [1] - The company has licensed a unique RNA splice variant of the alpha-Klotho gene from the Autonomous University of Barcelona, which is crucial for developing gene therapies [2] - Animal studies have shown that amplifying the levels of secreted alpha-Klotho (s-KL) through gene therapy leads to favorable therapeutic outcomes in models of ALS and other neurodegenerative diseases [2] - The company anticipates an eight-month timeline to complete process development and manufacturing, followed by four to six months for FDA-related activities, aiming to start clinical trials in ALS patients by Q3 of the following year [3] - The CEO highlighted a more efficient method for producing the AAV vector to deliver the s-KL gene directly to motor neurons, which are significantly affected by ALS [4] Company Overview - Klotho Neurosciences, Inc. focuses on innovative cell and gene therapies derived from the human Klotho gene, targeting neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease [5] - The company's portfolio includes proprietary cell and gene therapy programs utilizing DNA and RNA therapeutics, along with genomics-based diagnostic assays [5] - The management team consists of experienced individuals in biopharmaceutical product development and commercialization [5]

Core Viewpoint - Genprex, Inc. has presented promising preclinical research on its diabetes gene therapy candidate GPX-002, demonstrating its potential to improve glucose homeostasis by reprogramming alpha cells into beta-like cells, as showcased at the 2025 American Diabetes Association Scientific Session [1][2]. Group 1: Research Findings - The research indicates that alpha cells in animal models of Type 1 diabetes (T1D) can transdifferentiate into beta-like cells after being treated with GPX-002, maintaining improved glucose control for three months [2][6]. - The gene therapy utilizes recombinant adeno-associated virus (rAAV) to deliver Pdx1 and MafA genes directly into the pancreatic duct, effectively converting alpha cells into insulin-secreting beta-like cells without the need for immunosuppression in mouse models [3][12]. - In non-human primate studies, the infusion of rAAV resulted in improved glucose tolerance and reduced insulin requirements one month post-infusion, with ongoing evaluations of immune responses to the therapy [5][8]. Group 2: Clinical Development - Genprex is advancing GPX-002 for both Type 1 and Type 2 diabetes, with the same gene therapy approach applied to both conditions, aiming to rejuvenate exhausted beta cells in Type 2 diabetes [9][12]. - The company is currently conducting preclinical studies to gather additional data on the efficacy of GPX-002 after six months of immunosuppression [8][9]. - The therapy is designed to be administered via a routine endoscopy procedure in humans, enhancing its potential for clinical application [9]. Group 3: Company Overview - Genprex, Inc. is a clinical-stage gene therapy company focused on developing innovative therapies for cancer and diabetes, collaborating with leading institutions to advance its drug candidates [10][11]. - The company’s oncology program includes the Oncoprex® Delivery System, which encapsulates gene-expressing plasmids for intravenous administration, targeting tumor cells [11]. - Genprex aims to provide new treatment options for patients with limited alternatives, leveraging its gene therapy technologies [10].

Core Insights - ZEVASKYN™ (prademagene zamikeracel) is the first FDA-approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][2][9] - The pivotal Phase 3 VIITAL study demonstrated significant wound healing and pain reduction after a single treatment, with 81% of treated wounds showing at least 50% healing compared to 16% in control wounds [5][13] - The publication of the VIITAL study results in The Lancet marks a significant milestone for Abeona Therapeutics as it prepares for the U.S. launch of ZEVASKYN [3][4] Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being a key product in its portfolio [15] - The company operates a fully integrated cGMP cell and gene therapy manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [15] Study Details - The VIITAL study was an intra-patient randomized, open-label, controlled Phase 3 trial involving 11 patients with RDEB, assessing 43 pairs of large chronic wounds [3][4] - The study's co-primary endpoints of wound healing and pain reduction were successfully met, with significant statistical differences observed [5][13] Clinical Results - At week 24, 65% of treated wounds achieved 75% or more healing compared to 7% of control wounds, indicating a substantial improvement in treatment efficacy [13] - Mean change in wound pain was -3.1 in treated wounds versus -0.9 in control wounds, demonstrating a significant reduction in pain levels [13] Safety Profile - No serious ZEVASKYN-related adverse events were reported, consistent with previous clinical experiences, and no cases of squamous cell carcinoma were observed in treated wounds [13][14]

Financial Status - Adverum held approximately $174 million in cash, cash equivalents, and short-term investments as of June 2024, which is expected to fund operations into late 2025[8] Ixo-vec Clinical Development (Wet AMD) - Ixo-vec demonstrates a potential best-in-class product profile for wet AMD treatment, aiming for a one-time IVT injection to turn the eye into an anti-VEGF biofactory[10] - In the LUNA trial, 76% of patients at the 6E10 dose remained injection-free at 26 weeks, and 88% preferred Ixo-vec to prior injections[10] - The LUNA Phase 2 study results show a 90% to 95% reduction in anti-VEGF treatment burden across both the 6E10 and 2E11 Ixo-vec doses, aligning with OPTIC study results[70] - In the LUNA trial, 76% to 83% of patients were injection-free across both Ixo-vec doses, consistent with the OPTIC trial[73] - Among patients with ≤ 6 prior injections, 90% were injection-free at Week 26 in the LUNA study[78] - 100% of patients previously receiving Faricimab (Vabysmo) remained injection-free across both Ixo-vec doses in the LUNA study[81] - Both Ixo-vec doses in the LUNA study maintained visual and anatomic outcomes through 26 weeks[84] - Local corticosteroid prophylaxis was effective in minimizing inflammation, with 91% of participants having no or minimal inflammation at any study visit through Week 26[105] Market Opportunity - The global wet AMD market is large and growing, with approximately 1.5 million patients in the U S and 20 million worldwide, and about 200,000 new cases annually[14] - The global sales for wet AMD are projected to be around $9 billion by 2025[17] - A 1% market share for a gene therapy like Ixo-vec could translate to approximately $260 million to $440 million in sales, assuming a 3-5 year benefit[18]

Core Insights - Opus Genetics, Inc. has secured up to $2 million in non-dilutive funding from the Retinal Degeneration Fund to advance its OPGx-MERTK program aimed at treating retinitis pigmentosa caused by MERTK gene mutations [1][2] - The funding will support the preclinical development of OPGx-MERTK, which is expected to progress towards Investigational New Drug (IND) enabling studies [2] - Opus anticipates that this funding will extend its cash runway into the second half of 2026, ensuring operational continuity [3] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene and small molecule therapies for inherited retinal diseases (IRDs) [5] - The company's pipeline includes AAV-based gene therapies targeting various IRDs, with its lead candidate OPGx-LCA5 currently in a Phase 1/2 trial [5] - Opus is also advancing therapies for diabetic retinopathy and presbyopia, showcasing a diverse approach to vision-threatening conditions [5] Program Details - The OPGx-MERTK program utilizes a modern AAV vector to target MERTK-associated IRD, which affects approximately 600 individuals in the U.S. [4] - The funding from the RD Fund highlights the collaboration between venture philanthropy and industry to accelerate the development of treatments for rare diseases [2]

Core Insights - Passage Bio, Inc. reported updated data from the Phase 1/2 upliFT-D clinical trial for PBFT02, showing robust and durable increases in cerebrospinal fluid (CSF) progranulin (PGRN) levels and improvements in plasma neurofilament (NfL) levels, indicating potential efficacy in treating frontotemporal dementia (FTD) with granulin mutations [1][2][10] Clinical Trial Data - Dose 1 of PBFT02 increased CSF PGRN levels from below 3 ng/mL at baseline to a mean of 12.4 ng/mL at one month, 19.4 ng/mL at six months, 25.9 ng/mL at 12 months, and 23.8 ng/mL at 18 months [5] - The first patient treated with Dose 2 (50% of Dose 1) saw CSF PGRN levels rise from 1.5 ng/mL at baseline to 7.6 ng/mL at one month, nearing the upper limit of a healthy adult reference range [5] - Plasma NfL levels increased by an average of 4% at 12 months post-treatment, compared to an expected increase of 28% to 29% per year in untreated symptomatic FTD-GRN patients [5] Safety Profile - Treatment emergent adverse events were mostly mild to moderate, with three serious adverse events reported among eight patients, including venous sinus thrombosis and pulmonary embolism [5][6] - No evidence of dorsal root ganglion toxicity was observed, and no complications occurred during the administration of PBFT02 [5] Study Protocol Amendments - The company plans to amend the upliFT-D protocol to include a prophylactic course of low-dose anticoagulation and modify inclusion criteria to allow earlier-stage patients [6][7] - The amended protocol is expected to be submitted to health authorities in early July 2025, with plans to begin enrollment in additional cohorts thereafter [7] Anticipated Milestones - The company aims to seek regulatory feedback on the pivotal trial design for FTD-GRN in the first half of 2026 and report updated interim safety and biomarker data from Dose 2 in the same timeframe [14]

DISCLAIMER Various statements in this presentation concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward- looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Al ...

Financial Data and Key Metrics Changes - In Q1 2025, total revenues reached $3.03 billion with non-GAAP diluted earnings per share at $8.22, reflecting strong financial performance [40]. Business Line Data and Key Metrics Changes - DUPIXENT's net product sales grew 20% globally on a constant currency basis compared to Q1 2024, with a 19% increase in the US, indicating strong demand across all approved indications [40][41]. - EYLEA faced challenges due to new competition, but enhancements are expected to strengthen its market position [41]. - Libtayo grew 21% in the US compared to the previous year, establishing itself as a cornerstone therapy in oncology [42]. Market Data and Key Metrics Changes - The branded anti-VEGF category contracted in Q1 2025, presenting challenges for EYLEA, while Libtayo is gaining market share in advanced non-small cell lung cancer [41][42]. Company Strategy and Development Direction - The company focuses on scientific innovation, investing over 30% of revenues into R&D, which is significantly above the industry average [31]. - Regeneron aims to expand its manufacturing capacity with a $3 billion investment in North Carolina and a $3.6 billion expansion in New York [48][49]. Management's Comments on Operating Environment and Future Outlook - The management acknowledges headwinds in the industry but emphasizes resilience and the importance of advocacy for fair pricing and patient access [33][34]. - The company is optimistic about its pipeline, with approximately 45 candidates in development, and believes in the potential of its existing products to drive future growth [39][43]. Other Important Information - Regeneron has secured regulatory approval for 14 homegrown medicines and has built a robust clinical pipeline [39]. - The company emphasizes the importance of protecting innovations through patents and ensuring that developed nations contribute fairly to the costs of innovative treatments [36][37]. Q&A Session Summary Question: What will happen with the drug last week that failed to meet its primary endpoint in the study? Will you conduct a new drug trial? - The drug candidate ipilimumab showed promising data initially, but efficacy waned during the trial. The company is analyzing the data and considering future steps based on findings [54][56][58]. - The management remains optimistic about the therapeutic candidate's potential and is in discussions with regulators regarding the next steps [59].