EVERY BREAKTHROUGH BEGINS WITH BELIEF January 2026 Corporate Presentation Disclaimer Forward Looking Statements This communication contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and ...

Core Insights - Ocugen, Inc. announced positive Phase 1 GARDian1 trial results for OCU410ST, a novel modifier gene therapy for Stargardt disease, published in the peer-reviewed journal Nature Eye [1][2] Group 1: Clinical Trial Results - The Phase 1 GARDian1 trial demonstrated robust efficacy and safety outcomes, indicating a favorable safety and tolerability profile for OCU410ST [3][4] - Among six patients with gradable Fundus Auto Fluorescence images, atrophic lesion growth was reduced by 54% in treated eyes compared to untreated eyes over 12 months [5] - Treated eyes showed a 50% slower lesion expansion rate compared to untreated eyes, below published natural history rates [5] - Visual acuity improved in treated eyes, with a gain of +6 letters in Best Corrected Visual Acuity (BCVA) compared to a decline in untreated eyes [5] - No drug-related serious adverse events were reported during the trial [5] Group 2: Disease Background - Stargardt disease is the most common form of inherited macular degeneration, affecting over 100,000 individuals in the U.S. and Europe, characterized by progressive central vision loss [3][7] - Currently, there are no FDA-approved treatments for Stargardt disease, highlighting a critical unmet medical need [3][7] Group 3: Future Developments - The ongoing Phase 2/3 GARDian3 trial is progressing ahead of schedule, with anticipated enrollment completion in the first quarter of 2026 [4] - The company plans to file a Biologics License Application (BLA) in the first half of 2027, aiming for three regulatory submissions within three years [4] Group 4: Technology Overview - OCU410ST utilizes an AAV5 delivery platform to deliver the RORA gene to the retina, addressing multiple pathophysiological pathways linked to Stargardt disease [6] - The therapy aims to provide a gene-agnostic modification strategy that could benefit patients regardless of their underlying ABCA4 mutation [4][6] Group 5: Company Overview - Ocugen, Inc. focuses on discovering and developing novel gene therapies for blindness diseases, aiming to address significant unmet medical needs through innovative approaches [8]

Core Viewpoint - Sangamo Therapeutics, Inc. (NASDAQ:SGMO) is experiencing a stabilization in its share price following a significant pipeline update, despite a year-long decline of nearly 64% in its stock value [2]. Group 1: Pipeline Update - On December 18, 2025, Sangamo announced the initiation of a rolling Biologics License Application (BLA) submission to the U.S. FDA for isaralgagene civaparvovec (ST-920) aimed at adults with Fabry disease, which could expedite the regulatory process [3]. - The registration STAAR study data supports the BLA, showing ST-920's potential as a one-time, durable gene therapy, with the FDA validating that the mean annualized eGFR slope at 52 weeks can serve as an accelerated approval endpoint [4]. Group 2: Company Overview - Sangamo Therapeutics is a clinical-stage biotech company focused on developing genomic medicines for inherited metabolic, neurologic, and immune diseases, utilizing gene therapy and genome engineering platforms for potentially curative treatments [5].

Core Insights - Sangamo Therapeutics, Inc. (NASDAQ:SGMO) has experienced a significant decline in share price, down nearly 64% over the past year, but has recently stabilized with a 7.19% gain over the last five trading sessions [2] - The company is advancing its lead gene therapy program, seeking accelerated approval for isaralgagene civaparvovec (ST-920) for adults with Fabry disease through a rolling Biologics License Application (BLA) submission to the FDA [3] - The STAAR study data supports the BLA, indicating that ST-920 may serve as a one-time, durable gene therapy, with the FDA validating the use of mean annualized eGFR slope at 52 weeks as an accelerated approval endpoint [4] Company Overview - Sangamo Therapeutics is a clinical-stage biotech company focused on developing genomic medicines for inherited metabolic, neurologic, and immune diseases, utilizing gene therapy and genome engineering platforms [5]

Core Insights - REGENXBIO Inc. is poised for a transformative year in 2026, entering the commercial stage with two near-term catalysts from its late-stage assets and a clear path to sustained growth [2] - The company has reported positive long-term data for its Duchenne program, indicating effective therapeutic benefits across its gene therapy pipeline [2] Clinical Program Updates and 2026 Anticipated Milestones - New functional data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202 shows that all four patients exceeded expected disease trajectory, improving an average of 7.4 points on the North Star Ambulatory Assessment (NSAA) [3] - The company plans to present additional safety, biomarker, and functional data at the MDA Clinical and Scientific Conference in March 2026 [3] - REGENXBIO expects to submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026, following the completion of enrollment in the pivotal trial [7] Regulatory and Commercial Readiness - The FDA PDUFA target date for the Duchenne program is February 8, 2026, with potential approval leading to a Priority Review Voucher (PRV) [7] - REGENXBIO is collaborating with Nippon Shinyaku for the commercialization of clemidsogene lanparvovec (RGX-121) upon potential approval [7] - The company is enhancing its manufacturing capabilities at its Manufacturing Innovation Center in Rockville, Maryland, to support commercial launches [8] Gene Therapy Advancements - REGENXBIO is advancing its AAV gene delivery technology through capsid discovery and engineering, approaching IND readiness for treating geographic atrophy [9] - The company is on track to make surabgene lomparvovec (sura-vec) the first gene therapy for wet age-related macular degeneration (AMD) [12] - A two-part Phase IIb/III trial for sura-vec in diabetic retinopathy is set to begin, with a $100 million milestone payment from AbbVie expected upon the first patient dosing [12]

Core Insights - Benitec Biopharma Inc. announced positive results from the 24-month follow-up of the first patient treated with BB-301 in a clinical study for oculopharyngeal muscular dystrophy (OPMD), showing robust disease-modifying outcomes [1][3][4] Group 1: Clinical Study Results - Patient 1 demonstrated deepening improvements in post-swallow pharyngeal residue and total dysphagic symptom burden at the 24-month follow-up compared to both pre-treatment and 12-month follow-up assessments [1][3][4] - All four patients in Cohort 1 completed the 12-month follow-up, with each showing durable responses to BB-301, meeting pre-specified statistical criteria for improvement [1][4][8] - Patient 1 exhibited a 27% improvement in Pharyngeal Area at Maximum Constriction (PhAMPC) at the 12-month mark, which was maintained at the 24-month follow-up [5] - Patient 1 showed a 35% reduction in post-swallow residue in the vallecular region at 12 months, improving to a 60% reduction at 24 months [7] - Total Pharyngeal Residue (TPR) improved by 32% at 12 months and 39% at 24 months, indicating enhanced swallowing efficiency over time [7] Group 2: Patient-Reported Outcomes - The Sydney Swallow Questionnaire (SSQ) total score indicated a 64% reduction in total dysphagic symptom burden at 12 months, improving to a 78% reduction at 24 months [7] - The Responder Analysis framework was utilized to evaluate the efficacy of BB-301, with all four patients classified as formal responders based on their improvements across multiple assessment categories [6][8] Group 3: Company Overview and Future Plans - Benitec Biopharma is focused on developing novel genetic medicines using its proprietary "Silence and Replace" platform, targeting chronic and life-threatening conditions like OPMD [10][11] - The company plans to engage with the U.S. Food and Drug Administration (FDA) in mid-2026 to confirm the pivotal study design for BB-301 and will present interim clinical results at future medical conferences [2]

Core Insights - Taysha Gene Therapies has initiated dosing in the REVEAL pivotal trial for TSHA-102, a gene therapy for Rett syndrome, with the first patient dosed in Q4 2025 and enrollment progressing across multiple sites [1][2] - The company has reached written alignment with the FDA regarding the inclusion of at least three months of safety data from the ASPIRE trial in the planned Biologics License Application (BLA) submission, aiming for a broad label for patients aged two years and older with Rett syndrome [2][5] - Completion of dosing in both the REVEAL and ASPIRE trials is anticipated by Q2 2026, with longer-term safety and efficacy data updates expected in H1 2026 [1][6] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on developing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases affecting the central nervous system [9] - The lead program, TSHA-102, is designed as a one-time treatment targeting the genetic root cause of Rett syndrome by delivering a functional form of the MECP2 gene [7] - The company has received multiple designations from the FDA, including Breakthrough Therapy and Fast Track, indicating its potential significance in addressing unmet medical needs [7] Disease Context - Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by severe developmental regression and loss of motor and communication skills [8] - It is estimated that between 15,000 and 20,000 patients in the U.S., EU, and U.K. are affected by Rett syndrome, highlighting a significant market opportunity for effective treatments [2][8]

Core Viewpoint - GenSight Biologics has received authorization from Israel's Ministry of Health for the early access treatment of its gene therapy candidate GS010/LUMEVOQ® for individual patients [1] Company Summary - GenSight Biologics is a biopharma company focused on developing and commercializing innovative gene therapies specifically for retinal neurodegenerative diseases and central nervous system disorders [1]

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Group 1 - UniQure (NASDAQ:QURE) is considered a promising small-cap stock with significant upside potential, despite recent price target reductions by analysts [1][2] - Stifel reduced its price target for UniQure from $50 to $40 while maintaining a Buy rating, citing refreshed diligence on biotechnology companies [1] - Mizuho analyst Uy Ear lowered the price target from $60 to $33 but kept an Outperform rating, indicating a decreased probability of success for the company's AMT-130 therapy [2] Group 2 - UniQure announced on December 4 that it received final meeting minutes from the FDA regarding the pre-Biologics License Application (BLA) meeting for AMT-130, an investigational gene therapy for Huntington's disease [3] - The FDA indicated that the data from Phase I/II studies of AMT-130 is unlikely to support a BLA submission, prompting UniQure to plan a follow-up meeting with the FDA for fiscal Q1 2026 [4] - UniQure focuses on developing innovative gene therapies for conditions such as Huntington's disease, hemophilia, and cardiovascular issues [5]