Core Thesis - REGENXBIO Inc. presents a bullish investment opportunity due to its strong pipeline, strategic partnerships, and favorable market positioning in gene therapies [1][5]. Company Overview - REGENXBIO is a clinical-stage biotechnology company focused on gene therapies utilizing its proprietary NAV Technology platform, specifically AAV8 and AAV9 vectors [2]. - The lead program, suragene lomparvovec (sura-vec, ABBV-RGX-314), targets wet age-related macular degeneration and diabetic retinopathy, significantly reducing the need for repeated injections [2]. Clinical Programs - The company has the largest retinal gene therapy program to date, with over 1,200 patients enrolled in pivotal trials ATMOSPHERE and ASCENT [3]. - REGENXBIO is also advancing RGX-202 for Duchenne muscular dystrophy and RGX-121 for MPS II, with RGX-121 showing an 82% median reduction in a cerebrospinal fluid biomarker linked to neurodevelopmental stabilization [3]. Financial Position - As of early 2026, REGENXBIO has a solid liquidity position of approximately $302 million, supported by recent non-dilutive financings [4]. - This capital is expected to fund operations into early 2027, excluding significant milestone payments from AbbVie related to late-stage trials [4]. Market Position and Strategy - The company is strategically positioned in competitive markets favoring durable, long-acting therapies, with Phase 2 data in wet AMD showing favorable comparisons to emerging peers [4]. - AbbVie's commercial infrastructure provides a significant execution advantage for REGENXBIO [4]. Upcoming Catalysts - An FDA PDUFA date for RGX-121 is set for February 2026, with pivotal readouts for wet AMD expected in late 2026, indicating a catalyst-rich investment profile [5]. - The current valuation of REGENXBIO is seen as underappreciating the depth of its pipeline and the strength of its partnerships [5].

Core Insights - Ocugen, Inc. announced positive preliminary 12-month data from the Phase 2 ArMaDa clinical trial for OCU410, a gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD) [1][4] - The global prevalence of dAMD is 266 million, with GA affecting approximately 2-3 million people in the U.S. and Europe [1][8] Clinical Trial Findings - Phase 2 results show a 46% reduction in lesion growth compared to control, with a 50% responder rate achieving over 50% lesion size reduction [5][6] - No serious adverse events related to OCU410 were reported in both Phase 1 and Phase 2 trials [2][5] - The medium dose achieved a 54% lesion reduction, while the high dose achieved a 36% reduction compared to control [6] Treatment Landscape - Current treatment options for dAMD in the U.S. are limited and involve frequent injections with side effects, leaving approximately 2 million patients in Europe without approved treatments [2][8] - OCU410 is a multifunctional gene therapy targeting multiple pathways associated with GA, contrasting with existing therapies that target a single mechanism [3][9] Future Developments - Ocugen plans to file a Biologics License Application (BLA) for OCU410 in 2028, aiming for three regulatory submissions for marketing authorization within three years [7][10] - Full data from the Phase 2 clinical trial is expected to be reported later this quarter, with Phase 3 trials anticipated to begin in 2026 [4][7]

Summary of Rocket Pharmaceuticals FY Conference Call (January 14, 2026) Company Overview - **Company**: Rocket Pharmaceuticals (NasdaqGM:RCKT) - **Industry**: Biotechnology, specifically focused on gene therapies for rare genetic diseases - **Core Focus**: Development of gene therapies targeting complex and rare genetic diseases, particularly in the cardiovascular space Key Points and Arguments Financial Position - As of September 30, 2025, Rocket reported **$222.8 million** in cash, cash equivalents, and restricted cash, expected to fund operations into the second quarter of **2027** [2] - Anticipated approval of **Kresladi** on March 28, 2026, could yield a **PRV** (Priority Review Voucher), providing strategic non-dilutive capital to extend the cash runway [2] Strategic Focus - 2026 is designated as a year of execution, with a concentrated effort on advancing three cardiovascular programs [2] - The company has deprioritized investments in **Fanconi anemia** to focus on areas with immediate impact [3] Cardiovascular Programs - **Danon Disease**: - A severe X-linked cardiomyopathy with a prevalence of **15,000 to 30,000** in the U.S. and Europe [8] - The lead program shows **100% LAMP2 expression** durability in treated patients, with a median reduction of **24%** in LV mass index over time [9][11] - The program is viewed as a significant near-to-medium-term value driver due to its clear differentiation and market potential [28] - **PKP2 Arrhythmogenic Cardiomyopathy (ACM)**: - Estimated to affect **50,000** adults and children in the U.S. and Europe [17] - The program is currently in phase one, with preliminary safety and efficacy results indicating well-tolerated gene therapy [18] - The company aims to define a registrational pathway for this program in 2026 [20] - **BAG3 Dilated Cardiomyopathy (DCM)**: - Considered a potential largest market opportunity with around **30,000** individuals affected in the U.S. [24] - The program is in the process of initiating a phase one trial, focusing on safety and preliminary efficacy [25] Regulatory and Clinical Development - The company is working towards resuming patient dosing for Danon disease, with logistics being the primary gating factor [29] - A concurrent natural history study for Danon is ongoing, validating the rapid decline of untreated patients [35] - The regulatory review for Kresladi is mid-cycle, with no current showstoppers noted [37] Manufacturing Capabilities - Rocket has in-house manufacturing capabilities for AAV, which helps control costs and timelines [38] - The facility is designed to support current and future programs, ensuring high margins and process improvements over time [38] Future Outlook - The anticipated approval of Kresladi is seen as a pivotal moment for transitioning to a commercial-stage gene therapy company [26] - The company plans to maintain a steady pipeline with additional programs ready to advance in the future [40] Additional Important Insights - The company emphasizes the importance of achieving meaningful myocardial transduction and protein localization for clinical benefit [21] - There is a strong community interest in the Danon program, despite past challenges, indicating a significant unmet medical need [32] - The company is cautious but optimistic about the regulatory landscape and the potential for future growth [37] This summary encapsulates the key points discussed during the conference call, highlighting Rocket Pharmaceuticals' strategic focus, financial position, and advancements in their gene therapy programs.

Neurogene FY Conference Summary Company Overview - **Company**: Neurogene (NasdaqGM:NGNE) - **Focus**: Development of gene therapies for neurological diseases, specifically targeting Rett syndrome Key Industry Insights - **Rett Syndrome**: A rare neurological disorder affecting primarily females, characterized by developmental delays and loss of previously acquired skills. Estimated prevalence is 15,000-20,000 patients in major markets with an incidence rate of 1 in 10,000 females worldwide [7][24]. - **Market Opportunity**: The gene therapy market for Rett syndrome is projected to be multi-billion dollars, with no current disease-modifying treatments available [5][7]. Core Company Insights - **Lead Program**: NGN401 for Rett syndrome, currently in the Embolden registrational trial aimed at FDA approval [4][5]. - **Clinical Progress**: - Dosing of patients commenced in Q4 2025, following alignment with the FDA on trial design [4]. - Interim data from phase 1/2 trial shows 100% of participants achieved functional improvements in key domains (hand function, gross motor function, communication) [9][10]. - 35 developmental milestones gained, with improvements durable for up to 24 months [9][10]. Clinical Trial Details - **Embolden Trial**: - Single-arm, baseline-controlled, open-label trial evaluating NGN401 in 20 females aged three years and older [15]. - Primary endpoint: Composite of Clinician Global Impression of Improvement (CGII) and developmental milestones, measured at 12 months [15]. - Anticipated completion of enrollment and dosing by Q2 2026 [5][29]. Financial and Commercial Readiness - **Financial Position**: Strong balance sheet expected to fund operations through Q1 2028, covering key milestones including BLA submission [19]. - **Commercial Strategy**: - Plans to convert clinical trial sites into commercial sites post-approval [18][32]. - Integrated manufacturing capabilities established for commercial product production [18][32]. - Strong reimbursement potential confirmed through payer research [18][26]. Future Outlook - **Upcoming Milestones**: - Completion of Embolden trial dosing in Q2 2026 [19][29]. - Presentation of new clinical data mid-2026, including 12 months of follow-up data for all participants [19][31]. - **Market Positioning**: Neurogene aims to establish itself as a leader in the Rett syndrome treatment landscape, with expectations of multiple gene therapy products entering the market [24][27]. Additional Insights - **Patient and Caregiver Impact**: Emphasis on improving daily living activities and reducing caregiver burden through meaningful functional changes [10][11][36]. - **Community Enthusiasm**: High levels of interest from physicians and caregivers regarding NGN401 and gene therapy options for Rett syndrome [29][30]. Conclusion - Neurogene is positioned for significant advancements in the treatment of Rett syndrome, with a clear regulatory path, strong clinical data, and a robust commercial strategy aimed at transforming the lives of affected families [20][36].

Summary of 4D Molecular Therapeutics FY Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (NasdaqGS:FDMT) - **Industry**: Biotechnology, specifically focusing on genetic medicines for retinal diseases Core Points and Arguments Product Pipeline and Development - **Lead Product**: 4D-150, targeting neovascular diseases of the retina, specifically wet AMD and diabetic macular edema (DME) [2][3] - **Clinical Trials**: Two ongoing Phase 3 trials for wet AMD with excellent enrollment; a Phase 3 trial for DME expected to initiate in Q3 2026 [4][6] - **Safety and Efficacy**: Strong Phase 1 and 2 results reported, with no significant inflammation observed in over 80 patients [5][6][26] - **Durability**: Promises a significant reduction in treatment burden, with an 80-90% reduction in the need for injections compared to traditional therapies [24][30] Market Opportunity - **Market Size**: The retinal vascular disease market is approximately $17 billion annually, with $14 billion specifically for wet AMD and DME [12] - **Patient Population**: Approximately 9 million people in the U.S., Europe, and Japan suffer from these diseases, with 600,000 new diagnoses each year [12][13] - **Unmet Needs**: Current treatments require lifelong injections, leading to high discontinuation rates (40% within a year) due to the burden of therapy [17][18] Regulatory and Commercial Strategy - **Regulatory Designations**: Received RMAT designation for DME and wet AMD, indicating recognition of the unmet medical need [7][32] - **Partnerships**: Collaboration with Otsuka for Asia-Pacific rights, retaining U.S. and European rights; the deal includes $135 million in upfront and cost coverage [4][49] - **Financial Position**: Closed a $100 million raise in Q4 2025, providing a runway into the second half of 2028 [8][39] Team and Expertise - **Leadership Team**: Comprised of industry veterans with extensive experience in retina therapeutics, including prior roles at Novartis and Iveric [10][11] - **External Engagement**: Strong pre-commercial team engaging with physicians, indicating high interest in gene therapy solutions [44][46] Additional Important Content - **Patient Journey**: Emphasizes the emotional and psychological burden of current treatment regimens on patients, highlighting the need for a more manageable solution [13][14][15] - **Innovative Delivery**: Utilizes a proprietary capsid for safe, low-dose intravitreal injections, aiming for continuous expression of aflibercept [25][41] - **Market Differentiation**: Positioned as a unique offering in the gene therapy space, distinct from traditional TKIs, with a focus on long-term durability and reduced treatment burden [46][47] Future Catalysts - **Upcoming Milestones**: Expected completion of Phase 3 enrollment for wet AMD in Q1 2026 and DME in Q2 2026, with top-line data anticipated in the first half of 2027 [38][39] - **Financial Outlook**: Strong cash position expected to support ongoing clinical activities and potential commercialization efforts [39]

The New Backbone of Retina Care: Moving Beyond Lifelong Injections Corporate Overview J.P. Morgan Healthcare Conference | January 2026 This Presentation shall not constitute an offer to sell or the solicitation of an offer to buy securities. © 2026 4D Molecular Therapeutics. All Rights Reserved. 2 Overview 1 4DMT Vision & Pipeline 2 4D-150 Overview & 2025 Accomplishments 3 Retinal Vascular Disease Market & Unmet Medical Needs 4 Derisking Phase 3 & Commercialization 5 2026-27 Catalysts 6 Summary © 2026 4D Mo ...

Corporate Presentation January 2026 © 2026 Solid Biosciences Cautionary Note Regarding Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company's goals, priorities and key clinical and preclinical milestones; strategies and expectations for the company's SGT ...

Core Insights - Beam Therapeutics' shares increased by 22.3% following the announcement of strategic priorities and expected milestones for its gene therapy pipeline candidates aimed for 2026 [1][8] Pipeline Developments - The lead candidate, BEAM-302, is in an open-label phase I/II study for treating alpha-1 antitrypsin deficiency (AATD), with over 25 patients treated so far [2][4] - AATD is a genetic disorder leading to early emphysema and liver disease, with no current curative treatments available [3] - The company has aligned with the FDA on a potential accelerated approval pathway for BEAM-302 based on 12-month AAT biomarker data, planning to enroll around 50 additional patients [4][5] - Updated data from the phase I/II study and next steps for BEAM-302 are expected by the end of Q1 2026 [5] - BEAM-301 is also in a phase I/II study for glycogen storage disease type 1a (GSD1a), with initial data expected later in 2026 [6] Financial Position - Beam Therapeutics ended 2025 with approximately $1.25 billion in cash and equivalents, providing financial flexibility to support ongoing clinical programs and anticipated expenses through 2029 [13] Market Performance - Over the past six months, Beam Therapeutics' shares have increased by 59.1%, outperforming the industry average increase of 21% [7]

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Financial Performance & Guidance - BRINSUPRI achieved preliminary unaudited U S net revenues of approximately $144 6 million in Q4 2025 and $172 7 million for the full year 2025[23] - ARIKAYCE generated worldwide revenue of approximately $433 8 million in FY 2025, representing a 19 3% increase year-over-year[27] - The company anticipates ARIKAYCE revenue to be between $450 million and $470 million for the full year 2026[28] Clinical Development Programs - The company expects topline data readout from the Phase 3 ENCORE trial for ARIKAYCE in March/April 2026, potentially leading to label expansion[30, 31] - Phase 3 trials for TPIP in PAH, PPF, and IPF are expected to initiate in early and the second half of 2026 respectively[13, 38] - Topline data readout from the Phase 2 CEDAR trial for brensocatib in Hidradenitis Suppurativa is expected in Q2 2026[46, 50] Pipeline Expansion - The company plans to file Investigational New Drug (IND) applications for RA and IBD in the second half of 2026[13, 50] - IND filing for Stargardt is anticipated in 2026[13, 59] - Phase 2 programs in ILD and moderate to severe asthma are planned for INS1148[13, 40] Commercial Expansion - BRINSUPRI is expected to launch in the EU and UK, pending regulatory approval[13, 44] - BRINSUPRI is also anticipated to launch in Japan, pending regulatory approval[13, 44] Cash Position - The company reported a cash position of $1 7 billion as of September 30, 2025[62]