Company Overview - 4D Molecular Therapeutics is focused on developing an adaptable genetic medicines portfolio, particularly in ophthalmology and pulmonology [1] Lead Asset - The lead asset, 4D-150, targets wet AMD and DME, with the potential to significantly reduce treatment burden for patients [2] - The market for this therapy is estimated at approximately $17 billion annually, indicating a substantial commercial opportunity [2] - 4D-150 is expected to transform the field due to its safety, clear efficacy, and seamless integration into busy retina practices, providing a competitive advantage for commercialization [2] Cost Structure - The cost of goods for 4D-150 is notably low compared to most gene therapies, allowing for greater pricing flexibility [3] Additional Product - 4D-710 is an aerosol-delivered genetic medicine for cystic fibrosis, designed to deliver a CFTR transgene throughout the airways with consistent transduction and gene expression [3] - Updates on the Phase I program for 4D-710 are anticipated in December [3]

Table of Contents As filed with the Securities and Exchange Commission on November 21, 2025 Registration No. 333- (State or other jurisdiction of incorporation or organization) (Primary Standard Industrial Classification Code Number) Delaware 2834 90-0772347 (I.R.S. Employer Identification Number) UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 GENPREX, INC. (Exact name of registrant as specified in its charter) Copies ...

Core Insights - Genprex, Inc. has added Gabrail Cancer Center in Canton, Ohio, as a new clinical trial site for its Acclaim-1 and Acclaim-3 clinical trials studying Reqorsa® Gene Therapy for lung cancer [1][2] - The company aims to expand its reach and expedite patient enrollment in these trials, which have received FDA Fast Track Designation [1][4] Acclaim-1 Clinical Trial - Acclaim-1 is a Phase 1/2 clinical trial evaluating the combination of Reqorsa and AstraZeneca's Tagrisso® for late-stage non-small cell lung cancer (NSCLC) patients with activating EGFR mutations [2][4] - The Phase 1 portion showed that Reqorsa was well tolerated with no dose-limiting toxicities, and early signs of efficacy were observed [3][4] - The trial plans to enroll approximately 33 patients, with an interim analysis expected after treating 19 patients, anticipated to be completed in the first half of 2026 [4] Acclaim-3 Clinical Trial - Acclaim-3 is a Phase 1/2 clinical trial evaluating the combination of Reqorsa and Genentech's Tecentriq® as maintenance therapy for extensive stage small cell lung cancer (ES-SCLC) [5][7] - The Phase 2 expansion is expected to enroll around 50 patients, with the primary endpoint being the 18-week progression-free survival rate [7] - An interim analysis will be conducted after the 25th patient reaches 18 weeks of follow-up, with completion of enrollment for this analysis also expected in the first half of 2026 [7] Company Overview - Genprex, Inc. is focused on developing gene therapies for cancer and diabetes, utilizing its Oncoprex® Delivery System to administer disease-fighting genes [8][9] - The company's lead product candidate, Reqorsa, is being evaluated in two clinical trials for NSCLC and SCLC, both of which have received FDA Fast Track Designation [9]

Summary of 4D Molecular Therapeutics Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (NasdaqGS:FDMT) - **Focus**: Developing adaptable genetic medicines, particularly in ophthalmology and pulmonology - **Lead Asset**: 4D-150, targeting wet Age-related Macular Degeneration (AMD) and Diabetic Macular Edema (DME) with a market potential of approximately $17 billion annually [2][5][6] Core Insights and Arguments 4D-150 - **Product Description**: 4D-150 is designed to reduce treatment burden for patients with wet AMD and DME by providing continuous expression of aflibercept directly in the retina [5][6] - **Market Opportunity**: The product aims to address high unmet needs in a market that is currently burdened by frequent injections (average of 36 injections over five years) [11][12] - **Efficacy**: Clinical data shows a significant reduction in treatment burden, with patients receiving an average of 4.3 injections over two years compared to a projected 20 injections without the treatment, representing nearly 80% reduction [18][19] - **Safety Profile**: 4D-150 has demonstrated a favorable safety profile with low rates of intraocular inflammation, comparable to or better than current standard treatments [21][22] Clinical Trials - **Phase 3 Trials**: Two ongoing trials, Forefront One and Forefront Two, are designed to evaluate the efficacy and safety of 4D-150 in treatment-naive populations, with top-line data expected in 2027 [20][22] - **DME Data**: In a phase one study for DME, 4D-150 showed a treatment burden reduction of 78%, with an average of 1.6 injections compared to a projected seven injections with aflibercept [24][25] Commercial Strategy - **Cost of Goods**: The low cost of goods for 4D-150 allows for flexible pricing strategies, enhancing commercial viability [5][26] - **Licensing Agreement**: An exclusive licensing agreement with Otsuka for the Asia Pacific region has provided $85 million upfront, with additional potential milestones and royalties [27] 4D-710 - **Product Overview**: 4D-710 is an aerosol-delivered genetic medicine for cystic fibrosis, targeting patients with rare mutations or those intolerant to existing modulators [28][29] - **Clinical Development**: Promising early data shows high-level expression of the CFTR transgene, with updates expected in December [29][30] Additional Important Points - **Patient Perspective**: The treatment burden reduction is crucial for patient adherence and quality of life, as frequent injections can lead to anxiety and logistical challenges [12][25] - **Market Positioning**: 4D-150 is positioned as a transformative therapy compared to existing anti-VEGF treatments and tyrosine kinase inhibitors, offering potentially lifelong benefits with fewer injections [33][34] - **Future Milestones**: The company has a robust pipeline with upcoming data releases and enrollment updates for ongoing trials, indicating a strong catalyst calendar [30][31] This summary encapsulates the key points discussed during the conference call, highlighting the innovative approach of 4D Molecular Therapeutics in addressing significant medical needs in ophthalmology and pulmonology.

Core Insights - uniQure N.V. is viewed positively by Wall Street despite regulatory challenges from the FDA regarding its gene therapy AMT-130 [1][3] - The company presented significant results from a three-year study showing a reduction in Huntington's disease progression [2] - The FDA has expressed concerns about the adequacy of data from early Phase I/II studies for AMT-130, leading to uncertainty about the pre-Biologics License Application timeline [3] Analyst Ratings - Wells Fargo analyst Yanan Zhu reiterated a Buy rating on uniQure N.V. but lowered the price target from $80 to $60 [1] - TD Cowen analyst Joseph Thome maintained a Buy rating without disclosing a price target, expressing confidence in the company's data and potential [1][4] Company Background - uniQure N.V. is a Netherlands-based biotechnology company focused on developing gene therapies aimed at curing severe genetic diseases with a single administration [4]

Core Insights - Solid Biosciences has entered into a non-exclusive worldwide license and collaboration agreement with Andelyn Biosciences for the use of its proprietary capsid, AAV-SLB101, aimed at enhancing gene therapy development [1][2] Group 1: Agreement Details - The agreement allows Andelyn to utilize AAV-SLB101 in conjunction with its AAV Curator® Platform, which optimizes gene therapy manufacturing processes [2] - Financial terms of the agreement have not been disclosed [2] Group 2: AAV-SLB101 Characteristics - AAV-SLB101 is designed for improved skeletal muscle and cardiac tropism while minimizing liver biodistribution [3][5] - The capsid has shown good tolerability in a Phase 1/2 clinical trial involving 23 pediatric participants, with safety data reported as of October 31, 2025 [3] Group 3: Company Statements - The collaboration is expected to accelerate the development of next-generation gene therapies, enhancing safety and efficacy [4] - Andelyn aims to integrate AAV-SLB101 into its platform to provide advanced gene therapy vectors, potentially leading to faster and more cost-effective development [4] Group 4: Company Background - Solid Biosciences focuses on developing precision genetic medicines for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy [6][7] - Andelyn Biosciences specializes in the development and production of viral vectors for gene therapy, with extensive experience in clinical batch production [8]

Core Points - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Waskyra™, an ex-vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency [1][5] - Fondazione Telethon is the first non-profit organization to successfully navigate the entire process from laboratory research to regulatory approval, collaborating with industry partners to bring gene therapies from discovery to patients [2][10] - Waskyra™ represents a significant scientific and clinical achievement, providing new hope for patients affected by WAS [3][4] Company Information - Fondazione Telethon has been supporting impactful scientific research for over 35 years, focusing on developing innovative treatment methods for rare and complex genetic diseases [10] - The therapy was developed at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, which is recognized as a center of excellence for gene therapy [3][4] Therapy Details - Waskyra™ involves a one-time administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector encoding the WAS gene [8] - The therapy has shown to reduce the frequency of severe and moderate bleeding as well as severe infections in patients with WAS compared to the period before treatment [9] - The current treatment options for WAS are limited, with hematopoietic stem cell transplantation being the only potentially curative option, which is not always available due to donor compatibility issues [7]

Core Insights - Neurogene reported positive interim data from the NGN-401 Phase 1/2 gene therapy trial for Rett syndrome, showing multidomain, durable gains with 35 developmental milestones acquired across eight participants [1][5] - The first participant has been dosed in the Embolden registrational trial of NGN-401, with complete enrollment expected in three to six months [1][2] - The company maintains a strong cash position, with $265.4 million in cash and equivalents, providing a runway through the first quarter of 2028 [11] Clinical Development Highlights - The NGN-401 gene therapy program for Rett syndrome has advanced significantly, with 12 of 13 trial sites initiated [2][5] - Interim clinical data from the pediatric cohort (ages 4-10) demonstrated functional improvements across all participants, with gains in hand function, language, and ambulation [5] - The therapy has been well-tolerated in both pediatric and adolescent/adult cohorts, with no significant adverse effects reported [5] Financial Results - For the third quarter of 2025, R&D expenses were $17.2 million, up from $16.3 million in the same period in 2024, primarily due to increased clinical trial costs [11] - General and administrative expenses rose to $6.6 million from $5.9 million year-over-year, driven by higher employee-related costs [11] - The net loss for the quarter was $21.0 million, compared to $20.2 million in the prior year [11] Regulatory Designations - NGN-401 has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designation, indicating its potential as a significant treatment for Rett syndrome [8]

Core Insights - MeiraGTx Holdings plc announced its financial and operational results for Q3 2025, highlighting significant advancements in its gene therapy programs and strategic collaborations, particularly with Eli Lilly in ophthalmology [1][2][3] Financial Performance - As of September 30, 2025, the company reported cash and cash equivalents of approximately $14.8 million, a decrease from $105.7 million as of December 31, 2024 [13] - Service revenue for Q3 2025 was $0.4 million, down from $10.9 million in Q3 2024, primarily due to decreased activity related to the asset purchase agreement with Johnson & Johnson [14] - General and administrative expenses increased to $13.6 million in Q3 2025 from $12.7 million in Q3 2024, attributed to higher rent, consulting fees, and share-based compensation [16] - Research and development expenses rose to $32.5 million in Q3 2025 from $26.2 million in Q3 2024, driven by increased manufacturing costs and clinical program activities [17] - The net loss attributable to ordinary shareholders for Q3 2025 was $50.5 million, or $0.62 per share, compared to a net loss of $39.3 million, or $0.55 per share, in Q3 2024 [21] Strategic Collaborations - The company entered a strategic collaboration with Eli Lilly, granting exclusive rights to its AAV-AIPL1 program for treating Leber congenital amaurosis 4 (LCA4), along with access to other gene therapy technologies [2][3][8] - Under the collaboration, MeiraGTx will receive an upfront payment of $75 million and is eligible for over $400 million in milestone payments, along with tiered royalties on licensed products [8] Clinical Development - The pivotal Phase 2 study of AAV-hAQP1 for radiation-induced xerostomia is on track for target enrollment by the end of 2025, with potential BLA filing expected in early 2027 [2][8] - A Phase 3 study for AAV-GAD in Parkinson's disease is anticipated to start soon, following positive data from previous studies [2][8] - The company has optimized its riboswitch program for delivering native human leptin, addressing a significant unmet need in leptin deficiency treatment [2][8] Recent Developments - MeiraGTx has developed a second Specials program in the UK for treating BBS10-associated retinal dystrophy, which has received Rare Pediatric Disease Designation from the FDA [2][3] - The company is forming a joint venture with Hologen AI, which includes a $200 million upfront payment and additional funding for developing the AAV-GAD program [11][12]

Core Viewpoint - Abeona Therapeutics' stock surged over 25% following strong third-quarter earnings and positive updates regarding its treatment for Recessive Dystrophic Epidermolysis Bullosa (RDEB), Zevaskyn [1][7]. Group 1: Treatment Overview - RDEB is a rare inherited skin disorder with an incidence of 3.05 per million births and a prevalence of 1.35 per million, leading to a high mortality rate among sufferers due to complications [2]. - Zevaskyn is a gene therapy derived from a patient's own skin, modified to produce a healthy collagen gene, and applied to wounds [3]. Group 2: Patient and Coverage Updates - Management identified 30 patients at treatment centers, with 12 patients currently scheduling treatments, indicating strong interest in the therapy among RDEB patients [5]. - Major commercial insurers, including UnitedHealthcare, Cigna, Aetna, Anthem, and most Blue Cross Blue Shield plans, have published policies covering Zevaskyn, enhancing treatment accessibility [5]. Group 3: Market Reaction and Future Outlook - The market has largely ignored a one-quarter delay in treatment initiation due to a resolved issue, with Abeona expected to start patient treatments this quarter [6]. - Significant revenue growth is anticipated in 2026 as treatment progresses [6].