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]

AUSTIN, Texas, Nov. 19, 2025 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the Company has added Gabrail Cancer Center in Canton, Ohio, as a new clinical trial site for the Acclaim-1 and Acclaim-3 clinical trials studying its lead drug candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), in lung cancer. In addition, the Company expect ...

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.

​uniQure N.V. (NASDAQ:QURE) is one of the Hottest Small Cap Stocks to Buy Now. Wall Street has a positive outlook on uniQure N.V. (NASDAQ:QURE) despite the regulatory challenges from the FDA for its experimental gene therapy called AMT-130. On November 12, Yanan Zhu from Wells Fargo also reiterated a Buy rating on uniQure N.V. (NASDAQ:QURE), but lowered the price target from $80 to $60. A day earlier, TD Cowen analyst Joseph Thome had maintained a Buy rating on the stock without disclosing any price target ...

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]

Accessibility StatementSkip Navigation ROM, 15. November 2025 /PRNewswire/ -- Fondazione Telethon gibt die positive Stellungnahme des Ausschusses für Humanarzneimittel (CHMP) der Europäischen Arzneimittelagentur (EMA) bekannt, in der die Zulassung von Waskyra™ in der Europäischen Union empfohlen wird, einer ex-vivo -Gentherapie für das Wiskott-Aldrich-Syndrom (WAS), eine seltene und lebensbedrohliche primäre Immundefizienz. Die Fondazione Telethon ist die erste gemeinnützige Organisation, die den gesa ...

Nov 13, 2025 5:10 PM Eastern Standard Time Neurogene Reports Third Quarter 2025 Financial Results and Highlights Recent Updates NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced third quarter 2025 financial results and highlighted recent corporate updates. "We significantly advanced our NGN-401 gene therapy clinical program for Rett syndrome over ...

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].

Core Insights - Opus Genetics has reported positive clinical data from its OPGx-LCA5 Phase 1/2 trial, indicating potential for restoring cone-mediated vision in patients with inherited retinal diseases [1][6] - The company successfully completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA, which may facilitate an accelerated regulatory pathway for OPGx-LCA5 [1][6] Pipeline Updates - The OPGx-LCA5 program is focused on gene therapy for Leber Congenital Amaurosis (LCA) and has shown significant improvements in visual function in both pediatric and adult participants [3][6] - Recruitment is ongoing for the OPGx-BEST1 gene therapy program, targeting BEST1-related inherited retinal diseases [4][5] - The company plans to submit a Supplemental New Drug Application for Phentolamine Ophthalmic Solution 0.75% for presbyopia treatment by the end of 2025 [5][8] Financial Results - As of September 30, 2025, Opus Genetics reported cash and cash equivalents of $30.8 million, with an additional $23.0 million raised through a direct equity offering [10] - License and collaborations revenue for Q3 2025 was $3.1 million, a decrease from $3.9 million in Q3 2024, primarily due to lower research and development service reimbursements [11] - General and Administrative expenses increased to $5.0 million in Q3 2025 from $2.9 million in Q3 2024, attributed to higher legal, payroll, and professional service costs [12] - Research and Development expenses decreased to $6.4 million in Q3 2025 from $9.0 million in Q3 2024, reflecting lower costs in clinical research and manufacturing [13] Net Loss - The net loss for Q3 2025 was $17.5 million, or $(0.25) per share, compared to a net loss of $7.5 million, or $(0.29) per share, in Q3 2024 [15][20]