MALVERN, Pa., Oct. 23, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that it will host a conference call and live webcast to discuss the Company’s third quarter 2025 financial results and provide a business update at 8:30 a.m. ET on Wednesday, November 5, 2025. Ocugen will issue a pre-market earnings announcement on the same day. Attendees are invited to participate on the call usi ...

Summary of Conference Call on Gene Therapy for Neurodegenerative Diseases Industry Overview - The conference focused on gene therapy applications for neurodegenerative diseases, featuring speakers from three companies: Mira, uniQure, and Passage Bio [1][2][6]. Key Points by Company Mira - **Company Overview**: Mira specializes in genetic medicines, focusing on local delivery of small doses to treat severe indications, which enhances safety and reduces costs [2]. - **Recent Milestones**: Mira has two pivotal studies and two awaiting Biologics License Application (BLA) filings, including treatments for rare and common eye diseases and a Parkinson's treatment that has shown three positive studies [3][4]. - **Innovative Approach**: The company utilizes AI technology to analyze data from the largest neurohospital in Europe, demonstrating physiological changes in the brain related to their Parkinson's treatment [4][24]. - **Potential Applications**: Mira's gene therapy can convert glutamate to GABA, potentially treating various neurodegenerative diseases by calming hyperactive neurons [22][24]. uniQure - **Company Overview**: uniQure is a pioneer in genomic medicine, known for developing the first approved AAV gene therapies, including Glybera and Hemgenix [6][9]. - **Lead Program**: The company is focused on Huntington's disease, utilizing a platform called MyCure to suppress the aberrant Huntingtin protein [8]. - **Clinical Findings**: In a study involving 45 patients, uniQure reported a 75% statistically significant slowing of disease progression based on the UHDRS [9]. - **Regulatory Plans**: A pre-BLA meeting is expected in Q4, with a BLA submission planned for Q1 of the following year [9]. Passage Bio - **Company Overview**: Passage Bio is based on technology from the University of Pennsylvania, focusing on gene therapy for frontotemporal dementia and Huntington's disease [14]. - **Clinical Approach**: The company delivers AAV1 via a minimally invasive procedure, showing high levels of target engagement and stabilization of neurofilaments [14][15]. - **Future Directions**: Passage Bio is also developing a preclinical program targeting the DNA damage response pathway in Huntington's disease [15]. Challenges in Gene Therapy - **Delivery Issues**: The primary challenge remains delivering therapies to the correct brain regions, especially for diseases like Huntington's [18][19]. - **Slow Disease Progression**: Designing clinical studies for slowly progressing neurodegenerative diseases is difficult, as changes in function are hard to detect [19][20]. - **Heterogeneity**: Variability among patients complicates the assessment of treatment efficacy, necessitating innovative statistical methods to account for differences [31][32]. Regulatory Considerations - **FDA Flexibility**: The FDA is showing increased willingness to consider alternative trial designs and endpoints for rare diseases with high unmet needs [44][46]. - **Natural History Data**: Utilizing natural history datasets can help in understanding disease progression and support regulatory submissions [30][31]. Safety Concerns - **Patient Safety**: The panel acknowledged the importance of safety in gene therapy, especially following recent patient deaths in trials [35][37]. - **Local Delivery Advantages**: Localized delivery methods may reduce systemic exposure and associated risks, as demonstrated by lower doses used in certain therapies [38][41]. Access and Implementation - **Training and Infrastructure**: Successful implementation of gene therapies will depend on training healthcare providers and establishing treatment centers [52][54]. - **Data-Driven Adoption**: Strong clinical data will be crucial for gaining acceptance among medical professionals and ensuring broad access to therapies [55][56]. Conclusion - The conference highlighted the potential of gene therapy in treating neurodegenerative diseases, the challenges faced in delivery and study design, and the evolving regulatory landscape that may facilitate the development of these innovative treatments [1][17][42].

Summary of 4D Molecular Therapeutics FY Conference Call Company and Industry Overview - **Company**: 4D Molecular Therapeutics (NasdaqGS:FDMT) - **Industry**: Gene therapy, specifically targeting cystic fibrosis and other respiratory diseases - **Market Context**: Cystic fibrosis generated approximately $11 billion in sales in 2024, primarily driven by Vertex Pharmaceuticals, which is a major player in this space [1][2] Key Points and Arguments Gene Therapy Advances - The potential of gene therapy for cystic fibrosis has gained traction in recent years, particularly for patients with CFTR gene mutations that current modulators cannot address [2][4] - 4D Molecular Therapeutics is focused on developing customized vectors for gene delivery, with their lead product, 4D-710, targeting cystic fibrosis through aerosol delivery [4][41] Challenges in Gene Delivery - Historical challenges in gene delivery to the lungs include transduction efficiency, safety, and the complexity of lung tissue, which consists of over 10 different cell types [11][12] - The mucus barrier and pre-existing antibodies in patients pose significant hurdles for effective gene delivery [13][14] Clinical Insights and Data - 4D-710 has shown promising results in clinical trials, with 50% to 95% of cells expressing CFTR protein and a strong safety profile at lower dose levels [41][42] - The company is exploring the durability of gene expression in the lungs, with ongoing studies to understand lung turnover rates and the potential for re-dosing [42][49] Market Segmentation and Unmet Needs - The focus is on patients with class 1 mutations and those who are non-responders or intolerant to current modulators, representing 10% to 15% of the cystic fibrosis population [28][29] - There is a potential for combination therapies in the future, which could enhance treatment efficacy for a broader patient population [55][56] Regulatory Considerations - The discussion highlighted the need for a robust panel of endpoints for regulatory approval, moving beyond traditional metrics like FEV1, which can be variable and effort-dependent [30][34] - Alternative endpoints such as lung clearance index and quality of life measures are being considered to support clinical efficacy [31][35] Competitive Landscape - The gene therapy space is evolving with various approaches, including non-viral methods and RNA delivery systems. However, viral vectors like AAV are seen as having advantages in terms of efficiency and safety [69][70] - The sentiment around gene therapy is cautiously optimistic, with a recognition of the need for continued education about the safety and efficacy of localized delivery methods compared to systemic approaches [78][80] Manufacturing and Cost Considerations - The importance of early investment in manufacturing processes was emphasized, particularly for scalability and cost of goods sold (COGS) [59][61] - 4D Molecular Therapeutics aims to reduce treatment costs significantly through efficient vector design and manufacturing processes [62][63] Additional Important Insights - The collaboration with the Cystic Fibrosis Foundation has been crucial for advancing research and development efforts [15][49] - The potential for gene therapy to fundamentally modify the disease at its foundation, combined with modulators, could lead to significant improvements in patient outcomes [56][57] - The discussion underscored the importance of understanding immune responses and cellular turnover rates in the context of re-dosing strategies for gene therapies [51][53] This summary encapsulates the key discussions and insights from the conference call, highlighting the advancements, challenges, and future directions in the gene therapy landscape for cystic fibrosis.

Tenaya Therapeutics (NasdaqGS:TNYA) FY Conference October 21, 2025 12:00 PM ET Speaker3I'm Gula Lifschitz, a biotech analyst at Chardan, and welcome to this session on cardiac gene therapy. The space now has several programs that have delivered some exciting proof-of-concept data. As with other segments in the space, there have been some setbacks. For example, safety signals in the adenosine therapy program and discussion about the extent of read-through on risks from other systemically administered gene th ...

Summary of 4D Molecular Therapeutics FY Conference Call Company and Industry Overview - **Company**: 4D Molecular Therapeutics (NasdaqGS:FDMT) - **Industry**: Ocular Gene Therapy - **Key Focus**: Development of gene therapies for ophthalmic conditions, particularly age-related macular degeneration (AMD) and diabetic macular edema (DME) Core Points and Arguments Market Landscape - The panel discussed the competitive landscape for large ophthalmic indications, including geographic atrophy (GA), wet AMD, and DME, highlighting existing approved options and the unmet needs in these areas [13][14][15] - There is a significant unmet medical need in GA, with current therapies failing to show functional benefits, leading to patient reluctance to return for treatment [20][21] Clinical Development and Differentiation - **4D-150**: 4D Molecular Therapeutics' lead program is in phase three trials for wet AMD, aiming to provide long-lasting treatment benefits compared to existing therapies [11][15] - **Durability**: The need for improved durability in treatments is emphasized, with existing therapies providing only incremental benefits [14][15] - **Gene Therapy Potential**: The promise of gene therapy is to provide long-term solutions with potentially one-time treatments that could stabilize or improve vision, representing a paradigm shift in treatment [19][20] Regulatory Challenges - The FDA and EMA are supportive of gene therapies, with a focus on safety and efficacy as critical factors for approval [52][53] - There is a need for alignment on clinical endpoints, particularly for GA, where traditional measures may not adequately reflect treatment benefits [57][59] Commercial and Access Considerations - The transition to one-time therapies poses challenges for existing business models in the U.S., where ongoing treatments generate significant revenue for practices [63][68] - The economic impact of vision loss diseases is substantial, with potential savings and productivity gains from effective one-time therapies [65][66] - The market for GA is currently under-treated, with only about 15% of patients receiving treatment, indicating significant growth potential for new therapies [64] Additional Important Insights - **Heterogeneity of Disease**: The panelists noted the complexity and variability of diseases like GA and RP, which necessitate tailored treatment approaches [61][62] - **Emerging Therapies**: There is a recognition that multiple therapies targeting different pathways may coexist in the market, allowing for a segmented approach to treatment [30][39] - **Patient-Centric Focus**: Emphasis on the importance of patient outcomes and the need for therapies that improve quality of life, rather than solely focusing on economic models [66][70] This summary encapsulates the key discussions and insights from the conference call, highlighting the current state and future potential of gene therapies in the ocular space.

Market Movements - Australian Oilseeds Holdings Limited Warrant (NASDAQ:COOTW) surged by 494.86% to $0.18 with a trading volume of 3,759,243, while its ordinary shares (COOT) increased by 244.87% to $3.32 with a trading volume of 167,502,947, despite facing a Nasdaq notification regarding non-compliance with the minimum stockholders' equity requirement of $10 million [1][4] - Genprex, Inc. (NASDAQ:GNPX) saw a 193.90% increase in its stock price to $0.74 with a trading volume of 825,729,912, driven by anticipation of promising preclinical data on its Reqorsa® Gene Therapy, which targets ALK-EML4 positive translocated non-small cell lung cancer [1][4] - Omeros Corporation (NASDAQ:OMER) experienced a 170.73% increase in its stock price to $11.1 with a trading volume of 107,994,321, following a significant licensing agreement with Novo Nordisk valued at up to $2.1 billion, granting exclusive global rights to develop and commercialize Omeros' MASP-3 inhibitor [1][4] Investor Interest - Bonk, Inc. Warrant (BNKKW) experienced a 125.16% price increase to $0.17 with a volume of 127,897, indicating investor interest in the company's prospects [2] - The reasons behind these surges include positive clinical trial results, strategic corporate developments, and broader market trends favoring these sectors [2] - The market environment reflects significant investor interest across various sectors, including biotechnology and consumer defensive, with analysts closely monitoring these companies for further developments [3]

Gene Therapy Stock Genprex, Inc. (NASDAQ: GNPX) Makes Nasdaq Top Gainers List on News of Positive Preclinical Data on the Use of Reqorsa October 15, 2025- (Investorideas.com Newswire), a go-to investing platform covering biotech and medtech stocks releases a news and trading alert for Genprex, Inc. ( NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. The stock makes the Nasdaq top gainers list today following news ...

Previously disclosed 100% response rate across all 10 patients in Part A for pivotal trial primary endpoint of gain/regain of ≥ one natural history defined developmental milestone assessed via video-evidenced review by independent central raters New supplemental analysis of validated, structured efficacy scales provides supportive evidence of additional functional gains, with 100% of patients demonstrating multiple skill gains/improvements outside the natural history defined developmental milestones Finding ...

, /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present interim data from the Phase II ALTITUDE trial evaluating suprachoroidal delivery of surabgene lomparvovec (ABBV-RGX-314, sura-vec) for the treatment of diabetic retinopathy (DR) at the American Academy of Ophthalmology 2025 Annual Meeting. Sura-vec, developed in collaboration with AbbVie, is an investigational one-time gene therapy and potential first-in-class treatment for wet age-related macular degeneration (wet AMD) an ...

Company Overview - uniQure N.V. (NASDAQ:QURE) specializes in developing gene therapies for rare and severe diseases, including an approved treatment for hemophilia B called HEMGENIX and clinical candidates for Huntington's disease, epilepsy, ALS, and Fabry disease [2] Clinical Data - On September 24, uniQure announced positive Phase I/II results for its gene therapy candidate AMT-130 targeting Huntington's disease, demonstrating a 75% slowing of disease progression on the composite Unified Huntington's Disease Rating Scale and a 60% improvement on Total Functional Capacity after 36 months compared to a matched control group [2] Market Sentiment - Jim Cramer highlighted the positive clinical data but expressed caution due to an insider selling approximately $9 million worth of stock, indicating potential concerns about the stock's future performance after a significant price increase [1]