Sarepta said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company https://t.co/DSR52u0ort ...

Klotho Neurosciences Maintains Listing on the NASDAQ NEW YORK, July 16, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. ("Klotho" or the "Company") (NASDAQ: KLTO), a gene and cell therapy company focused on the treatment of neurodegenerative and other aging-related diseases, today announced that it had received a notification letter from The NASDAQ Stock Market LLC confirming that the Company has regained compliance with the minimum $1.00 bid price under NASDAQ Listing Rule 5550(a)(2), and the minimum stock ...

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [1][3] - The company granted equity awards to two new non-executive employees as an inducement for employment, consisting of stock options and restricted stock units (RSUs) [1][2] Equity Awards Details - The equity awards include stock options for 240,000 shares and 150,000 RSUs, with the stock options having an exercise price of $0.94, equal to the closing price on the grant date [2] - Stock options vest over four years, with 25% vesting on the one-year anniversary and the remaining 75% vesting quarterly thereafter; RSUs vest in four equal installments on each anniversary of the grant date [2] Company Pipeline - Opus Genetics is developing AAV-based gene therapies targeting inherited retinal diseases such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - The lead candidate, OPGx-LCA5, is in a Phase 1/2 trial for LCA5-related mutations and has shown promising early results [3] - Additional programs include OPGx-BEST1 for BEST1-related retinal degeneration and a Phase 3-ready small molecule therapy for diabetic retinopathy, developed under a Special Protocol Assessment with the FDA [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and dim light vision disturbances [3]

NEW YORK, June 26, 2025 (GLOBE NEWSWIRE) -- Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, held today a shareholders general meeting at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4th floor, 75013 Paris, France. At the meeting, during which approximately 57% of voting rights were exercised, resolutions 1 through 23 and resolutions 25 and 26 were adopted, while resolu ...

Company Overview - Ocugen Inc. is a clinical-stage biotechnology company focused on developing modifier gene therapies for rare inherited retinal diseases that lead to progressive vision loss [1]. Pipeline and Focus - The company is particularly concentrating on therapies for diseases that cause vision impairment, indicating a specialized approach within the biotechnology sector [1].

Core Insights - Klotho Neurosciences, Inc. has terminated its Share Exchange Agreement with SkyBell Technologies, allowing the company to focus on its core R&D programs and enhance shareholder value [2][3] Company Overview - Klotho Neurosciences, Inc. is a biogenetics company specializing in innovative, disease-modifying cell and gene therapies derived from the anti-aging human Klotho gene, targeting neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease [4] Strategic Implications - The termination of the agreement eliminates the need to issue new common stock to SkyBell, which would have resulted in SkyBell owning 90% of Klotho, thereby preventing significant dilution of existing shareholders [7] - The company will not incur any liability as a result of this termination, allowing it to refocus its strategy and allocate resources towards advancing its primary therapeutic and longevity programs [7]

So what most people realize is, you know, there's amazing cell and gene therapies coming through in the market at the moment and they're done and actuated in different ways. Uh crisper gene editing is a way in which you can make very precise edits to a patient's DNA. And what that allows you to do is identify new targets, but also to correct some diseases, more importantly expand the number of treatable diseases.And we're working with our partners. We've built the world's most powerful crisper gene editing ...

Crispr Technology & Applications - Crispr 基因编辑技术能够对患者的 DNA 进行精确编辑，从而识别新的治疗靶点并纠正某些疾病，扩大可治疗疾病的范围 [2] - Crispr 技术与 AI 结合，加速识别靶点和疗法，从而加快新疗法上市的速度 [3] - 癌症，尤其是无法以其他方式治疗的儿童疾病和罕见癌症，可能是 Crispr 技术首先产生影响的领域 [5] Regulatory Landscape - FDA 对新的治疗方法和技术平台持支持态度，并倾向于快速批准 [5] - 美国卫生与公众服务部 (HHS) 部长承诺支持细胞和基因治疗领域，这对行业来说是一个巨大的推动力 [7] - 众议院拨款委员会也表达了对细胞和基因治疗领域的支持，释放了积极信号 [7] Manufacturing & Supply Chain - 该公司主要在美国本土进行生产，对进口尤其是来自中国的依赖程度不高 [9] - 公司在马萨诸塞州进行生产，并在匹兹堡进行扩张 [9] - 匹兹堡的团队致力于支持当地学校的 STEM (科学、技术、工程和数学) 项目 [9]

Lexeo Therapeutics (LXEO) Conference Call Summary Company Overview - Lexeo Therapeutics is a gene therapy company focused on genetic cardiovascular diseases, specifically targeting Friedreich's ataxia and arrhythmogenic cardiomyopathy [2][4] Core Points and Arguments Gene Therapy Approach - Lexeo utilizes AAV vectors for gene delivery, which have shown significant improvements in cardiac symptoms and function at safe doses [3][10] - The company has reached an agreement with the FDA for an accelerated approval path, with a registrational study expected to launch in early 2026 and data readout anticipated in 2027 [4][48] Friedreich's Ataxia (FA) - Approximately 5,000 patients in the U.S. are diagnosed with Friedreich's ataxia, with 70% developing cardiomyopathy, leading to high mortality rates [7][13] - The treatment aims to address the cardiac component of FA, which is responsible for 70% of deaths in these patients [13][15] - Clinical data indicates a 25% reduction in left ventricular mass index (LVMI) in the phase one study, exceeding the FDA's required 10% reduction for approval [22][26] Arrhythmogenic Cardiomyopathy (ACM) - The PKB2 mutation accounts for about 70% of the arrhythmogenic cardiomyopathy population, representing a significant commercial opportunity with around 60,000 patients in the U.S. [8][33] - The gene therapy approach involves delivering a functional copy of the PKP2 gene to restore desmosomal function, which has shown promise in preclinical studies [35][36] - Early clinical data from the first cohort of patients showed a 70% reduction in premature ventricular contractions (PVCs), indicating a potential shift in treatment paradigms [45][46] Additional Important Content Safety and Efficacy - Lexeo reports a compelling safety profile across its clinical programs, with no serious adverse events beyond grade two observed in 23 patients dosed [11] - The AAVrh10 capsid used in therapies has shown 1.5 to 2 times greater biodistribution in the heart compared to other vectors, allowing for lower doses and reduced immune suppression [10][11] Future Milestones - The company plans to provide updates on the statistical analysis plan for the FA program and expects to initiate the registrational study in early 2026 [48] - A broad range of endpoints will be evaluated in the ongoing ACM program, with significant data readouts expected in the second half of the year [49] Financial Position - Lexeo completed an equity financing, providing approximately $181 million in capital, which supports operations into 2028 [50] Community Engagement - There is a passionate patient community advocating for treatments for Friedreich's ataxia, highlighting the urgency and unmet need for effective therapies [14][15] This summary encapsulates the key points discussed during the Lexeo Therapeutics conference call, focusing on the company's innovative gene therapy approaches, clinical progress, and future plans in addressing significant cardiovascular diseases.

NEW YORK, May 21, 2025 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that it will hold its annual general meeting on June 26, 2025 at 2:30 p.m. CET at the Biopark auditorium, 11 rue Watt, 4th floor, 75013 Paris, France. The notice convening the annual general meeting stating the detailed agenda and modalities of participati ...