Third-Quarter 2025 Earnings Presentation October 30, 2025 1 Forward Looking Statements The forward-looking statements in this presentation are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timings discussed, projected, anticipated or indicated in any fo ...

Accessibility StatementSkip Navigation ROCKVILLE, Md., Oct. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the completion of enrollment in the AFFINITY DUCHENNE pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, as well as the successful production of the first batches intended for commercial supply. "The Duchenne community urgently needs new treatment options that provide durable, safe outcomes and can meaningfully change the ...

Key Takeaways BioMarin's Q3 EPS of $0.12 beat estimates but fell 87% year over year from Inozyme-related expenses.Revenue rose 4% to $776M, driven by Voxzogo and Palynziq growth, yet came in below consensus views.The company will divest Roctavian to sharpen its focus on strategic growth and future partnerships.BioMarin Pharmaceutical (BMRN) reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks Consensus Estimate of a loss of 15 cents. Quarterly earnings plummeted nearly ...

Accessibility StatementSkip Navigation $3.4 million upfront with up to an additional $6.6 million of potential aggregate gross proceeds upon the exercise in full of short-term warrants AUSTIN, Texas, Oct. 28, 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 that it has entered into definitive agreements for the issuance and sale of  377, ...

-- Data demonstrates sustained clinical improvements in pain, stiffness and function for up to 156 weeks following a single administration of PCRX-201 -- BRISBANE, Calif., Oct. 28, 2025 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (NASDAQ: PCRX), the industry leader in the delivery of innovative, non-opioid pain therapies to transform the lives of patients, today announced updated three-year results and a new subset analyses demonstrating sustained efficacy and safety across all patient subgroups, including ...

Accessibility StatementSkip Navigation Reqorsa® Gene Therapy is a Potential Treatment for ALK-EML4 Positive Translocated Non-Small Cell Lung Cancer Combining REQORSA with Alectinib Further Increased Apoptosis and Improved Treatment Outcomes in a Mouse Model AUSTIN, Texas, Oct. 28, 2025 /PRNewswire/ --Â 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 that its r ...

Taysha Gene Therapies Inc. (NASDAQ:TSHA) is one of the best NASDAQ stocks under $5 to buy now. On October 16, Taysha Gene Therapies announced that it had regained full global rights to its lead program, TSHA-102, which is in clinical evaluation for the treatment of Rett syndrome. The 2022 Option Agreement between Taysha and Astellas has officially expired. The agreement granted Astellas an exclusive option to negotiate an exclusive license to TSHA-102 for Rett syndrome. Taysha now holds unencumbered right ...

My last coverage of Crispr Therapeutics (NASDAQ: CRSP ) and Intellia Therapeutics (NASDAQ: NTLA ) was published in May 2025 . At the time, gene therapy stocks were suffering from regulatory uncertainties, potential pricingOriginally a Biologist, M.Sc in Biomedicine, PhD in Bioengineering, with +20 years experience in the research and development of novel Cell & Gene Therapies (CGT) tackling several clinical needs. As an investor, I have been utilising my background in life sciences to assess the potential o ...

Accessibility StatementSkip Navigation $2.7 million upfront with up to an additional $5.4 million of potential aggregate gross proceeds upon the exercise in full of short-term warrants AUSTIN, Texas, Oct. 23, 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 that it has entered into definitive agreements for the issuance and sale of 243,62 ...

核心观点 - Genprex公司宣布其领先候选药物Reqorsa基因疗法在治疗ALK-EML4阳性非小细胞肺癌的临床前研究中显示出积极数据，肿瘤缩小率达79% [1][2] - 数据表明Reqorsa可能作为单药或与阿来替尼联合使用，为对ALK抑制剂产生耐药性的晚期患者提供新的治疗策略 [2][4] - 这些临床前研究结果支持了公司未来开展针对该适应症的临床试验的路径 [2][8] 数据呈现详情 - 在临床前研究中，Reqorsa单药或与阿来替尼联合使用使肿瘤缩小79%，而阿来替尼单药使肿瘤缩小60%，显示Reqorsa疗效优于阿来替尼23% [7] - 研究通过皮下注射NCI-H2228 ALK+细胞至裸鼠模型进行，当肿瘤生长至约100mm³时，将小鼠分为四组进行不同治疗 [6] - 治疗结束两周后的记录显示，接受单药治疗的小鼠肿瘤再生速度比接受Reqorsa与阿来替尼联合治疗的小鼠更快 [7] 药物作用机制 - Reqorsa基因疗法是一种含有TUSC2基因的质粒，封装于非病毒脂质纳米颗粒中，通过上调TUSC2表达诱导癌细胞凋亡 [5][9] - 临床前研究表明，Reqorsa介导的TUSC2过表达通过增加caspase 3/7活性、促凋亡标志物蛋白表达、DNA碎片化以及降低细胞集落形成能力，显著诱导ALK+细胞系和患者来源类器官凋亡 [6][8] - 实验室研究显示，Reqorsa治疗后肿瘤细胞对TUSC2的摄取量是正常细胞的10至33倍 [9] 临床开发背景 - ALK-EML4融合突变发生在大约5%的非小细胞肺癌患者中，这些肿瘤最初对ALK酪氨酸激酶抑制剂敏感但最终会产生耐药性 [4] - Genprex是一家临床阶段基因治疗公司，专注于为癌症和糖尿病患者开发疗法，其Oncoprex®递送系统采用脂质纳米颗粒封装基因表达质粒 [10] - 公司领先候选药物Reqorsa正在两项针对非小细胞肺癌和小细胞肺癌的临床试验中进行评估，且每个肺癌临床项目均获得了FDA的快速通道认定 [10] 学术合作与数据发布 - 这些积极临床前数据将由Genprex的研究合作方密歇根大学罗格尔癌症中心在2025年EORTC-NCI-AACR分子靶点与癌症治疗国际会议上以海报形式展示 [1][3] - 展示会议定于2025年10月25日美国东部时间下午12:30至4:00进行 [3] - 海报标题为"Quaratusugene ozeplasmid介导的TUSC2上调在携带EML4-ALK的非小细胞肺癌中诱导凋亡并在临床前研究中显示高效" [3]