Quaratusugene ozeplasmid

如果你以为生物技术还停留在「实验室里慢慢磨」的阶段，那最近密集公布的几组数字可能会彻底刷新你的认知。 先看最让人坐不住的一条：礼来旗下的基因编辑疗法 VERVE-102，在 I 期临床中用单次注射把坏胆固醇干掉了 62%。一次治疗，替代终生服药。而它靶向的 PCSK9 基因，关联的是全球头号死因——心血管疾病。 这不是实验室里的论文。这是 III 期临床就在眼前的产品。 再看第二条：Precedence Research 最新报告把全球生物技术市场的 2035 年目标定在了 6.34 万亿美元。2025 年是 1.77 万亿——十年翻 3.6 倍，年复合增长率 13.61%。 凭什么？ 答案不在某一个突破里，而在三个引擎同时点火。 第一台引擎：AI 把研发从十年压进几个月 Nature 最近发表了一篇标题很直白的文章——《编程生物学：下一代 AI 公司正在筹集数十亿美元来设计更好的药物》。 文章的核心结论就一句话：AI 正在把药物发现从「碰运气」变成「可编程」。 具体发生了什么？ 5 月 20 日，Incyte 签下一笔 1.2 亿美元的 AI 药物开发大单；同一天，QIAGEN 宣布与 NVIDIA 合作，把 BioNeMo 平台和自己的生物医学知识图谱对接，目标是用 GPU 集群直接「算」出新药分子。Benchling 和 Baseten 同期联手，把 GPU 推理能力塞进了生物技术公司的日常研发管线。 更关键的是，FDA 在 4 月底破天荒地公开征求意见，准备启动一个 AI 辅助早期临床试验的试点项目。这意味着监管端已经开始为「AI 造药」铺路。 从找靶点、设计分子、到上临床、过审批——AI 不是在某一个环节提效，而是在整条链上重构游戏规则。Precedence Research 的首席顾问 Rohan Patil 的原话更狠：「下一波生物技术领导者，将不再只由科学创新定义，而是由他们整合 AI、可规模化生物制造和数据驱动临床开发的能力来定义。」 第二台引擎：基因编辑从罕见病变大众病 就在本周，ASCO 2026 年会上扔出了好几颗重磅炸弹。 Genprex 的 Reqorsa 基因疗法在肺癌患者中拿出积极生物标志物数据——Trop-2 高表达、PTEN 低表达的患者无进展生存期显著延长。这不是动物实验，是人体数据。 然后是礼来。这家全球市值最高的药企刚刚宣布，从 Verve Therapeutics 拿来的 VERVE-102 在 I 期临床中表现惊艳：高剂量组一次注射，低密度脂蛋白胆固醇降幅达到 62%。关键是一次治疗，不是每天吃药。 这才是基因编辑真正的拐点：从治疗几千人的罕见遗传病，切换到影响几亿人的慢性病。 同一天，礼来还宣布以最高 38 亿美元一口气收购三家传染病疫苗开发商——这家公司正在把基因技术和免疫学武装到牙齿。 全球生物技术市场报告里写得很清楚：组织工程与再生医学板块贡献了最大的技术份额（19.17%），生物制药应用占了 42%。这两个板块的共同底色，就是基因和细胞层面的精准操作。 第三台引擎：生物导弹 ADC 进入爆发期 如果你还没关注 ADC（抗体偶联药物），现在该补课了。 简单说，ADC 就是把化疗毒药挂在抗体上，像导弹一样精准炸掉癌细胞，正常细胞几乎不受影响。 5 月 22 日，阿斯利康/第一三共的 DATROWAY 获 FDA 批准，成为全球首个用于一线转移性三阴性乳腺癌的 TROP2 靶向 ADC。三阴性乳腺癌是乳腺癌里最难治的一种，这个批文可以说是在最难打的战场上撕开了一道口子。 同一周，ASCO 2026 年会的日程表被 ADC 占据了至少三分之一：SystImmune 一口气拿出 4 个 ADC 管线的临床数据；Formosa Pharmaceuticals 展示了新型双特异性 ADC（能同时打两个靶点）；Adcendo 宣布和默沙东合作，把自家 ADC 和「药王」KEYTRUDA 联用。 Gilead/Kite 更绝——同时在 ADC 和 CAR-T 细胞疗法两条线亮数据，Trodelvy（ADC）+ Anito-cel（CAR-T）双线作战。 如果说 AI 解决的是「怎么更快找到药」，基因编辑解决的是「怎么从根上修好病」，那 ADC 和细胞疗法解决的就是「怎么把药精准送到该去的地方」。三台引擎，对应研发、治疗、递送三个核心瓶颈，同时被突破。 中国在哪里？ 这份 6.34 万亿美元的报告有一个容易被忽略的关键数据：亚太地区是增速最快的市场，CAGR 14.8%，超过全球平均的 13.61%。 不是偶然。本周 ASCO 2026 上，亚盛医药（Ascentage Pharma）发布了多项临床试验的最新数据。这家总部在苏州、纳斯达克和港交所双重上市的中国药企，正在全球最大的肿瘤学会上跟跨国巨头同台竞技。 而这只是中国生物技术正在发生的事的一个剪影。过去五年，中国在基因编辑、CAR-T、AI 制药、ADC 上建立了完整的产业链——从上游的基因测序，到中游的 CDMO 代工，到下游的商业化销售。 Precedence Research 的报告还指出，欧洲制药 CDMO 市场也将在 2035 年达到 949 亿美元——生物制剂和先进疗法的代工需求正在爆炸。而中国 CDMO 的成本优势和技术积累，正在全球供应链里切走越来越大的蛋糕。 十年翻 3.6 倍，不是线性外推，是三台引擎叠加的指数效应。 AI 加速发现 → 基因编辑根治疾病 → ADC 精准递送 → 更多临床成功 → 更多资本涌入 → AI 再加速——这不是三个独立的趋势，而是一条正在闭环的飞轮。 6.34 万亿美元听起来很大。但如果你仔细看这三台引擎现在的转速，它可能还保守了。 点评新视界 - 深度洞察，科技先行 专注全球科技前沿，带来专业视角与深度解读 助力把握时代脉搏，发现未来机遇

Trop-2 H-scores Above 100 and PTEN H-scores Below 100 Correlated With Longer Progression Free Survival in Non-Small Cell Lung Cancer Patients Identified Biomarkers May Predict Patient Response, Enable Targeted Patient Selection and Allow for Precision Medicine AUSTIN, Texas, May 26, 2026 /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 its research collaborators' abstract was published at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. The abstract details positive clinical data from studies of predictive biomarkers in patients receiving its lead drug candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), for the treatment of lung cancer. "This clinical validation, derived from patients in our Acclaim clinical trials, substantiates earlier preclinical evidence revealing that Non-Small Cell Lung Cancer (NSCLC) patients receiving REQORSA who exhibit high Trop-2 levels and low PTEN levels experience prolonged Progression Free Survival (PFS), underscoring the critical role these biomarkers play in predicting treatment efficacy and advancing our understanding of this novel gene therapy," said Ryan Confer, President and Chief Executive Officer at Genprex. "These findings represent a substantial leap forward for personalized medicine in lung cancer, allowing for a targeted approach to potentially improve treatment outcomes and optimize resource allocation within the therapeutic landscape." The featured Genprex-supported abstract at ASCO 2026: Title: "Predictive biomarkers for PFS in patients receiving quaratusugene ozeplasmid" Abstract Number: e15184 Quaratusugene ozeplasmid is a gene therapy that delivers a plasmid coding for the TUSC2 tumor suppressor gene to lung cancer cells, as >80% of lung cancers have been shown to have decreased or absent TUSC2 protein. TUSC2 protein levels have not correlated with PFS, presumably because of the complexities of TUSC2 protein regulation. Preclinical studies have identified higher levels of Trop-2 protein in organoids and lower levels of PTEN protein in lung cancer cell lines as correlating with response (AACR 2026). Tumor tissue from patients in clinical trials with quaratusugene ozeplasmid were evaluated for Trop-2 and PTEN protein expression. Monoclonal antibodies against Trop-2 (BSB148 from BioSB) and PTEN (138G6 from Cell Signaling Technology) were used for immunohistochemistry in paraffin sections from archival tumor samples in patients enrolled in three clinical trials with quaratusugene ozeplasmid and results expressed as H-scores. H-scores were calculated by evaluating diaminobenzidine staining intensity using the formula [1 × (% cells 1+) + 2 × (% cells 2+) + 3 × (% cells 3+)]. Data on Trop-2 and PTEN protein expression and data on PFS were available from 18 patients enrolled in clinical trials with quaratusugene ozeplasmid. Six patients with NSCLC were enrolled in the Acclaim-1 trial in combination with osimertinib. One patient was enrolled in the Acclaim-2 trial in combination with pembrolizumab. Eleven patients with small cell lung cancer (SCLC) were enrolled in the Acclaim-3 trial in combination with atezolizumab. In patients with NSCLC, Trop-2 H-scores above 100 correlated with prolonged PFS (p=0.05), and PTEN H-scores below 100 correlated with prolonged PFS (p=0.03). In patients with SCLC, Trop-2 H-scores were universally low, and thus non-evaluable. PTEN H-scores in patients with SCLC did not correlate with prolonged PFS (p=0.53). Following up on preclinical cell line and organoid models indicating that Trop-2 and PTEN protein expression correlated with response, levels of Trop-2 and PTEN protein were evaluated in patients treated with quaratusugene ozeplasmid. In conclusion, both Trop-2 H-scores above 100 and PTEN H-scores below 100 correlated with longer PFS in patients with NSCLC, but not in patients with SCLC. Beyond the ASCO 2026 Abstract: Following the clinical studies outlined above, Genprex completed additional analysis to evaluate the relationship between NSCLCs with high intensity staining (3+) and PFS. NSCLCs with 3+ Trop-2 staining had a strong relationship with PFS that was just outside the bounds for significance (p=0.053) and those with 3+ PTEN staining exhibited a trend for a negative relationship with PFS that was not statistically significant (p=0.309). These results are consistent with the H-score analysis regarding a strong positive relationship between Trop-2 expression and PFS. High (3+) Trop-2 expression will be investigated further as a potential biomarker for REQORSA. "We look forward to additional studies using intensity staining to understand the correlation between Trop-2 expression and PFS, offering more concrete data for optimized patient selection," said Mark S. Berger, Chief Medical Officer at Genprex. About Acclaim-1 The Acclaim-1 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating REQORSA in combination with AstraZeneca's Tagrisso® (osimertinib) in patients with late-stage NSCLC with activating epidermal growth factor receptor (EGFR) mutations whose disease progressed after treatment with Tagrisso or or Tagrisso-containing regimens. Acclaim-1 received Fast Track Designation by the U.S. Food and Drug Administration (FDA) for use of REQORSA in combination with TKI Tagrisso for the treatment of NSCLC patients with EGFR mutations whose tumors progressed after treatment with Tagrisso. About Acclaim-3 The Acclaim-3 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating REQORSA in combination with Genentech, Inc.'s Tecentriq® (atezolizumab) as maintenance therapy in patients with extensive stage small cell lung cancer (ES-SCLC) who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Acclaim-3 received Fast Track Designation by the FDA for the Acclaim-3 treatment combination of REQORSA and Tecentriq as maintenance therapy in patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. The FDA also granted Orphan Drug Designation to REQORSA for the treatment of SCLC. About Genprex, Inc. Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex's technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex's oncology program utilizes its systemic, non-viral Oncoprex® Delivery System which encapsulates the gene-expressing plasmids using lipid-based nanoparticles in a lipoplex form. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company's lead product candidate, Reqorsa® Gene Therapy (quaratusugene ozeplasmid), is being evaluated in two clinical trials as a treatment for NSCLC and SCLC. Each of Genprex's lung cancer clinical programs has received a Fast Track Designation from the FDA for the treatment of that patient population, and Genprex's SCLC program has received an FDA Orphan Drug Designation. Genprex's diabetes gene therapy approach is comprised of a novel infusion process that uses an AAV vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body's immune system. In a similar approach for Type 2 diabetes, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells. Interested investors and shareholders are encouraged to sign up for press releases and industry updates by visiting the Company Website, registering for Email Alerts and by following Genprex on Twitter, Facebook and LinkedIn. Cautionary Language Concerning Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made on the basis of the current beliefs, expectations and assumptions of management, are not guarantees of performance and are subject to significant risks and uncertainty. These forward-looking statements should, therefore, be considered in light of various important factors, including those set forth in Genprex's reports that it files from time to time with the Securities and Exchange Commission and which you should review, including those statements under "Item 1A – Risk Factors" in Genprex's Annual Report on Form 10-K for the year ended December 31, 2025. Because forward-looking statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: Genprex's ability to advance the clinical development, manufacturing and commercialization of its product candidates in accordance with projected timelines and specifications; the timing and success of Genprex's clinical trials, its intended regulatory submissions and any resulting regulatory approvals; the effect of Genprex's product candidates, alone and in combination with other therapies, on cancer and diabetes, including allowing for a targeted approach to potentially improve treatment outcomes and optimize resource allocation within the therapeutic landscape and High (3+) Trop-2 expression as a potential biomarker for REQORSA; Genprex's future growth and financial status, including Genprex's ability to maintain compliance with the continued listing requirements of The Nasdaq Capital Market and to continue as a going concern and to obtain capital to meet its long-term liquidity needs on acceptable terms, or at all; Genprex's commercial and strategic partnerships, including those with its third party vendors, suppliers and manufacturers and their ability to successfully perform and scale up the manufacture of its product candidates; Genprex's intellectual property and licenses; and Genprex's current expectations, estimates, forecasts and projections about the industry and markets in which it operates. These forward-looking statements should not be relied upon as predictions of future events and Genprex cannot assure you that the events or circumstances discussed or reflected in these statements will be achieved or will occur. If such forward-looking statements prove to be inaccurate, the inaccuracy may be material. You should not regard these statements as a representation or warranty by Genprex or any other person that Genprex will achieve its objectives and plans in any specified timeframe, or at all. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Genprex disclaims any obligation to publicly update or release any revisions to these forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release or to reflect the occurrence of unanticipated events, except as required by law. Genprex, Inc. (877) 774-GNPX (4679) GNPX Investor Relations [email protected] GNPX Media Contact Kalyn Dabbs [email protected] SOURCE Genprex, Inc. 21% more press release views with Request a Demo