~ Pivotal study met primary endpoint; high-dose AMT-130 demonstrated statistically significant 75% disease slowing at 36 months as measured by cUHDRS compared to a propensity score-matched external control ~ ~ High-dose AMT-130 also demonstrated statistically significant slowing of disease progression as measured by TFC, a key secondary endpoint, and favorable trends across additional clinical measures ~ ~ Mean cerebrospinal fluid NfL levels were below baseline at 36 months ~ ~ AMT-130 continued to be gener ...

- Non-exclusive license for Solid’s proprietary, next generation capsid, AAV-SLB101, to accelerate development of Kinea Bio’s gene therapy for dysferlin-related limb-girdle muscular dystrophy - - Solid to receive an upfront payment and is eligible for certain development and sales milestones and tiered royalties on net sales -- Solid continues to expand collaborative efforts for AAV-SLB101 with more than 25 agreements and licenses executed to date - CHARLESTOWN, Mass., Sept. 23, 2025 (GLOBE NEWSWIRE) -- Sol ...

Screening closing ahead of expectations, with full enrollment now anticipated in 4Q 2025 (previously 1Q 2026)ARTEMIS topline data readout accelerated to 1Q 2027 REDWOOD CITY, Calif., Sept. 22, 2025 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of intravitreal gene therapy as a potential One And DoneTM treatment to preserve sight for life, today announced it has notified sites that it plans to complete screening in ARTEMIS, its first pivotal Ph ...

BERLIN--(BUSINESS WIRE)--Bayer announced today progress for two potential therapies against Parkinson's disease (PD). A first participant received randomized treatment in the pivotal Phase III clinical trial, exPDite-2, of bemdaneprocel, an investigational cell therapy for PD. At the same time, first European participants have been randomized in REGENERATE-PD, a Phase II clinical trial of AB-1005, an investigational gene therapy. Both therapies are focused on treating moderate-stage PD and are. ...

Adverum Biotechnologies, Inc. (NASDAQ:ADVM) is among the best fundamentally strong penny stocks to buy right now. During the first quarter, Trexquant Investment LP reduced its stake in Adverum Biotechnologies, Inc. (NASDAQ:ADVM) by 80.5%. Following the offload of 77,303 shares, the institutional investor now owns 18,694 shares of the company’s stock, valued at approximately $82,000. According to the recent 13F filing with the SEC, the firm has an ownership of 0.09%. Last month, Adverum Biotechnologies, In ...

Key Takeaways Krystal Biotech gained FDA approval to expand Vyjuvek's label to DEB patients from birth.The update allows at-home administration and flexibility in managing wound dressings.Approvals in Europe and Japan, plus U.S. growth, are expected to boost Vyjuvek sales.Krystal Biotech (KRYS) announced that the FDA has approved a label update of its marketed product, Vyjuvek (beremagene geperpavec-svdt), to include dystrophic epidermolysis bullosa ("DEB") patients from birth.Shares of Krystal Biotech were ...

FDA批准标签更新 - 美国食品药品监督管理局批准Krystal Biotech公司Vyjuvek的标签更新 将适用患者群体扩大至出生起的营养不良性大疱性表皮松解症患者[1] - 标签更新为患者提供用药和伤口敷料管理的完全灵活性 允许在下次换药时移除敷料而非等待24小时[1][3] - 此次更新基于上市后真实世界数据及今年发表的开放标签扩展研究结果 证实了各年龄段患者长期安全性和有效性[4] 疾病背景 - 大疱性表皮松解症是一组罕见遗传性疾病 会导致皮肤和粘膜脆弱 轻微摩擦或创伤即引起疼痛性水疱和皮肤糜烂[2] 市场反应 - William Blair分析师认为家庭给药方式、患者行为改善潜力及海外市场机会将推动收入增长[5] - 分析师Sami Corwin指出公司当前估值未反映Vyjuvek长期收入潜力及未来12个月临床管线催化剂[5] - KRYS股价周一上涨6.01%至152.32美元 市场对标签更新反应积极[6]

公司动态 - 公司宣布FDA批准其基因疗法Vyjuvek（beremagene geperpavec）的更新标签 [6] - 新批准将Vyjuvek的患者资格扩大到包括出生时患有营养不良性大疱性表皮松解症的个体 [6] - 新标签还允许患者从出生开始接受治疗 [6]

SANTA ANA, Calif.--(BUSINESS WIRE)--FUJIFILM Biosciences, a global leader in the innovation and manufacture of cell culture solutions for the Life Science market, today announced the commercial launch of BalanCD HEK293 Perfusion A. Designed to expand and augment the company's portfolio of gene therapy application solutions, BalanCD HEK293 Perfusion A uses suspension HEK293 cells and perfusion technology to provide reliable, efficient, and scalable production of viral vectors for development of. ...

公司财务与资金状况 - Sana Biotechnology通过市场发售和公开发行筹集资金 显著扩大财务运营空间 第二季度末现金7270万美元 增资后增至1.772亿美元 预计资金可支持运营至2026年下半年[1] - Iovance Biotherapeutics第二季度末持有现金及投资3.071亿美元 通过每年1亿美元成本节约措施 将现金运营时间延长至2026年第四季度[11] - Bicycle Therapeutics第二季度末现金及等价物7.215亿美元 通过30%成本削减计划（包括裁员）将财务运营时间延长至2028年[15] 产品研发与临床试验 - Sana Biotechnology糖尿病突破性研究开发无需终身免疫抑制的功能性治疗方法 同时开展异体CAR-T疗法项目：SC291（GLEAM试验）治疗B细胞介导自身免疫疾病 SC262（VIVID试验）治疗复发/难治性B细胞恶性肿瘤 两项I期临床试验正在招募患者 预计2025年公布结果[2] - UP421试验使用低免疫（HIP）修饰胰岛细胞治疗1型糖尿病 旨在实现"无需免疫抑制的广泛可及单次治疗 达到长期正常血糖且无需外源性胰岛素"[3] - Iovance Biotherapeutics开发肿瘤浸润淋巴细胞（TIL）疗法治疗实体瘤 FDA已批准Amtagvi（lifileucel）作为首个实体瘤T细胞疗法 用于转移性黑色素瘤 正在拓展非小细胞肺癌 子宫内膜癌和前线晚期黑色素瘤适应症[7][9][11] - Bicycle Therapeutics采用双环肽技术开发新型药物 主要项目zelenectide pevedotin靶向Nectin-4肿瘤抗原 正在进行转移性尿路上皮癌 非小细胞肺癌等实体瘤的Duravelo I/II/III期临床试验[14] 市场表现与估值 - Sana Biotechnology市值8.376亿美元 年内股价上涨100% 华尔街认为仍有上涨三倍空间[3][4] - 华尔街对SANA股票给出"强力买入"评级 9位分析师中7位建议"强力买入" 2位建议"持有" 平均目标价9.17美元隐含169.7%上涨空间 最高目标价15美元隐含341.2%上涨潜力[6] - Iovance Biotherapeutics市值8.757亿美元 年内股价下跌69% 华尔街最高目标价隐含700%上涨空间[7][8] - 华尔街对IOVA股票给出"适度买入"评级 13位分析师中7位建议"强力买入" 5位建议"持有" 1位建议"强力卖出" 平均目标价9.10美元隐含276%上涨空间 最高目标价20美元隐含726.4%上涨潜力[12] - Bicycle Therapeutics市值4.865亿美元 年内股价下跌51%[13] - 华尔街对BCYC股票给出"适度买入"评级 13位分析师中8位建议"强力买入" 2位建议"适度买入" 3位建议"持有" 平均目标价24.90美元隐含254.7%上涨空间 最高目标价44美元隐含526.7%上涨潜力[16] 商业进展与营收表现 - Iovance第二季度产品总收入6000万美元 同比增长近一倍 其中Amtagvi收入5410万美元（治疗102名美国商业患者） Proleukin（IL-2治疗）额外贡献590万美元销售额 重申全年2.5-3亿美元销售目标[9][10] - Iovance第二季度净亏损1.117亿美元[11] - Bicycle Therapeutics尚未有获批产品 第二季度净亏损7900万美元 研发费用因zelenectide pevedotin及其他管线项目增加[15]