排名前五的靶点	数量

IL-12(白细胞介素-12)	1
COL3A1(collagen type III alpha 1 chain)	1
COL7A1(VII型胶原蛋白α-1链)	1
TGM1(蛋白-谷氨酰胺γ-谷氨酰转移酶K)	1
elastin	1

关联

项与 Krystal Biotech, Inc. 相关的药物

Beremagene geperpavec

靶点

COL7A1

作用机制

COL7A1基因转移

在研机构

Krystal Biotech, Inc.

原研机构

Krystal Biotech, Inc.

在研适应症

营养不良性大疱性表皮松解

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2023-05-19

KB-105

靶点

TGM1

作用机制

TGM1替代物 [+1]

在研机构

Krystal Biotech, Inc.

原研机构

Krystal Biotech, Inc.

在研适应症

常染色体隐性鱼鳞病

非在研适应症-

最高研发阶段临床2期

首次获批国家/地区-

首次获批日期-

KB-408

靶点

A1AT

作用机制

A1AT刺激剂

在研机构

Krystal Biotech, Inc.

原研机构

Krystal Biotech, Inc.

在研适应症

α1-抗胰蛋白酶缺乏症

非在研适应症-

最高研发阶段临床1期

首次获批国家/地区-

首次获批日期-

项与 Krystal Biotech, Inc. 相关的临床试验

NCT06563414 / RecruitingN/A

A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)

This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). Corneal abrasion symptomology, frequency, and outcomes will be evaluated for up to 6 months.

开始日期2024-08-02

申办/合作机构

Krystal Biotech, Inc.

NCT06228326 / Recruiting临床1期

KB707-02: a Phase 1 Study of Inhaled KB707 in Patients with Advanced Solid Tumor Malignancies Affecting the Lungs

The Sponsor is developing KB707, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector that is designed to stimulate an anti-tumor immune response through the production of cytokines delivered to the airways of people with advanced solid tumor malignancies affecting the lungs via nebulization. This Phase 1, open-label, multicenter, dose escalation and expansion study is designed to evaluate the safety and tolerability of KB707 in adults with with advanced solid tumor malignancies affecting the lungs who have progressed on standard of care therapy, cannot tolerate standard of care therapy, or refused standard of care therapy. The study will include a dose escalation portion for single agent KB707 using a standard 3+3 design followed by an expansion portion to further evaluate single agent KB707 at a dose determined by preliminary data in the dose escalation phase. Subjects in both the dose escalation and dose expansion cohorts will receive KB707 via nebulization weekly for three weeks, then every three weeks for up to two years until tumor progression, death, unacceptable toxicity, symptomatic deterioration, achievement of maximal response, subject choice, Investigator decision to discontinue treatment, or the Sponsor determines to terminate the study.

开始日期2024-04-17

申办/合作机构

Krystal Biotech, Inc.

NCT06049082 / Recruiting临床1期

A Phase 1 Study of Inhaled KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI*ZZ or PI*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. At the high dose, two cohorts will be conducted in parallel to evaluate patients on and off IV augmentation therapy.

开始日期2024-02-15

申办/合作机构

Krystal Biotech, Inc.

100 项与 Krystal Biotech, Inc. 相关的临床结果

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0 项与 Krystal Biotech, Inc. 相关的专利（医药）

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项与 Krystal Biotech, Inc. 相关的文献（医药）

2021-04-01·The Journal of investigative dermatology1区 · 医学

Preclinical Evaluation of a Modified Herpes Simplex Virus Type 1 Vector Encoding Human TGM1 for the Treatment of Autosomal Recessive Congenital Ichthyosis

1区 · 医学

Article

作者: Freedman, John C ; Regula, Lauren K ; O'Malley, Mark ; Ramasamy, Sureshkumar ; Krishnan, Suma ; Agarwal, Pooja ; Zhang, Peipei ; Coghlan, Sarah ; Majumdar, Avijit ; Yogesha, S D ; Parry, Trevor J

Autosomal recessive congenital ichthyosis (ARCI) is a diverse group of cornification diseases associated with severe clinical complications and decreased quality of life. Germline mutations in the TGM1 gene, which encodes the enzyme TGM1, are the predominant cause of ARCI. These TGM1 mutations trigger the abnormal epidermal differentiation and impaired cutaneous barrier function observed in patients with ARCI. Unfortunately, current ARCI therapies focus solely on symptomatic relief. Thus, there is a significant unmet need for therapeutic strategies aimed at correcting the TGM1 deficiency underlying ARCI. In this study, we investigated the ability of KB105, a gene therapy vector encoding full-length human TGM1, to deliver functional human TGM1 to keratinocytes. In vitro, KB105 efficiently infected TGM1-deficient human keratinocytes, produced TGM1 protein, and rescued transglutaminase enzyme function. In vivo studies demonstrated that both single and repeated topical KB105 administration induced TGM1 protein expression in the target epidermal layer without triggering fibrosis, necrosis, or acute inflammation. Toxicity and biodistribution assessments on repeat dosing indicated that KB105 was well-tolerated and restricted to the dose site. Overall, our results demonstrate that rescuing TGM1 deficiency in patients with ARCI through topical KB105 application represents a promising strategy for safely and noninvasively treating this debilitating disease.

2019-07-01·The Journal of investigative dermatology1区 · 医学

Thrombospondin-1 Is a Major Activator of TGF-β Signaling in Recessive Dystrophic Epidermolysis Bullosa Fibroblasts

1区 · 医学

Article

作者: Salas-Alanis, Julio C ; Fuentes, Ignacia ; Agarwal, Pooja ; Russell, Rebecca J ; McGrath, John A ; Guttmann-Gruber, Christina ; South, Andrew P ; Fertala, Andrzej ; Krishnan, Suma ; Cao, Qingqing ; Lim, Yok Zuan ; Webster, Timothy G ; Atanasova, Velina S

Mutations in the gene encoding collagen VII cause the devastating blistering disease recessive dystrophic epidermolysis bullosa (RDEB). RDEB is characterized by severe skin fragility and nonhealing wounds aggravated by scarring and fibrosis. We previously showed that TSP1 is increased in RDEB fibroblasts. Because transforming growth factor-β (TGF-β) signaling is also increased in RDEB, and TSP1 is known to activate TGF-β, we investigated the role of TSP1 in TGF-β signaling in RDEB patient cells. Knockdown of TSP1 reduced phosphorylation of smad3 (a downstream target of TGF-β signaling) in RDEB primary fibroblasts, whereas overexpression of collagen VII reduced phosphorylation of smad3. Furthermore, inhibition of TSP1 binding to the LAP/TGF-β complex decreased fibrosis in engineered extracellular matrix formed by RDEB fibroblasts, as evaluated by picrosirius red staining and analyses of birefringent collagen fibrillar deposits. We show that collagen VII binds TSP1, which could potentially limit TSP1-LAP association and subsequent TGF-β activation. Our study suggests a previously unreported mechanism for increased TGF-β signaling in the absence of collagen VII in RDEB patient skin. Moreover, these data identify TSP1 as a possible target for reducing fibrosis in the tumor-promoting dermal microenvironment of RDEB patients.

PLoS Neglected Tropical Diseases

Transmission of viable Haemophilus ducreyi by Musca domestica

Article

作者: Donlan, Rachel A ; Gaston, Jordan R ; McCandless, Kayla ; Stabile, Haley D ; Humphreys, Tricia L

Haemophilus ducreyi was historically known as the causative agent of chancroid, a sexually-transmitted disease causing painful genital ulcers endemic in many low/middle-income nations. In recent years the species has been implicated as the causative agent of nongenital cutaneous ulcers affecting children of the South Pacific Islands and West African countries. Much is still unknown about the mechanism of H. ducreyi transmission in these areas, and recent studies have identified local insect species, namely flies, as potential transmission vectors. H. ducreyi DNA has been detected on the surface and in homogenates of fly species sampled from Lihir Island, Papua New Guinea. The current study develops a model system using Musca domestica, the common house fly, as a model organism to demonstrate proof of concept that flies are a potential vector for the transmission of viable H. ducreyi. Utilizing a green fluorescent protein (GFP)-tagged strain of H. ducreyi and three separate exposure methods, we detected the transmission of viable H. ducreyi by 86.11% ± 22.53% of flies sampled. Additionally, the duration of H. ducreyi viability was found to be directly related to the bacterial concentration, and transmission of H. ducreyi was largely undetectable within one hour of initial exposure. Push testing, Gram staining, and PCR were used to confirm the identity and presence of GFP colonies as H. ducreyi. This study confirms that flies are capable of mechanically transmitting viable H. ducreyi, illuminating the importance of investigating insects as vectors of cutaneous ulcerative diseases.

187

项与 Krystal Biotech, Inc. 相关的新闻（医药）

2024-09-13

·GlobeNewswire-Health Care

Krystal Biotech to Present at 2024 Cantor Global Healthcare Conference

PITTSBURGH, Sept. 13, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the Company will participate in the 2024 Cantor Global Healthcare Conference on September 18, 2024, in New York. Krish S. Krishnan, Chairman and Chief Executive Officer, will take part in a fireside chat scheduled at 1:20 pm ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 1:20 pm ET on Wednesday, September 18, 2024 and will be posted on the Investors section of the Company’s website. About Krystal Biotech, Inc.Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). CONTACTInvestors and Media:Stéphane PaquetteKrystal Biotechspaquette@krystalbio.com

上市批准

2024-09-06

·GlobeNewswire-Health Care

Krystal Biotech to Present at H.C. Wainwright 26th Annual Global Investment Conference

PITTSBURGH, Sept. 06, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that the Company will participate in the H.C. Wainwright 26th Annual Global Investment Conference on September 10, 2024, in New York. Krish S. Krishnan, Chairman and Chief Executive Officer, is scheduled to give a company presentation at 12:30 pm ET and host investor meetings throughout the day. A webcast of the presentation will be available here beginning at 12:30 pm ET on Tuesday, September 10, 2024 and will be posted on the Investors section of the Company’s website. About Krystal Biotech, Inc. Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). CONTACTInvestors and Media:Stéphane PaquetteKrystal Biotechspaquette@krystalbio.com

上市批准基因疗法

2024-09-02

·派真生物PackGene

一周要闻丨基因治疗要闻速递第118期

行业动态速览派真新闻 01 基因治疗与小核酸药物论坛暨派真生物与益诺思战略合作签约仪式在北京成功举办！ 2024年8月28日下午，2024同写意CGT产业大会在北京盛大开幕。作为峰会的重要组成部分，由上海益诺思生物技术股份有限公司（以下简称“益诺思”）承办的基因治疗与小核酸药物论坛隆重举行。会上，派真生物与益诺思正式宣布达成战略合作并举行签约仪式，此次合作标志着双方将携手共进，共同推动创新药产业快速发展，为人类健康事业贡献更多力量。推荐阅读：基因治疗与小核酸药物论坛暨派真生物与益诺思战略合作签约仪式在北京成功举办！热点聚焦 01 基因治疗逆转衰老，I期临床数据亮眼！近日，Krystal Biotech的全资子公司Jeune Aesthetics宣布了其I期临床试验PEARL-1中关于基因疗法KB301用于眼角皱纹（队列3）和颈部动态皱纹（队列4）治疗的安全性和有效性数据。推荐阅读：基因治疗逆转衰老，I期临床数据亮眼 02 3期临床成功！诺华PCSK9 siRNA计划拓展适应症 2024年8月28日，诺华宣布在III期V-MONO研究中一年两次Leqvio（Inclisiran）取得了积极的初步结果，达到了主要终点。与安慰剂和依折麦布相比，Leqvio单药治疗对动脉粥样硬化性心血管疾病（ASCVD）中低风险且未接受降脂治疗的患者的低密度脂蛋白胆固醇（LDL-C）的降低具有临床意义和统计学意义。推荐阅读：3期临床成功！诺华PCSK9 siRNA计划拓展适应症 03 创新siRNA疗法3期结果登《新英格兰医学杂志》，监管申请递交在即近日，Alnylam Pharmaceuticals公布了其HELIOS-B临床3期研究的积极成果。分析结果显示，与安慰剂相比，皮下注射的siRNA疗法Amvuttra（vutrisiran）显著降低了伴有心肌病的转甲状腺素蛋白淀粉样变性（ATTR-CM）患者因任何原因死亡或复发性心血管事件的风险，同时维持了患者的身体功能和生活质量。这些数据在2024年欧洲心脏病学会（ESC）年会上公布，并同步发表于《新英格兰医学杂志》。基于这一积极结果，Alnylam计划在今年晚些时候使用优先审评券（PRV）向美国FDA递交补充新药申请（sNDA）。推荐阅读：创新siRNA疗法3期结果登《新英格兰医学杂志》，监管申请递交在即 04 加速中美双报！艾美疫苗mRNA RSV与mRNA带状孢疹疫苗向FDA提交临床试验预申请 2024年8月29日，艾美疫苗股份有限公司（“艾美疫苗”）发布公告，该集团按照既定公司战略积极推进疫苗产品管线的开发，利用mRNA技术平台优势，通过持续的技术创新，快速推进mRNA疫苗系列产品的研发，并已于2024年8月首次向美国食品和药品监督管理局(FDA)提交了mRNA RSV(呼吸道合胞病毒)疫苗以及mRNA带状疱疹疫苗的临床试验预申请。推荐阅读：加速中美双报！艾美疫苗mRNA RSV与mRNA带状疱疹疫苗向FDA提交临床试验预申请 05 CDE发布《预防用mRNA疫苗非临床研究技术指导原则（征求意见稿）》意见的通知 2024年8月29日，国家药品监督管理局药品审评中心发布了关于公开征求《预防用mRNA疫苗非临床研究技术指导原则（征求意见稿）》意见的通知。推荐阅读：CDE发布《预防用mRNA疫苗非临床研究技术指导原则（征求意见稿）》意见的通知 06 9月1日起施行！上海发布CAR-T细胞治疗药品监督管理规定 8月28日，上海市药监局发布了《上海市自体嵌合抗原受体T细胞（CAR-T）治疗药品监督管理规定》以及这一监管规定的政策解读通知，本规定自2024年9月1日起施行，有效期5年，有效期至2029年8月31日。推荐阅读：9月1日起施行！上海发布CAR-T细胞治疗药品监督管理规定 07 九天生物AAV基因疗法SKG1108治疗视网膜色素变性获FDA孤儿药资格认定 2024年9月2日 — 九天生物（Skyline Therapeutics）自主研发的腺相关病毒（AAV）基因治疗药物SKG1108，一款用于治疗视网膜色素变性（Retinitis Pigmentosa，RP）的突破性基因疗法，已获得美国食品药品监督管理局（FDA）孤儿药资格认定（ODD）。这一认定标志着SKG1108这一创新AAV载体在治疗RP方面的潜力得到了FDA的认可，并使其在未来的开发、上市过程中及上市后有资格享受一系列激励政策，获得FDA的持续支持。推荐阅读：九天生物AAV基因疗法SKG1108治疗视网膜色素变性获FDA孤儿药资格认定创新突破 01 悉尼大学开发rAAV+睡美人转座子基因递送系统，可用于治疗新生儿遗传性肝病腺相关病毒（rAAV）载体已经成为体内基因治疗的首选工具，尤其在成人肝脏等分裂不活跃的细胞中，rAAV基因治疗药物的疗效显著。然而，在快速增殖的细胞（如新生儿肝脏）中，传统rAAV载体的疗效非常有限，主要原因是rAAV载体携带的外源基因组处于游离状态，容易在细胞复制时发生丢失或稀释，这一局限性也使得一些需要早期干预的遗传性肝脏疾病的治疗成功率较低。为了应对这一挑战，悉尼大学儿童医学研究所研究人员开发了一种rAAV/ Sleeping Beauty（SB）转座子系统，它结合了rAAV的高效肝脏靶向能力和SB系统的基因组整合能力，可用于新生小鼠肝脏的稳定基因转导，相关研究结果发表在The Journal of Gene Medicine杂志上。推荐阅读：悉尼大学开发rAAV+睡美人转座子基因递送系统，可用于治疗新生儿遗传性肝病 02 郑大一附院唐俊楠/哥大程柯ACS Nano: 生物衍生纳米颗粒在心脏修复中的应用与策略郑州大学第一附属医院唐俊楠&哥伦比亚大学程柯已经提出无细胞策略作为心脏组织修复的替代策略。无细胞策略主要基于细胞治疗的旁分泌效应，已经显示出其在抑制心肌梗死后的细胞凋亡、减少炎症、促进现场细胞迁移和增殖以及血管生成方面的潜力，并已在临床前和临床上进行了探索。在各种无细胞方式中，包括腺相关病毒（AAV）、细胞外囊泡、细胞膜包被的纳米颗粒和外泌体模拟纳米囊泡在内的生物衍生纳米颗粒，因其改善的生物学功能和治疗效果而成为有希望的策略。推荐阅读：郑大一附院唐俊楠/哥大程柯ACS Nano: 生物衍生纳米颗粒在心脏修复中的应用与策略 03 最新Nature NBE：低免疫原性环状RNA高效生产新方法加州大学圣地亚哥分校Prashant Mali课题组通过体外和体内两种途径实现了cRNA的高效生产和纯化，提高了cRNA的产量并保持了低免疫反应，同时展示了cRNA在多种细胞类型中的广泛应用，特别是在非分裂细胞中的持久性和表达性。此外，作者还描述了一种新的蛋白工程方法LORAX，用于筛选降低免疫原性的Cas9蛋白变体，从而提高了CRISPR-Cas9系统在基因编辑中的安全性和有效性。相关内容以“Robust genome and cell engineering via in vitro and in situ circularized RNAs”为题发表在《Nature Biomedical Engineering》上。推荐阅读：最新Nature NBE：低免疫原性环状RNA高效生产新方法 04 张锋团队揭示首个真核基因编辑系统Fanzor的结构多样性和DNA切割机制 2024年8月28日，张锋团队（徐沛雨和Makoto Saito为论文共同第一作者）在 Cell 期刊发表了题为：Structural Insights into the Diversity and DNA Cleavage Mechanism of Fanzor 的研究论文【2】。该研究展示了Fanzor蛋白在不同生物中所表现出的分子多样性，并深入解析了其通过RNA引导的DNA切割机制，为进一步的基因工程改造和开发奠定了基础。推荐阅读：张锋团队揭示首个真核基因编辑系统Fanzor的结构多样性和DNA切割机制 05 颠覆教科书！张锋最新Science论文：发现“隐藏基因”，揭开全新遗传调控方式 2024年8月28日，张锋团队（徐沛雨和Makoto Saito为论文共同第一作者）在 Cell 期刊发表了题为：Structural Insights into the Diversity and DNA Cleavage Mechanism of Fanzor 的研究论文【1】。该研究展示了Fanzor蛋白在不同生物中所表现出的分子多样性，并深入解析了其通过RNA引导的DNA切割机制，为进一步的基因工程改造和开发奠定了基础。推荐阅读：颠覆教科书！张锋最新Science论文：发现“隐藏基因”，揭开全新遗传调控方式资本速递 01 开发潜在"first-in-class"抗癌小分子疗法！拜耳达成近5.5亿美元合作拜耳和NextRNA Therapeutics近期宣布，双方已达成一项总额近5.5亿美元的合作和许可协议，将共同开发两项靶向长链非编码RNA（lncRNA）的潜在"first-in-class"小分子疗法，进一步增强拜耳的精准肿瘤学研发管线。推荐阅读：开发潜在"first-in-class"抗癌小分子疗法！拜耳达成近5.5亿美元合作 02 10.35亿元！信立泰引进罕见病基因编辑新药 8月27日，尧唐（上海）生物科技有限公司（下称“尧唐生物”）宣布与深圳信立泰药业股份有限公司（股票代码：002294）（以下简称“信立泰”）就尧唐生物自主研发的针对PCSK9靶点的碱基编辑药物YOLT-101，签订了在中国内地的开发及商业化授权协议，总交易金额近10.35亿元人民币。推荐阅读：10.35亿元！信立泰引进罕见病基因编辑新药参考资料： https://mp.weixin.qq.com/s/0Eu4tgexcU2UWT17mCXCJg https://mp.weixin.qq.com/s/PaGmtZhcUVzFUAwf3fTGig https://mp.weixin.qq.com/s/JstfUFKfthiLCtyqrPvk7Q https://mp.weixin.qq.com/s/ZeDT4iJzH0MLpEwzbwGqAA https://mp.weixin.qq.com/s/jGDDY3Aeo1_ssTdYDaODvg https://mp.weixin.qq.com/s/ykdZrVexdC19q4rBP7-F9A https://mp.weixin.qq.com/s/cpyOwivbZHjfT_8WSZv2ww https://mp.weixin.qq.com/s/NNxng0JMvz18rWBgd0E_sg https://mp.weixin.qq.com/s/CiKQih5oHBk2P-uCvv5OJQ https://mp.weixin.qq.com/s/jO_RK5wTsGPxVo5IFE9zkA https://mp.weixin.qq.com/s/VMHXN1J9T1BcKfj5QlA8ug https://mp.weixin.qq.com/s/5_rgOnTiez3k89K-hYnmBA https://mp.weixin.qq.com/s/lVZotmzMfOwI6QoqgtP0og https://mp.weixin.qq.com/s/U42oDbOABatpiUGlcuVehA https://mp.weixin.qq.com/s/0kMzwpEQOriqxFtZ2h2sFQ 关于派真生物

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药物(靶点)	适应症	全球最高研发状态

Beremagene geperpavec ( COL7A1 )	营养不良性大疱性表皮松解更多	批准上市
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