爆料：诺华首次公布CAR-T细胞制备全过程
来源:杭州联科生物技术股份有限公司
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日/瑞士巴塞尔当地时间8月30日，诺华宣布FDA正式批准其突破性CAR-T疗法Kymriah (tisagenlecleucel) 上市，曾用名CTL019。用于治疗25岁以下的难治复发性B细胞前体急性淋巴性白血病（ALL）患者，定价47.5万美元。
而FDA的这一决定比预定的时间提前了大约1个月，FDA在新闻公告中明确将CAR-T疗法 Kymriah划分为以细胞工程为基础的基因治疗产品，至此，Kymriah成为了人类历史上批准的首款CAR-T疗法，也是在美国境内FDA批准的首款基因疗法！
Kymriah，作为一款需要基因改造的自体T细胞免疫疗法，需要先从个体患者的体内提取出T细胞，并在生产中心进行遗传改造。在那里，这些T细胞会被引入全新的嵌合抗原受体（CAR），使T细胞有能力直接靶向并杀伤带有CD19抗原的白血病细胞。当这些T细胞改造完成后，就会被回输到患者体内，进行治疗。
病人专用的Kymriah包装袋(图片来源 novartis)
当患者的体重&50kg时，Kymriah的给药剂量基于患者的体重，超过50kg时，Kymriah的给药剂量则为固定剂量。
Forpatients 50 kg or less: administer 0.2 to 5.0 x 10^6 CAR-positive viable Tcells per kg body weight
Forpatients above 50 kg: administer 0.1 to 2.5 x 10^ 8 CAR-positive viable T cells
副作用管理策略
除此之外，FDA将细胞因子释放综合症(CRS)列为 Kymriah的黑框警告，提供了风险评估与减轻策略，同时还提供了一种对抗CRS的有效解决方案&罗氏的Actemra（tocilizumab，IL-6R单抗）。
副作用应对
为此，FDA甚至还直接扩大批准了tocilizumab的适应症，用于对抗2岁以上患者接受CAR-T治疗后产生的严重或威胁生命的细胞因子释放综合症(CRS)。在用CAR-T细胞治疗的患者的临床试验中，69％的患者CRS在一个或两个剂量的Actemra后两周内完全消除。
定价47.5万美元
在获批上市之前，CAR-T疗法的定价问题一直很棘手，而此次诺华公司宣布Kymriah（tisagenlecleucel）的单次治疗费用为47.5万美元，是处于预期的40万到75万美元之间的较低价格。
此外，诺华表示正在与CMS（美国医疗保险与医疗补助服务中心）合作，将采用&按疗效付费&的定价方案，只对治疗有效的ALL患者收费，并且付费时间在治疗后的一个月。同时，诺华还表示Kymriah一个月内将在20个中心内提供，之后会达到35个中心。
受此消息影响，刚刚成功牵手Kite的这家公司的股价也一路飙升!
119亿美元全现金收购Kite
8月28日正值中国七夕节，Gilead(吉利德)和Kite Pharma联合宣布，双方已经达成最终收购协议，吉利德将以每股180美元的现金价格收购Kite，总计119亿美元，相比Kite Pharma上周五的收盘价溢价29%。
值得一提的是，此次收购采用的是全现金收购，该交易得到了吉利德公司董事会和风筝医药公司董事会的一致批准，预计将在2017年第四季度完成。同时，本次交易也标志着Gilead正式入主细胞治疗领域。
John Milligan
对此，Gilead公司的CEO John Milligan表示：&收购Kite Pharma将使得Gilead成为细胞治疗领域的领导者，并为推动惠及晚期癌症患者的持续创新提供坚实的基础，细胞治疗领域发展很快，科学技术已开辟了一条明确的可以为患者提供治疗方案的途径，我们对Kite Pharma团队和他们所取得的成就印象深刻，相信细胞治疗将成为治疗癌症的基石，未来，细胞治疗将转型成为我们的主要战略之一。&
众所周知，Kite Pharma作为CAR-T领域的领导者之一，足以与诺华并驾齐驱。这也就是Gilead为何如此钟情Kite，创下最壕收购记录。
2017年8月，FDA已经完成了对Kite Pharma公司的CAR-T疗法axicabtagene ciloleucel（axi-cel，KTE-C19）的制造基地以及治疗中心的严格审查，并表示其公司的重磅CAR-T疗法不需要再经过专家委员会评审了。(Kite公司公布CAR-T细胞制备过程最新视频，非常震撼)
基于此，Kite公司的高层领导也表示他们已经为axi-cel提前上市做好了充分的准备，其CAR-T疗法的制造与物流管理工作在9月份将完全准备好，随时待命。
可见第二款CAR-T细胞药物获批上市在即，Kite和Juno的专利纷争也是时候完结了。(Kite和Juno CAR-T专利之争愈演愈烈，能否顺利上市就看FDA了)
总而言之，&细胞免疫疗法是一个极其疯狂的领域，竞争激烈，未来发展值得期待&，这句话并没有丝毫夸张，细胞免疫疗法这个新兴的癌症克星正得到前所未有的关注
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邮政编码：310012Novartis AG:開發中產品分析
Novartis AG - Product Pipeline Review - 2016
出版日期: 日
內容資訊: 英文 489 Pages
Novartis AG在國際的醫藥品企業，集中性地致力於新醫藥品解決方案的藥物研發·開發。供給著還有人·動物用的所有疾病·障礙作為對象的種類的處方藥·非專利產品。作為成為該公司的產品的對象的具體的疾病·障礙，癌症·心血管疾病·皮膚病·神經病變·眼科疾病·呼吸疾病·免疫障礙·感染疾病等能舉出(舉行)。在該公司還有預防用疫苗和OTC (一般藥)也銷售著。主要的活動地區成為美國·歐洲和其他的國? (RoW) 。
本報告提供美國的生物醫藥品企業，Novartis AG的醫藥品的研究開發的進展相關分析，與該公司的產品平台結構，及臨床實驗的整體進展，的主要開發中產品簡介與開發情形，該公司簡介調查著最新趨勢等。
Novartis AG:現狀
研究開發(R&D)概況
主要的治療領域
開發平台·檢討
各開發階段的開發中產品
開發中產品:單劑治療藥
開發中產品:並用治療模式
開發中產品:合作夥伴產品
開發中產品:轉出授權產品
開發中產品的概要
後期階段的開發中產品
登記前產品/並用治療模式
登記駁回的產品/並用治療模式
第三階段產品/並用治療模式
臨床實驗階段的開發中產品
第二階段產品/並用治療模式
第一階段產品/並用治療模式
初期階段的開發中產品
臨床實驗前產品/並用治療模式
藥物研發階段的產品/並用治療模式
開發階段未公開的開發中產品
非公開產品/並用治療模式
藥物簡介 (全170種)
(indacaterol maleate + glycopyrrolate)
研究開發(R&D)的發展
(sacubitril + valsartan)
eltrombopag olamine
erismodegib
glycopyrrolate
haemophilus influenzae [serotype B] vaccine
influenza [strains A/H1N1 + A/H3N2 + B] (trivalent, split virion) vaccine
omalizumab
panobinostat
ruxolitinib phosphate
trametinib dimethyl sulfoxide
imatinib mesylate
pazopanib hydrochloride
(vildagliptin + metformin hydrochloride)
aliskiren fumarate
bimagrumab
buparlisib hydrochloride
canakinumab
dabrafenib mesylate
everolimus
fingolimod hydrochloride
influenza [strains A/H1N1 + A/H3N2 + B] (trivalent) vaccine
midostaurin
ofatumumab
osilodrostat
pasireotide
pasireotide LAR
pradigastat sodium
ranibizumab
ribociclib
seasonal influenza (quadrivalent) vaccine
secukinumab
zoledronic acid
(VAK-694 + dectrekumab)
aldesleukin
capmatinib
cipargamin
dectrekumab
fevipiprant
GSK-2285921
infigratinib
influenza [strain H5N1] vaccine
ligelizumab
mavoglurant
octreotide acetate long acting
seasonal influenza (trivalent) vaccine
selurampanel
tisagenlecleucel-T
lapatinib ditosylate
sotrastaurin acetate
Cell Therapy to Target c-Met for Cancer
Cell Therapy to Target EGFR for Glioblastoma
influenza [strain A/H3N2] (monovalent) vaccine
influenza [strain H7N9] (monovalent) vaccine
octreotide acetate LAR
Antibody to Inhibit PD-L1 for Cancer
Antibody to Inhibit TIM-3 for Cancer
Cell Therapy Program for Cancer
Cell Therapy to Target 5T4 for Oncology
Cell Therapy to Target GD2 for Neuroblastoma
cosyntropin ER
GNFPf-5069
Kartogenin
Monoclonal Antibody Conjugated 3 for Cancer
Monoclonal Antibody to Inhibit CXCR2 for Respiratory Diseases
NVPQBE-170
NVPTNKS-656
Small Molecule to Activate Nurr1 for Multiple Sclerosis
Small Molecule to Agonize GPR119 for Type 2 Diabetes
Small Molecule to Agonize GPR39 for Type 2 Diabetes
Small Molecule to Inhibit LRRK2 for Parkinsons Disease
Small Molecule to Inhibit OX2R for Insomnia
Small Molecules to Antagonize CSF1R for Cancer and Osteolysis
Small Molecules to Inhibit Dipeptidyl Peptidase IV for Type 2 Diabetes
Small Molecules to Inhibit Pan-RAF Kinase for Oncology
Antibodies to Inhibit CTLA4 and PD-1 for Cancer
ESBA-903 SR
Small Molecule to Agonize Adrenergic Beta-2 Receptor for COPD
Small Molecules 1 for Undisclosed Indication
Small Molecules 2 for Undisclosed Indication
Small Molecules for Malaria
Small Molecules for Rhinovirus Infection
Small Molecules for Tuberculosis
Small Molecules to Inhibit Acetyl-CoA Carboxylase for Metabolic, Cardiovascular and Infections
Small Molecules to Inhibit CFTR for Secretory Diarrhea
Small Molecules to Inhibit PI4KIIIbeta for HCV
Small Molecules to Inhibit Renin for Hypertension
Small Molecules for Mycobacterium Tuberculosis
開發平台分析
各分子類型
各作用機制
開發中產品的最新趨勢
開發暫停的產品
開發中止的產品
開發中止的產品簡介
企業的聲明
公司所在地及子公司
其他的公司所在地·子公司
Product Code: GMDHC07987CDBSummary
Global Markets Direct's, 'Novartis AG - Product Pipeline Review - 2016', provides an overview of the Novartis AG's pharmaceutical research and development focus.
The report provides comprehensive information on the therapeutics under development by Novartis AG, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and the dormant and discontinued projects.
Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.
Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.
The report provides a snapshot of the pipeline therapeutic landscape of Novartis AG
The report provides overview of Novartis AG including its business description, key facts, and locations and subsidiaries
The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
The report assesses Novartis AG's pipeline therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
The report features Novartis AG's out-licensed and partnered product portfolio and summarizes its dormant and discontinued projects
Reasons to buy
Evaluate Novartis AG's strategic position with total access to detailed information on its product pipeline
Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
Identify and understand important and diverse types of therapeutics under development for Novartis AG
Identify potential new clients or partners in the target demographic
Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
Devise corrective measures for pipeline projects by understanding Novartis AG's pipeline depth and focus of pipeline therapeutics
Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
Table of Contents
Table of Contents
List of Tables
List of Figures
Novartis AG Snapshot
Novartis AG Overview
Key Information
Novartis AG - Research and Development Overview
Key Therapeutic Areas
Novartis AG - Pipeline Review
Pipeline Products by Stage of Development
Pipeline Products - Monotherapy
Pipeline Products - Combination Treatment Modalities
Pipeline Products - Partnered Products
Pipeline Products - Out-Licensed Products
Novartis AG - Pipeline Products Glance
Novartis AG - Late Stage Pipeline Products
Novartis AG - Clinical Stage Pipeline Products
Novartis AG - Early Stage Pipeline Products
Novartis AG - Drug Profiles
dabrafenib mesylate + trametinib dimethyl sulfoxide
eltrombopag olamine
everolimus
trametinib dimethyl sulfoxide
zoledronic acid
imatinib mesylate
panobinostat
pazopanib hydrochloride
(glycopyrrolate + indacaterol maleate + mometasone fuorate)
(indacaterol maleate + mometasone furoate)
(sacubitril + valsartan)
bimagrumab
buparlisib hydrochloride
canakinumab
dabrafenib mesylate
fevipiprant
fingolimod hydrochloride
midostaurin
ofatumumab
osilodrostat phosphate
pasireotide
pasireotide ER
ranibizumab
ribociclib
secukinumab
clofazimine
(dectrekumab + VAK-694)
afuresertib hydrochloride
aldesleukin
capmatinib
cipargamin
dectrekumab
elgemtumab
GSK-2285921
iloperidone
infigratinib
ligelizumab
mavoglurant
octreotide acetate long acting
pradigastat sodium
ruxolitinib phosphate
selurampanel
sonidegib phosphate
tesidolumab
tisagenlecleucel-T
trametinib dimethyl sulfoxide + uprosertib
uprosertib
lapatinib ditosylate
sotrastaurin acetate
Cellular Immunotherapy to Target c-Met for Oncology
Cellular Immunotherapy to Target CD22 for Acute Lymphoblastic Leukemia
Cellular Immunotherapy to Target EGFR for Glioblastoma
octreotide acetate LAR
Cellular Immunotherapy to Target B-Cell Maturation Factor for Multiple Myeloma
Antibody to Inhibit PD-L1 for Oncology
Cellular Immunotherapy Program for Oncology
Cellular Immunotherapy to Target GD2 for Neuroblastoma
cosyntropin ER
GNFPf-5069
Kartogenin
Monoclonal Antibody to Inhibit CXCR2 for Respiratory Diseases
NVPQBE-170
NVPTNKS-656
Small Molecule to Agonize GPR119 for Type 2 Diabetes
Small Molecule to Agonize GPR39 for Type 2 Diabetes
Small Molecule to Agonize Nurr1 for Multiple Sclerosis
Small Molecule to Inhibit LRRK2 for Parkinson's Disease
Small Molecule to Inhibit OX2R for Insomnia
Small Molecules to Antagonize VEGFR2 for Solid Tumors
Small Molecules to Inhibit Cytochrome bc1 Complex for Tuberculosis
Small Molecules to Inhibit Dipeptidyl Peptidase IV for Type 2 Diabetes
Small Molecules to Inhibit Pan PI3K for Ovarian Cancer
Small Molecules to Inhibit Pan-RAF Kinase for Oncology
Small Molecules to Inhibit Renin for Hypertension
ESBA-903 SR
Monoclonal Antibodies to Inhibit CTLA4 and PD-1 for Oncology
Small Molecule to Agonize Adrenergic Beta-2 Receptor for COPD
Small Molecules for Tuberculosis
Small Molecules for Undisclosed Indication
Small Molecules to Antagonize NR2B for Depression
Small Molecules to Inhibit Acetyl-CoA Carboxylase for Metabolic, Cardiovascular and Infections
Small Molecules to Inhibit CFTR for Secretory Diarrhea
Small Molecules to Inhibit FPPS for Soft Tissue Cancer
Small Molecules to Inhibit PfATP4 for Malaria
Small Molecules to Inhibit PI4KIIIbeta for HCV
Novartis AG - Pipeline Analysis
Novartis AG - Pipeline Products by Target
Novartis AG - Pipeline Products by Route of Administration
Novartis AG - Pipeline Products by Molecule Type
Novartis AG - Pipeline Products by Mechanism of Action
Novartis AG - Recent Pipeline Updates
Novartis AG - Dormant Projects
Novartis AG - Discontinued Pipeline Products
Novartis AG - Discontinued Pipeline Products
Discontinued Pipeline Product Profiles
Novartis AG - Company Statement
Novartis AG - Locations And Subsidiaries
Head Office
Other Locations & Subsidiaries
Methodology
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Novartis AG, Key Information
Novartis AG, Key Facts
Novartis AG - Pipeline by Indication, 2016
Novartis AG - Pipeline by Stage of Development, 2016
Novartis AG - Monotherapy Products in Pipeline, 2016
Novartis AG - Combination Treatment Modalities in Pipeline, 2016
Novartis AG - Partnered Products in Pipeline, 2016
Novartis AG - Partnered Products/ Combination Treatment Modalities, 2016
Novartis AG - Out-Licensed Products in Pipeline, 2016
Novartis AG - Out-Licensed Products/ Combination Treatment Modalities, 2016
Novartis AG - Pre-Registration, 2016
Novartis AG - Filing rejected/Withdrawn, 2016
Novartis AG - Phase III, 2016
Novartis AG - Phase II, 2016
Novartis AG - Phase I, 2016
Novartis AG - Phase 0, 2016
Novartis AG - Preclinical, 2016
Novartis AG - Discovery, 2016
Novartis AG - Pipeline by Target, 2016
Novartis AG - Pipeline by Route of Administration, 2016
Novartis AG - Pipeline by Molecule Type, 2016
Novartis AG - Pipeline Products by Mechanism of Action, 2016
Novartis AG - Recent Pipeline Updates, 2016
Novartis AG - Dormant Developmental Projects,2016
Novartis AG - Discontinued Pipeline Products, 2016
Novartis AG, Subsidiaries
List of Figures
Novartis AG - Pipeline by Top 10 Indication, 2016
Novartis AG - Pipeline by Stage of Development, 2016
Novartis AG - Monotherapy Products in Pipeline, 2016
Novartis AG - Combination Treatment Modalities in Pipeline, 2016
Novartis AG - Partnered Products in Pipeline, 2016
Novartis AG - Out-Licensed Products in Pipeline, 2016
Novartis AG - Pipeline by Top 10 Target, 2016
Novartis AG - Pipeline by Top 10 Route of Administration, 2016
Novartis AG - Pipeline by Top 10 Molecule Type, 2016
Novartis AG - Pipeline Products by Top 10 Mechanism of Action, 2016美食, 餐厅餐饮, 团购,生活,优惠券-大众点评网
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