Crigler-Najjar Syndrome - Pipeline Review, H2 2017

Publisher Name :
Date: 08-Aug-2017
No. of pages: 36
Inquire Before Buying

Crigler-Najjar Syndrome - Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Crigler-Najjar Syndrome - Pipeline Review, H2 2017, provides an overview of the Crigler-Najjar Syndrome (Genetic Disorders) pipeline landscape.

Crigler-Najjar syndrome is a severe condition characterized by high levels of a toxic substance called bilirubin in the blood (hyperbilirubinemia). Symptoms include yellow skin (jaundice) and yellow in the whites of the eyes (icterus). It occurs when the enzyme that normally converts bilirubin into a form that can easily be removed from the body does not work correctly. Treatment includes phototherapy, liver transplantation and blood transfusions.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Crigler-Najjar Syndrome - Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Crigler-Najjar Syndrome (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Crigler-Najjar Syndrome (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Crigler-Najjar Syndrome and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed and Preclinical stages are 1, 1 and 4 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Crigler-Najjar Syndrome (Genetic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Crigler-Najjar Syndrome (Genetic Disorders).

- The pipeline guide reviews pipeline therapeutics for Crigler-Najjar Syndrome (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

- The pipeline guide reviews key companies involved in Crigler-Najjar Syndrome (Genetic Disorders) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Crigler-Najjar Syndrome (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

- The pipeline guide reviews latest news related to pipeline therapeutics for Crigler-Najjar Syndrome (Genetic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Find and recognize significant and varied types of therapeutics under development for Crigler-Najjar Syndrome (Genetic Disorders).

- Classify potential new clients or partners in the target demographic.

- Develop tactical initiatives by understanding the focus areas of leading companies.

- Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.

- Formulate corrective measures for pipeline projects by understanding Crigler-Najjar Syndrome (Genetic Disorders) pipeline depth and focus of Indication 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.

- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Crigler-Najjar Syndrome - Pipeline Review, H2 2017

Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Crigler-Najjar Syndrome - Overview
Crigler-Najjar Syndrome - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Crigler-Najjar Syndrome - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Crigler-Najjar Syndrome - Companies Involved in Therapeutics Development
Audentes Therapeutics Inc
Genethon SA
International Stem Cell Corp
Promethera Biosciences SA
Crigler-Najjar Syndrome - Drug Profiles
ALXN-1540 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ALXN-1630 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-342 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Glucuronosyltransferase for Crigler-Najjar Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate UGT1A1 for Crigler-Najjar Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HepaStem - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Alpha-Antitrypsin Deficiency, Crigler-Najjar Syndrome and Liver Cirrhosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Crigler-Najjar Syndrome - Product Development Milestones
Featured News & Press Releases
May 03, 2017: Audentes Therapeutics Announces Presentation of Data on at the American Society of Gene and Cell Therapy Annual Meeting
Mar 01, 2017: Audentes Therapeutics Announces Commencement of LUSTRO, a Clinical Assessment and Phase 1/2 Run-in Study of Patients with Crigler-Najjar Syndrome
Feb 01, 2017: Audentes Therapeutics Announces FDA Clearance of Investigational New Drug Application for AT342 to Treat Crigler-Najjar Syndrome
May 07, 2015: Evidence of efficacy of gene therapy in rodents affected by a rare genetic liver disease, Crigler-Najjar syndrome
Feb 12, 2013: International Stem Cell Demonstrates Positive Animal Efficacy Results In Metabolic Liver Disease Program
Jan 07, 2013: International Stem Cell Announces Positive Animal Efficacy Results In Liver Disease Program
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Crigler-Najjar Syndrome, H2 2017
Number of Products under Development by Companies, H2 2017
Number of Products under Development by Universities/Institutes, H2 2017
Products under Development by Companies, H2 2017
Products under Development by Universities/Institutes, H2 2017
Number of Products by Stage and Target, H2 2017
Number of Products by Stage and Mechanism of Action, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Crigler-Najjar Syndrome - Pipeline by Audentes Therapeutics Inc, H2 2017
Crigler-Najjar Syndrome - Pipeline by Genethon SA, H2 2017
Crigler-Najjar Syndrome - Pipeline by International Stem Cell Corp, H2 2017
Crigler-Najjar Syndrome - Pipeline by Promethera Biosciences SA, H2 2017

List of Figures
Number of Products under Development for Crigler-Najjar Syndrome, H2 2017
Number of Products under Development by Companies, H2 2017
Number of Products by Targets, H2 2017
Number of Products by Stage and Targets, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017
  • Sarcopenia - Pipeline Review, H2 2018
    Published: 17-Jul-2018        Price: US 2000 Onwards        Pages: 71
    Sarcopenia - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Sarcopenia - Pipeline Review, H2 2018, provides an overview of the Sarcopenia (Musculoskeletal Disorders) pipeline landscape. Sarcopenia is an aging-associated condition which is characterized by loss of muscle mass, strength and function. Symptoms include weakness, loss of endurance and poor balance. Causes of sarcopenia include......
  • Degenerative Disc Disease - Pipeline Review, H2 2018
    Published: 17-Jul-2018        Price: US 2000 Onwards        Pages: 72
    Degenerative Disc Disease - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Degenerative Disc Disease - Pipeline Review, H2 2018, provides an overview of the Degenerative Disc Disease (Musculoskeletal Disorders) pipeline landscape. Degenerative disc disease is refers to degeneration of intervertebral discs of the spinal column. The main symptom of degenerative disc disease is pain, which o......
  • Systemic Sclerosis (Scleroderma) - Pipeline Review, H2 2018
    Published: 17-Jul-2018        Price: US 2000 Onwards        Pages: 319
    Systemic Sclerosis (Scleroderma) - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Systemic Sclerosis (Scleroderma) - Pipeline Review, H2 2018, provides an overview of the Systemic Sclerosis (Scleroderma) (Immunology) pipeline landscape. Scleroderma is a group of rare diseases that involve the hardening and tightening of the skin and connective tissues. Symptoms include muscle pains, joint......
  • Myasthenia Gravis - Pipeline Review, H2 2018
    Published: 17-Jul-2018        Price: US 2000 Onwards        Pages: 133
    Myasthenia Gravis - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Myasthenia Gravis - Pipeline Review, H2 2018, provides an overview of the Myasthenia Gravis (Immunology) pipeline landscape. Myasthenia gravis is autoimmune neuromuscular disease characterized by weakness and rapid fatigue of any of the muscles under voluntary control. Symptoms include drooping of one or both eyelids, dipl......
  • Birch Pollen Allergy - Pipeline Review, H2 2018
    Published: 17-Jul-2018        Price: US 2000 Onwards        Pages: 45
    Birch Pollen Allergy - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Birch Pollen Allergy - Pipeline Review, H2 2018, provides an overview of the Birch Pollen Allergy (Immunology) pipeline landscape. Birch pollen allergy is a reaction which occurs soon after exposure to pollen of birch trees. Signs and symptoms include skin reactions, such as hives, redness or swelling, itching or tingli......
  • House Dust Mite Allergy - Pipeline Review, H2 2018
    Published: 17-Jul-2018        Price: US 2000 Onwards        Pages: 51
    House Dust Mite Allergy - Pipeline Review, H2 2018 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide House Dust Mite Allergy - Pipeline Review, H2 2018, provides an overview of the House Dust Mite Allergy (Immunology) pipeline landscape. House dust mite allergy is an allergic reaction to tiny bugs that commonly live in house dust. Symptoms include frequent sneezing, a runny, stuffy, itchy nose and irritated eyes. As......
  • Proton Therapy Market Outlook, Actual & Potential Market, Actual and Potential Candidate for Proton Therapy, List of Proton Therapy Centers, Start of Treatment, Number of Patients Treated, Specifications & Company Analysis - Global Forecast to 2024
    Published: 12-Jul-2018        Price: US 2390 Onwards        Pages: 429
    The rise in popularity of proton therapy is continuing across the globe. It is estimated that more than 165,000 patients suffering from a variety of cancers, such as prostate cancer, brain tumors, etc. have already been successfully treated using this method. In fact, the proton therapy market is on track to become a multibillion-dollar industry by 2024. The number of proton therapy centers is increasing globally. Still, industry experts believe that players will miss out on a majority of can......
  • Global and United States Infantile Spasms Therapeutics Market Research by Company, Type & Application 2013-2025
    Published: 08-Jul-2018        Price: US 2000 Onwards        Pages: 111
    Summary An infantile spasm (IS), also known as West Syndrome, is a specific type of seizure seen in an epilepsy syndrome of infancy and childhood. West Syndrome is characterized by infantile spasms, developmental regression, and a specific pattern on electroencephalography (EEG) testing called hypsarrhythmia (chaotic brain waves). The onset of infantile spasms is usually in the first year of life, typically between 4-8 months. The seizures primarily consist of a sudden bending for......
  • 2013-2023 Global Infrared light therapy device Industry Report
    Published: 02-Jul-2018        Price: US 4000 Onwards        Pages: 150
    The global market size of Infrared Light Therapy Device is $XX million in 2017 with XX CAGR from 2013 to 2017, and it is expected to reach $XX million by the end of 2023 with a CAGR of XX% from 2018 to 2023. There are 3 key segments covered in this report: geography segment, end use/application segment and competitor segment. For geography segment, regional supply, application-wise and type-wise demand, major players, price is presented from 2013 to 2023. This repor......
  • SERVICES
    Value for Money
    We believe in "optimum utilization of available budget and resources". While servicing our clients' (your) market research requirements, we keep the same approach in focus to help you get the best value for your $$s.
    Ever Growing Inventory
    Ranging from the smallest feasible / required data (datasheets, data facts, SWOT analysis, company profiles, etc) to full research reports that help you make decisions, our inventory is updated almost on a daily basis with the latest industry reports from domain experts that track more than 5000 niche sectors.
    One Stop Solution
    Need a custom research report on medical devices market? Require all available business intelligence on 3D printing industry? Exploring F&B sector of a particular country/region? RnRMarketResearch.com is your one-stop-solution to all market intelligence needs. We not only offer custom research and consulting services, we also "bundle" reports to meet your needs and help you fetch the data analysis you require for your business.
    Dedicated Client Engagement
    Not limited to only "finding" relevant reports for you, our client engagement team dedicates its efforts to understand your "business need" and accordingly maps available research data to help you move forward. Call "your" client engagement executive any time of your day and get your questions answered in order to make the correct business decision.
    Saving Time and Efforts
    Simply share your research requirement details with us and let us do all the hard work to find required intelligence for you. When you add up our "one stop solution" and "dedicated client engagement" services mentioned above, you obviously know the time and effort saving you do by working with us.
    Payment Flexibility
    Working with Fortune 500 organizations, we understand the importance of being flexible for payments. Share your payment terms with us and we will surely match up to them to ensure you get access to required business intelligence data without having to wait for the payment to be done.
    Post-Purchase Research Support
    Have questions after reading a report / datasheet bought through us? Not sure about the methodology used for data available in the research? Talk to us / Share your questions with us and if required, we will connect you with the analyst(s)/author(s) of the report(s) and ensure you get satisfactory answers for the same. Need more data / analysis / report(s) on the topic of your research/project? The RnRMarketResearch.com team is here for you 24X7 to support you with your post-purchase requirements. Subscription Offers & Packages (Get in touch with us for more details - sales@rnrmarketresearch.com / +1 888 391 5441 )
    • Ad Hoc
    • Pay - as - you - go / Bucket Subscriptions
    • Fixed Cost for #of reports
    • Customize / Personalize as per your needs