Sickle Cell Disease - Pipeline Review, H2 2017

  • ID: 4419035
  • Report
  • 190 pages
  • Global Markets Direct
1 of 5

FEATURED COMPANIES

  • Acceleron Pharma Inc
  • Bioverativ Inc
  • CRISPR Therapeutics
  • Gamida Cell Ltd
  • MaxCyte Inc
  • Novartis AG
  • MORE
Sickle Cell Disease - Pipeline Review, H2 2017

Summary

The latest Pharmaceutical and Healthcare disease pipeline guide Sickle Cell Disease - Pipeline Review, H2 2017, provides an overview of the Sickle Cell Disease (Hematological Disorders) pipeline landscape.

Sickle cell anemia is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Signs and symptoms include anemia, delayed growth, vision problems, pain and frequent infections. Treatment includes antibiotics, pain relievers, blood transfusion and stem cell transplant.

Report Highlights

The latest Pharmaceutical and Healthcare latest pipeline guide Sickle Cell Disease - Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Sickle Cell Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 5, 4, 11, 25, 4 and 1 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 5 and 2 molecules, respectively.

Sickle Cell Disease (Hematological 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 our 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 Sickle Cell Disease (Hematological Disorders).
  • The pipeline guide reviews pipeline therapeutics for Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological 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 Sickle Cell Disease (Hematological 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.
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Note: Product cover images may vary from those shown
2 of 5

FEATURED COMPANIES

  • Acceleron Pharma Inc
  • Bioverativ Inc
  • CRISPR Therapeutics
  • Gamida Cell Ltd
  • MaxCyte Inc
  • Novartis AG
  • MORE
Introduction

Sickle Cell Disease - Overview

Sickle Cell Disease - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Sickle Cell Disease - Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Sickle Cell Disease - Companies Involved in Therapeutics Development

Acceleron Pharma Inc

Addmedica SAS

Angiocrine Bioscience Inc

Annexin Pharmaceuticals AB

ArQule Inc

Bio Products Laboratory Ltd

Bioverativ Inc

bluebird bio Inc

Bristol-Myers Squibb Co

Calimmune Inc

CRISPR Therapeutics

CSL Ltd

Daiichi Sankyo Co Ltd

Editas Medicine Inc

Errant Gene Therapeutics LLC

Gamida Cell Ltd

Genethon SA

Gilead Sciences Inc

Global Blood Therapeutics Inc

Incyte Corp

Ironwood Pharmaceuticals Inc

La Jolla Pharmaceutical Company

MaxCyte Inc

Merck & Co Inc

Morphogenesis Inc

NKT Therapeutics Inc

Novartis AG

Orphagen Pharmaceuticals Inc

Prolong Pharmaceuticals LLC

Protagonist Therapeutics Inc

ReveraGen BioPharma Inc

Sancilio & Company Inc

Sangamo Therapeutics Inc

Sickle Cell Disease - Drug Profiles

(decitabine + tetrahydrouridine) - Drug Profile

AB-110 - Drug Profile

ACY-957 - Drug Profile

ambrisentan - Drug Profile

AP-01 - Drug Profile

apixaban - Drug Profile

ARQ-092 - Drug Profile

BB-305 - Drug Profile

benserazide - Drug Profile

BIVV-003 - Drug Profile

CAL-H - Drug Profile

CordIn - Drug Profile

crizanlizumab - Drug Profile

CTX-001 - Drug Profile

didox - Drug Profile

Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile

DRX-194 - Drug Profile

GBT-440 - Drug Profile

Gene Therapy for Sickle Cell Anemia - Drug Profile

Gene Therapy for Sickle Cell Disease - Drug Profile

Gene Therapy for Sickle Cell Disease - Drug Profile

Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile

Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile

Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia - Drug Profile

haptoglobin (human) - Drug Profile

haptoglobin + hemopexin - Drug Profile

HBI-002 - Drug Profile

HBI-137 - Drug Profile

hydroxyurea - Drug Profile

hydroxyurea - Drug Profile

IMR-687 - Drug Profile

INCB-59872 - Drug Profile

IW-1701 - Drug Profile

LJPC-401 - Drug Profile

luspatercept - Drug Profile

M-012 - Drug Profile

NiCord - Drug Profile

NKTT-120 - Drug Profile

panobinostat - Drug Profile

pentosan polysulfate sodium - Drug Profile

Polysaccharide to Inhibit Coagulation Factor X for Deep Vein Thrombosis and Sickle Cell Disease - Drug Profile

prasugrel hydrochloride - Drug Profile

Protein for Sickle Cell Disease - Drug Profile

PTG-300 - Drug Profile

RCY-1497 - Drug Profile

RN-1 - Drug Profile

RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile

Sanguinate - Drug Profile

SC-411 - Drug Profile

SCD-101 - Drug Profile

SCPF - Drug Profile

Small Molecules for Sickle Cell Disease - Drug Profile

Small Molecules for Sickle Cell Disease - Drug Profile

Small Molecules to Inhibit HDAC for Oncology and Sickle Cell Disease - Drug Profile

Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile

sotatercept - Drug Profile

Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile

Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile

Trichosic - Drug Profile

vamorolone - Drug Profile

Sickle Cell Disease - Dormant Projects

Sickle Cell Disease - Product Development Milestones

Featured News & Press Releases

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

Number of Products under Development for Sickle Cell Disease, H2

Number of Products under Development by Companies, H2

Number of Products under Development by Universities/Institutes, H2

Products under Development by Companies, H2

Products under Development by Universities/Institutes, H2

Number of Products by Stage and Target, H2

Number of Products by Stage and Mechanism of Action, H2

Number of Products by Stage and Route of Administration, H2

Number of Products by Stage and Molecule Type, H2

Sickle Cell Disease - Pipeline by Acceleron Pharma Inc, H2

Sickle Cell Disease - Pipeline by Addmedica SAS, H2

Sickle Cell Disease - Pipeline by Angiocrine Bioscience Inc, H2

Sickle Cell Disease - Pipeline by Annexin Pharmaceuticals AB, H2

Sickle Cell Disease - Pipeline by ArQule Inc, H2

Sickle Cell Disease - Pipeline by Bio Products Laboratory Ltd, H2

Sickle Cell Disease - Pipeline by Bioverativ Inc, H2

Sickle Cell Disease - Pipeline by bluebird bio Inc, H2

Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Co, H2

Sickle Cell Disease - Pipeline by Calimmune Inc, H2

Sickle Cell Disease - Pipeline by CRISPR Therapeutics, H2

Sickle Cell Disease - Pipeline by CSL Ltd, H2

Sickle Cell Disease - Pipeline by Daiichi Sankyo Co Ltd, H2

Sickle Cell Disease - Pipeline by Editas Medicine Inc, H2

Sickle Cell Disease - Pipeline by Errant Gene Therapeutics LLC, H2

Sickle Cell Disease - Pipeline by Gamida Cell Ltd, H2

Sickle Cell Disease - Pipeline by Genethon SA, H2

Sickle Cell Disease - Pipeline by Gilead Sciences Inc, H2

Sickle Cell Disease - Pipeline by Global Blood Therapeutics Inc, H2

Sickle Cell Disease - Pipeline by Incyte Corp, H2

Sickle Cell Disease - Pipeline by Ironwood Pharmaceuticals Inc, H2

Sickle Cell Disease - Pipeline by La Jolla Pharmaceutical Company, H2

Sickle Cell Disease - Pipeline by MaxCyte Inc, H2

Sickle Cell Disease - Pipeline by Merck & Co Inc, H2

Sickle Cell Disease - Pipeline by Morphogenesis Inc, H2

Sickle Cell Disease - Pipeline by NKT Therapeutics Inc, H2

Sickle Cell Disease - Pipeline by Novartis AG, H2

Sickle Cell Disease - Pipeline by Orphagen Pharmaceuticals Inc, H2

Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals LLC, H2

Sickle Cell Disease - Pipeline by Protagonist Therapeutics Inc, H2

Sickle Cell Disease - Pipeline by ReveraGen BioPharma Inc, H2

Sickle Cell Disease - Pipeline by Sancilio & Company Inc, H2

Sickle Cell Disease - Pipeline by Sangamo Therapeutics Inc, H2

Sickle Cell Disease - Dormant Projects, H2

List of Figures

Number of Products under Development for Sickle Cell Disease, H2

Number of Products under Development by Companies, H2

Number of Products under Development by Universities/Institutes, H2

Number of Products by Top 10 Targets, H2

Number of Products by Stage and Top 10 Targets, H2

Number of Products by Top 10 Mechanism of Actions, H2

Number of Products by Stage and Top 10 Mechanism of Actions, H2

Number of Products by Routes of Administration, H2

Number of Products by Stage and Routes of Administration, H2

Number of Products by Top 10 Molecule Types, H2

Number of Products by Stage and Top 10 Molecule Types, H
Note: Product cover images may vary from those shown
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  • Acceleron Pharma Inc
  • Addmedica SAS
  • Angiocrine Bioscience Inc
  • Annexin Pharmaceuticals AB
  • ArQule Inc
  • Bio Products Laboratory Ltd
  • Bioverativ Inc
  • bluebird bio Inc
  • Bristol-Myers Squibb Co
  • Calimmune Inc
  • CRISPR Therapeutics
  • CSL Ltd
  • Daiichi Sankyo Co Ltd
  • Editas Medicine Inc
  • Errant Gene Therapeutics LLC
  • Gamida Cell Ltd
  • Genethon SA
  • Gilead Sciences Inc
  • Global Blood Therapeutics Inc
  • Incyte Corp
  • Ironwood Pharmaceuticals Inc
  • La Jolla Pharmaceutical Company
  • MaxCyte Inc
  • Merck & Co Inc
  • Morphogenesis Inc
  • NKT Therapeutics Inc
  • Novartis AG
  • Orphagen Pharmaceuticals Inc
  • Prolong Pharmaceuticals LLC
  • Protagonist Therapeutics Inc
  • ReveraGen BioPharma Inc
  • Sancilio & Company Inc
  • Sangamo Therapeutics Inc
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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