Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) – Drugs In Development, 2024

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Familial hypercholesterolemia (FH) is a genetic disorder that affects the way the body processes cholesterol. People with FH have very high levels of low-density lipoprotein (LDL) cholesterol, also known as “bad” cholesterol, in their blood. This can lead to early and severe cardiovascular diseases, such as heart attacks and strokes. FH is caused by a mutation in one or more genes that are involved in removing LDL cholesterol from the blood. The most common gene affected is LDLR, which encodes the LDL receptor protein. Other genes that can cause FH are APOB, PCSK9, and LDLRAP1. FH is inherited in an autosomal dominant pattern, which means that having one copy of the mutated gene is enough to cause the condition. People who inherit two copies of the mutated gene have a more severe form of FH. FH affects about 1 in 250 people worldwide, but the prevalence may vary depending on the population and the genetic background. FH can be diagnosed by measuring the blood cholesterol levels, examining the physical signs of cholesterol deposits (such as xanthomas and arcus corneae), and performing genetic testing to confirm the mutation. FH can be treated with medications that lower cholesterol levels, such as statins, bile acid sequestrants, ezetimibe, and PCSK9 inhibitors. Some people with FH may also need other treatments, such as LDL apheresis or liver transplantation, to reduce their risk of complications. FH also requires lifestyle modifications, such as eating a healthy diet, exercising regularly, avoiding smoking, and managing other risk factors for cardiovascular diseases.

The Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) drugs in development market research report provide comprehensive information on the therapeutics under development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia), 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 and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) and features dormant and discontinued products.

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Quick View – Familial Hypercholesterolemia (Type II Hyperlipoproteinemia)
Key Targets
· Proprotein Convertase Subtilisin/Kexin Type 9; Angiopoietin Related Protein 3; Cholesteryl Ester Transfer Protein; Low Density Lipoprotein Receptor; NPC1 Like Intracellular Cholesterol Transporter 1; Microsomal Triglyceride Transfer Protein Large Subunit; Liver X Receptor; Probable G Protein Coupled Receptor 146; Oxidized Low Density LipoproteiN; and ATP Citrate Synthase
Key Mechanisms of Action
· Proprotein Convertase Subtilisin/Kexin Type 9 Inhibitor; Angiopoietin Related Protein 3 Inhibitor; Cholesteryl Ester Transfer Protein Inhibitor; NPC1 Like Intracellular Cholesterol Transporter 1 Inhibitor; Oxidized Low Density LipoproteiN Inhibitor; Probable G Protein Coupled Receptor 146 Inhibitor; Angiopoietin Related Protein 4 Inhibitor; 3 Hydroxy 3 Methylglutaryl Coenzyme A Reductase Inhibitor; ATP Citrate Synthase Inhibitor; and Microsomal Triglyceride Transfer Protein Large Subunit Inhibitor
Key Routes of Administration
· Subcutaneous; Oral; Intravenous; Intraperitoneal; and Parenteral
Key Molecule Types
· Gene Therapy; Monoclonal Antibody; Small Molecule; Antisense RNAi Oligonucleotide; Fusion Protein; Peptide; Antisense Oligonucleotide; Biologic; and Synthetic Peptide
Major Companies
· Verve Therapeutics Inc; Jiangsu Hengrui Medicine Co Ltd; NewAmsterdam Pharma Company NV; Arrowhead Pharmaceuticals Inc; CRISPR Therapeutics AG; Daewoong Pharmaceutical Co Ltd; Esperion Therapeutics Inc; Epicrispr Biotechnologies Inc; EmendoBio Inc; and EDIRNA Inc

Scope

  • Therapeutics in Development: Covering 45 molecules, with 41 developed by companies and the rest by universities/institutes.
  • Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.
  • Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.
  • Development History: Access the complete research and development history.
  • Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.

Reasons to Buy

  • Holistic insights: Understand the broad spectrum of Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
  • Pipeline Exploration: Explore the detailed Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.
  • Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Familial Hypercholesterolemia (Type II Hyperlipoproteinemia) treatments for adaptable decision-making.
  • Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.

Key Players

Akeso Inc
Amgen Inc
Arrowhead Pharmaceuticals Inc
AstraZeneca Plc
Beijing Mabworks Biotech Co Ltd
Chiesi Farmaceutici SpA
Daewoong Pharmaceutical Co Ltd
EDIRNA Inc
EmendoBio Inc
Epicrispr Biotechnologies Inc
Esperion Therapeutics Inc
Gruthan Bioscience LLC
Hanmi Science Co Ltd
iMetabolic Biopharma Corp
Innovent Biologics Inc
Jiangsu Hengrui Medicine Co Ltd
LIB Therapeutics LLC
Lipigon Pharmaceuticals AB
LipimetiX Development Inc
Merck & Co Inc
NeuroBo Pharmaceuticals Inc
NewAmsterdam Pharma Company NV
Novartis AG
Novo Nordisk AS
OrsoBio Inc
PRD Therapeutics Inc
Precision Biosciences Inc
Repair Biotechnologies Inc
Saliogen Therapeutics Inc
San Raffaele Telethon Institute for Gene Therapy
Sanofi
Shanghai Junshi Biosciences Co Ltd
Suzhou Ribo Life Sciences Co Ltd
Tangdu Hospital
Ultragenyx Pharmaceutical Inc
University Hospitals Leuven
Verve Therapeutics Inc
Vesicle Therapeutics Inc
Visirna Therapeutics HK Ltd
Xi'an Jiaotong University
Yaotang (Shanghai) Biotechnology Co Ltd

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Overview

Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Drugs Under Development by Companies

Drugs under Development by Universities/Institutes

Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Companies Involved in Therapeutic Development

Drug Profiles

Dormant Projects

Discontinued Drugs

Product Development Milestones

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Drugs under Development by Companies, 2024

Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Stage and Target, 2024

Number of Drugs by Stage and Mechanism of Action, 2024

Number of Drugs by Stage and Route of Administration, 2024

Number of Drugs by Stage and Molecule Type, 2024

Dormant Projects, 2024

Discontinued Drugs, 2024

Figures

List of Figures

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Top 10 Targets, 2024

Number of Drugs by Stage and Top 10 Targets, 2024

Number of Drugs by Top 10 Mechanism of Actions, 2024

Number of Drugs by Stage and Top 10 Mechanism of Actions, 2024

Number of Drugs by Top 10 Routes of Administration, 2024

Number of Drugs by Stage and Top 10 Routes of Administration, 2024

Number of Drugs by Top 10 Molecule Types, 2024

Number of Drugs by Stage and Top 10 Molecule Types, 2024

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