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Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2020

  • ID: 5215145
  • Drug Pipelines
  • December 2020
  • Region: Global
  • 60 pages
  • DelveInsight
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FEATURED COMPANIES

  • Arrowhead Pharmaceuticals
  • Catabasis Pharmaceuticals
  • iMetabolic Biopharma
  • Ionis Pharmaceuticals
  • Novartis
  • Precision Biosciences
  • MORE
This “Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2020,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Familial Lipoprotein Lipase Deficiency pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered
  • Global coverage
Familial Lipoprotein Lipase Deficiency Understanding

Familial Lipoprotein Lipase Deficiency: Overview

Familial Lipoprotein Lipase Deficiency (LPLD) is a rare inherited condition, in which the normal breakdown of fats in the body is affected. The condition is inherited in an autosomal recessive pattern. This deficiency is usually caused by a defect in the LPL gene, which encodes for an enzyme called lipoprotein lipase. Lipoprotein lipase (LPL) is a key enzyme that ensures triglycerides are unloaded from chylomicrons. Absence of this enzyme leads to increased blood triglyceride and chylomicron levels. Persons with LPLD cannot break down fat from digested food and therefore fat particles called chylomicrons build up in the blood.

Symptoms

The symptoms of Familial Lipoprotein Lipase Deficiency include:
  • Abdominal pain (may appear as colic in infancy) and swelling
  • Pain in the muscles and bones (musculoskeletal pain)
  • Nausea and Vomiting
  • Recurrent acute pancreatitis
  • Reduced or absent lipoprotein lipase
  • Skin xanthomas
  • Increased cholesterol and/or triglyceride level
  • Whitish-yellow nodules
  • White fatty retinal deposits
Diagnosis

A diagnosis of Familial Lipoprotein Lipase Deficiency may be done based upon identification of characteristic symptoms, a detailed patient history, and a thorough clinical evaluation. Blood tests can reveal reduced activity of the lipoprotein lipase enzyme in the plasma. Molecular genetic testing can confirm the diagnosis of Familial Lipoprotein Lipase Deficiency.

Treatment

Treatment for Familial Lipoprotein Lipase Deficiency aims to control the symptoms and blood triglyceride levels with a very low-fat diet. Drugs that lower lipid levels in the body are not effective in reducing fat levels. Alcohol and drugs that increase triglyceride levels should be avoided. Gene Therapy is approved for the treatment, specifically for patients who have not responded to other treatments

Familial Lipoprotein Lipase Deficiency Emerging Drugs Chapters

This segment of the Familial Lipoprotein Lipase Deficiency report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Familial Lipoprotein Lipase Deficiency Emerging Drugs

ARO-APOC3: Arrowhead Pharmaceuticals

ARO-APOC3 is a subcutaneously administered RNAi therapeutic that targets apolipoprotein C-III (apoC-III), and reduces VLDL synthesis and assembly, enhances the breakdown of triglyceride rich lipoproteins, and improve clearance of VLDL and chylomicron remnants.

Vupanorsen: Ionis Pharmaceuticals

Vupanorsen is an investigational antisense therapy to reduce the production of angiopoietin-like 3 (ANGPTL3) protein, a key regulator of triglyceride and cholesterol metabolism, in the liver. The therapy was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform.

Familial Lipoprotein Lipase Deficiency: Therapeutic Assessment

This segment of the report provides insights about the different Familial Lipoprotein Lipase Deficiency drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Familial Lipoprotein Lipase Deficiency

There are approx. 8+ key companies which are developing the therapies for Familial Lipoprotein Lipase Deficiency. The companies which have their Familial Lipoprotein Lipase Deficiency drug candidates in the most advanced stage, i.e. phase III include, Ionis Pharmaceuticals.

Phases

This report covers around 8+ products under different phases of clinical development like
  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration
Familial Lipoprotein Lipase Deficiency pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
  • Oral
  • Parenteral
  • intravitreal
  • Subretinal
  • Topical.
  • Molecule Type
Products have been categorized under various Molecule types such as
  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Familial Lipoprotein Lipase Deficiency: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Familial Lipoprotein Lipase Deficiency therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Familial Lipoprotein Lipase Deficiency drugs.

Report Highlights
  • The companies and academics are working to assess challenges and seek opportunities that could influence Familial Lipoprotein Lipase Deficiency R&D. The therapies under development are focused on novel approaches to treat/improve Familial Lipoprotein Lipase Deficiency.
  • In August, 2020 Ionis Pharmaceuticals and its affiliate Akcea Therapeutics announced that they have entered into a definitive agreement under which Ionis will acquire all of the outstanding shares of Akcea common stock. In November 2019, Akcea and Ionis announced the closing of a worldwide exclusive licensing agreement with Pfizer Inc. for vupanorsen development. Pfizer is responsible for all development and regulatory activities and costs beyond those associated with the Phase 2 study.
Familial Lipoprotein Lipase Deficiency Report Insights
  • Familial Lipoprotein Lipase Deficiency Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs
Familial Lipoprotein Lipase Deficiency Report Assessment
  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs
Key Questions Answered

Current Treatment Scenario and Emerging Therapies:
  • How many companies are developing Familial Lipoprotein Lipase Deficiency drugs?
  • How many Familial Lipoprotein Lipase Deficiency drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Familial Lipoprotein Lipase Deficiency?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Familial Lipoprotein Lipase Deficiency therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Familial Lipoprotein Lipase Deficiency and their status?
  • What are the key designations that have been granted to the emerging drugs?
Key Players
  • Arrowhead Pharmaceuticals
  • Novartis
  • Ionis Pharmaceuticals
  • Catabasis Pharmaceuticals
  • iMetabolic Biopharma
  • Precision Biosciences
Key Products
  • ARO-APOC3
  • Pradigastat
  • Vupanorsen
  • CAT-2003
  • iMBP-001
  • ApoC3 gene editing therapeutic
  • IONIS APOCIII LRx

This report will be delivered within 1-3 business days
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Arrowhead Pharmaceuticals
  • Catabasis Pharmaceuticals
  • iMetabolic Biopharma
  • Ionis Pharmaceuticals
  • Novartis
  • Precision Biosciences
  • MORE
Introduction

Executive Summary

Familial Lipoprotein Lipase Deficiency: Overview
  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management
Pipeline Therapeutics
  • Comparative Analysis
Therapeutic Assessment
  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type
Familial Lipoprotein Lipase Deficiency- Analytical Perspective

In-depth Commercial Assessment
  • Familial Lipoprotein Lipase Deficiency companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Familial Lipoprotein Lipase Deficiency Collaboration Deals
  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
  • Comparative Analysis
IONIS APOCIII LRx: Ionis Pharmaceuticals
  • Product Description
  • Research and Development
  • Product Development Activities
Mid Stage Products (Phase II)
  • Comparative Analysis
Vupanorsen: Ionis Pharmaceuticals
  • Product Description
  • Research and Development
  • Product Development Activities
Early Stage Products (Phase I/II)
  • Comparative Analysis
ARO-APOC3: Arrowhead Pharmaceuticals
  • Product Description
  • Research and Development
  • Product Development Activities
Inactive Products
  • Comparative Analysis
Familial Lipoprotein Lipase Deficiency Key Companies

Familial Lipoprotein Lipase Deficiency Key Products

Familial Lipoprotein Lipase Deficiency- Unmet Needs

Familial Lipoprotein Lipase Deficiency- Market Drivers and Barriers

Familial Lipoprotein Lipase Deficiency- Future Perspectives and Conclusion

Familial Lipoprotein Lipase Deficiency Analyst Views

Familial Lipoprotein Lipase Deficiency Key Companies

Appendix

List of Tables
Table 1 Total Products for Familial Lipoprotein Lipase Deficiency
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

List of Figures
Figure 1 Total Products for Familial Lipoprotein Lipase Deficiency
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Note: Product cover images may vary from those shown
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  • Arrowhead Pharmaceuticals
  • Novartis
  • Ionis Pharmaceuticals
  • Catabasis Pharmaceuticals
  • iMetabolic Biopharma
  • Precision Biosciences
Note: Product cover images may vary from those shown
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