DUBLIN–(BUSINESS WIRE)–The “Therapeutic Advances in Amyloidosis” report has been added to ResearchAndMarkets.com’s offering.
This research service provides an overview of therapeutic advancements enabling amyloidosis research.
Amyloidosis is a rare disease caused by the accumulation and deposition of proteins such as immunoglobulin light-chain protein in AL amyloidosis and misfolded transthyretin (TTR) protein in ATTR amyloidosis. There are a few other types of amyloidosis as well, which are either a result of a preexisting condition or are not highly prevalent.
This study discusses various new and emerging therapeutics for amyloidosis, trends, challenges, and clinical pipelines; it also makes recommendations to pharmaceutical/biotechnological and research organizations to leverage growth opportunities.
Various challenges exist in terms of finding a cure for this debilitating disease, such as multiple mutations in the TTR gene, resulting in diverse etiologies across different geographies. Research efforts have decreased disease progression significantly, and many researchers are focusing on treatment options to deplete amyloid deposition on organs that cause their dysfunction.
Developments in amyloidosis treatment have resulted in many pharmaceutical and biotechnological companies trying different drug classes targeting various stages of pathways that lead to misfolded protein, including stabilizers, inhibitors, and silencers. Recently, gene editing drugs, such as RNA interference drugs, have been receiving approval, indicating the success rate of targeted therapies and creating a manufacturer monopoly for the development of a certain class of drugs for treatment.
Therapeutic options being considered are a mix of drug combinations, drugs used in multiple myeloma, other repurposed drugs, novel drug molecules, advanced therapeutics such as gene editing, CAR T-cells, and transplants. As healthcare advances toward modular methods, several upcoming companies are being spun off of universities that have developed technology platforms to target amyloid formation and deposition.
For new targets to be explored, research efforts have to run in parallel to the growing number of treatment approvals. Several funding organizations exist in parts of the United States and the United Kingdom, and grants are being offered by leading pharmaceutical companies for this effort. Drug development is also translating into collaborations and acquisitions between companies.
One of the many challenges associated with patients is the high cost of these therapies, which necessitates financial assistance programs by manufacturers. In addition, the ongoing COVID-19 pandemic is having a major impact, and the fear of contracting infection due to hospital exposure is making people averse to hospitalization; this is a major hindrance as many drugs are delivered as infusions.
Development of new target drugs and increased understanding of the disease have to be coupled with evidence-based treatment pathways so that there is uniformity in treatment and ease in measuring outcomes to facilitate streamlined treatment over the next 5-10 years. Furthermore, a treatment that is suitable for patients at all stages of the disease should be developed.
Growth opportunities exist in terms of having clinical trials and disease registries in geographies where the disease is prevalent as this will boost the market access to certain drugs based on the manifestation that the drug can treat. Screening of members at high risk and asymptomatic transthyretin gene mutations can curb the disease at its onset.
Key Topics Covered:
1. Strategic Imperatives
- Why is it increasingly difficult to grow? The Strategic Imperative: Factors Creating Pressure on Growth
- The Strategic Imperative
- The Impact of the Top 3 Strategic Imperatives on the Development of New Therapies for Amyloidosis
- Growth Opportunities Fuel the Growth Pipeline Engine
- Research Methodology
2. Growth Opportunity Analysis
- Scope of Analysis
- Segmentation
- Growth Drivers
- Growth Restraints
3. Growth Environment Analysis
- Introduction to Amyloidosis
- Existing Therapies in Amyloidosis
- Existing Challenges in Amyloidosis
4. Emerging Therapeutic Modalities in Amyloidosis – Technology Snapshot
- Therapies in the Pipeline to Address Challenges
- Repurposed Drugs to Address Amyloidosis’ Treatment Challenges
- Drug Class Adoption for AL Amyloidosis
- Drug Class Adoption for ATTR Amyloidosis
- Snapshot of Monoclonal Antibody (mAb) Development
- Developments in mAb
- Snapshot of Developing Gene Therapies
- Snapshot of RNAi Drug Development
- Developments in RNAi and siRNA Therapy
- Developments in RNAi and ASO Therapy
- Snapshot of Small Molecule Development
- Developments in Small Molecules
- Snapshot of CAR T-Cell Therapy Development
- Developments in CAR T-Cell Therapy
- Other Drugs under Development
- New Approaches in Targeted Delivery Systems
5. Pipeline/Clinical Trial Analysis
- Drugs Approved for Amyloidosis Treatment
- Drugs under Investigation in Clinical Trials
- Drug Distribution in Clinical Trials for AL and ATTR Amyloidosis
- Top Therapeutic Modalities for AL Amyloidosis in Clinical Trials
- Top Therapeutic Modalities for ATTR Amyloidosis in Clinical Trials
- Top Therapeutic Modalities for Cardiomyopathy and Neuropathy in ATTR Amyloidosis in Clinical Trials
6. Stakeholder Ecosystem
- Strategic Collaborations Bolster Product Developments and Commercialization
- Spin-off Companies Grow from Significant Research Developments
- Manufacturers Enter into Contracts with Various Stakeholders to Ease Market Access
- Consortia and Public/Private Partnerships to Expand Awareness and Impart Knowledge of Rare Diseases
7. Funding Landscape
- Grants and Research Funding Recipients and Organizations
- Research Grants by Universities and Biopharma/Pharma Companies for Amyloidosis
- Financing from Pharmaceutical/Biopharmaceutical Companies
- Patient Assistance Programs
- Takeaways from Developments and Trends in the Stakeholder Environment
- Takeaways from the Funding Landscape
8. Growth Opportunity Universe
- Growth Opportunity 1: Creating More Strategic Contracts with Stakeholders to Improve Market Access
- Growth Opportunity 2: Acceleration of Advanced Therapeutics Research
9. Next Steps
For more information about this report visit https://www.researchandmarkets.com/r/ev3ksk
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