淀粉样变性的治疗进展

本报告提供淀粉样变性研究分析，深入了解新疗法、趋势、挑战、临床管道以及增长机会分析。

内容

战略要务

• 为什么成长越来越难？
• 战略要务 8 (TM)
• 三大战略要务对开发淀粉样变性新疗法的影响
• 增长机会推动增长管道引擎 (TM)
• 调查方法

增长机会分析

• 分析范围
• 细分
• 增长驱动力
• 抑制增长的因素

成长环境分析

• 淀粉样变性简介
• 现有的淀粉样变性治疗方法
• 淀粉样变性的现有挑战

淀粉样变性的新疗法 - 技术概览

• 应对挑战的管道疗法
• 重新利用药物来应对淀粉样变性治疗的挑战
• 采用 AL 淀粉样变性药物类别
• 采用 ATTR 淀粉样变性药物类别
• 单克隆抗体 (mAb) 开发概览
• 单克隆抗体的发展
• 基因治疗发展概况
• RNAi 药物开发概览
• 开发 RNAi/siRNA 疗法
• 开发 RNAi 和 ASO 疗法
• 小分子开发快照
• 小分子的开发
• CAR-T 细胞疗法开发概览
• CAR-T 细胞疗法的发展
• 正在开发的其□□他药物
• 定向投放系统的新方法

管道/临床试验分析

• 获批用于治疗淀粉样变性的药物
• 正在临床试验中研究的药物
• AL/ATTR 淀粉样变性临床试验药物分布
• 临床试验中 AL 淀粉样变性的主要治疗方法
• 临床试验中 ATTR 淀粉样变性的主要治疗方式
• 临床试验中 ATTR 淀粉样变性中心肌病和神经病的初步治疗

利益相关者生态系统

• 战略联盟加强产品开发和商业化
• 衍生公司从重要的研发中成长
• 製造商与各利益相关方签订合同以促进市场进入
• 联盟和公私合作伙伴关係提高了对罕见病的认识并传授知识

资金情况

• 资助和研究基金的获得者和组织
• 大学和生物製药/製药公司对淀粉样变性的研究资助
• 来自製药/生物製药公司的贷款
• 患者支持计划
• 我们可以从利益相关者环境的发展和趋势中学到什么
• 我们可以从资金状况中学到什么

增长机会

• 增长机会 1：与利益相关方签署更多战略协议以改善市场准入
• 增长机会 2：加速先进疗法的研究
• 增长机会 3：不断发现新的治疗方式

下一步

Research Efforts and Technology Developments through Partnerships and Collaborations will Push New Therapeutic Modalities into Treatment Regimens.

Amyloidosis is a rare disease caused by the accumulation and the 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. 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.

This Frost & Sullivan research service provides an overview of therapeutic advancements enabling amyloidosis research.

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 of 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.

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.

Table of Contents

Strategic Imperatives

• Why is it increasingly difficult to grow?The Strategic Imperative 8™: Factors Creating Pressure on Growth
• The Strategic Imperative 8™
• 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

Growth Opportunity Analysis

• Scope of Analysis
• Segmentation
• Growth Drivers
• Growth Restraints

Growth Environment Analysis

• Introduction to Amyloidosis
• Existing Therapies in Amyloidosis
• Existing Challenges in Amyloidosis

Emerging Therapeutic Modalities in Amyloidosis-Technology Snapshot

• Therapies in the Pipeline to Address Challenges
• Therapies in the Pipeline to Address Challenges (continued)
• 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

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

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

Funding Landscape

• Grants and Research Funding Recipients and Organizations
• Grants and Research Funding Recipients and Organizations (continued)
• Research Grants by Universities and Biopharma/Pharma Companies for Amyloidosis
• Research Grants by Universities and Biopharma/Pharma Companies for Amyloidosis (continued)
• Financing from Pharmaceutical/Biopharmaceutical Companies
• Patient Assistance Programs
• Takeaways from Developments and Trends in the Stakeholder Environment
• Takeaways from the Funding Landscape

Growth Opportunity Universe

• Growth Opportunity 1: Creating More Strategic Contracts with Stakeholders to Improve Market Access
• Growth Opportunity 1: Creating More Strategic Contracts with Stakeholders to Improve Market Access (continued)
• Growth Opportunity 2: Acceleration of Advanced Therapeutics Research
• Growth Opportunity 2: Acceleration of Advanced Therapeutics Research (continued)
• Growth Opportunity 3: Continued Discovery of New Modalities for Treatment
• Growth Opportunity 3: Continued Discovery of New Modalities for Treatment (continued)

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