NPHP5 视网膜变性治疗的全球市场：按治疗类型、按适应症、按分销管道、按地区

全球 NPHP5 视网膜变性治疗市场规模预计将从 2024 年的 1,570 万美元增加到 2031 年的 1.313 亿美元，预测期内年复合成长率(CAGR)为 35.4%。

图 1. NPHP5 视网膜变性治疗的全球市场占有率（%），按地区划分，2024 年

NPHP5 视网膜变性，也称为高级洛肯综合症 7，是一种罕见的遗传性纤毛疾病，会导致进行性视力障碍，并可能导致完全失明。这种疾病是由 NPHP5 基因突变引起的，该基因编码肾囊肿素 5（对纤毛功能很重要的蛋白质）。此病的特征是视网膜色素变性、肾病变和其他全身性病变。目前还没有核准的NPHP5 视网膜变性治疗方法，治疗方法包括使用低视力辅助设备和支持性护理。然而，最近调查致病机制的研究正在揭示潜在的药物标靶，例如纤毛功能，并为治疗开发开闢了新的途径。几家生物製药公司已经开始临床试验，以评估基因疗法和其他新的治疗方法，以恢復这些患者的视力。 Senior Loken (SL) 是一种体染色体隐性遗传综合征，其中囊性肾病变是伴随视网膜营养不良（视网膜色素变性或莱伯氏先天性黑朦症）的肾病相关疾病的一种变体。这是一种爆发性疾病，可导致失明和肾衰竭。

市场动态：

未满足的需求市场：未满足的需求视网膜变性市场是由对这种使人衰弱的疾病的有效治疗的高度未满足的医疗需求推动的。据估计，全球整体NPHP5 视网膜退化的盛行率为 20 万分之一。对睫状途径的研究不断增加，以及视网膜基因治疗的最新突破，透过扩大治疗机会进一步推动市场成长。然而，与眼部基因转移相关的技术挑战以及开发治疗罕见疾病的药物的高成本继续限制市场收益。另一方面，生物技术公司和研究机构之间的合作，以及对罕见疾病和研发的公共和私人资金的增加，为市场进入者提供了利润丰厚的机会。

本研究的主要特点

• 本报告对全球NPHP5视网膜变性治疗药物市场进行了详细分析，并列出了以2023年为基准年的预测期（2024-2031）的市场规模和復合年增长率。
• 它还揭示了各个细分市场的潜在商机，并为该市场说明了一系列有吸引力的投资提案。
• 它还提供了有关市场驱动因素、限制因素、机会、新产品发布和核准、市场趋势、区域前景、主要企业采取的竞争策略等的重要见解。
• 该报告根据公司亮点、产品系列、主要亮点、财务表现和策略等参数，介绍了全球 NPHP5 视网膜变性治疗市场的主要参与者。这项研究的主要目标公司包括 ProQR Therapeutics、Editas Medicine、Nanscope Therapeutics、jCyte、Biogen、Novartis AG、Spark Therapeutics、MeiraGTx、NightstaRx、Beacon Therapeutics，其中包括 Applied Genetic Technologies Corporation、ViGeneron 和 RetinAI Medical。
• 该报告的见解使行销人员和公司负责人能够就未来的产品发布、类型升级、市场扩张和行销策略做出明智的决策。
• 全球 NPHP5 视网膜变性治疗市场报告迎合了该行业的各种相关人员，如投资者、供应商、产品製造商、经销商、新进业者和财务分析师。
• 相关人员可以透过用于分析全球 NPHP5 视网膜变性治疗市场的各种策略矩阵来促进他们的决策。

目录

第一章 研究目的与前提

• 研究目标
• 先决条件
• 简称

第二章 市场展望

• 报告说明
  • 市场定义和范围
• 执行摘要
• Coherent Opportunity Map（COM）

第三章市场动态、法规及趋势分析

• 市场动态
  • 加强宣传活动力度
  • 核准时间长
  • 亚太地区和拉丁美洲新兴市场
• 主要亮点
• 监管场景
• 市场趋势
• PEST分析
• 波特的分析
• 产品发表会
• 流行病学
• 合併、收购和合作

第四章全球 NPHP5 视网膜退化治疗市场 – 冠状病毒 (COVID-19) 大流行的影响

• 新型冠状病毒感染疾病（COVID-19）的流行病学
• 供需面分析
• 经济影响

第5章全球NPHP5视网膜变性治疗市场，依治疗类型，2019-2031

• 基因治疗
• 药物治疗
• 其他的

第 6 章 2019-2031 年全球 NPHP5 视网膜退化治疗市场（依适应症）

• 色素性视网膜炎
• 莱伯氏先天性黑朦症
• 尤塞氏症候群
• 其他的

第7章2019-2031年全球NPHP5视网膜变性治疗市场（依通路）

• 零售药房
• 医院药房
• 网路药房

第8章2019-2031年全球NPHP5视网膜变性治疗市场（按地区）

• 北美洲
• 美国
• 加拿大
• 欧洲
• 德国
• 英国
• 西班牙
• 法国
• 义大利
• 俄罗斯
• 其他欧洲国家
• 亚太地区
• 中国
• 印度
• 日本
• 澳洲
• 韩国
• ASEAN
• 其他亚太地区
• 拉丁美洲
• 巴西
• 阿根廷
• 墨西哥
• 其他拉丁美洲
• 中东
• 海湾合作委员会国家
• 以色列
• 其他中东地区
• 非洲
• 北非
• 中部非洲
• 南非

第9章 竞争形势

• 公司简介
  • ProQR Therapeutics
  • Editas Medicine
  • Nanoscope Therapeutics, Inc.
  • jCyte, Inc.
  • Biogen
  • Novartis AG
  • Spark Therapeutics
  • MeiraGTx
  • NightstaRx
  • Beacon Therapeutics
  • Applied Genetic Technologies Corporation
  • ViGeneron
  • RetinAI Medical

第10章

• 参考
• 调查方法
• 关于出版商

The global NPHP5 retinal degeneration treatment market size is expected to reach US$ 131.3 Mn by 2031 from US$ 15.7 Mn in 2024, exhibiting a compound annual growth rate (CAGR) of 35.4% during the forecast period.

Figure 1. Global NPHP5 Retinal Degeneration Treatment Market Share (%), By Region, 2024

NPHP5 retinal degeneration, also known as Senior-Loken syndrome 7, is a rare genetic ciliopathy that causes progressive visual impairment and can lead to complete blindness. It is caused by mutations in the NPHP5 gene, which encodes for nephrocystin-5, a protein critical for cilia function. The condition is characterized by retinitis pigmentosa, nephronophthisis, and other systemic involvement. Currently, there is no approved treatment for NPHP5 retinal degeneration, and management involves low vision aids and supportive care. However, recent research investigating the pathogenic mechanisms has uncovered potential drug targets such as cilia function, which is opening up new avenues for treatment development. Several biopharmaceutical companies have started clinical trials evaluating gene therapy and other novel therapeutic approaches to restore vision in these patients. Senior-Loken (S-L) is an autosomal recessive syndrome and a variant of the nephronophthisis-associated disorders, in which the cystic kidney disease is associated with retinal dystrophy (retinitis pigmentosa or Leber congenital amaurosis). It is a deleterious disease that culminates in blindness and renal failure.

Market Dynamics:

The NPHP5 retinal degeneration treatment market is driven by the high unmet medical need for an effective treatment for this debilitating condition. As per estimates, NPHP5 retinal degeneration has a prevalence of 1 in 200,000 live births globally. The increasing research focus on cilia pathways and recent breakthroughs in retinal gene therapy are further boosting market growth by expanding therapeutic opportunities. However, technical challenges associated with ocular gene delivery and the high costs of developing orphan disease therapies continue to restrain market revenues. On the other hand, partnerships between biotech firms and research institutions, growing public-private funding for rare disease, research and development present lucrative opportunities for market players.

Key features of the study:

• This report provides in-depth analysis of the global NPHP5 retinal degeneration treatment market, and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2024-2031), considering 2023 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
• It profiles key players in the global NPHP5 retinal degeneration treatment market based on the following parameters - company highlights, products portfolio, key highlights, financial performance, and strategies - Key companies covered as a part of this study include ProQR Therapeutics, Editas Medicine, Nanoscope Therapeutics, Inc., jCyte, Inc., Biogen, Novartis AG, Spark Therapeutics, MeiraGTx, NightstaRx, Beacon Therapeutics, Applied Genetic Technologies Corporation, ViGeneron and RetinAI Medical
• Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• The global NPHP5 retinal degeneration treatment market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global NPHP5 retinal degeneration treatment market

Detailed Segmentation:

• Global NPHP5 Retinal Degeneration Treatment Market, By Treatment Type
  • Gene Therapy
  • Cell Therapy
  • Drug Therapy
  • Others
• Global NPHP5 Retinal Degeneration Treatment Market, By Indication
  • Retinitis Pigmentosa
  • Leber Congenital Amaurosis
  • Usher Syndrome
  • Others
• Global NPHP5 Retinal Degeneration Treatment Market, By Distribution Channel
  • Retail Pharmacies
  • Hospital Pharmacies
  • Online Pharmacies
• Global NPHP5 Retinal Degeneration Treatment Market, By Region
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East
  • Africa
• Top Companies in the Global NPHP5 Retinal Degeneration Treatment Market:
  • ProQR Therapeutics
  • Editas Medicine
  • Nanoscope Therapeutics, Inc.
  • jCyte, Inc.
  • Biogen
  • Novartis AG
  • Spark Therapeutics
  • MeiraGTx
  • NightstaRx
  • Beacon Therapeutics
  • Applied Genetic Technologies Corporation
  • ViGeneron
  • RetinAI Medical

Table of Contents

1. Research Objectives and Assumptions

• Research Objectives
• Assumptions
• Abbreviations

2. Market Purview

• Report Description
  • Market Definition and Scope
• Executive Summary
  • Market Snippet, By Treatment Type
  • Market Snippet, By Indication
  • Market Snippet, By Distribution Channel
  • Market Snippet, By Region
• Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

• Market Dynamics
  • Increasing Launch of Awareness Campaigns
  • Long approval timelines
  • Emerging markets in Asia Pacific and Latin America
• Key Highlights
• Regulatory Scenario
• Market Trends
• PEST Analysis
• PORTER's Analysis
• Product Launches
• Epidemiology
• Mergers, Acquisitions, and Collaborations

4. Global NPHP5 Retinal Degeneration Treatment Market - Impact of Coronavirus (COVID-19) Pandemic

• COVID-19 Epidemiology
• Supply Side and Demand Side Analysis
• Economic Impact

5. Global NPHP5 Retinal Degeneration Treatment Market, By Treatment Type, 2019-2031, (US$ Mn)

• Introduction
  • Market Share Analysis, 2024 and 2031 (%)
  • Y-o-Y Growth Analysis, 2020 - 2031
  • Segment Trends
• Gene Therapy
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
• Drug Therapy
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
• Others
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)

6. Global NPHP5 Retinal Degeneration Treatment Market, By Indication, 2019-2031, (US$ Mn)

• Introduction
  • Market Share Analysis, 2024 and 2031 (%)
  • Y-o-Y Growth Analysis, 2020 - 2031
  • Segment Trends
• Retinitis Pigmentosa
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
• Leber Congenital Amaurosis
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
• Usher Syndrome
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
• Others
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)

7. Global NPHP5 Retinal Degeneration Treatment Market, By Distribution Channel, 2019-2031, (US$ Mn)

• Introduction
  • Market Share Analysis, 2024 and 2031 (%)
  • Y-o-Y Growth Analysis, 2020 - 2031
  • Segment Trends
• Retail Pharmacies
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
• Hospital Pharmacies
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
• Online Pharmacies
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)

8. Global NPHP5 Retinal Degeneration Treatment Market, By Region, 2019-2031, (US$ Mn)

• Introduction
  • Market Share Analysis, By Region, 2024 and 2031 (%)
  • Y-o-Y Growth Analysis, For Region, 2020 -2031
  • Country Trends
• North America
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
• U.S.
• Canada
• Europe
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
• Germany
• U.K.
• Spain
• France
• Italy
• Russia
• Rest of Europe
• Asia Pacific
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
• China
• India
• Japan
• Australia
• South Korea
• ASEAN
• Rest of Asia Pacific
• Latin America
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
• Brazil
• Argentina
• Mexico
• Rest of Latin America
• Middle East
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
• GCC Countries
• Israel
• Rest of Middle East
• Africa
  • Introduction
  • Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Indication, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  • Market Size and Forecast, and Y-o-Y Growth, By Country/Region, 2019-2031,(US$ Mn)
• North Africa
• Central Africa
• South Africa

9. Competitive Landscape

• Company Profile
  • ProQR Therapeutics
• Company Highlights
• Product Portfolio
• Key Highlights
• Financial Performance
• Strategies
  • Editas Medicine
  • Nanoscope Therapeutics, Inc.
  • jCyte, Inc.
  • Biogen
  • Novartis AG
  • Spark Therapeutics
  • MeiraGTx
  • NightstaRx
  • Beacon Therapeutics
  • Applied Genetic Technologies Corporation
  • ViGeneron
  • RetinAI Medical
• Analyst Views

10. Section

• References
• Research Methodology
• About us