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全球 NPHP5 视网膜变性治疗市场（按治疗类型、适应症、分销管道和地区划分）

Global NPHP5 Retinal Degeneration Treatment Market, By Treatment Type, By Indication, By Distribution Channel, By Geography

出版日期: 2025年03月04日 | 出版商:

Coherent Market Insights | 英文 170 Pages | 商品交期: 2-3个工作天内

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简介目录

2025 年全球 NPHP5 视网膜变性治疗市场规模估计为 1,580 万美元，预计到 2032 年将达到 1.333 亿美元，2025 年至 2032 年的复合年增长率为 35.6%。

报告范围		报告详细信息
基准年	2024	2025年的市场规模	1580万美元
效能数据	从2020年到2024年	预测期	2025年至2032年
预测期：2025-2032年复合年增长率：	35.60%	2032年价值预测	1.333亿美元

数字。 2025年NPHP5视网膜变性治疗全球市场占有率（%）

NPHP5 视网膜变性，也称为 Senior-Loken 症候群 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。该疾病是由 NPHP5 基因突变引起的，该基因编码肾囊素-5，这是对纤毛功能很重要的蛋白质。此疾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，尚无针对 NPHP5 视网膜退化的核准治疗方法，治疗包括低视力辅助和支持性护理。然而，最近对发病机制的研究揭示了潜在的药物标靶，例如纤毛功能，这为治疗开闢了新的途径。几家生物製药公司已经启动临床试验，评估基因疗法和其他新型治疗方法，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，是肾病变相关疾病的变体，其中囊性肾病变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）。这是一种毁灭性的疾病，可能导致失明和肾衰竭。

市场动态：

NPHP5 视网膜变性治疗市场受到对这种衰弱性疾病的有效治疗的未满足医疗需求的推动。据估计，全球整体每 20 万名新生儿中就有 1 名患有 NPHP5 视网膜退化。纤毛通路研究的不断增加以及视网膜基因治疗的最新突破正在扩大治疗机会，进一步推动市场成长。然而，将基因输送到眼睛的技术挑战和罕见疾病药物的高开发成本继续限制市场收益。同时，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，为市场进入者创造了有利可图的机会。

本研究的主要特点

本报告对全球 NPHP5 视网膜变性治疗市场进行了详细分析，并以 2024 年为基准年，给出了预测期（2025-2032 年）的市场规模和年复合成长率(CAGR)。
它还强调了各个领域的潜在商机，并说明了该市场的有吸引力的投资提案矩阵。
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、区域前景和主要企业采用的竞争策略的重要见解。
该报告根据公司亮点、产品系列、关键亮点、财务表现和策略等参数，概述了全球 NPHP5 视网膜变性治疗市场的领导者。研究涉及的主要公司包括 ProQR Therapeutics、Editas Medicine、Nanoscope Therapeutics、jCyte、Biogen、Novartis AG、Spark Therapeutics、MeiraGTx、NightstaRx、Beacon Therapeutics、Applied Genetic Technologies Corporation、ViGeneron 和 RetinAI Medical。
本报告的见解将使负责人和公司经营团队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涉及该行业的各个相关人员，例如投资者、供应商、产品製造商、经销商、新进业者和金融分析师。
相关人员将透过用于分析全球 NPHP5 视网膜变性治疗市场的各种策略矩阵来做出决策。

Report Coverage	Report Details
Base Year:	2024	Market Size in 2025:	USD 15.8 Mn
Historical Data for:	2020 To 2024	Forecast Period:	2025 To 2032
Forecast Period 2025 to 2032 CAGR:	35.60%	2032 Value Projection:	USD 133.3 Mn

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 (2025-2032), considering 2024 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
Company Profiles
- 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

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, 2020-2032, (US$ Mn)

Introduction
- Market Share Analysis, 2025 and 2032 (%)
- Y-o-Y Growth Analysis, 2021 - 2032
- Segment Trends
Gene Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
Drug Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
Others
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)

6. Global NPHP5 Retinal Degeneration Treatment Market, By Indication, 2020-2032, (US$ Mn)

Introduction
- Market Share Analysis, 2025 and 2032 (%)
- Y-o-Y Growth Analysis, 2021 - 2032
- Segment Trends
Retinitis Pigmentosa
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
Leber Congenital Amaurosis
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
Usher Syndrome
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
Others
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)

7. Global NPHP5 Retinal Degeneration Treatment Market, By Distribution Channel, 2020-2032, (US$ Mn)

Introduction
- Market Share Analysis, 2025 and 2032 (%)
- Y-o-Y Growth Analysis, 2021 - 2032
- Segment Trends
Retail Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
Hospital Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)
Online Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2020-2032,(US$ Mn)

8. Global NPHP5 Retinal Degeneration Treatment Market, By Region, 2020-2032, (US$ Mn)

Introduction
- Market Share Analysis, By Region, 2025 and 2032 (%)
- Y-o-Y Growth Analysis, For Region, 2021 -2032
- Country Trends
North America
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
  - U.S.
  - Canada
Europe
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(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, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(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, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
  - Brazil
  - Argentina
  - Mexico
  - Rest of Latin America
Middle East
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032,(US$ Mn)
  - GCC Countries
  - Israel
  - Rest of Middle East
Africa
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment Type, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032,(US$ Mn)
- Market Size and Forecast, and Y-o-Y Growth, By Country/Region, 2020-2032,(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
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- Nanoscope Therapeutics, Inc.
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- jCyte, Inc.
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- Biogen
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- Novartis AG
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- Spark Therapeutics
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- MeiraGTx
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- NightstaRx
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- Beacon Therapeutics
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- Applied Genetic Technologies Corporation
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- ViGeneron
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
- RetinAI Medical
Company Highlights
Product Portfolio
Key Highlights
Financial Performance
Strategies
Analyst Views

10. Section

References
Research Methodology
About us

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FAQ

全球 NPHP5 视网膜变性治疗市场（按治疗类型、适应症、分销管道和地区划分）

Global NPHP5 Retinal Degeneration Treatment Market, By Treatment Type, By Indication, By Distribution Channel, By Geography

市场动态：

本研究的主要特点

目录

第一章调查目的与前提条件

第二章市场展望

第三章市场动态、法规与趋势分析

4. 全球 NPHP5 视网膜退化治疗市场-冠状病毒 (COVID-19) 大流行的影响

5. 2020 年至 2032 年全球 NPHP5 视网膜退化治疗市场（依治疗类型）

6. 全球 NPHP5 视网膜退化治疗市场（依适应症划分），2020 年至 2032 年

7. 2020 年至 2032 年全球 NPHP5 视网膜退化治疗市场（依分销管道）

8. 全球 NPHP5 视网膜变性治疗市场（按地区），2020 年至 2032 年

第九章竞争态势

第 10 章章节

Market Dynamics:

Key features of the study:

Detailed Segmentation:

Table of Contents

1. Research Objectives and Assumptions

2. Market Purview

3. Market Dynamics, Regulations, and Trends Analysis

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

5. Global NPHP5 Retinal Degeneration Treatment Market, By Treatment Type, 2020-2032, (US$ Mn)

6. Global NPHP5 Retinal Degeneration Treatment Market, By Indication, 2020-2032, (US$ Mn)

7. Global NPHP5 Retinal Degeneration Treatment Market, By Distribution Channel, 2020-2032, (US$ Mn)

8. Global NPHP5 Retinal Degeneration Treatment Market, By Region, 2020-2032, (US$ Mn)

9. Competitive Landscape

10. Section

全球 NPHP5 视网膜变性治疗市场（按治疗类型、适应症、分销管道和地区划分）

Global NPHP5 Retinal Degeneration Treatment Market, By Treatment Type, By Indication, By Distribution Channel, By Geography

市场动态：

本研究的主要特点

目录

第一章 调查目的与前提条件

第二章 市场展望

第三章市场动态、法规与趋势分析

4. 全球 NPHP5 视网膜退化治疗市场-冠状病毒 (COVID-19) 大流行的影响

5. 2020 年至 2032 年全球 NPHP5 视网膜退化治疗市场（依治疗类型）

6. 全球 NPHP5 视网膜退化治疗市场（依适应症划分），2020 年至 2032 年

7. 2020 年至 2032 年全球 NPHP5 视网膜退化治疗市场（依分销管道）

8. 全球 NPHP5 视网膜变性治疗市场（按地区），2020 年至 2032 年

第九章 竞争态势

第 10 章 章节

Market Dynamics:

Key features of the study:

Detailed Segmentation:

Table of Contents

1. Research Objectives and Assumptions

2. Market Purview

3. Market Dynamics, Regulations, and Trends Analysis

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

5. Global NPHP5 Retinal Degeneration Treatment Market, By Treatment Type, 2020-2032, (US$ Mn)

6. Global NPHP5 Retinal Degeneration Treatment Market, By Indication, 2020-2032, (US$ Mn)

7. Global NPHP5 Retinal Degeneration Treatment Market, By Distribution Channel, 2020-2032, (US$ Mn)

8. Global NPHP5 Retinal Degeneration Treatment Market, By Region, 2020-2032, (US$ Mn)

9. Competitive Landscape

10. Section

第一章调查目的与前提条件

第二章市场展望

第九章竞争态势

第 10 章章节