NPHP5 視網膜變性治療的全球市場：按治療類型、按適應症、按分銷管道、按地區

全球 NPHP5 視網膜變性治療市場規模預計將從2024年的 1,570 萬美元增加到2031年的 1.313 億美元，預測期內年複合成長率（CAGR）為 35.4%。

圖 1. NPHP5 視網膜變性治療的全球市場佔有率（%），依地區分類，2024年

NPHP5 視網膜變性，也稱為高級洛肯症候群 7，是一種罕見的遺傳性纖毛疾病，會導致進行性視力障礙，並可能導致完全失明。這種疾病是由對纖毛功能很重要的蛋白質編碼腎囊腫素5NPHP5基因突變引起的。此病的特徵是視網膜色素變性、腎病變和其他全身性病變。目前還沒有核准的NPHP5 視網膜變性治療方法，治療方法包括使用低視力輔助設備和支持性護理。然而，最近調查致病機制的研究揭示潛在的藥物標靶，例如纖毛功能，並為治療開發開闢了新的途徑。幾家生物製藥公司已經開始臨床試驗，以評估基因療法和其他新的治療方法，以恢復這些患者的視力。 Senior Loken（SL）是一種體染色體隱性遺傳症候群，其中囊性腎病變是伴隨視網膜營養不良（視網膜色素變性或萊伯氏先天性黑朦症）的腎病相關疾病的一種變體。這是一種爆發性疾病，可導致失明和腎衰竭。

市場動態：

未滿足的需求市場：未滿足的需求視網膜變性市場是由對這種使人衰弱的疾病的有效治療的高度未滿足的醫療需求推動的。據估計，全球整體NPHP5 視網膜退化的盛行率為20 萬分之一。對睫狀途徑的研究不斷增加，以及視網膜基因治療的最新突破，透過擴大治療機會進一步推動市場成長。然而，與眼部基因轉移相關的技術挑戰以及開發治療罕見疾病的藥物的高成本繼續限制市場收益。另一方面，生物技術公司和研究機構之間的合作，以及對罕見疾病和研發的公共和私人資金的增加，為市場進入者提供了利潤豐厚的機會。

本研究的主要特點

• 本報告對全球NPHP5視網膜變性治療藥物市場進行了詳細分析，並列出了以2023年為基準年的預測期（2024-2031）的市場規模和年複合成長率。
• 揭示了各個細分市場的潛在商機，並為該市場說明了一系列有吸引力的投資提案。
• 提供了有關市場促進因素、限制因素、機會、新產品發布和核准、市場趨勢、區域前景、主要企業採取的競爭策略等的重要見解。
• 本報告根據公司亮點、產品系列、主要亮點、財務表現和策略等參數，介紹了全球 NPHP5 視網膜變性治療市場的主要參與者。這項研究的主要目標公司包括 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。
• 本報告的見解使行銷人員和公司負責人能夠就未來的產品發布、類型升級、市場擴張和行銷策略做出明智的決策。
• 全球 NPHP5 視網膜變性治療市場報告迎合了該行業的各種相關人員，如投資者、供應商、產品製造商、經銷商、新進業者和財務分析師。
• 相關人員可以透過用於分析全球 NPHP5 視網膜變性治療市場的各種策略矩陣來促進他們的決策。

目錄

第1章 研究目的與前提

• 研究目標
• 先決條件
• 簡稱

第2章 市場展望

• 報告說明
  • 市場定義和範圍
• 執行摘要
• Coherent Opportunity Map（COM）

第3章 市場動態、法規及趨勢分析

• 市場動態
  • 加強宣傳活動力度
  • 核准時間長
  • 亞太地區和拉丁美洲新興市場
• 主要亮點
• 監管場景
• 市場趨勢
• PEST分析
• 波特的分析
• 產品發表會
• 流行病學
• 合併、收購和合作

第4章 全球 NPHP5 視網膜退化治療市場 – 冠狀病毒（COVID-19）大流行的影響

• 新型冠狀病毒感染疾病（COVID-19）的流行病學
• 供需面分析
• 經濟影響

第5章 全球NPHP5視網膜變性治療市場，依治療類型，2019-2031

• 基因治療
• 藥物治療
• 其他

第6章 2019-2031年全球 NPHP5 視網膜退化治療市場，依適應症

• 色素性視網膜炎
• 萊伯氏先天性黑朦症
• 尤塞氏症候群
• 其他

第7章 2019-2031年全球NPHP5視網膜變性治療市場，依通路

• 零售藥房
• 醫院藥房
• 網路藥房

第8章 2019-2031年全球NPHP5視網膜變性治療市場，依地區

• 北美洲
• 美國
• 加拿大
• 歐洲
• 德國
• 英國
• 西班牙
• 法國
• 義大利
• 俄羅斯
• 其他歐洲國家
• 亞太地區
• 中國
• 印度
• 日本
• 澳洲
• 韓國
• ASEAN
• 其他亞太地區
• 拉丁美洲
• 巴西
• 阿根廷
• 墨西哥
• 其他拉丁美洲
• 中東
• 海灣合作理事會國家
• 以色列
• 其他中東地區
• 非洲
• 北非
• 中部非洲
• 南非

第9章 競爭形勢

• 公司簡介
  • 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

• 參考
• 調查方法
• 關於出版商

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