DelveInsight's analysts estimate that the inherited retinal diseases market is poised to show significant growth, mainly attributed to increasing prevalence, drug approvals, and anticipated launch of novel therapies during the forecast period (2023–2034).

LAS VEGAS, May 22, 2024 /PRNewswire/ -- DelveInsight's Inherited Retinal Disease Market Insights report includes a comprehensive understanding of current treatment practices, inherited retinal disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

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Key Takeaways from the Inherited Retinal Disease Market Report

  • According to DelveInsight's analysis, the market size for inherited retinal disease is expected to grow significantly by 2034.
  • As per DelveInsight's estimates, the prevalent population of retinitis pigmentosa and choroideremia in the US was found to be approximately 112K and 6.6K, respectively in 2021, which as per DelveInsight's analysts, might increase by 2034.
  • Leading inherited retinal disease companies such as HuidaGene Therapeutics Co., Ltd., Cholgene Therapeutics, Inc., Endogena Therapeutics, Inc., Opus Genetics, Inc., Editas Medicine, Inc., Spark Therapeutics, Inc., ProQR Therapeutics, Alkeus Pharmaceuticals, Inc., Gyroscope Therapeutics Limited, Novartis Pharmaceuticals, SparingVision, Unity Biotechnology, Inc., Beacon Therapeutics, GenSight Biologics, Ocugen, AbbVie, Belite Bio, Inc, IVERIC bio, Inc., ReNeuron Limited, and others are developing novel inherited retinal disease drugs that can be available in the inherited retinal disease market in the coming years.
  • The promising inherited retinal disease therapies in the pipeline include HG004, EA-2353, AAV8.hLCA5, EDIT-101, AAV2-hRPE65v2,voretigene neparvovec-rzyl, QR-1123, ALK-001, GT005, SPVN06, Aflibercept, rAAV2tYF-GRK1-hRPGRco, GS010, OCU410ST, RST-001, Tinlarebant, Zimura, hRPC, among others.

Discover which therapies are expected to grab the major inherited retinal disease market share @ Inherited Retinal Disease Market Report

Inherited Retinal Disease Overview

Inherited retinal diseases (IRDs) encompass a diverse range of disorders marked by either the degeneration or malfunction of photoreceptors. These conditions often lead to significant vision impairment, which may worsen gradually, manifesting from birth to later stages of adulthood. The root cause of IRDs lies in genetic mutations, disrupting the normal function of retinal cells and consequently resulting in vision loss over time. Presently, approximately 250 genes have been identified as contributors to IRD, though many more remain undiscovered. Common symptoms include challenges in low-light environments, diminished peripheral vision leading to collisions, sensitivity to glare, and various other visual difficulties.

Inherited Retinal Disease Epidemiology Segmentation

The inherited retinal disease epidemiology section provides insights into the historical and current inherited retinal disease patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.

The inherited retinal disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

  • Total Prevalent Cases of Inherited Retinal Diseases 
  • Type-specific Prevalent Cases of Inherited Retinal Diseases
  • Diagnosed and Treatable Cases of Inherited Retinal Diseases

Inherited Retinal Disease Treatment Market 

So far, progress in developing treatments and cures to alter the speed of disease advancement has been limited. However, there have been some notable successes in clinical trials, particularly in the realms of neurotrophic factor therapy and gene therapies. One outstanding example of treatment success in inherited retinal diseases is gene augmentation therapy. After almost twenty years of development in both preclinical models and clinical trials, the FDA approved the first gene-augmentation therapy for an IRD (Retinitis Pigmentosa) in December 2017. This treatment, named LUXTURNA, is intended for individuals affected by retinal dystrophies caused by mutations in the RPE65 gene.

Researchers are actively engaged in the development and evaluation of drugs to combat Choroideremia and similar retinal conditions, aiming to impede or halt the deterioration of eyesight. The genetic anomaly in Choroideremia leads to the gradual dysfunction and subsequent death of specific retinal cells, resulting in vision impairment. Investigations are underway into various compounds that could sustain the vitality of these cells. These treatments, known as neuroprotective agents, enhance retinal health by prolonging the functionality and lifespan of affected cells, thus decelerating the advancement of vision loss.

Regenerative therapy in the field of medicine shows great potential in rejuvenating visual capabilities for individuals affected by degenerative retinal conditions like Retinitis Pigmentosa, Stargardt's macular dystrophy, and age-related macular degeneration. Phase I/II trials involving stem cell treatments have demonstrated significant success, with no observed systemic side effects across the studies.

One field currently under investigation involves the utilization of neuroprotective substances. These encompass a diverse array of treatments designed to impede or halt the degenerative process by fostering cell survival. Neuroprotective substances include N-acetylcysteine (NAC)/N-acetylcysteine-amide (NACA), Tauroursodeoxycholate acid (TUDCA), Idebenone (Raxone), and various antioxidants. NAC has demonstrated notable efficacy as an antioxidant in a mouse model of Retinitis Pigmentosa. Both oral and topical administration of NAC are viable for extended treatment in humans, exhibiting a favorable safety profile. Thus, there is solid justification for exploring NAC's potential in treating Retinitis Pigmentosa patients.

To know more about inherited retinal disease treatment guidelines, visit @ Inherited Retinal Disease Management 

Inherited Retinal Disease Pipeline Therapies and Key Companies

  • HG004: HuidaGene Therapeutics Co., Ltd./Cholgene Therapeutics, Inc.
  • EA-2353: Endogena Therapeutics, Inc
  • AAV8.hLCA5: Opus Genetics, Inc
  • EDIT-101: Editas Medicine, Inc.
  • AAV2-hRPE65v2,voretigene neparvovec-rzyl: Spark Therapeutics, Inc.
  • QR-1123: ProQR Therapeutics
  • ALK-001: Alkeus Pharmaceuticals, Inc.
  • GT005: Gyroscope Therapeutics Limited/Novartis Pharmaceuticals
  • SPVN06: SparingVision
  • Aflibercept: Unity Biotechnology, Inc.
  • rAAV2tYF-GRK1-hRPGRco: Beacon Therapeutics
  • GS010: GenSight Biologics
  • OCU410ST: Ocugen
  • RST-001: AbbVie
  • Tinlarebant: Belite Bio, Inc
  • Zimura: IVERIC bio, Inc.
  • hRPC: ReNeuron Limited

Learn more about the FDA-approved drugs for inherited retinal disease @ Drugs for Inherited Retinal Disease Treatment 

Inherited Retinal Disease Market Dynamics

The dynamics of the inherited retinal disease market are expected to change in the coming years. With over 250 genetic mutations identified to date, the landscape of IRD diagnosis and treatment is rapidly evolving. Genetic testing plays a pivotal role in early detection and personalized treatment strategies, allowing for targeted interventions tailored to individual patients' genetic profiles. This has led to a shift towards precision medicine approaches, with pharmaceutical companies investing heavily in research and development to bring innovative therapies to market.

Moreover, the growing interest from biotechnology and pharmaceutical companies in the IRD market is fueling competition and driving innovation. Gene therapies, in particular, have emerged as a promising treatment modality for IRDs, with several candidates in various stages of clinical development. The potential for gene therapies to address the underlying genetic causes of IRDs offers hope for patients with previously untreatable conditions. 

Furthermore, patient advocacy groups and collaborations between academia, industry, and regulatory agencies are playing an increasingly important role in shaping the IRD landscape. These collaborations facilitate knowledge sharing, streamline clinical trial processes, and advocate for improved access to care for patients with IRDs. Additionally, initiatives aimed at raising awareness about the importance of early diagnosis and treatment are helping to reduce diagnostic delays and improve outcomes for patients affected by these debilitating diseases. As the IRD market continues to evolve, collaboration and innovation will be key drivers in unlocking new treatment modalities and improving the lives of patients worldwide.

Furthermore, many potential therapies are being investigated for the treatment of inherited retinal disease, and it is safe to predict that the treatment space will significantly impact the inherited retinal disease market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the inherited retinal disease market in the 7MM.

However several factors may impede the growth of the inherited retinal disease market. Scientifically, the intricate genetic underpinnings of IRDs present challenges in developing targeted therapies for the diverse array of conditions within this category. The rarity and genetic heterogeneity of IRDs further complicate clinical trial recruitment and therapeutic development. Additionally, the high cost of research and development, coupled with the small patient populations, renders IRD therapies economically challenging for pharmaceutical companies. Moreover, regulatory hurdles and the need for specialized infrastructure for diagnosis and treatment further impede market access. Overcoming these barriers demands collaborative efforts among stakeholders, innovative financing models, and advancements in genetic diagnostics and therapeutic technologies.

Inherited Retinal Disease Market Report Metrics

Details

Study Period

2020–2034

Coverage

7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Inherited Retinal Disease Companies

HuidaGene Therapeutics Co., Ltd., Cholgene Therapeutics, Inc., Endogena Therapeutics, Inc, Opus Genetics, Inc, Editas Medicine, Inc., Spark Therapeutics, Inc., ProQR Therapeutics, Alkeus Pharmaceuticals, Inc., Gyroscope Therapeutics Limited, Novartis Pharmaceuticals, SparingVision, Unity Biotechnology, Inc., Beacon Therapeutics, GenSight Biologics, Ocugen, AbbVie, Belite Bio, Inc, IVERIC bio, Inc., ReNeuron Limited, and others

Key Pipeline Inherited Retinal Disease Therapies

HG004, EA-2353, AAV8.hLCA5, EDIT-101, AAV2-hRPE65v2,voretigene neparvovec-rzyl, QR-1123, ALK-001, GT005, SPVN06, Aflibercept, rAAV2tYF-GRK1-hRPGRco, GS010, OCU410ST, RST-001, Tinlarebant, Zimura, hRPC, and others

Scope of the Inherited Retinal Disease Market Report

  • Therapeutic Assessment: Inherited Retinal Disease current marketed and emerging therapies
  • Inherited Retinal Disease Market Dynamics: Key Market Forecast Assumptions of Emerging Inherited Retinal Disease Drugs and Market Outlook
  • Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
  • Unmet Needs, KOL's views, Analyst's views, Inherited Retinal Disease Market Access and Reimbursement

Discover more about inherited retinal disease drugs in development @ Inherited Retinal Disease Clinical Trials

Table of Contents

1.

Inherited Retinal Disease Key Insights

2.

Inherited Retinal Disease Report Introduction

3.

Inherited Retinal Disease Overview at a Glance

4.

Inherited Retinal Disease Executive Summary

5

Inherited Retinal Disease Key Events

6

Epidemiology and Market Forecast Methodology

6.

Disease Background and Overview

7.

Inherited Retinal Disease Treatment and Management

8.

Inherited Retinal Disease Guidelines

9.

Inherited Retinal Disease Epidemiology and Patient Population

10.

Patient Journey

11.

Key Endpoints in Inherited Retinal Disease 

12.

Inherited Retinal Disease Marketed Drugs

13.

Inherited Retinal Disease Emerging Drugs

14.

7MM Inherited Retinal Disease Market Analysis

15.

Market Access and Reimbursement

16.

KOL Views

17.

Unmet Needs

18.

SWOT Analysis

19.

Appendix

20.

DelveInsight Capabilities

21.

Disclaimer

22.

About DelveInsight

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