유전자 치료의 AAV(아데노 부속 바이러스) 벡터 : 시장 인사이트, 역학, 시장 예측(-2034년)

Key Highlights:

• AAVs have emerged as the predominant vectors for delivering genes of interest to target tissues with improved specificity, efficiency, and safety.
• The first Adeno Associated Virus Vectors in Gene Therapy drug, GLYBERA, was approved by the European Medicines Agency (EMA) in 2012 but later in 2020, it was withdrawn from the market mainly due to commercial failure. Many AAV-based gene therapies are currently FDA-approved, ELEVIDYS and ROCTAVIAN were approved in 2023, HEMGENIX was approved in 2022, LUXTURNA was approved in 2020 for a rare inherited retinal dystrophy, ZOLGENSMA was approved in 2019 for spinal muscular atrophy and GLYBERA was approved by EMA.
• In November 2023, REGENXBIO Presented positive one-year data from the Phase II ALTITUDE trial of ABBV-RGX-314 for the treatment of diabetic retinopathy using suprachoroidal delivery
• In November 2023, Pfizer announced dosing completed in the Phase III AFFINE trial of giroctocogene fitelparvovec; pivotal data read-out expected in mid-2024; BLA and MAA submissions anticipated in the second half of 2024.
• From the perspective of unmet medical needs, the majority of rAAV gene therapy programs are centered around the liver, striated muscles, and the CNS. Essentially all characteristics of AAV capsids can transduce liver productively following systemic administration.
• Companies are offering innovative payment plans for one-time gene therapies, which can lead to better uptake of gene therapies.

DelveInsight's "Adeno-Associated Virus Vector in Gene Therapy - Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of Adeno-Associated Virus Vector in Gene Therapy, historical and forecasted epidemiology as well as Adeno-Associated Virus Vector in Gene Therapy market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The Adeno-Associated Virus Vector in Gene Therapy market report provides current treatment practices, emerging drugs, Adeno-Associated Virus Vector in Gene Therapy market share of individual therapies, and current and forecasted Adeno-Associated Virus Vector in Gene Therapy market size from 2020 to 2034, segmented by seven major markets. The report also covers current Adeno-Associated Virus Vector in Gene Therapy treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Adeno-Associated Virus Vector in Gene Therapy Understanding

Adeno-Associated Virus Vector in Gene Therapy Overview

Recent advancements in genetic analysis, enabling rapid and cost-effective sequencing of genomes, have facilitated the identification of genes underlying various diseases. In particular, monogenic diseases caused by mutations in a single gene present promising targets for gene therapy interventions. However, realizing this potential has been hindered by challenges in safely delivering therapeutic nucleic acids into cells. While viral vectors, such as AAV, initially showed promise, concerns over immunogenicity and safety limitations have tempered their widespread use.

Alternative delivery methods, like nanoparticles, have also encountered hurdles. Despite these challenges, rAAV, a genetically engineered form of AAV devoid of viral DNA, has emerged as a promising gene therapy vehicle. Engineered to efficiently transport therapeutic DNA into target cells, rAAV circumvents integration into the host genome, thus minimizing the risk of unintended genetic alterations. Instead, rAAV-delivered DNA persists in the cell nucleus as episomal concatemers, ensuring sustained therapeutic effect without genomic integration. Despite ongoing research to enhance delivery efficiency and safety, rAAV holds significant potential for treating a wide range of genetic diseases, with ongoing clinical trials providing valuable insights for future therapeutic strategies.

Adeno-Associated Virus Vector in Gene Therapy Epidemiology

The Adeno-Associated Virus Vector in Gene Therapy epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the Total Prevalent Cases of Selected Indications Adeno-Associated Virus Vector in Gene Therapy, Total Diagnosed Prevalent Cases of Selected Indications of Adeno-Associated Virus Vector in Gene Therapy, Total Treated Cases of Selected Indications for Adeno-Associated Virus Vector in Gene Therapy, in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• Retinitis Pigmentosa, is the leading cause of visual disability and blindness in subjects less than 60 years old.
• Hemophilia A is more common than Hemophilia B, representing approximately 80-85% of the total hemophilia population.

Adeno-Associated Virus Vector in Gene Therapy Drug Chapters

The drug chapter segment of the adeno-associated virus vector in gene therapy report encloses a detailed analysis of the late-stage (Phase III ) and mid-stage (Phase II/III and Phase II) pipeline drugs. The current key players include Sangamo and Pfizer (Giroctocogene fitelparvovec), MEIRAGTx/J&J (Bota-vec), Johnson & Johnson (JNJ-81201887) and others.

The drug chapter also helps understand the adeno-associated virus vector in gene therapy clinical trial details, pharmacological action, agreements and collaborations, approval, and patent details, and the latest news and press releases.

Adeno Associated Virus Vectors in Gene Therapy Marketed Drugs

LUXTURNA: Spark Therapeutics

LUXTURNA (voretigene neparvovec-rzyl) is a suspension of an adeno-associated virus vector-based gene therapy for subretinal injection. LUXTURNA is a live, non-replicating adeno-associated virus serotype 2 which has been genetically modified to express the human RPE65 gene. LUXTURNA is derived from naturally occurring adeno-associated virus using recombinant DNA techniques.

LUXTURNA, is a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. The FDA approved Spark Therapeutics' LUXTURNA in December 2017.

ZOLGENSMA: Novartis

ZOLGENSMA is a suspension of an adeno-associated viral vector-based gene therapy for intravenous infusion. It is a recombinant self-complementary AAV9 containing a transgene encoding the human survival motor neuron (SMN) protein, under the control of a cytomegalovirus enhancer/chicken-B-actin hybrid promoter. ZOLGENSMA an AAV-delivered gene therapy used to treat spinal muscular atrophy (SMA), was approved for clinical use in the US by the FDA.

Adeno Associated Virus Vectors in Gene Therapy Emerging Drugs

LUMEVOQ: GenSight Biologics

LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector. According to Phase III results all treated participants showed sustained improvement over 4 years, but that patients treated with a bilateral injection of the gene therapy continued to have a better visual acuity than the patients treated with a unilateral injection, a disparity that had been seen in REFLECT since 1.5 years posttreatment.

The company is planning to submit to MHRA in the second half of 2024 and aiming to receive a decision from MHRA on LUMEVOQ in the second half of 2025, in the hopes of commercializing the product in the UK that same year.

Giroctocogene fitelparvovec: Sangamo and Pfizer

Giroctocogene fitelparvovec comprises of a recombinant AAV6 encoding the complementary deoxyribonucleic acid for B-domain deleted human FVIII. It is being developed as part of a collaboration agreement for the global development and commercialization of gene therapies for hemophilia A between Sangamo and Pfizer. Currently, the company is conducting Phase III trials to attain better and clear results about the efficacy of SB-525. A Phase III clinical trial (NCT03587116) evaluates the effectiveness and safety of preventive replacement therapy in the usual care setting in hemophilia A patients. A pivotal readout is expected in mid-2024, with Pfizer anticipating BLA and MAA submissions in the second half of 2024 if the pivotal readout is supportive.

Adeno-Associated Virus Vector in Gene Therapy Market Outlook

Adeno Associated Virus Vectors in Gene Therapy has provided a unique opportunity to treat and even cure degenerative diseases, offering hope to the millions of people either affected by inherited disorders or carrying disease-causing mutations. Addressing optimal intervention timing, standardized outcome assessments, inflammation mitigation, awareness enhancement, and equitable access are key to advancing inherited retinal disease treatments and reshaping the landscape of visual impairment.

Gene therapies have brought about a change in the treatment paradigm for genetic diseases by providing lasting therapeutic effects with a single intervention. Gene therapy holds significant potential for addressing various eye diseases. However, individuals considering this treatment should be mindful of certain challenges and crucial factors. Although the recent successes in rare disease therapy approvals have provided momentum for AAV therapy research and funding, several limitations make this a difficult, capital-intensive platform to develop.

In December 2017, Spark Therapeutics announced that their drug candidate LUXTURNA received US FDA approval for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. LUXTURNA is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease, and the first AAV vector gene therapy approved in the United States. The drug was developed and commercialized in the United States by Spark Therapeutics. In Europe, Novartis is currently marketing LUXTURNA as per a licensing agreement covering the development, registration, and commercialization rights of LUXTURNA in markets outside the United States. The US FDA's advisory panel had all the way commended LUXTURNA approval in October 2017.

In the past few years, the treatment landscape of many diseases has rapidly changed. Now companies are developing gene and cell therapies that will have a promising role in the future, especially for the treatment of rare genetic diseases. The task of defining appropriate candidates for given gene therapy and cell therapy will need to await the enrollment and long-term follow-up of a sufficient number of study subjects to provide acceptable clarity about its safety and efficacy. To summarize, the outlook for gene and cell therapies is promising. Various clinical trials have been fairly positive in terms of safety and efficacy. The results of these studies are encouraging further investigation into multiple indications. The current scenario also anticipates a positive shift in the market for the forecast period.

Key Findings of Adeno Associated Virus Vectors in Gene Therapy Treatment Market

• The United States captured the major market shared of AAV gene therapy market among the 7MM.
• Recently, Pfizer BEQVEZ approved or the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
• Pfizer set the similar price of BEQVEZ as HEMGENIX, i.e., around USD 3.5 million.
• Advancements in viral vector research, including targeted cell-based delivery, hold potential for enhancing treatment efficacy
• Larger firms have an edge in the gene therapy industry because they have the resources and expertise to organize intricate development pathways.

Adeno-Associated Virus Vector in Gene Therapy Pipeline Development Activities

The report provides insights into therapeutic candidates in Phase III, Phase II/III, and Phase II. It also analyzes key players involved in developing targeted therapeutics. Companies like Sangamo and Pfizer, MEIRAGTx/J&J, Johnson & Johnson, and others actively engage in late and mid-stage research and development efforts for Adeno-Associated Virus Vector in Gene Therapy pipeline possesses potential drugs. However, there is a positive outlook for the therapeutics market, with expectations of growth during the forecast period (2024-2034).

Adeno Associated Virus Vectors in Gene Therapy Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Adeno-Associated Virus Vector in Gene Therapy emerging therapy.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Adeno Associated Virus Vectors in Gene Therapy Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis, Conjoint analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Adeno Associated Virus Vectors in Gene Therapy Market Access and Reimbursement

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In the report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

Since the patients' healthcare payments are substantial and such high healthcare expenditure is burdensome for the patients and their families. To help these patients, various third parties and nonprofits also have reimbursement schemes based on the demonstration of acceptable cost-effectiveness.

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of Adeno-Associated Virus Vector in Gene Therapy, explaining currently used therapies.
• Additionally, an all-inclusive account of the emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the Adeno-Associated Virus Vector in Gene Therapy market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views around Adeno-Associated Virus Vector in Gene Therapy market.
• Adeno-Associated Virus Vector in Gene Therapy Report Insights
• Patient Population
• Therapeutic Approaches
• Adeno-Associated Virus Vector in Gene Therapy Pipeline Analysis
• Adeno-Associated Virus Vector in Gene Therapy Market Size and Trends
• Existing and Future Market Opportunity

Adeno-Associated Virus Vector in Gene Therapy Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage
• Adeno-Associated Virus Vector in Gene Therapy Epidemiology Segmentation
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Adeno-Associated Virus Vector in Gene Therapy Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Analyst Views

Reasons to Buy Adeno Associated Virus Vectors in Gene Therapy Market Report

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving Adeno-Associated Virus Vector in Gene Therapy
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis ranking of class-wise potential current and emerging therapies under the analyst view section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Table of Contents

1 Key Insights

2 Report Introduction

3 Key Highlights from Report

4 Executive Summary of AAV in Gene Therapy

5 Key Events

6 Epidemiology and Market Forecast Methodology

7 AAV in Gene Therapy Market Overview at a Glance in the 7MM

• 7.1 Market Share (%) Distribution by Therapies in 2020
• 7.2 Market Share (%) Distribution by Therapies in 2034

8 Disease Background and Overview

• 8.1 Introduction to and Gene Therapies
• 8.2 Adenoviral vectors
• 8.3 Limitations of vectors
• 8.4 Challenges in Adenoviral vectors

9 Epidemiology and Patient Population

• 9.1 Key Findings
• 9.2 Assumptions and Rationale: 7MM
• 9.3 Epidemiology Scenario in the 7MM
  • 9.3.1 Total Prevalent Cases of Selected Indications for AAV in Gene Therapy in the 7MM
  • 9.3.2 Total Diagnosed Prevalent Cases Selected Indications for AAV in Gene Therapy in the in the 7MM
  • 9.3.3 Indication-wise Treated Cases of AAV in Gene Therapy in the 7MM
• 9.4 United States
  • 9.4.1 Total Prevalent Cases of Selected Indications for AAV in Gene Therapy in the US
  • 9.4.2 Total Diagnosed Prevalent Cases of Selected Indications for AAV in Gene Therapy in the US
  • 9.4.3 Indication-wise Treated Cases of AAV in Gene Therapy in the US
• 9.5 EU4 and the UK
  • 9.5.1 Total Prevalent Cases of Selected Indications for AAV in Gene Therapy in EU4 and the UK
  • 9.5.2 Total Diagnosed Prevalent Cases of Selected Indications for AAV in Gene Therapy in EU4 and the UK
  • 9.5.3 Indication-wise Treated Cases of AAV in Gene Therapy in EU4 and the UK
• 9.6 Japan
  • 9.6.1 Total Prevalent Cases of Selected Indications for AAV in Gene Therapy in Japan
  • 9.6.2 Total Diagnosed Prevalent Cases of Selected Indications for AAV in Gene Therapy in Japan
  • 9.6.3 Indication-wise Treated Cases of AAV in Gene Therapy in Japan

10 Marketed Drugs

• 10.1 Retinitis Pigmentosa
  • 10.1.1 LUXTURNA: Sparks Therapeutics (a company of Roche)/ Novartis
    • 10.1.1.1 Product description
    • 10.1.1.2 Regulatory milestones
    • 10.1.1.3 Others developmental activities
    • 10.1.1.4 Ongoing clinical developmental activities
    • 10.1.1.5 Safety and efficacy
    • 10.1.1.6 Product profile
• 10.2 Hemophilia A
  • 10.2.1 ROCTAVIAN: BioMarin Pharmaceutical
    • 10.2.1.1 Product description
    • 10.2.1.2 Regulatory milestones
    • 10.2.1.3 Others developmental activities
    • 10.2.1.4 Ongoing clinical developmental activities
    • 10.2.1.5 Safety and efficacy
    • 10.2.1.6 Product profile

11 Emerging Drugs

• 11.1 Key Competitors
• 11.2 Hemophilia B
  • 11.2.1 Giroctocogene fitelparvovec: Sangamo and Pfizer
    • 11.2.1.1 Product description
    • 11.2.1.2 Other developmental activity
    • 11.2.1.3 Clinical developmental activities
    • 11.2.1.3.1 Clinical trial information
    • 11.2.1.4 Safety and efficacy
• 11.3 Retinitis Pigmentosa
  • 11.3.1 Botaretigene sparoparvovec (bota-vec): MeiraGTx Limited
    • 11.3.1.1 Product description
    • 11.3.1.2 Other developmental activities
    • 11.3.1.3 Clinical developmental activities
    • 11.3.1.3.1 Clinical trials information
    • 11.3.1.4 Safety and efficacy
• 11.4 Duchenne Muscular Dystrophy
  • 11.4.1 PF-06939926: Pfizer
    • 11.4.1.1 Product description
    • 11.4.1.2 Other developmental activities
    • 11.4.1.3 Clinical developmental activities
    • 11.4.1.3.1 Clinical trials information
    • 11.4.1.4 Safety and efficacy

List to be continued in the full report

12 AAV in Gene Therapy: 7MM analysis

• 12.1 Key Findings
• 12.2 Market Outlook
• 12.3 Conjoint Analysis
• 12.4 Key Market Forecast Assumptions
• 12.5 Market Size by Indications in the 7MM
• 12.6 Market Size by therapies in the 7MM
• 12.7 United States Market Size
  • 12.7.1 Market Size by Indications in the US
  • 12.7.2 Market Size by therapies in the US
• 12.8 EU4 and the UK Market Size
  • 12.8.1 Market Size by Indications in EU4 and the UK
  • 12.8.2 Market Size by therapies in EU4 and the UK
• 12.9 Japan Market Size
  • 12.9.1 Market Size by Indications in Japan
  • 12.9.2 Market Size by therapies in Japan

13 Unmet Needs

14 SWOT Analysis

15 KOL Views

16 Market Access and Reimbursement

17 Appendix

• 17.1 Bibliography
• 17.2 Report Methodology

18 DelveInsight Capabilities

19 Disclaimer

20 About DelveInsight