Adeno Associated Virus Vectors In Gene Therapy Market

DelveInsight's "Adeno-Associated Virus Vectors in Gene Therapy - Market Insights, Epidemiology, and Market Forecast-2030" report delivers an in-depth understanding of the Adeno-Associated Virus Vectors in Gene Therapy, historical and forecasted epidemiology as well as the Adeno-Associated Virus Vectors in Gene Therapy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Adeno-Associated Virus Vectors in Gene Therapy market report provides current treatment practices, emerging drugs, Adeno-Associated Virus Vectors in Gene Therapy market share of the individual therapies, current and forecasted Adeno-Associated Virus Vectors in Gene Therapy market Size from 2017 to 2030 segmented by seven major markets. The Report also covers current Adeno-Associated Virus Vectors in Gene Therapy treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.


Geography Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan


Study Period: 2017-2030


Adeno-Associated Virus Vectors in Gene Therapy Disease Understanding and Treatment Algorithm

Gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections). Gene therapy involves “vectors” which can be either viral or non-viral vectors. Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. There are currently two classes of recombinant AAVs (rAAVs) in use: single-stranded AAV (ssAAV) and self-complementary AAV (scAAV). rAAV gene therapy strategies include Gene replacement, Gene silencing, Gene addition and Gene editing. There are 12 naturally occurring serotypes (AAV serotype 1 [AAV-1] to AAV-12)and more than 100 variants of AAV, each of which differs in its amino acid sequence, particularly within the hypervariable regions of the capsid proteins, and, thus, also differ slightly in their gene delivery properties. AAV2 is the most extensively studied serotype among all. The tissue tropism of the different AAV serotypes is determined by the different cell surface receptors used for the attachment to the target cell. According to certain comparative studies between AAV serotypes, when it comes to targeting tissues, AAV3 and AAV4 are the slowest, and among all the serotypes, AAV2, 3, 4, and 5 have the lowest transduction efficiency.


The first AAV-based gene therapy, Glybera, was approved by the European Medicines Agency (EMA) in 2012 but later in 2017, it was withdrawn from the market mainly due to commercial failure. Only two AAV-based gene therapies are currently FDA-approved. Luxturna was approved in 2017 for a rare inherited retinal dystrophy, and Zolgensma was approved in 2019 for spinal muscular atrophy. Keeping in mind, the numerous advantages of AAV-vectors, currently, these vectors are being tested to cure diseases which were earlier thought to be nearly impossible to treat. Be it any Ocular disorder, blood disorder, metabolic disorder or muscular disorder expectations are high from AAV-vector gene therapy and thus, clinical trials for each mentioned disorders are positively going on.


Adeno-Associated Virus Vectors in Gene Therapy Epidemiology  

The Adeno-Associated Virus Vectors in Gene Therapy epidemiology division provide insights about historical and current Adeno-Associated Virus Vectors in Gene Therapy patient pool and forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.  


Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.


Country Wise- Adeno-Associated Virus Vectors in Gene Therapy Epidemiology 

Estimates show that the highest cases of AAV vector based gene therapy in the 7MM were in the United States, followed by Germany, Japan, France, the United kingdom, Italy, and Spain in 2017.

  • In the year 2017, the total prevalent cases of selected indications for AAV Gene Therapies were 2,718,559 cases in the 7MM which are expected to grow during the study period, i.e., 2017–2030.
  • In the United States, the total number of prevalent cases of AAV vector based gene therapy was 1,333,147 cases in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.
  • In EU-5 countries the highest number of prevalent cases of AAV vector based gene therapy were in Germany i.e. 428,198 in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.
  • In Japan, the total number of prevalent cases of AAV vector based gene therapy was 233,892 cases in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.
  • Also, in the 7MM, the total treated cases of indication wise AAV Gene Therapies were 736,173 in the year 2017 which are expected to grow during the study period, i.e., 2017–2030.


Adeno-Associated Virus Vectors in Gene Therapy Drug Chapters

Drug chapter segment of the Adeno-Associated Virus Vectors in Gene Therapy report encloses the detailed analysis of Adeno-Associated Virus Vectors in Gene Therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Adeno-Associated Virus Vectors in Gene Therapy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.


AAV Vector Based Gene Therapy Approved Drugs

Zolgensma/AVXS-101 (Novartis (AveXis))


Zolgensma (onasemnogene abeparvovec-xioi), previously known as AVXS-101, is a proprietary gene therapy of Novartis, designed for the treatment of pediatric patients less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. It is a suspension of an adeno-associated viral vector-based gene therapy for intravenous (IV) infusion. It address the genetic root cause of SMA and halt the disease progression through sustained SMN protein expression with a single, one-time IV 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-β-actin hybrid promoter


Luxturna (Spark Therapeutics/ Novartis)


Luxturna (AAV2-hRPE65v2) known as voretigene neparvovec-rzyl, 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. It provides a copy of the RPE65 gene to act in place of the mutated RPE65 gene. The drug is administered as a subretinal single injection below the retina in patients who have confirmed RPE65 mutations and viable retinal cells. Luxturna is an AAV2 gene therapy vector with a cytomegalovirus (CMV) enhancer and chicken beta-actin promoter driving expression of normal human retinal pigment epithelium 65 kDa protein (hRPE65) gene. It is indicated for the treatment of patients with vision loss due to Leber’s congenital amaurosis or retinitis pigmentosa inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations

Note: Detailed Current therapies assessment will be provided in the full report of AAV Vector Based Gene Therapy


Adeno-Associated Virus Vectors in Gene Therapy Emerging Drugs 

Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical

Valoctocogene Roxaparvovec is under development by Biomarin Pharmaceutical, which is an experimental gene therapy based on the use of adeno-associated virus (AAV) vectors. It delivers a functional copy of clotting factor VIII that people with hemophilia A are missing. Valoctocogene roxaparvovec is administered as a single infusion. Currently, Phase III GENEr8-3 trial is going on which started in June 2020, including 20 participants.


RG6357 (SPK-8011): Roche (Spark Therapeutics)


RG6357 (SPK-8011), which is under development by Roche’s subsidiary Spark Therapeutics, is an intravenously administered novel bio-engineered AAV vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter for hemophilia A, or factor VIII deficiency


Fidanacogene elaparvovec (SPK-9001/ PF-06838435): Pfizer (Spark Therapeutics)


Fidanacogene elaparvovec, previously SPK-9001 or PF-06838435 is a novel, investigational bio-engineered AAV vector utilizing a high-activity F9 transgene for hemophilia B, or factor IX deficiency. Currently, SPK-9001 is under Phase III (NCT03861273) clinical trials. This study will evaluate the efficacy and safety of SPK-9001 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less).


AT-GTX-502/scAAV9.P546.CLN3: Amicus Therapeutics


Amicus Therapeutics is developing adeno-associated virus serotype 9 AAV9-CLN3 (AAV9-CLN3) gene therapy for children with CLN3 Batten disease. The company is currently leading a Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, delivering the CLN3 Gene by Self-Complementary AAV9. AT-GTX-502, also known as AAV9-CLN3, is a gene therapy that works by delivering a working copy of the gene CLN3. In May 2020, the US FDA granted fast track designation to AT-GTX-502 for Batten disease caused by mutations in the CLN3 gene.


Timrepigene emparvovec/BIIB111/AAV2-REP1: NightstaRx Ltd, a Biogen Company


Timrepigene emparvovec is in clinical development by Biogen for the treatment of choroideremia. This gene therapy enables cells to generate the missing REP-1 protein when injected into the eye. The early phases of cell death can be slowed down or reversed with REP-1 present, avoiding the gradual loss of vision seen in choroideremia.


SGT-001: Solid Biosciences


SGT-001, lead candidate of Solid Biosciences, is a novel AAV vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD, mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of DMD,


FLT190: Freeline Therapeutics


FLT190 is a next-generation gene therapy that uses an AAV8 as a vehicle to deliver a healthy copy of the GLA gene in the hopes that it will induce the production of normal alpha-GAL A. In contrast to the regular infusions of enzyme replacement therapy (ERT), this gene therapy is designed to be given in a single dose. Preclinical data using gene therapy platform for expression of the enzyme a-galactosidase A (GLA) has demonstrated safety and efficacy in various animal models.


RGX-121: Regenxbio


RGX-121 is being developed as a novel, one-time, treatment for MPS II, which is directly administered intra-cisternally into the CNS. It includes the NAV AAV9 vector encoding for human IDS (iduronate-2-sulfatase). Treatment with RGX-121 has been shown to restore IDS enzyme activity in animal models of MPS II to levels equivalent to or greater than those in non-affected animals. The extent of CNS correction in animal studies suggests that RGX-121 has the potential to be an important and suitable therapeutic option for MPS II patients.

Note: Detailed emerging therapies assessment will be provided in the final report.


Adeno-Associated Virus Vectors in Gene Therapy Market Outlook

The Adeno-Associated Virus Vectors in Gene Therapy market outlook of the report helps to build the detailed comprehension of the historic, current, and forecasted Adeno-Associated Virus Vectors in Gene Therapy market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology. 

This segment gives a thorough detail of Adeno-Associated Virus Vectors in Gene Therapy market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight. 

According to DelveInsight, Adeno-Associated Virus Vectors in Gene Therapy market in 7MM is expected to change in the study period 2017-2030.


Key Findings

The AAV Vector Based Gene Therapy market size in the 7MM is expected to change during the forecast period 2020–2030. The therapeutic market of AAV Vector Based Gene Therapy in the seven major markets was USD 428 million in 2019 which is expected to increase during forecast period (2020–2030). According to the estimates, the highest market size of AAV Vector Based Gene Therapy is found in the United States.


The United States Market Outlook

In 2019, the total market size of AAV Vector Based Gene Therapy therapies was USD 418.5 million in the United States. 


EU-5 Countries: Market Outlook

In 2019, the total market size of AAV Vector Based Gene Therapy therapies was USD 9.0 million in the EU-5 countries. 


Japan Market Outlook 

In 2020, the total market size of AAV-vector-based gene therapy was USD 24 million which is expected to increase by 2030. 


Adeno-Associated Virus Vectors in Gene Therapy Drugs Uptake

Among the selected indications, hemophilia A is expected to contribute maximum in terms of revenue by 2030 owing to maximum number of gene therapies which are expected to enter in to the market, precedence of existing high treatment cost and also expected high cost for these therapies and also significant residual unmet need. Whereas, among the emerging AAV gene therapies, Valoctocogene Roxaparvovec (BMN 270) expected to generate the maximum revenue by 2030 in the 7MM from the hemophilia A.


Adeno-Associated Virus Vectors in Gene Therapy Pipeline Development Activities 

The report provides insights into different therapeutic candidates in Phase II, and Phase III stage. It also analyses Adeno-Associated Virus Vectors in Gene Therapy key players involved in developing targeted therapeutics. 


Pipeline Development Activities

The drugs which are in pipeline include:

  • Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical: Phase III
  • PF-06939926: Pfizer: Phase III
  • Fidanacogene elaparvovec: Pfizer (initiated by Spark Therapeutics): Phase III
  • AMT-061: uniQure/CSL Behring: Phase III
  • Timrepigene emparvovec (BIIB111): NightstaRx Ltd, a Biogen Company: Phase III
  • Giroctocogene fitelparvovec (SB-525): Pfizer (previously Sangamo Biosciences): Phase III
  • BIIB112 (AAV8-RPGR): NightstaRx Ltd, a Biogen Company: Phase II/III
  • NLX P101 (AAV-GAD): MeiraGTx: Phase II
  • VY-AADC (NBIb-1817/ AAV2-hAADC): Neurocrine Biosciences/ Voyager Therapeutics: Phase II
  • SPK-8011: Roche (previously Spark Therapeutics)/Pfizer: Phase I/II
  • ST-920: Sangamo Therapeutics: Phase I/II
  • FLT190: Freeline Therapeutics: Phase I/II
  • SPK-3006 (AAV-sec-GAA): Spark Therapeutics: Phase I/II
  • ACTUS-101: Asklepios Biopharmaceutical (Actus Therapeutics): Phase I/II
  • AT845: Audentes Therapeutics: Phase I/II
  • SRP-9001: Roche/Sarepta Therapeutics: Phase I/IIa
  • HORA-PDE6B: Horama S.A.: Phase I/II
  • AAV-RPGR (AAV2/5-RPGR): MeiraGTx UK II Ltd: Phase I/II
  • RGX-121: RegenxBio: Phase I/II
  • SB-913: Sangamo Therapeutics: Phase I/II
  • AT-GTX-501 (scAAV9.CB.CLN6): Amicus Therapeutics: Phase I/IIa
  • AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics: Phase I/IIa


Reimbursement Scenario in Adeno-Associated Virus Vectors in Gene Therapy

  • In 2019, Luxturna was recommended by NICE, within its marketing authorization, as an option for treating RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. It was only recommended if the company provides Luxturna according to the commercial arrangement. In addition, Luxturna received ‘considerable added benefit’ from G-BA, the German health technology assessment. Luxturna was launched in Germany in April 2019 at the manufacturer price of €345,000 per eye per patient.
  • In Germany, Glybera was initially positioned as a community product as it does not require hospital admission for administration. It was thus formally evaluated through AMNOG (the German Health Technology Assessment process) and was granted “unquantifiable additional benefit.” G-BA concluded that clinical data did not support an additional benefit, and thus the “unquantifiable” category was chosen. The positioning was then changed to a hospital-only product, allowing direct price negotiations between hospitals and payers. While these negotiations are highly resource-intensive and time-consuming, national price negotiations and coverage decisions are avoided.


KOL- Views 

To keep up with current market trends, we take KOLs and SME's opinion working in Adeno-Associated Virus Vectors in Gene Therapy domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Adeno-Associated Virus Vectors in Gene Therapy market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs. 


Competitive Intelligence Analysis 

We perform Competitive and Market Intelligence analysis of the Adeno-Associated Virus Vectors in Gene Therapy Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.


Scope of the Report

  • The report covers the descriptive overview of Adeno-Associated Virus Vectors in Gene Therapy, explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
  • Comprehensive insight has been provided into the Adeno-Associated Virus Vectors in Gene Therapy epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for Adeno-Associated Virus Vectors in Gene Therapy are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
  • A detailed review of Adeno-Associated Virus Vectors in Gene Therapy market; historical and forecasted is included in the report, covering drug outreach in the 7MM
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Adeno-Associated Virus Vectors in Gene Therapy market


Report Highlights

  • In the coming years, Adeno-Associated Virus Vectors in Gene Therapy market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence Adeno-Associated Virus Vectors in Gene Therapy R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition       
  • Major players are involved in developing therapies for Adeno-Associated Virus Vectors in Gene Therapy. Launch of emerging therapies will significantly impact the Adeno-Associated Virus Vectors in Gene Therapy market
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Adeno-Associated Virus Vectors in Gene Therapy
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities


Adeno-Associated Virus Vectors in Gene Therapy Report Insights

  • Patient Population
  • Therapeutic Approaches
  • AAV Vector Based Gene Therapy Pipeline Analysis
  • AAV Vector Based Gene Therapy Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies


Adeno-Associated Virus Vectors in Gene Therapy Report Key Strengths

  • 11 Years Forecast
  • 7MM Coverage 
  • Adeno-Associated Virus Vectors in Gene Therapy Epidemiology Segmentation
  • Key Cross Competition 
  • Highly Analyzed Market
  • Drugs Uptake


Adeno-Associated Virus Vectors in Gene Therapy Report Assessment

  • SWOT Analysis
  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Conjoint Analysis
  • Market Attractiveness
  • Market Drivers and Barriers


Key Questions

Market Insights:

  • What was the Adeno-Associated Virus Vectors in Gene Therapy market share (%) distribution in 2017 and how it would look like in 2030?
  • What would be the Adeno-Associated Virus Vectors in Gene Therapy total market size as well as market size by therapies across the 7MM during the forecast period (2017-2030)?
  • What are the key findings pertaining to the market across 7MM and which country will have the largest Adeno-Associated Virus Vectors in Gene Therapy market size during the forecast period (2017-2030)?
  • At what CAGR, the Adeno-Associated Virus Vectors in Gene Therapy market is expected to grow in 7MM during the forecast period (2017-2030)?
  • What would be the Adeno-Associated Virus Vectors in Gene Therapy market outlook across the 7MM during the forecast period (2017-2030)?
  • What would be the Adeno-Associated Virus Vectors in Gene Therapy market growth till 2030, and what will be the resultant market Size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

  • What is the disease risk, burden and unmet needs of the Adeno-Associated Virus Vectors in Gene Therapy?
  • What is the historical Adeno-Associated Virus Vectors in Gene Therapy patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What would be the forecasted patient pool of Adeno-Associated Virus Vectors in Gene Therapy in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Adeno-Associated Virus Vectors in Gene Therapy?
  • Out of all 7MM countries, which country would have the highest prevalent population of Adeno-Associated Virus Vectors in Gene Therapy during the forecast period (2017-2030)?
  • At what CAGR the population is expected to grow in 7MM during the forecast period (2017-2030)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

  • What are the current options for the Adeno-Associated Virus Vectors in Gene Therapy treatment, along with the approved therapy?
  • What are the current treatment guidelines for the treatment of Adeno-Associated Virus Vectors in Gene Therapy in the USA, Europe, and Japan?
  • What are the Adeno-Associated Virus Vectors in Gene Therapy marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing therapies for the treatment of Adeno-Associated Virus Vectors in Gene Therapy?
  • How many therapies are developed by each company for Adeno-Associated Virus Vectors in Gene Therapy treatment?
  • How many are emerging therapies in mid-stage, and late stage of development for Adeno-Associated Virus Vectors in Gene Therapy treatment?
  • What are the key collaborations (Industry - Industry, Industry - Academia), Mergers and acquisitions, licensing activities related to the Adeno-Associated Virus Vectors in Gene Therapy therapies? 
  • What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies? 
  • What are the clinical studies going on for Adeno-Associated Virus Vectors in Gene Therapy and their status?
  • What are the key designations that have been granted for the emerging therapies for Adeno-Associated Virus Vectors in Gene Therapy?
  • What are the global historical and forecasted market of Adeno-Associated Virus Vectors in Gene Therapy?


Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Adeno-Associated Virus Vectors in Gene Therapy market
  • To understand the future market competition in the Adeno-Associated Virus Vectors in Gene Therapy market and Insightful review of the key market drivers and barriers
  • Organize sales and marketing efforts by identifying the best opportunities for Adeno-Associated Virus Vectors in Gene Therapy in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors
  • Organize sales and marketing efforts by identifying the best opportunities for Adeno-Associated Virus Vectors in Gene Therapy market
  • To understand the future market competition in the Adeno-Associated Virus Vectors in Gene Therapy market

1. Key Insights

2. Key Highlights from Report

3. Executive Summary of Adeno-Associated Virus (AAV) Vector Based Gene Therapy

4. SWOT Analysis

5. AAV Vector Based Gene Therapy: Market Share (%) Distribution Overview at a Glance: By Country

6. Epidemiology and Market Methodology

7. Adeno-Associated Virus (AAV) Vector Based Gene Therapy: Background and Overview

7.1. Introduction

7.2. Viral Vectors

7.3. Adeno-Associated Virus (AAV): Biology

7.4. Adeno-associated Virus (AAV): Vector

7.4.1. rAAV transduction pathway

7.5. rAAV gene therapy strategies

7.5.1. Gene replacement

7.5.2. Gene silencing

7.5.3. Gene addition

7.5.4. Gene editing

7.6. AAV serotypes and Tissue tropism

7.7. Pros and Cons of AAV Gene Therapy

7.8. AAV-vector gene therapy in Clinical Trials

7.8.1. Eye diseases

7.8.2. Hemophilia

7.8.3. Neurological diseases

7.8.4. Muscular diseases

7.8.5. Heart diseases

7.8.6. Other disorders

7.9. Considerations for successful AAV-mediated gene therapies

8. Epidemiology and Patient Population

8.1. Epidemiology Key Findings

8.2. Assumptions and Rationale: 7MM

9. Epidemiology Scenario: 7MM

9.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in the 7MM

9.2. Total Indication wise eligible cases in the 7MM

9.3. Indication wise Treated Cases of AAV Gene Therapies in the 7MM

10. The United States Epidemiology

10.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in the United States

10.2. Total Indication wise eligible cases in the United States

10.3. Indication wise Treated Cases of AAV Gene Therapies in the United States

11.  EU-5 Epidemiology

11.1.Germany

11.1.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Germany

11.1.2. Total Indication wise eligible cases in Germany

11.1.3. Indication wise Treated Cases of AAV Gene Therapies in Germany

11.2.France

11.2.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in France

11.2.2. Total Indication wise eligible cases in France

11.2.3. Indication wise Treated Cases of AAV Gene Therapies in France

11.3.Italy

11.3.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Italy

11.3.2. Total Indication wise eligible cases in Italy

11.3.3. Indication wise Treated Cases of AAV Gene Therapies in Italy

11.4.Spain

11.4.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Spain

11.4.2. Total Indication wise eligible cases in Spain

11.4.3. Indication wise Treated Cases of AAV Gene Therapies in Spain

11.5.The United Kingdom

11.5.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in the United Kingdom

11.5.2. Total Indication wise eligible cases in the United Kingdom

11.5.3. Indication wise Treated Cases of AAV Gene Therapies in the United Kingdom

12.  Japan Epidemiology

12.1. Total Prevalent Cases of selected indications for AAV Gene Therapies in Japan

12.2. Total Indication wise eligible cases in Japan

12.3. Indication wise Treated Cases of AAV Gene Therapies in Japan

13.  Unmet Needs

14.  Key Endpoints in AAV Vector Based Gene Therapy Clinical Trials

15.  Marketed Therapy

15.1. Zolgensma (AVXS-101): Novartis (AveXis)

15.1.1. Product Description

15.1.2. Regulatory Milestones

15.1.3. Other Developmental Activities

15.1.4. Pivotal Clinical Trials

15.1.5. Ongoing Current Pipeline Activity

15.1.6. Safety and Efficacy

15.2. Luxturna: Spark Therapeutics/ Novartis

15.2.1. Product Description

15.2.2. Regulatory Milestones

15.2.3. Other Developmental Activities

15.2.4. Pivotal clinical Trials

15.2.5. Ongoing Current Pipeline Activity

16.  Emerging Therapies

16.1.Key Cross

16.2. Achromatopsia Emerging Therapies

16.2.1. AAV-CNGA3: MeiraGTx UK II Ltd/Janssen Research & Development

16.2.2. AAV-CNGB3: MeiraGTx UK II Ltd

16.2.3. AGTC-402 (ACHM CNGA3): Applied Genetic Technologies Corporation

16.2.4. ACHM B3 (rAAV2tYF-PR1.7-hCNGB3): Applied Genetic Technologies Corporation (AGTC)

16.3. Age-related Macular Degeneration Emerging Therapy

16.3.1. ADVM-022 (AAV.7m8-aflibercept): Adverum Biotechnologies, Inc.

16.4. Batten Disease Emerging Therapies

16.4.1. AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics

16.4.2. AT-GTX-501 (scAAV9.CB.CLN6): Amicus Therapeutics

16.5. Charcot-Marie-Tooth Neuropathy Emerging Therapies

16.5.1. scAAV1.tMCK.NTF3: Sarepta Therapeutics/ Nationwide Children's Hospital

16.6. Choroiderma Emerging Therapies

16.6.1. Timrepigene emparvovec (BIIB111, AAV2-REP1): NightstaRx Ltd, a Biogen Company

16.6.2. SPK 7001 (AAV2-hCHM): Spark Therapeutics

16.6.3. 4D-110: 4D Molecular Therapeutics| Roche Pharma AG

16.7. Diabetic Macular Edema Emerging Therapies

16.7.1. ADVM-022 (AAV.7m8-aflibercept): Adverum Biotechnologies, Inc.

16.8. Duchenne Muscular Dystrophy Emerging Therapies

16.8.1. PF-06939926: Pfizer

16.8.2. Delandistrogene moxeparvovec (SRP-9001): Roche/Sarepta Therapeutics

16.8.3. GALGT2 (rAAVrh74.MCK.GALGT2): Sarepta Therapeutics/ Nationwide Children's Hospital

16.8.4. scAAV9.U7.ACCA: Audentes Therapeutics

16.8.5. SGT-001: Solid Biosciences

16.9. Fabry Disease Emerging Drugs

16.9.1. 4D-310: 4D Molecular Therapeutics

16.9.2. ST-920: Sangamo Therapeutics

16.9.3. FLT190: Freeline Therapeutics

16.10. Gangliosidosis Emerging Therapies

16.10.1. AXO-AAV-GM1: Sio Gene Therapies

16.10.2. AXO-AAV-GM2: Sio Gene Therapies

16.11. Glycogen Storage Disease Emerging Therapies

16.11.1. DTX401 (AAV8G6PC): Ultragenyx Pharmaceutical Inc

16.12. Hemophilia A Emerging Therapies

16.12.1. Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical

16.12.2. Giroctocogene fitelparvovec (SB-525 or PF-07055480): Pfizer/Sangamo Therapeutics

16.12.3. RG6357 (SPK-8011): Roche (Spark Therapeutics)

16.12.4. RG6358 (SPK-8016): Roche (Spark Therapeutics)

16.12.5. TAK-754 (BAX 888/SHP654): Takeda (Shire)

16.12.6. BAY2599023 (DTX201 AAV FVIII): Bayer/Ultragenyx Pharmaceutical

16.13. Hemophilia B Emerging Therapies

16.13.1. Fidanacogene elaparvovec (SPK-9001/ PF-06838435): Pfizer (Spark Therapeutics)

16.13.2. Etranacogene dezaparvovec (AMT-061): UniQure

16.13.3. Verbrinacogene setparvovec (FLT-180a): Freeline Therapeutics

16.13.4. AskBio009 (BAX 335, AAV8.sc-TTR-FIXR338Lopt): Asklepios BioPharmaceutical

16.14. Huntington Disease Emerging Therapy

16.14.1. AMT-130: UniQure Biopharma B.V.

16.15. Leber Hereditary Optic Neuropathy Emerging Therapies

16.15.1. LUMEVOQ (GS010; lenadogene nolparvovec): GenSight Biologics

16.16. Limb-girdle muscular dystrophy Emerging Therapies

16.16.1. SRP-9004 (MYO 102/ scAAVrh74.tMCK.hSGCA): Sarepta Therapeutics

16.17. Mucopolysaccharidosis II/Hunter Syndrome Emerging Therapies

16.17.1. RGX-121: Regenxbio

16.17.2. SB-913: Sangamo Therapeutics

16.18. Mucopolysaccharidosis Type I Emerging Therapies

16.18.1. RGX-111: REGENXBIO

16.18.2. SB-318: Sangamo Therapeutics

16.19. Mucopolysaccharidosis Type III Emerging Therapies

16.19.1. ABO-101 (rAAV9.CMV.hNAGLU): Abeona Therapeutics

16.19.2. LYS-SAF302 (Olenasufligene Relduparvovec): Lysogene and Sarepta Therapeutics

16.19.3. EGT-101 (AAV9-CAG-coh-SGSH): Esteve

16.19.4. ABO-102 (scAAV9.U1a.hSGSH): Abeona Therapeutics

16.20. Myotubular Myopathy Emerging Therapy

16.20.1. AT132: Audentes Therapeutics

16.21. OTC Deficiency/Urea Cycle Disorder Emerging Therapy

16.21.1. DTX301 (scAAV8OTC): Ultragenyx Pharmaceutical

16.22. Parkinson ’s disease Emerging Therapies

16.22.1. NBIb-1817 (VY-AADC): Neurocrine Biosciences/ Voyager Therapeutics

16.22.2. NLX P101 (AAV-GAD): Meira Gtx

16.23. Phenylketonuria (PKU) Emerging Therapies

16.23.1. HMI-102: Homology Medicines

16.23.2. BMN 307: BioMarin Pharmaceutical

16.24. Pompe Disease Emerging Therapies

16.24.1. SPK-3006: Spark Therapeutics (acquired by Roche)

16.24.2. AT845: Astellas Therapeutics

16.24.3. ACTUS-101: Actus Therapeutics

16.25. Retinitis Pigmentosa Emerging Therapies

16.25.1. AAV8-RPGR/BIIB112: Nightstar Therapeutics/Biogen

16.25.2. GS030: GenSight Biologics

16.25.3. AAV-RPGR: MeiraGTx

16.25.4. AAV-RPE65: MeiraGTx

16.25.5. 4D-125: 4D Molecular Therapeutics/Roche

16.25.6. ATGC-501 (rAAV2tYF-GRK1-RPGR): Applied Genetic Technologies Corporation

16.25.7. HORA-PDE6B: Horama

16.26. Retinoschisis Emerging Therapy

16.26.1. rAAV-hRS1: Applied Genetic Technologies Corporation

16.27. Wilson’s Disease Emerging Therapies

16.27.1. VTX-801: Vivet Therapeutics and Pfizer

17.  AAV Vector Based Gene Therapy: Seven Major Market Analysis

17.1.Key Findings

17.2.Market Outlook

17.3.7MM Market Size

17.3.1. Indication wise Market size of AAV-vector-based gene therapy in the 7MM

17.3.2. Total Market size of AAV-vector-based gene therapy by Therapies in the 7MM

17.4.The United States Market Size

17.4.1. Indication wise Market size of AAV-vector-based gene therapy in the United States

17.4.2. Total Market size of AAV-vector-based gene therapy by Therapies in the United States

17.5.EU-5 Market Size

17.6.Germany

17.6.1. Indication wise Market size of AAV-vector-based gene therapy in Germany

17.6.2. Total Market size of AAV-vector-based gene therapy by Therapies in Germany

17.7.France

17.7.1. Indication wise Market size of AAV-vector-based gene therapy in France

17.7.2. Total Market size of AAV-vector-based gene therapy by Therapies in France

17.8.Italy

17.8.1. Indication wise Market size of AAV-vector-based gene therapy in Italy

17.8.2. Total Market size of AAV-vector-based gene therapy by Therapies in Italy

17.9.Spain

17.9.1. Indication wise Market size of AAV-vector-based gene therapy in Spain

17.9.2. Total Market size of AAV-vector-based gene therapy by Therapies in Spain

17.10. The United Kingdom

17.10.1. Indication wise Market size of AAV-vector-based gene therapy in the United Kingdom

17.10.2. Total Market size of AAV-vector-based gene therapy by Therapies in the United Kingdom

17.11. Japan

17.11.1. Indication wise Market size of AAV-vector-based gene therapy in Japan

17.11.2. Total Market size of AAV-vector-based gene therapy by Therapies in Japan

18.  Market Access and Reimbursement of AAV Vector Based Gene Therapy

19.  Market Drivers

20.  Market Barriers

21.  Appendix

21.1. Bibliography

21.2. Report Methodology

22.  DelveInsight Capabilities

23.  Disclaimer

24.  About DelveInsight

Table 1: Summary of AAV Vectors in Gene Therapy, Epidemiology, and Key Events (2019–2030)

Table 2: List of sources Used for Epidemiology Evaluation for shortlisted Indication

Table 3: Total Prevalent Cases of selected indications for AAV Gene Therapies in the 7MM (2017–2030)

Table 4: Total Indication wise eligible cases in the 7MM (2017–2030)

Table 5: Total Indication wise Treated Cases of AAV Gene Therapies in the 7MM (2017–2030)

Table 6: Total Prevalent Cases of selected indications for AAV Gene Therapies in the United States (2017–2030)

Table 7: Total Indication wise eligible cases in the United States (2017–2030)

Table 8: Total Indication wise Treated Cases of AAV Gene Therapies in the United States (2017–2030)

Table 9: Total Prevalent Cases of selected indications for AAV Gene Therapies in Germany (2017–2030)

Table 10: Total Indication wise eligible cases in Germany (2017–2030)

Table 11: Total Indication wise Treated Cases of AAV Gene Therapies in Germany (2017–2030)

Table 12: Total Prevalent Cases of selected indications for AAV Gene Therapies in France (2017–2030)

Table 13: Total Indication wise eligible cases in France (2017–2030)

Table 14: Total Indication wise Treated Cases of AAV Gene Therapies in France (2017–2030)

Table 15: Total Prevalent Cases of selected indications for AAV Gene Therapies in Italy (2017–2030)

Table 16: Total Indication wise eligible cases in Italy (2017–2030)

Table 17: Total Indication wise Treated Cases of AAV Gene Therapies in Italy (2017–2030)

Table 18: Total Prevalent Cases of selected indications for AAV Gene Therapies in Spain (2017–2030)

Table 19: Total Indication wise eligible cases in Spain (2017–2030)

Table 20: Total Indication wise Treated Cases of AAV Gene Therapies in Spain (2017–2030)

Table 21: Total Prevalent Cases of selected indications for AAV Gene Therapies in the United Kingdom (2017–2030)

Table 22: Total Indication wise eligible cases in the United Kingdom (2017–2030)

Table 23: Total Indication wise Treated Cases of AAV Gene Therapies in the United Kingdom (2017–2030)

Table 24: Total Prevalent Cases of selected indications for AAV Gene Therapies in Japan (2017–2030)

Table 25: Total Indication wise eligible cases in Japan (2017–2030)

Table 26: Total Indication wise Treated Cases of AAV Gene Therapies in Japan (2017–2030)

Table 27: Zolgensma (AVXS-101); Clinical Trial Description, 2020

Table 28: Luxturna; Clinical Trial Description, 2020

Table 29: Key Cross of Emerging Therapies

Table 30: AAV- CNGA3; Clinical Trial Description, 2020

Table 31: AAV-CNGB3; Clinical Trial Description, 2020

Table 32: AGTC-402, Clinical Trial Description, 2020

Table 33: rAAV2tYF-PR1.7-hCNGB3, Clinical Trial Description, 2020

Table 34: ADVM-022 (AAV.7m8-aflibercept); Clinical Trial Description, 2020

Table 35: AT-GTX-502, Clinical Trial Description, 2020

Table 36: AT-GTX-501, Clinical Trial Description, 2020

Table 37: scAAV1.tMCK.NTF3, Clinical Trial Description, 2020

Table 38: Timrepigene emparvovec (BIIB111, AAV2-REP1); Clinical Trial Description, 2020

Table 39: SPK 7001 (AAV2-hCHM); Clinical Trial Description, 2020

Table 40: 4D-110; Clinical Trial Description, 2020

Table 41: ADVM-022 (AAV.7m8-aflibercept); Clinical Trial Description, 2020

Table 42: PF-06939926, Clinical Trial Description, 2020

Table 43: SRP-9001; Clinical Trial Description, 2020

Table 44: GALGT2 (rAAVrh74.MCK.GALGT2); Clinical Trial Description, 2020

Table 45: scAAV9.U7.ACCA; Clinical Trial Description, 2020

Table 46: SGT-001; Clinical Trial Description, 2020

Table 47: 4D-310; Clinical Trial Description, 2020

Table 48: ST-920; Clinical Trial Description, 2020

Table 49: FLT 190; Clinical Trial Description, 2020

Table 49: AXO-AAV-GM1; Clinical Trial Description, 2020

Table 49: DTX401 (AAV8G6PC); Clinical Trial Description, 2020

Table 52: Valoctocogene Roxaparvovec (BMN 270); Clinical Trial Description, 2020

Table 53: Giroctocogene fitelparvovec (SB-525 or PF-07055480); Clinical Trial Description, 2020

Table 54: RG6357 (SPK-8011); Clinical Trial Description, 2020

Table 55: RG6358 (SPK-8016); Clinical Trial Description, 2020

Table 56: TAK754 (SHP654 or BAX 888); Clinical Trial Description, 2020

Table 57: BAY2599023 (DTX201 AAV FVIII); Clinical Trial Description, 2020

Table 58: Fidanacogene elaparvovec (SPK-9001/ PF-06838435); Clinical Trial Description, 2020

Table 59: Etranacogene dezaparvovec (AMT-061); Clinical Trial Description, 2020

Table 60: Verbrinacogene setparvovec (FLT-180a); Clinical Trial Description, 2020

Table 61: AskBio009 (BAX 335, AAV8.sc-TTR-FIXR338Lopt); Clinical Trial Description, 2020

Table 62: AMT-130; Clinical Trial Description, 2020

Table 63: LUMEVOQ (GS010); Clinical Trial Description, 2020

Table 64: scAAVrh74.tMCK.hSGCA, Clinical Trial Description, 2020

Table 65: RGX-121; Clinical Trial Description, 2020

Table 66: SB-913; Clinical Trial Description, 2020

Table 67: RGX-111, Clinical Trial Description, 2020

Table 68: SB-318, Clinical Trial Description, 2020

Table 69: ABO-101, Clinical Trial Description, 2020

Table 70: LYS-SAF302, Clinical Trial Description, 2020

Table 71: EGT-101, Clinical Trial Description, 2020

Table 72: ABO-102, Clinical Trial Description, 2020

Table 73: AT132, Clinical Trial Description, 2020

Table 74: DTX301 (scAAV8OTC); Clinical Trial Description, 2020

Table 75: VY-AADC; Clinical Trial Description, 2020

Table 76: AAV-GAD; Clinical Trial Description, 2020

Table 77: HMI-102, Clinical Trial Description, 2020

Table 78: BMN 307, Clinical Trial Description, 2020

Table 79: SPK-3006; Clinical Trial Description, 2020

Table 80: AT845; Clinical Trial Description, 2020

Table 81: ACTUS-101; Clinical Trial Description, 2020

Table 82: AAV8-RPGR/BIIB112; Clinical Trial Description, 2020

Table 83: GS030, Clinical Trial Description, 2020

Table 84: AAV8-RPGR/BIIB112; Clinical Trial Description, 2020

Table 86: AAV RPE65; Clinical Trial Description, 2020

Table 85: 4D-125, Clinical Trial Description, 2020

Table 87: rAAV2tYF-GRK1-RPGR; Clinical Trial Description, 2020

Table 88: HORA-PDE6B; Clinical Trial Description, 2020

Table 89: rAAV-hRS1; Clinical Trial Description, 2020

Table 90: VTX-801, Clinical Trial Description, 2020

Table 91: List of Forecasted Therapies

Table 92: 7MM Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 93: 7MM Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 94: The United States Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 95: The United States Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 96: Germany Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 97: Germany Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 98: France Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 99: France Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 100: Italy Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 101: Italy Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 102: Spain Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 103: Spain Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 104: The United Kingdom Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 105: The United Kingdom Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 106: Japan Indication wise Market Size of AAV-vector-based gene therapy, in USD Million (2017–2030)

Table 107: Japan Market Size of AAV-vector-based gene therapy by Therapies, in USD Million (2017–2030)

Table 108: Reimbursement Scenario of Glybera

Figure 1: AAV Vector Gene Therapy SWOT Analysis

Figure 2: Gene Therapy – Therapeutic Gene Delivery via Viral Vector

Figure 3: Examples of viral vectors applied for gene therapy

Figure 4: Timeline of AAV Gene Therapies

Figure 5: AAV-2 Genome Map

Figure 6: AAV vector in gene therapy

Figure 7: Gene therapy strategies

Figure 8: Tissue tropisms by AAV serotypes

Figure 9: Spinal muscular atrophy gene therapy

Figure 10: Neurodegenerative diseases

Figure 11: Total Prevalent Cases of selected indications for AAV Gene Therapies in the 7MM (2017–2030)

Figure 12: Total Indication wise eligible cases in the 7MM (2017–2030)

Figure 13: Indication wise Treated Cases of AAV Gene Therapies in the 7MM (2017–2030)

Figure 14: Total Prevalent Cases of selected indications for AAV Gene Therapies in the United States (2017–2030)

Figure 15: Total Indication wise eligible cases in the United States (2017–2030)

Figure 16: Indication wise Treated Cases of AAV Gene Therapies in the United States (2017–2030)

Figure 17: Total Prevalent Cases of selected indications for AAV Gene Therapies in Germany (2017–2030)

Figure 18: Total Indication wise eligible cases in Germany (2017–2030)

Figure 19: Indication wise Treated Cases of AAV Gene Therapies in Germany (2017–2030)

Figure 20: Total Prevalent Cases of selected indications for AAV Gene Therapies in France (2017–2030)

Figure 21: Total Indication wise eligible cases in France (2017–2030)

Figure 22: Indication wise Treated Cases of AAV Gene Therapies in France (2017–2030)

Figure 23: Total Prevalent Cases of selected indications for AAV Gene Therapies in Italy (2017–2030)

Figure 24: Total Indication wise eligible cases in Italy (2017–2030)

Figure 25: Indication wise Treated Cases of AAV Gene Therapies in Italy (2017–2030)

Figure 26: Total Prevalent Cases of selected indications for AAV Gene Therapies in Spain (2017–2030)

Figure 27: Total Indication wise eligible cases in Spain (2017–2030)

Figure 28: Indication wise Treated Cases of AAV Gene Therapies in Spain (2017–2030)

Figure 29: Total Prevalent Cases of selected indications for AAV Gene Therapies in the United Kingdom (2017–2030)

Figure 30: Total Indication wise eligible cases in the United Kingdom (2017–2030)

Figure 31: Indication wise Treated Cases of AAV Gene Therapies in the United Kingdom (2017–2030)

Figure 32: Total Prevalent Cases of selected indications for AAV Gene Therapies in Japan (2017–2030)

Figure 33: Total Indication wise eligible cases in Japan (2017–2030)

Figure 34: Indication wise Treated Cases of AAV Gene Therapies in Japan (2017–2030)

Figure 35: Unmet Needs of AVV-vector gene therapy

Figure 36: scAAV1.tMCK.NTF3 gene therapy

Figure 37: Sangamo Therapeutics' in vivo genome editing approach

Figure 38: Indication wise Market Size of AAV-vector-based gene therapy in the 7MM, in USD Million (2017–2030)

Figure 39: Market Size of AAV-vector-based gene therapy in the 7MM by Therapy, in USD Million (2017–2030)

Figure 40: Indication wise Market Size of AAV-vector-based gene therapy in the United States, in USD Million (2017–2030)

Figure 41: Market Size of AAV-vector-based gene therapy in the United States by Therapy, in USD Million (2017–2030)

Figure 42: Indication wise Market Size of AAV-vector-based gene therapy in Germany, in USD Million (2017–2030)

Figure 43: Market Size of AAV-vector-based gene therapy in Germany by Therapy, in USD Million (2017–2030)

Figure 44: Indication wise Market Size of AAV-vector-based gene therapy in France, in USD Million (2017–2030)

Figure 45: Market Size of AAV-vector-based gene therapy in France by Therapy, in USD Million (2017–2030)

Figure 46: Indication wise Market Size of AAV-vector-based gene therapy in Italy, in USD Million (2017–2030)

Figure 47: Market Size of AAV-vector-based gene therapy in Italy by Therapy, in USD Million (2017–2030)

Figure 48: Indication wise Market Size of AAV-vector-based gene therapy in Spain, in USD Million (2017–2030)

Figure 49: Market Size of AAV-vector-based gene therapy in Spain by Therapy, in USD Million (2017–2030)

Figure 50: Indication wise Market Size of AAV-vector-based gene therapy in the United Kingdom, in USD Million (2017–2030)

Figure 51: Market Size of AAV-vector-based gene therapy in the United Kingdom by Therapy, in USD Million (2017–2030)

Figure 52: Indication wise Market Size of AAV-vector-based gene therapy in Japan, in USD Million (2017–2030)

Figure 53: Market Size of AAV-vector-based gene therapy in Japan by Therapy, in USD Million (2017–2030)

Figure 54: Spark Therapeutics innovative payment programs for Luxturna

Figure 55: Market Drivers of AAV-Vector gene therapy

Figure 56: Market Barriers of AVV-vector gene therapy

  • Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical: Phase III
  • PF-06939926: Pfizer: Phase III
  • Fidanacogene elaparvovec: Pfizer (initiated by Spark Therapeutics): Phase III
  • AMT-061: uniQure/CSL Behring: Phase III
  • Timrepigene emparvovec (BIIB111): NightstaRx Ltd, a Biogen Company: Phase III
  • Giroctocogene fitelparvovec (SB-525): Pfizer (previously Sangamo Biosciences): Phase III
  • BIIB112 (AAV8-RPGR): NightstaRx Ltd, a Biogen Company: Phase II/III
  • NLX P101 (AAV-GAD): MeiraGTx: Phase II
  • VY-AADC (NBIb-1817/ AAV2-hAADC): Neurocrine Biosciences/ Voyager Therapeutics: Phase II
  • SPK-8011: Roche (previously Spark Therapeutics)/Pfizer: Phase I/II
  • ST-920: Sangamo Therapeutics: Phase I/II
  • FLT190: Freeline Therapeutics: Phase I/II
  • SPK-3006 (AAV-sec-GAA): Spark Therapeutics: Phase I/II
  • ACTUS-101: Asklepios Biopharmaceutical (Actus Therapeutics): Phase I/II
  • AT845: Audentes Therapeutics: Phase I/II
  • SRP-9001: Roche/Sarepta Therapeutics: Phase I/IIa
  • HORA-PDE6B: Horama S.A.: Phase I/II
  • AAV-RPGR (AAV2/5-RPGR): MeiraGTx UK II Ltd: Phase I/II
  • RGX-121: RegenxBio: Phase I/II
  • SB-913: Sangamo Therapeutics: Phase I/II
  • AT-GTX-501 (scAAV9.CB.CLN6): Amicus Therapeutics: Phase I/IIa
  • AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics: Phase I/IIa
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