(New York, USA) DelveInsight predicts that the Adeno-Associated Virus Vector-Based Gene Therapy market is set to gain traction due to extensive efforts by global companies in developing new AAV-gene therapies to address various diseases. Leading pharmaceutical and biotech firms like Biomarin Pharmaceutical, Roche (Spark Therapeutics), Sangamo, Pfizer, and others are actively researching AAV vector-based gene therapy for diverse indications, including Hemophilia A and B, MPS, and more. This surge in research and development indicates a growing interest in utilizing AAV vector-based gene therapy as a promising approach to treat a wide spectrum of medical conditions.

 

DelveInsight’s Adeno-Associated Virus (AAV) Vector-Based Gene Therapy Market report offers a clear picture of the market position of the AAV vectors in the Gene therapy, emerging pipeline therapies, AAV vector-based Gene Therapy market share occupied by individual diseases, current and forecasted market share in the 7MM (the US, EU5 (the UK, Germany, France, Italy and Spain) and Japan) for the study period 2019-32.

The report focuses on the factors driving and limiting the current AAV Vector-Based Gene Therapy market, as well as identifying unmet medical needs that create opportunities for key players to explore the market’s underlying potential.

 

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Key Highlights of AAV Vector-Based Gene Therapy Market:

• The Adeno-Associated Viral vector is the safest and effective vehicle that is able to maintain long-term gene and protein expression following a single injection of the vector.
• Currently, two classes of recombinant AAVs (rAAVs) are 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.
• The first AAV-based gene therapy drug, Glybera, was approved by the European Medicines Agency (EMA) in 2012 but later in 2017, it was withdrawn from the market mainly due to commercial failure.
• The present AAV-based Gene therapy market owns two FDA-approved AAV-based gene therapies namely, Luxturna, approved in 2017 for a rare inherited retinal dystrophy, and Zolgensma, approved in 2019 for spinal muscular atrophy.
• Leading AAV Vector-Based Gene Therapy companies are Biomarin Pharmaceutical, Sarepta Therapeutics, Roche (Spark Therapeutics), Sangamo, Pfizer, NightstaRx, Freeline Therapeutics, Horama S.A, MeiraGTx, RegenxBio, Asklepios Biopharmaceutical, Audentes Therapeutics, and others.
• Key indications in which the most clinical trials are ongoing include Hemophilia A, Hemophilia B, Choroideremia, Retinitis pigmentosa, Fabry disease, Pompe disease, Hunter Syndrome, Parkinson’s disease, Batten Disease, Duchenne Muscular Dystrophy (DMD), and Spinal Muscular Atrophy (SMA).
• Among all the indication, Hemophilia A is expected to contribute maximally to the growth of the market size in terms of revenue by 2032 owing to a maximum number of gene therapies with mostly in late phase of development, which are expected to enter into the market, the precedence of existing high treatment cost and also expected high cost for these upcoming gene 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 2032 in the 7MM from the haemophilia A owing to visibility around the clinical profile and also early mover advantage.

 

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Adeno-Associated Virus (AAV) Overview

Adeno-associated virus hails from the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae. They are small viruses (25-nm) with a genome of single-stranded DNA (~4.7 kb) that can either be the plus (sense) or minus (anti-sense) strand. AAV is replication-defective and depends on a helper virus for effective and productive replication in mammalian cells. However, for choosing AAV as a gene delivery vector depends on:

• which cell/tissue types are being targeted;
• the safety profile associated with the delivered gene;
• the choice of systemic versus local delivery; and
• the use of tissue-specific or constitutively active promoters.

 

Adeno-associated virus (AAV) Vector-Based Gene Therapy

With advances in bioengineering and genetics, the horizon of the medical approaches giving rise to novel treatment options, such as Gene therapy has also widened. Gene therapy has emerged out as a promising treatment approach for a number of inherited disorders, certain types of cancer, and certain viral infections. The delivery of gene therapy involves “vectors” which can be either viral or non-viral vectors. Out of the several viral vectors, Adeno-associated virus (AAV) vectors are currently among the most frequently used, safest and effective viral vectors for gene therapy delivery. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases.

Key Indications Covered:

• Haematology
• Hemophilia A
• Hemophilia B
• Ophthalmology
• Choroideremia
• Retinitis pigmentosa
• Lysosomal Storage Disorders
• Fabry disease
• Pompe disease
• Hunter Syndrome
• Neurology
• Parkinson’s disease
• Neurological Disorders
• Batten Disease
• Musculoskeletal
• Duchenne Muscular Dystrophy (DMD)
• Spinal Muscular Atrophy (SMA)

 

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Epidemiology Segmentation

In the year 2020, the total prevalent cases of selected indications in which AAV Gene Therapies were administered were estimated to be 2,863,103 in the 7MM. DelveInsight’s Adeno-Associated Virus Vector-Based Gene Therapy Market Report provides historical as well as forecasted epidemiological analysis for the study period 2019-32 for the 7MM segmented into:

• Total prevalent cases of selected indications (Hemophilia A, Hemophilia B, Fabry disease, Pompe disease, Choroideremia, Retinitis Pigmentosa, Hunter syndrome, Batten disease, Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Parkinson’s disease) in the 7MM [2019-2032]
• Total Indication wise eligible cases in the 7MM [2019–2032]
• Indication wise Treated Cases of AAV Gene Therapies in the 7MM [2019–2032]

 

Adeno-Associated Virus Vector-Based Gene Therapy Market

Currently, the AAV Vector-Based Gene Therapy Market has only two FDA-approved therapies. One is Luxturna by Spark Therapeutics, which treats patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Luxturna is the first FDA-approved gene therapy for a genetic disease, the sole pharmacologic treatment for an inherited retinal disease, and the first AAV vector gene therapy approved in the United States.

 

The second approved therapy is Zolgensma by Novartis/AveXis, an AAV-delivered gene therapy indicated for pediatric patients under 2 years of age with SMA and bi-allelic mutations in the SMN1 gene. Zolgensma is currently being investigated in global Phase III trials, including STR1VE and SPR1NT, to evaluate its efficacy in different patient populations with SMA.

 

Before Luxturna and Zolgensma emerged as prominent therapies in the AAV Vector-Based Gene Therapy Market, there was another candidate called Glybera. Glybera received approval from the EMA to treat an ultra-rare, hereditary lipoprotein lipase deficiency (LPLD) and became the first launched gene therapy. However, due to the high costs associated with maintenance and development, the company decided not to renew its market authorization after it expired in October 2017, highlighting the challenges in sustaining gene therapies despite their potential to offer definitive cures.

 

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Key Pipeline Adeno-Associated Virus Vector-Based Gene Therapies:

• 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/II
• AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics: Phase I/II

 

There is not a shred of doubt that clinical successes in AAV-mediated gene replacement have helped Adeno-Associated Viruses to get recognized as an ideal therapeutic vector for delivery of gene therapies. With two AAV vector-based gene therapies having won regulatory approval, their popularity as the predominant vectors that deliver genes of interest to target tissues with improved specificity, efficiency, and safety in the Gene therapy market further fuelled. However, the production and formulation of AAV products require specified conditions so as to ensure good stability and yield, and even after caution, some of its processing methods (filtration or lyophilization) may lead to aggregation or loss of AAV titer. Further, storing the AAV products can also prove to be quite challenging.

Conclusively, Gene therapy has proved to be an ingenious tool in the healthcare sector and with dramatic advancements being made in the domain, its potential is unfolding at a rapid pace. Thus, it is not wrong to say that Gene therapy is set to emerge as a novel therapeutic option.

 

Scope of the Adeno-Associated Virus Vector-Based Gene Therapies report:

• Geography Coverage: The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), Japan.
• Study Period: 3-year historical and 11-year forecasted analysis (2019-2032).
• Adeno-Associated Virus Vectors in Gene Therapy Market Segmentation: By Selected Indication Patient Pool, Market Size by Therapies, Market Size by Indication, Market Size by Country
• Key Players Involved: BioMarin Pharmaceutical, Sangamo Therapeutics, Amicus Therapeutics, Roche, Pfizer, NightstaRx, MeiraGTx UK II Ltd, Horama S.A, Sarepta Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Asklepios Biopharmaceutical, Spark Therapeutics, and several others.
• Analysis: Comparative and conjoint analysis of Adeno-Associated Virus Vectors in Gene Therapy Emerging therapies
• Tools used: SWOT analysis, Porter’s Five Forces, PESTLE analysis, BCG Matrix analysis methods.
• Case Studies
• KOL’s Views
• Analyst’s Views

 

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Table of Content

1.  Key Insights

2. Executive Summary of Adeno-Associated Virus Vectors in Gene Therapy

3. Competitive Intelligence Analysis for AAV Vector-Based Gene Therapy Market

4. AAV Vector-Based Gene Therapy Market SWOT Analysis

5. Adeno-Associated Virus Vector-Based Gene Therapy Market Overview at a Glance

6. Adeno-Associated Virus Vector-Based Gene Therapy: Disease Background and Overview

7. Adeno-Associated Virus Vectors in Gene Therapy Patient Journey

8. Adeno-Associated Virus Vectors in Gene Therapy Epidemiology and Patient Population

9. AAV Vector-Based Gene Therapy Market: Treatment Algorithm, Current Treatment, and Medical Practices

10. AAV Vector-Based Gene Therapy Market Unmet Needs

11. Key Endpoints of Adeno-Associated Virus Vectors in Gene Therapy Treatment

12. Marketed Products in AAV Vector-Based Gene Therapy Market

13. Emerging Adeno-Associated Virus Vector-Based Gene Therapies

14. Adeno-Associated Virus Vectors in Gene Therapy: Seven Major Market Analysis

15. Attribute analysis

16. Adeno-Associated Virus Vector-Based Gene Therapy Market Outlook: 7MM

17. Access and Reimbursement Overview of Adeno-Associated Virus Vector-Based Gene Therapy Market

18. Adeno-Associated Virus Vectors in Gene Therapy KOL Views

19. Adeno-Associated Virus Vector-Based Gene Therapy Market Drivers

20. Adeno-Associated Virus Vector-Based Gene Therapy Market Barriers

21. Appendix

22. DelveInsight Capabilities

23. Disclaimer

24. About DelveInsight

 

About DelveInsight

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