AAV-Vectors in gene therapy market are expected to grow at a significantly high rate and are mainly driven by an increase in approval of a growing number of gene therapies and readily adoption on approval, the ability to treat a broad array of conditions, an increase in a number of cases, expected one-time dosing approach and curative treatment options.
LAS VEGAS, Jan. 16, 2024 /PRNewswire/ -- DelveInsight's AAV Vectors in Gene Therapy Market Insights report includes a comprehensive understanding of current treatment practices, emerging AAV vectors in gene therapy, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Key Takeaways from the AAV Vectors in Gene Therapy Market Report
- According to DelveInsight's analysis, the market size for AAV vectors in gene therapy across the 7MM is expected to grow with a significant CAGR by 2032.
- DelveInsight's analysis reveals that the prevalent cases of selected indications for AAV gene therapies in the 7MM were reported as ~3 million in 2020.
- Leading AAV vectors in gene therapy companies such as MeiraGTx UK II Ltd, Janssen Research & Development, Adverum Biotechnologies, Inc., Amicus Therapeutics, Audentes Therapeutics, Solid Biosciences, Sio Gene Therapies, Takeda (Shire), Bayer, Pfizer, Freeline Therapeutics, Asklepios BioPharmaceutical, UniQure Biopharma B.V., GenSight Biologics, Regenxbio, Sangamo Therapeutics, Lysogene, Sarepta Therapeutics, Esteve, Abeona Therapeutics, Audentes Therapeutics, Ultragenyx Pharmaceutical, Homology Medicines, BioMarin Pharmaceutical, Spark Therapeutics, Astellas Therapeutics, Actus Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Roche, Coave Therapeutics, Applied Genetic Technologies Corporation, Vivet Therapeutics, and others are developing novel AAV vectors in gene therapy that can be available in the AAV vectors in gene therapy market in the coming years.
- The promising AAV vectors in gene therapies in the pipeline include AAV-CNGA3, AAV-CNGB3, AGTC-402 (ACHM CNGA3), ACHM B3 (rAAV2tYF-PR1.7-hCNGB3), ADVM-022 (AAV.7m8-aflibercept), AT-GTX-502 (scAAV9.P546.CLN3), AT-GTX-501 (scAAV9.CB.CLN6), scAAV1.tMCK.NTF3, SPK 7001 (AAV2-hCHM), 4D-110, PF-06939926, Delandistrogene moxeparvovec (SRP-9001), scAAV9.U7.ACCA, SGT-001, 4D-310, Isaralgagene civaparvovec, FLT190, AXO-AAV-GM1, AXO-AAV-GM2, DTX401 (AAV8G6PC), Valoctocogene Roxaparvovec (BMN 270), Giroctocogene fitelparvovec (SB-525 or PF-07055480), RG6357 (SPK-8011), RG6358 (SPK-8016), TAK-754 (BAX 888/SHP654), BAY2599023 (DTX201 AAV FVIII), Fidanacogene elaparvovec (SPK-9001/ PF-06838435), Verbrinacogene setparvovec (FLT-180a), AskBio009 (BAX 335, AAV8.sc-TTR-FIXR338Lopt), AMT-130, LUMEVOQ (GS010; lenadogene nolparvovec), SRP-9004 (MYO 102/ scAAVrh74.tMCK.hSGCA), RGX-121, RGX-111, SB-318, ABO-101 (rAAV9.CMV.hNAGLU), LYS-SAF302 (Olenasufligene Relduparvovec), EGT-101 (AAV9-CAG-coh-SGSH), ABO-102 (scAAV9.U1a.hSGSH), AT132, DTX301 (scAAV8OTC), NLX P101 (AAV-GAD), HMI-102, BMN 307, SPK-3006, AT845, ACTUS-101, GS030, AAV-RPGR, AAV-RPE65, 4D-125, ATGC-501 (rAAV2tYF-GRK1-RPGR), CTx-PDE6b, rAAV-hRS1, VTX-801, and others.
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AAV Vectors in Gene Therapy Overview
Gene therapy shows great promise as a potential treatment for various diseases, including inherited disorders, certain types of cancer, and specific viral infections. The treatment utilizes carriers known as "vectors," which can be viral or non-viral. Among the viral vectors employed for delivering target genes, the Adeno-associated viral (AAV) vector stands out as a particularly safe and efficient vehicle. Notably, AAV vectors have demonstrated the ability to sustain prolonged gene and protein expression after a single injection. Consequently, AAV vectors are currently at the forefront of viral vectors used for gene delivery, offering significant potential for treating a range of human diseases.
The selection of a specific AAV for gene transfer is significantly influenced by several crucial factors. These include (1) the targeted cell/tissue types; (2) the safety profile associated with the delivered gene; (3) the decision between systemic and local delivery; and (4) the utilization of tissue-specific or constitutively active promoters. Currently, two classes of recombinant AAVs (rAAVs) are employed: single-stranded AAV (ssAAV) and self-complementary AAV (scAAV). Strategies for rAAV gene therapy encompass gene replacement, gene silencing, gene addition, and gene editing.
AAV Vectors in Gene Therapy Epidemiology Segmentation
The AAV vectors in gene therapy epidemiology section provide insights into the historical and current AAV vectors in gene therapy 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 AAV vectors in gene therapy market report proffer epidemiological analysis for the study period 2019-2032 in the 7MM segmented into:
- Total Prevalent Cases of Selected Indications
- Total Indication-wise Eligible Cases
- Indication-wise Treated Cases of AAV Gene Therapies
AAV Vectors in Gene Therapy Treatment Market
The FDA has approved three marketing applications for gene therapy products based on AAV, namely Luxturna, Zolgensma, and most recently, Hemgenix. In 2019, the overall market value of AAV-vector-based gene therapy was approximately USD 428 million. Projections suggest that the largest market share for AAV vector-based gene therapy is anticipated to come from the United States.
In 2012, the EMA granted initial approval to Glybera for the treatment of hereditary lipoprotein lipase deficiency (LPLD), and it was administered through a series of intramuscular injections. Nevertheless, this therapeutic approach was later withdrawn from the market for various reasons. UniQure and Chiesi Farmaceutici collaborated to sell and promote Glybera in Europe. The drug received orphan drug designation for addressing homozygous LPL deficiency, which includes a subgroup of patients with familial chylomicronemia syndrome (FCS). However, the marketing authorization for Glybera lapsed in October 2020.
Recently, in November 2022, the FDA approved Hemgenix (etranacogene dezaparvovec), a gene therapy utilizing an adeno-associated virus vector. This approval is for the treatment of adults diagnosed with Hemophilia B, a congenital condition characterized by Factor IX deficiency. Specifically, Hemgenix is intended for individuals currently undergoing Factor IX prophylaxis therapy, those with a history of life-threatening hemorrhages, or those experiencing recurrent and severe spontaneous bleeding episodes. Administered as a one-time treatment through a single intravenous infusion, Hemgenix involves the delivery of a clotting Factor IX gene via a viral vector.
Discover more about AAV vectors in gene therapy in development @ AAV Vectors in Gene Therapy Clinical Trials
AAV Vectors in Gene Therapy Pipeline Therapies and Key Companies
- AAV-CNGA3: MeiraGTx UK II Ltd/Janssen Research & Development
- AAV-CNGB3: MeiraGTx UK II Ltd
- AGTC-402 (ACHM CNGA3): Applied Genetic Technologies Corporation
- ACHM B3 (rAAV2tYF-PR1.7-hCNGB3): Applied Genetic Technologies Corporation (AGTC)
- ADVM-022 (AAV.7m8-aflibercept): Adverum Biotechnologies, Inc.
- AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics
- AT-GTX-501 (scAAV9.CB.CLN6): Amicus Therapeutics
- scAAV1.tMCK.NTF3: Sarepta Therapeutics/ Nationwide Children's Hospital
- SPK 7001 (AAV2-hCHM): Spark Therapeutics
- 4D-110: 4D Molecular Therapeutics| Roche Pharma AG
- PF-06939926: Pfizer
- Delandistrogene moxeparvovec (SRP-9001): Roche/Sarepta Therapeutics
- scAAV9.U7.ACCA: Audentes Therapeutics
- SGT-001: Solid Biosciences
- 4D-310: 4D Molecular Therapeutics
- Isaralgagene civaparvovec: Sangamo Therapeutics
- FLT190: Freeline Therapeutics
- AXO-AAV-GM1: Sio Gene Therapies
- AXO-AAV-GM2: Sio Gene Therapies
- DTX401 (AAV8G6PC): Ultragenyx Pharmaceutical Inc
- Valoctocogene Roxaparvovec (BMN 270): BioMarin Pharmaceutical
- Giroctocogene fitelparvovec (SB-525 or PF-07055480): Pfizer/Sangamo Therapeutics
- RG6357 (SPK-8011): Roche (Spark Therapeutics)
- RG6358 (SPK-8016): Roche (Spark Therapeutics)
- TAK-754 (BAX 888/SHP654): Takeda (Shire)
- BAY2599023 (DTX201 AAV FVIII): Bayer/Ultragenyx Pharmaceutical
- Fidanacogene elaparvovec (SPK-9001/ PF-06838435): Pfizer (Spark Therapeutics)
- Verbrinacogene setparvovec (FLT-180a): Freeline Therapeutics
- AskBio009 (BAX 335, AAV8.sc-TTR-FIXR338Lopt): Asklepios BioPharmaceutical
- AMT-130: UniQure Biopharma B.V.
- LUMEVOQ (GS010; lenadogene nolparvovec): GenSight Biologics
- SRP-9004 (MYO 102/ scAAVrh74.tMCK.hSGCA): Sarepta Therapeutics
- RGX-121: Regenxbio
- RGX-111: REGENXBIO
- SB-318: Sangamo Therapeutics
- ABO-101 (rAAV9.CMV.hNAGLU): Abeona Therapeutics
- LYS-SAF302 (Olenasufligene Relduparvovec): Lysogene and Sarepta Therapeutics
- EGT-101 (AAV9-CAG-coh-SGSH): Esteve
- ABO-102 (scAAV9.U1a.hSGSH): Abeona Therapeutics
- AT132: Audentes Therapeutics
- DTX301 (scAAV8OTC): Ultragenyx Pharmaceutical
- NLX P101 (AAV-GAD): Meira Gtx
- HMI-102: Homology Medicines
- BMN 307: BioMarin Pharmaceutical
- SPK-3006: Spark Therapeutics (acquired by Roche)
- AT845: Astellas Therapeutics
- ACTUS-101: Actus Therapeutics
- GS030: GenSight Biologics
- AAV-RPGR: MeiraGTx
- AAV-RPE65: MeiraGTx
- 4D-125: 4D Molecular Therapeutics/Roche
- ATGC-501 (rAAV2tYF-GRK1-RPGR): Applied Genetic Technologies Corporation
- CTx-PDE6b: Coave Therapeutics
- rAAV-hRS1: Applied Genetic Technologies Corporation
- VTX-801: Vivet Therapeutics and Pfizer
Learn more about the AAV vectors for gene therapy in clinical trials @ AAV Vectors for Gene Therapy
AAV Vectors in Gene Therapy Market Dynamics
The dynamics of the AAV vectors in gene therapy market are expected to change in the coming years. AAVs have emerged as the predominant vectors for delivering genes of interest to target tissues with improved specificity, efficiency, and safety, leading to clinical successes in AAV-mediated gene replacement. This success has helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States.
Furthermore, many potential AAV vectors in gene therapies are being investigated, and it is safe to predict that the treatment space will significantly impact the AAV vectors in gene therapy 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 AAV vectors in gene therapy market in the 7MM.
However several factors may impede the growth of the AAV vectors in gene therapy market. The formulation of AAV products necessitates meticulously chosen conditions to guarantee optimal stability and yield. Commonly employed processing methods for biologics, such as filtration, may induce aggregation or a decline in AAV titer. Additionally, the limited understanding of immune responses to AAV vectors and the safety implications associated with administering high vector doses to humans restrict their use in treatment.
Moreover, AAV vectors in gene therapy treatment pose a significant economic burden and disrupt patients' overall well-being and QOL. Furthermore, the AAV vectors in gene therapy market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the AAV vectors in gene therapy market growth.
AAV Vectors in Gene Therapy Market Report Metrics | Details |
Study Period | 2019-2032 |
Coverage | 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. |
Key AAV Vectors in Gene Therapy Companies | MeiraGTx UK II Ltd, Janssen Research & Development, Adverum Biotechnologies, Inc., Amicus Therapeutics, Audentes Therapeutics, Solid Biosciences, Sio Gene Therapies, Takeda (Shire), Bayer, Pfizer, Freeline Therapeutics, Asklepios BioPharmaceutical, UniQure Biopharma B.V., GenSight Biologics, Regenxbio, Sangamo Therapeutics, Lysogene, Sarepta Therapeutics, Esteve, Abeona Therapeutics, Audentes Therapeutics, Ultragenyx Pharmaceutical, Homology Medicines, BioMarin Pharmaceutical, Spark Therapeutics, Astellas Therapeutics, Actus Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Roche, Coave Therapeutics, Applied Genetic Technologies Corporation, Vivet Therapeutics, and others |
Key Pipeline AAV Vectors in Gene Therapies | AAV-CNGA3, AAV-CNGB3, AGTC-402 (ACHM CNGA3), ACHM B3 (rAAV2tYF-PR1.7-hCNGB3), ADVM-022 (AAV.7m8-aflibercept), AT-GTX-502 (scAAV9.P546.CLN3), AT-GTX-501 (scAAV9.CB.CLN6), scAAV1.tMCK.NTF3, SPK 7001 (AAV2-hCHM), 4D-110, PF-06939926, Delandistrogene moxeparvovec (SRP-9001), scAAV9.U7.ACCA, SGT-001, 4D-310, Isaralgagene civaparvovec, FLT190, AXO-AAV-GM1, AXO-AAV-GM2, DTX401 (AAV8G6PC), Valoctocogene Roxaparvovec (BMN 270), Giroctocogene fitelparvovec (SB-525 or PF-07055480), RG6357 (SPK-8011), RG6358 (SPK-8016), TAK-754 (BAX 888/SHP654), BAY2599023 (DTX201 AAV FVIII), Fidanacogene elaparvovec (SPK-9001/ PF-06838435), Verbrinacogene setparvovec (FLT-180a), AskBio009 (BAX 335, AAV8.sc-TTR-FIXR338Lopt), AMT-130, LUMEVOQ (GS010; lenadogene nolparvovec), SRP-9004 (MYO 102/ scAAVrh74.tMCK.hSGCA), RGX-121, RGX-111, SB-318, ABO-101 (rAAV9.CMV.hNAGLU), LYS-SAF302 (Olenasufligene Relduparvovec), EGT-101 (AAV9-CAG-coh-SGSH), ABO-102 (scAAV9.U1a.hSGSH), AT132, DTX301 (scAAV8OTC), NLX P101 (AAV-GAD), HMI-102, BMN 307, SPK-3006, AT845, ACTUS-101, GS030, AAV-RPGR, AAV-RPE65, 4D-125, ATGC-501 (rAAV2tYF-GRK1-RPGR), CTx-PDE6b, rAAV-hRS1, VTX-801, and others |
Scope of the AAV Vectors in Gene Therapy Market Report
- Therapeutic Assessment: AAV Vectors in Gene Therapy current marketed and emerging therapies
- AAV Vectors in Gene Therapy Market Dynamics: Key Market Forecast Assumptions of Emerging AAV Vectors in Gene Therapy Drugs and Market Outlook
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL's views, Analyst's views, AAV Vectors in Gene Therapy Market Access and Reimbursement
To know more about AAV vectors in gene therapy, visit @ AAV Vector Technology
Table of Contents
1. | AAV Vectors in Gene Therapy Key Insights |
2. | AAV Vectors in Gene Therapy Report Introduction |
3. | AAV Vectors in Gene Therapy Overview at a Glance |
4. | AAV Vectors in Gene Therapy Executive Summary |
5 | AAV Vectors in Gene Therapy Key Events |
6 | Epidemiology and Market Forecast Methodology |
6. | Disease Background and Overview |
7. | AAV Vectors in Gene Therapy Treatment and Management |
8. | AAV Vectors in Gene Therapy Guidelines |
9. | AAV Vectors in Gene Therapy Epidemiology and Patient Population |
10. | Patient Journey |
11. | Key Endpoints in AAV Vectors in Gene Therapy |
12. | AAV Vectors in Gene Therapy Marketed Drugs |
13. | AAV Vectors in Gene Therapy Emerging Drugs |
14. | 7MM AAV Vectors in Gene Therapy 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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