Adeno-Associated Virus Vectors in Gene Therapy Therapeutics Market Size is estimated to grow with CAGR by 2034

Adeno-Associated Virus Vectors in Gene Therapy Therapeutics Market

LAS VEGAS, NV, UNITED STATES, January 23, 2025 /EINPresswire.com/ -- 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.

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Key Takeaways from the Adeno-Associated Virus Vectors in Gene Therapy Market Report
• In January 2025:- MeiraGTx UK II Ltd:- An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/5-hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
• In January 2025:- CSL Behring:- The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10^13 gc/kg.
• 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.
• The leading Adeno-Associated Virus Vectors in Gene Therapy Companies such as Biomarin Pharmaceutical, Sarepta Therapeutics, Roche (Spark Therapeutics), Sangamo, Pfizer, NightstaRx, Freeline Therapeutics, Horama S.A, MeiraGTx, RegenxBio, Asklepios Biopharmaceutical, Audentes Therapeutics, and others.
• Promising Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies such as AAV - CNGB3, SB-525 (PF-07055480), BMN 307, GC301, and others.

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Adeno-Associated Virus Vector in Gene Therapy Epidemiology
• 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

Download the report to understand which factors are driving Adeno-Associated Virus Vectors in Gene Therapy epidemiology trends @ Adeno-Associated Virus Vectors in Gene Therapy Prevalence

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.

• 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-β-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.

• 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.

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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.

Adeno-Associated Virus Vectors in Gene Therapy Companies
Biomarin Pharmaceutical, Sarepta Therapeutics, Roche (Spark Therapeutics), Sangamo, Pfizer, NightstaRx, Freeline Therapeutics, Horama S.A, MeiraGTx, RegenxBio, Asklepios Biopharmaceutical, Audentes Therapeutics, and others.

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Scope of the Adeno-Associated Virus Vectors in Gene Therapy Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Adeno-Associated Virus Vectors in Gene Therapy Companies- Biomarin Pharmaceutical, Sarepta Therapeutics, Roche (Spark Therapeutics), Sangamo, Pfizer, NightstaRx, Freeline Therapeutics, Horama S.A, MeiraGTx, RegenxBio, Asklepios Biopharmaceutical, Audentes Therapeutics, and others.
• Adeno-Associated Virus Vectors in Gene Therapy Therapies- AAV - CNGB3, SB-525 (PF-07055480), BMN 307, GC301, and others.
• Adeno-Associated Virus Vectors in Gene Therapy Market Dynamics: Adeno-Associated Virus Vectors in Gene Therapy Market Drivers and Barriers
• Adeno-Associated Virus Vectors in Gene Therapy Market Access and Reimbursement, Unmet Needs and Future Perspectives

Table of Content
1. Key Insights
2. Executive Summary of Adeno-Associated Virus Vectors in Gene Therapy
3. Competitive Intelligence Analysis for Adeno-Associated Virus Vectors in Gene Therapy
4. Adeno-Associated Virus Vectors in Gene Therapy: Market Overview at a Glance
5. Adeno-Associated Virus Vectors in Gene Therapy: Disease Background and Overview
6. Patient Journey
7. Adeno-Associated Virus Vectors in Gene Therapy Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Adeno-Associated Virus Vectors in Gene Therapy Unmet Needs
10. Key Endpoints of Adeno-Associated Virus Vectors in Gene Therapy Treatment
11. Adeno-Associated Virus Vectors in Gene Therapy Marketed Products
12. Adeno-Associated Virus Vectors in Gene Therapy Emerging Therapies
13. Adeno-Associated Virus Vectors in Gene Therapy: Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Market Outlook
16. Access and Reimbursement Overview of Adeno-Associated Virus Vectors in Gene Therapy
17. KOL Views
18. Market Drivers
19. Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight

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