Adeno Associated Virus Vectors In Gene Therapy Market to Grow Positively at a Paltry CAGR During the Study Period (2020-2034) | Companies- Neurophth biological tech, Asklepios Biopharmaceutical, MeiraGTx, Nanoscope Therapeutics, Spark Therapeutics, Lysoge

DelveInsight's "Adeno Associated Virus Vectors In Gene Therapy - Market Insight, Epidemiology and Market Forecast - 2034" 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 2020 to 2034 segmented by seven major markets. The report also offers current Adeno Associated Virus Vectors In Gene Therapy therapy algorithms, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the Adeno Associated Virus Vectors In Gene Therapy market.
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Key highlights from the adeno associated virus vectors in gene therapy market report:
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in Gene Therapy drug, GLYBERA, was approved by the European Medicines Agency (EMA) in 2012 but later in 2020, it was withdrawn from the market mainly due to commercial failure.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.

Adeno Associated Virus Vectors In Gene Therapy Overview
Recent advancements in genetic analysis, which allow for rapid and cost-effective sequencing of genomes, have facilitated the identification of genes underlying various diseases. Monogenic diseases, caused by mutations in a single gene, are particularly promising targets for gene therapy interventions. However, the potential of gene therapy has been hindered by challenges in safely delivering therapeutic nucleic acids into cells.

Viral vectors, such as AAV (adeno-associated virus), initially showed promise but have raised concerns over immunogenicity and safety limitations, tempering their widespread use. Alternative delivery methods, like nanoparticles, have also encountered hurdles.

Despite these challenges, rAAV (recombinant AAV), a genetically engineered form of AAV that lacks viral DNA, has emerged as a promising gene therapy vehicle. Engineered to efficiently transport therapeutic DNA into target cells, rAAV avoids integration into the host genome, minimizing the risk of unintended genetic alterations. Instead, the DNA delivered by rAAV persists in the cell nucleus as episomal concatemers, ensuring a sustained therapeutic effect without genomic integration.

Ongoing research aims to enhance the delivery efficiency and safety of rAAV. This vector holds significant potential for treating a wide range of genetic diseases, with ongoing clinical trials providing valuable insights for future therapeutic strategies.
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Adeno associated virus vectors in gene therapy epidemiology insights:
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 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-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.

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.

Promising Therapies in the Adeno Associated Virus Vectors In Gene Therapy Pipeline
NFS-01
AAV2/8LSPhGAA
AAV CNGA3
MCO-010
SPK-8011
LYS-SAF302
And others
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Leading Companies Working in the Adeno Associated Virus Vectors In Gene Therapy Market
Neurophth biological tech
Asklepios Biopharmaceutical
MeiraGTx
Nanoscope Therapeutics
Spark Therapeutics
Lysogene
And others
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Adeno-Associated Virus Vector in Gene Therapy Market Outlook
Adeno-Associated Virus (AAV) vectors in gene therapy have provided a unique opportunity to treat and even cure degenerative diseases, offering hope to millions 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 revolutionized the treatment paradigm for genetic diseases by providing lasting therapeutic effects with a single intervention. They hold significant potential for addressing various eye diseases. However, individuals considering this treatment should be aware of certain challenges and crucial factors. Despite the recent successes in rare disease therapy approvals providing momentum for AAV therapy research and funding, several limitations make this a difficult and 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 under a licensing agreement covering the development, registration, and commercialization rights outside the United States. The US FDA's advisory panel had commended LUXTURNA's approval in October 2017.

In recent years, the treatment landscape for many diseases has rapidly evolved. Companies are now developing gene and cell therapies that will play a promising role in the future, especially for the treatment of rare genetic diseases. Defining appropriate candidates for given gene and cell therapies will require the enrollment and long-term follow-up of a sufficient number of study subjects to provide clarity about their safety and efficacy.

To summarize, the outlook for gene and cell therapies is promising. Various clinical trials have shown positive results in terms of safety and efficacy, encouraging further investigation into multiple indications. The current scenario also anticipates a positive shift in the market for the forecast period.

Scope of the Adeno Associated Virus Vectors In Gene Therapy Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), Japan]
Key Adeno Associated Virus Vectors In Gene Therapy Companies: Neurophth biological tech, Asklepios Biopharmaceutical, MeiraGTx, Nanoscope Therapeutics, Spark Therapeutics, Lysogene, and others
Key Adeno Associated Virus Vectors In Gene Therapy Pipeline Therapies: NFS-01, AAV2/8LSPhGAA, AAV CNGA3, MCO-010, SPK-8011, LYS-SAF302, and others
Therapeutic Assessment: Adeno Associated Virus Vectors In Gene Therapy current marketed and emerging therapies
Adeno Associated Virus Vectors In Gene Therapy Market Dynamics: Adeno Associated Virus Vectors In Gene Therapy market drivers and barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
Unmet Needs, KOL's views, Analyst's views, Adeno Associated Virus Vectors In Gene Therapy Market Access and Reimbursement
Table of Contents
1. Adeno Associated Virus Vectors In Gene Therapy Market Key Insights
2. Adeno Associated Virus Vectors In Gene Therapy Market Report Introduction
3. Adeno Associated Virus Vectors In Gene Therapy Market Overview at a Glance
4. Adeno Associated Virus Vectors In Gene Therapy Market Executive Summary
5. Disease Background and Overview
6. Adeno Associated Virus Vectors In Gene Therapy Treatment and Management
7. Adeno Associated Virus Vectors In Gene Therapy Epidemiology and Patient Population
8. Patient Journey
9. Adeno Associated Virus Vectors In Gene Therapy Emerging Drugs
10. 7MM Adeno Associated Virus Vectors In Gene Therapy Market Analysis
11. Adeno Associated Virus Vectors In Gene Therapy Market Outlook
12. Potential of Current and Emerging Therapies
13. KOL Views
14. Adeno Associated Virus Vectors In Gene Therapy Market Drivers
15. Adeno Associated Virus Vectors In Gene Therapy Market Barriers
16. Unmet Needs
17. SWOT Analysis
18. Appendix
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
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