Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Outlook Report 2024 (Updated)

DelveInsight has released its latest report titled "AAV Vectors in Gene Therapy Pipeline Insight 2024" offering extensive insights into over 70 companies and more than 235 pipeline drugs within the AAV vectors gene therapy landscape. This comprehensive report includes detailed profiles of pipeline drugs across clinical and nonclinical stages, alongside thorough assessments based on product type, development stage, route of administration, and molecule type. Additionally, it features an analysis of inactive pipeline products within this sector.

Key Takeaways from the Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Report

* DelveInsight's Adeno-Associated Virus Vectors in Gene Therapy pipeline report depicts a robust space with 70+ active players working to develop 235+ pipeline therapies for Adeno-Associated Virus Vectors in Gene Therapy treatment.
* The leading Adeno-Associated Virus Vectors in Gene Therapy companies such as BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark therapeuics (Roche), Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline therapeutics, Astellas Pharma, Aspa therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus therapeutics, 4D Molecular therapeutics, Taysha Gene Therapies, Atsena Therapeutics, BioMarin Pharmaceutical, Abeona Therapeutics, REGENXBIO, uniQure, Taysha Gene Therapies, Asklepios BioPharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Forge Biologics, Sangamo therapeutics, and others.
* Promising Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies such as Valoctocogene Roxaparvovec, AAV - CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, and others.
* June 2024:- UniQure Biopharma B.V.- A Phase Ib/II Randomized, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington's Disease. This is the second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon continued demonstration of safety of AMT-130 administration.

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy Overview

Adeno-Associated Virus (AAV) vectors have emerged as a pivotal tool in gene therapy due to their unique properties that make them highly effective and safe vehicles for delivering therapeutic genes into target cells. AAV vectors are derived from non-pathogenic viruses that do not cause disease in humans, making them ideal candidates for therapeutic applications. Their ability to infect both dividing and non-dividing cells, coupled with their minimal immunogenicity and long-lasting gene expression, further enhances their utility.

In gene therapy, AAV vectors are engineered to carry therapeutic genes that can correct genetic defects, modulate gene expression, or deliver specific proteins to treat a wide range of diseases, including genetic disorders, neurodegenerative diseases, and certain types of cancer. Ongoing research and clinical trials continue to explore and refine AAV vector technology, aiming to optimize delivery efficiency, target specificity, and safety profiles to unlock their full potential in revolutionizing medical treatments.

AAV vectors in Gene Therapy Emerging Drugs Profile

* GS010: Gensight Biologics

LUMEVOQ Registered (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 (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. "LUMEVOQ" was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

* Valoctocogene roxaparvovec: BioMarin Pharmaceutical

Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A. It is currently in preregistration stage of development. In June 2021, BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA). In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase III GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

* DTX401: Ultragenyx Pharmaceutical

DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase- under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase- activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I.

* AAV5-RPGR: MeiraGTx

AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase III clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15.

* Timrepigene emparvovec: Biogen

Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase III STAR study.

* AMT 061: UniQure

AMT-061 (etranacogene dezaparvovec) is an experimental gene therapy that uniQure is developing to treat hemophilia B. AMT-061 uses a modified and harmless adeno-associated virus 5 (AAV5) to deliver a highly functional copy of the F9 gene, called FIX-Padua, to patients' cells. The FIX-Padua gene version was shown to result in FIX clotting activity eight times greater than that associated with the standard F9 gene. As such, the one-time therapy - administrated directly into the bloodstream - is expected to increase FIX levels, helping to prevent and control bleeds. If approved, the therapy will be marketed globally by CSL Behring, which closed a commercialization and licensing agreement with uniQure in May 2021. UniQure and CSL Behring expect to file a regulatory application to the U.S. Food and Drug Administration (FDA) in early 2022 seeking AMT-061's approval for hemophilia B. Research and Development.

* RGX-314: REGENXBIO

RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector Registered from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye. According to the company's pipeline, the drug is currently in the Phase III stage of development.

* SPK-8011: Spark Therapeutics

Investigational SPK-8011, a novel bio-engineered adeno-associated viral (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. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the US, while the European Commission has granted orphan designation to SPK-8011.

* NFS-01: Neurophth

Investigational NR082, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND4 mutations. It is currently being evaluated in Phase II/III clinical trial.

* GT 005: Gyroscope Therapeutics

GT005 is designed as an AAV2-based one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. Complement overactivation has been strongly correlated with the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production could dampen the system's overactivity and reduce inflammation, with the goal of preserving a person's eyesight. Gyroscope is also evaluating GT005 in two Phase II clinical trials.

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Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapeutics Assessment

There are approx. 70+ key companies developing therapies for AAV vectors in gene therapy. The companies that have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include BioMarin Pharmaceutical.

DelveInsight's Adeno-Associated Vector Report covers around 235+ products under different phases of clinical development like

* Late stage products (Phase III)
* Mid-stage products (Phase II)
* Early-stage product (Phase I) along with the details of
* Pre-clinical and Discovery stage candidates
* Discontinued & Inactive candidates
* Route of Administration

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Adeno-Associated Vector pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.

* Intracerebral
* Intracerebroventricular
* Intracisternal
* Intramuscular
* Intraocular
* Intrapancreatic
* Intrastriatal
* Intrathecal
* Intravenous
* Intravitreous
* Introvitinreous
* Oral
* Parenteral
* Retinal
* Intra-arterial
* Molecule Type

Adeno-Associated Vector Products have been categorized under various Molecule types such as

* Monoclonal Antibody
* Peptides
* Polymer
* Small molecule
* Gene therapy
* Product Type

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Scope of the Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Report

* Coverage- Global
* Adeno-Associated Virus Vectors in Gene Therapy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Adeno-Associated Virus Vectors in Gene Therapy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
* Adeno-Associated Virus Vectors in Gene Therapy Companies- BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, Ultragenyx Pharmaceutical, MeiraGTx, Pfizer, Biogen, uniQure, Pfizer, Ultragenyx Pharmaceutical, REGENXBIO, Biogen, Spark therapeuics (Roche), Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, Freeline therapeutics, Astellas Pharma, Aspa therapeutics, Adrenas Therapeutics, ESTEVE, Sio Gene Therapies, Amicus therapeutics, 4D Molecular therapeutics, Taysha Gene Therapies, Atsena Therapeutics, BioMarin Pharmaceutical, Abeona Therapeutics, and others.
* Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapies- Valoctocogene Roxaparvovec, AAV - CNGB3, SB-525 (PF-07055480), AAV2/5-RPGR, BMN 307, GC301, rAAV-Olig001-ASPA, and others.

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Table of Content

* Introduction
* Executive Summary
* AAV vectors in gene therapy: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Late Stage Products (Preregistration)
* Valoctocogene roxaparvovec: BioMarin Pharmaceutical
* Drug profiles in the detailed report.....
* Late Stage Products (Phase III)
* DTX401: Ultragenyx Pharmaceutical
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* GT 005: Gyroscope Therapeutics
* Drug profiles in the detailed report.....
* Early stage products (Phase I/II)
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* HMI-203: Homology Medicine
* Drug profiles in the detailed report.....
* Preclinical stage products
* AXV101: Axovia Therapeutics
* Drug profiles in the detailed report.....
* Discovery stage products
* DINA-002: DiNAQOR
* Drug profiles in the detailed report.....
* Inactive Products
* AAV vectors in gene therapy Key Companies
* AAV vectors in gene therapy Key Products
* AAV vectors in gene therapy- Unmet Needs
* AAV vectors in gene therapy- Market Drivers and Barriers
* AAV vectors in gene therapy- Future Perspectives and Conclusion
* AAV vectors in gene therapy Analyst Views
* AAV vectors in gene therapy Key Companies
* Appendix

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