Adeno Associated Virus Vectors in Gene Therapy Pipeline Insight and Clinical Trial Analysis | 70+ Companies and 235+ Drugs

DelveInsight's, "Adeno Associated Virus Vectors in Gene Therapy Pipeline Insight 2023" report provides comprehensive insights about 70+ companies and 235+ pipeline drugs in AAV vectors in gene therapy pipeline landscape. It covers the Adeno Associated Virus Vectors in Gene Therapy pipeline drug profiles, including Adeno Associated Virus Vectors in Gene Therapy clinical trial and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

In the Adeno Associated Virus Vectors in Gene Therapy Pipeline Report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Adeno Associated Virus Vectors in Gene Therapy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Key takeaways from the Adeno Associated Virus 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 include 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 include AAV - CNGB3, AAV2/5-RPGR, AAV OPTIRPE65, BMN 307, AAV RPE65, AGTC-402, and others.

• The Adeno Associated Virus Vectors in Gene Therapy Companies and academics are working to assess challenges and seek opportunities that could influence AAV vectors in gene therapy R&D. The Adeno Associated Virus Vectors in Gene Therapy pipeline therapies under development are focused on novel approaches to treat/improve AAV vectors in gene therapy.

Explore more information on the latest breakthroughs in the Adeno Associated Virus Vectors in Gene Therapy treatment landscape of the report, visit here Adeno Associated Virus Vectors in Gene Therapy Pipeline Outlook @ https://www.delveinsight.com/sample-request/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Recent Developmental Activities in the Adeno Associated Virus Vectors in Gene Therapy Treatment Landscape

• 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.

• 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 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.

Adeno Associated Virus Vectors in Gene Therapy Overview

Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs, and harnessing revolutionary biotechnologies have contributed extensively to the growth of the gene therapy field.

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Adeno Associated Virus Vectors in Gene Therapy Emerging Drugs Profile

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.

Adeno Associated Virus Aav Vectors In Gene Therapy Therapeutics Assessment

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

For further information, refer to the detailed Adeno Associated Virus Vectors In Gene Therapy pipeline report, visit here for Adeno Associated Virus Vectors In Gene Therapy Treatment Landscape @ https://www.delveinsight.com/sample-request/adeno-associated-virus-aav-vectors-in-gene-therapy-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Adeno Associated Virus Vectors In Gene Therapy Pipeline Report

• Coverage- Global

• 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 Pipeline Therapies- AAV - CNGB3, AAV2/5-RPGR, AAV OPTIRPE65, BMN 307, AAV RPE65, AGTC-402, and others.

• Adeno Associated Virus Vectors in Gene Therapy Segmentation: Phases, Product Type, Molecule Type, Mechanism of Action, Route of Administration

Table of Content

1. Introduction
2. Executive Summary
3. AAV vectors in gene therapy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Late Stage Products (Preregistration)
7. Valoctocogene roxaparvovec: BioMarin Pharmaceutical
8. Drug profiles in the detailed report…..
9. Late Stage Products (Phase III)
10. DTX401: Ultragenyx Pharmaceutical
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II)
13. GT 005: Gyroscope Therapeutics
14. Drug profiles in the detailed report…..
15. Early stage products (Phase I/II)
16. AXO-AAV-GM1: Sio Gene Therapies
17. Drug profiles in the detailed report…..
18. Early Stage Products (Phase I)
19. HMI-203: Homology Medicine
20. Drug profiles in the detailed report…..
21. Preclinical stage products
22. AXV101: Axovia Therapeutics
23. Drug profiles in the detailed report…..
24. Discovery stage products
25. DINA-002: DiNAQOR
26. Drug profiles in the detailed report…..
27. Inactive Products
28. AAV vectors in gene therapy Key Companies
29. AAV vectors in gene therapy Key Products
30. AAV vectors in gene therapy- Unmet Needs
31. AAV vectors in gene therapy- Market Drivers and Barriers
32. AAV vectors in gene therapy- Future Perspectives and Conclusion
33. AAV vectors in gene therapy Analyst Views
34. AAV vectors in gene therapy Key Companies
35. Appendix

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