Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Therapeutics, Clinical Trials, Companies and Treatment Drugs 2024 (Updated)

DelveInsight's, "Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Pipeline Insight, 2024" report provides comprehensive insights about 180+ companies and 200+ pipeline drugs in Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline landscape. It covers the pipeline drug profiles, including clinical 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.

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Key Takeaways from the Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline Report
• In December 2024:- CSL Behring:- Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B.
• DelveInsight's Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline report depicts a robust space with 180+ active players working to develop 200+ pipeline therapies for Adeno-Associated Virus (AAV) Vectors in Gene Therapy treatment.
• The leading Adeno-Associated Virus (AAV) 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.

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AAV vectors in Gene Therapy Emerging Drugs Profile

• BBM-H901: Belief BioMed
BBM-H901 is indicated for prophylactic treatment of bleeding in adults with hemophilia B. BBM-H901 is designed to restore the production of factor IX (FIX) the blood-clotting protein that is faulty or missing in people with hemophilia B by delivering a functional copy of the F9 gene to liver cells, which are the main producers of clotting factors in the body. Gene therapy components are delivered to liver cells by a modified, harmless adeno-associated virus (AAV) that works as a vehicle. The drug is currently being registered for the treatment of hemophilia B.

• GS010: 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 (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. Currently the drug is in Phase III stage of its development for the treatment of Leber Hereditary Optic Neuropathy.

• AAV-CNGA3: Johnson & Johnson Innovative Medicine/ MeiraGTx
AAV-CNGA3, a gene therapy treatment designed to restore cone function, is delivered to the cone receptors at the back of the eye via subretinal injection. It was designed with a synthetic promoter associated with strong gene expression to account for the larger amount of protein needed to restore cone function in achromatopsia (ACHM) patients with a CNGA3 gene mutation. AAV-CNGA3 was granted Orphan Drug designation by the FDA and EMA, as well as Rare Pediatric Disease designation and Fast Track designation by the FDA, for the treatment of ACHM caused by mutations in the CNGA3 gene. According to company's pipeline the drug is being evaluated in a Phase II trial for the treatment of achromatopsia.

• PBGM01: Passage Bio
PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual β-gal enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a next-generation AAVhu68 capsid administered through the cisterna magna to deliver a functional GLB1 gene encoding β-gal to the brain and peripheral tissues. By reducing the accumulation of GM1 gangliosides, PBGM01 has the potential to reverse neuronal toxicity, thereby restoring developmental potential. In preclinical models, PBGM01 has demonstrated broad brain distribution and high levels of expression of the β-gal enzyme in both the CNS and critical peripheral organs, suggesting potential treatment for both the CNS and peripheral manifestations of GM1. The drug is currently being evaluated in Phase I/II stage of development to treat Gangliosidosis.

• IVB102: InnoVec Biotherapeutics
IVB102 is a drug for the treatment of X-linked retinoschisis (XLRS) developed based on a new vector independently developed by Innovecon. Preclinical data showed that the visual electrophysiological signals of model animals treated with IVB102 can be restored to a level comparable to that of wild-type animals, with the potential of being 'best in class'. The positive feedback received from the FDA on IVB102 proves the FDA's recognition of IVB102 and also reflects the urgent need for drugs from regulators and patients. Innovecon is rapidly advancing the clinical research of IVB102. The drug is currently being evaluated in a Phase I trial for the treatment of XLRS.

Learn more about Adeno-Associated Virus (AAV) Vectors in Gene Therapy Drugs opportunities in our groundbreaking Adeno-Associated Virus (AAV) Vectors in Gene Therapy Research and development projects @ Adeno-Associated Virus (AAV) Vectors in Gene Therapy Unmet Needs- 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

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

Adeno-Associated Vector pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Adeno-Associated Vector Products have been categorized under various Molecule types such as
• Monoclonal antibody
• Small molecule
• Peptide

Discover the latest advancements in Adeno-Associated Virus (AAV) Vectors in Gene Therapy Treatment by visiting our website. Stay informed about how we're transforming the future of disease @ Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market Drivers and Barriers, and Future Perspectives- 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 (AAV) Vectors in Gene Therapy Pipeline Report
• Coverage- Global
• Adeno-Associated Virus (AAV) Vectors in Gene Therapy Companies- 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.
• 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.
• Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Adeno-Associated Virus (AAV) Vectors in Gene Therapy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

For a detailed overview of our latest research findings and future plans, read the full details of Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline on our website @ Adeno-Associated Virus (AAV) Vectors in Gene Therapy Emerging Drugs and Companies- 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

Table of Content
1. Introduction
2. Executive Summary
3. Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Adeno-Associated Virus (AAV) Vectors in Gene Therapy- DelveInsight's Analytical Perspective
7. Late Stage Products (Registration)
8. BBM-H901: Belief BioMed
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. AAV-CNGA3: Johnson & Johnson Innovative Medicine/ MeiraGTx
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. IVB102: InnoVec Biotherapeutics
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug Name: Company Name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Companies
21. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Products
22. Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Unmet Needs
23. Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Market Drivers and Barriers
24. Adeno-Associated Virus (AAV) Vectors in Gene Therapy- Future Perspectives and Conclusion
25. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Analyst Views
26. Adeno-Associated Virus (AAV) Vectors in Gene Therapy Key Companies
27. Appendix

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