AAV Vectors in Gene Therapy Pipeline Experiences Momentum: DelveInsight Estimates a Diverse Pipeline Comprising 70+ Companies Working in the Domain
New York, USA, April 27, 2023 (GLOBE NEWSWIRE) — AAV Vectors in Gene Therapy Pipeline Experiences Momentum: DelveInsight Estimates a Diverse Pipeline Comprising 70+ Companies Working in the Domain
AAV-vector is currently being tested to cure diseases that were previously thought to be nearly impossible to treat. The best example is the involvement of AVV-vector gene therapy in treating neurological disorders like Spinal Muscular Atrophy, Huntington’s disease, Parkinson’s disease, Alzheimer’s Disease, etc. Either little is known about the exact cause of these diseases, or like in the case of Alzheimer’s disease, there are several competing hypotheses behind the cause. These neurodegenerative diseases represent a particularly devastating health problem for which a significant unmet medical need exists. AAV-vector gene therapy has already shown benefits in treating many diseases, but still, several unanswered questions need to be addressed. Current results are promising and suggest that investigators have now defined a population of patients that can expect long-lasting expression of a donated gene from an AAV vector.
DelveInsight’s ‘AAV Vectors in Gene Therapy Pipeline Insight 2023‘ report provides comprehensive global coverage of available pipeline AAV vectors in gene therapy in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the AAV vectors in gene therapy pipeline domain.
Key Takeaways from the AAV Vectors in Gene Therapy Pipeline Report
- DelveInsight’s AAV vectors in gene therapy pipeline report depicts a robust space with 70+ active players working to develop 210+ pipeline therapies.
- Key AAV vectors in gene therapy companies such as Gensight Biologics, PTC therapeutics, uniQure, Pfizer, Ultragenyx Pharmaceutical, Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, 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 therapeuics, Bayer, LogicBio therapeutics, Solid Biosciences, Applied Genetic Technologies, Spark therapeuics (Roche), Eli lilly and company, Lexeo Therapeutics, Spark therapeutics, Gensight Biologics, Alcyone Lifesciences, MeiraGTx, Audentes therapeutics, Vivet Therapeutics, Astellas Gene Therapies, Homology Medicines, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Brain Neurotherapy Bio, Inc., Ocugen, Asklepios BioPharmaceutical, Neurogene, Jaguar Gene Therapy, LLC, Axovia Therapeutics, Coave therapeutics, Novartis, Arctos medical, Novartis, Vedere Bio, REGENXBIO Inc., StrideBio, Abeona therapeutics, Sanofi, Sirion therapeutics, Capsida Biotherapeutics, Kriya therapeutics, Neurophth, Poseida Therapeutics, BridgeBio Pharma, DiNAQOR, Biomarin, ViGeneron, AskBio, Selecta Biosciences, Prevail therapeutics, Intas Pharmaceuticals, Tenaya Therapeutics, Scout Bio therapeutics, Ocugen, Oyster Point pharmaceuticals, Cyprium Therapeutics, Solid Biosciences, Amicus Therapeutics, Coave therapeutics, AvantiBio, Voyager Therapeutics, Pfizer, Aruvant Sciences, Alcyone Lifesciences, Gene Therapy Research Institution Co., Ltd., and others are evaluating new candidates to improve the treatment landscape.
- Promising AAV vectors in gene therapy in pipeline such as GS010, Eladocagene exuparvovec, DTX401, AAV5-RPGR, Fidanacogene elaparvovec, AMT 061, Giroctocogene fitelparvovec, UX701, RGX-314, SPK-8011, SRP-9001, NFS-01, LYS-SAF302, GT005, MCO-010, HMI-102, DTX301, PBGM01, PBKR03, FLT180a, PBFT02, AT702, BBP-812, BBP-631, EGT-101, AT-GTX-501, AT-GTX-502, AAV2-hCHM, 4D-125, BMN 331, TSHA-118, SAR439483, BMN 307, ABO-102, CTx-PDE6b, RGX-121, RGX-111, AMT-130, TSHA-120, TSHA-101, AAV2/8-LSPhGAA, AAV2-GDNF, SRP-9003, ABO-101, SRP-9004, FBX-101, Isaralgagene civaparvovec (ST-920), (BAY-2599023), LB-001, SGT-001, rAAV2tYF-PR1.7-hCNGB3, AGTC-402, SPK-8016, PR001, PR006, 4D-310, LX1004, SPK-3006, GS030, ACTX 401, AAV- CNGA3, AAV-CNGB3, AT132, rAAVrh74.MCK.GALGT2, scAAV9.U7.ACCA, VTX 801, TAK 754, AskBio009, AT 845, SRP-6004, HMI-203, ADVM-022, AAV-hTERT, LX1001, RP-A501, AAV2-GDNF gene therapy, 4D 110, 4D-710, OCU400, LION-10, AAV gene therapies, 4D-150, JAG101, JAG201, AXV101, CTx-ABCA4, Optogenetic AAV gene therapy, Research programme: AAV ocular gene therapy, RGX-202, RGX-181, RGX-381, AMT-210, AMT-161, AMT-191, AMT-150, STRX-210, STRX-110, STRX-230, ABO-201, SRP-9005, SRP-9006, AAV gene therapy, Research programme: adeno associated virus based gene therapies, KT-A112, KT-A522, KT-A832, NFS-02, NFS- 04, NFS-05, NFS-10, P-OTC-101, BBP-815, BBP-818, DiNA-001, EGT-201, EGT-301, VG901, VG801, MMA-101, FLT210, FLT201, PR004, AAV-FIX, AAV-FVIII, AAV-AntiVEGF, TN-201, PKP2 PROGRAM, DWORF GENE THERAPY PROGRAM, SB-004, OCU410, ETF™ Gene Therapy, TSHA-102, LX2020, LX2021, LX2022, LX1020, LX1021, AMT-240, AMT-260, UX810, STRX-310, STRX-330, AAV-ATP7A Gene Therapy, SGT-003, TSHA-119, TSHA-104, TSHA-112, TSHA-106, TSHA-103, TSHA-105, TSHA-111-LAFORIN, TSHA-111-MALIN, Research programme: AAV based gene therapies, CTx-GBA1, CTx-FUS, Program: XLRS, Program: USH1B, KT-A281, KT-A252, KT-A261, AVB-202, TRACERᵀᴹ AAV Capsids, AAV gene therapies, AT GTX 701, LX2006, ARU-2801, Anc 80L65-arylsulfatase A, Anc 80L65-trastuzumab, ACTX 101, JAG301, NFS-07, NFS-09, NFS-11, NFS-12, NFS-13, STRX-220, STRX-240, vgAAV, AAV-SMN1, AAV-DMD, AAV.103, AAV.104, DINA-002, DINA-003, DINA-004, STRX-340, TSHA-113, TSHA-115, TSHA-114, TSHA-116, TSHA-117, TSHA-107, TSHA-108, TSHA-109, and others are under different phases of AAV vectors in gene therapy clinical trials.
- In January 2023, Rocket Pharmaceuticals announced the addition of RP-A601 to Rocket’s cardiac gene therapy portfolio as well as anticipated highlights for the year ahead across the Company’s world-class pipeline of lentiviral and AAV gene therapy programs targeting rare hematologic and cardiovascular diseases.
- In October 2022, Astellas Pharma and Taysha Gene Therapies, Inc. announced a strategic investment to support the advancement of Taysha’s adeno-associated virus (AAV) gene therapy development programs for the treatment of Rett syndrome and GAN.
Request a sample and discover the recent advances in AAV vectors in gene therapy @ AAV Vectors in Gene Therapy Pipeline Report
The AAV vectors in gene therapy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage AAV vectors in gene therapy, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the AAV vectors in gene therapy clinical trial landscape.
AAV Vectors in Gene Therapy Overview
The Adeno-associated viral (AAV) vector appears to be the safest and most effective vehicle for delivering the genes of interest, and it can maintain long-term gene and protein expression following a single injection of the vector. AAV vectors are the most widely used viral vectors for gene delivery to treat a wide range of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs, and harnessing revolutionary biotechnologies have all contributed significantly to the field’s growth. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing, and gene editing have contributed to AAV’s popularity as an ideal therapeutic vector, with only a few AAV-based therapeutics gaining regulatory approval in Europe and the United States.
Recombinant AAVs (rAAV) are now used for in vivo gene therapy delivery. The groundbreaking effort and outcomes of gene therapy led to the approval of Glybera, the first gene therapy approved by the European Medicines Agency (EMA) in 2012 to treat lipoprotein lipase deficiency. Luxturna received regulatory approval from the US Food and Drug Administration after a few years (FDA). The approval of these therapies was a watershed moment for researchers developing successful gene therapy protocols for inherited human disorders.
Learn more about the AAV vectors for gene therapy in clinical trials @ AAV Vectors for Gene Therapy
A snapshot of the AAV Vectors in Gene Therapy Pipeline Drugs mentioned in the report:
| Drugs | Company | Indication | Phase | RoA |
| GS010 | Gensight Biologics | Leber’s hereditary optic atrophy | Preregistration | Intravitreal |
| DTX401 | Ultragenyx Pharmaceutical | Glycogen storage disease type I | Phase III | Intravenous |
| AAV5-RPGR | MeiraGTx | Retinitis pigmentosa | Phase III | Subretinal |
| RGX-314 | REGENXBIO | Wet age-related macular degeneration | Phase III | Subretinal |
| SPK-8011 | Spark Therapeutics | Hemophilia A | Phase III | Intravenous |
| NFS-01 | Neurophth | Leber’s hereditary optic atrophy | Phase III | Intravitreal |
| GT 005 | Gyroscope Therapeutics | Dry age-related macular degeneration | Phase II | Intraocular |
Discover more about AAV vectors in gene therapy in development @ AAV Vectors in Gene Therapy Clinical Trials
AAV Vectors in Gene Therapy Therapeutics Assessment
The AAV vectors in gene therapy pipeline report proffers an integral view of the emerging novel AAV vectors in gene therapy segmented by stage, product type, molecule type, mechanism of action, and route of administration.
Scope of the AAV Vectors in Gene Therapy Pipeline Report
- Coverage: Global
- AAV Vectors in Gene Therapy Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
- AAV Vectors in Gene Therapy Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- AAV Vectors in Gene Therapy Therapeutics Assessment By Route of Administration: Intracerebral, Intracerebroventricular, Intracisternal, Intramuscular, Intraocular, Intrapancreatic, Intrastriatal, Intrathecal, Intravenous, Intravitreous, Introvitinreous, Oral, Parenteral, Retinal, Intra-arterial
- AAV Vectors in Gene Therapy Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy
- Key AAV Vectors in Gene Therapy Companies: BioMarin Pharmaceutical, Gensight Biologics, PTC therapeutics, uniQure, Pfizer, Ultragenyx Pharmaceutical, Biogen, Sarepta therapeutics, Neurophth Therapeutics, LYSOGENE, Gyroscope Therapeutics, Nanoscope Therapeutics, Homology medicines, Ultragenyx Pharmaceutical, Passage Bio, 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 therapeuics, Bayer, LogicBio therapeutics, Solid Biosciences, Applied Genetic Technologies, Spark therapeuics (Roche), Eli lilly and company, Lexeo Therapeutics, Spark therapeutics, Gensight Biologics, Alcyone Lifesciences, MeiraGTx, Audentes therapeutics, Vivet Therapeutics, Astellas Gene Therapies, Homology Medicines, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Brain Neurotherapy Bio, Inc., Ocugen, Asklepios BioPharmaceutical, Neurogene, Jaguar Gene Therapy, LLC, Axovia Therapeutics, Coave therapeutics, Novartis, Arctos medical, Novartis, Vedere Bio, REGENXBIO Inc., StrideBio, Abeona therapeutics, Sanofi, Sirion therapeutics, Capsida Biotherapeutics, Kriya therapeutics, Neurophth, Poseida Therapeutics, BridgeBio Pharma, DiNAQOR, Biomarin, ViGeneron, AskBio, Selecta Biosciences, Prevail therapeutics, Intas Pharmaceuticals, Tenaya Therapeutics, Scout Bio therapeutics, Ocugen, Oyster Point pharmaceuticals, Cyprium Therapeutics, Solid Biosciences, Amicus Therapeutics, Coave therapeutics, AvantiBio, Voyager Therapeutics, Pfizer, Aruvant Sciences, Alcyone Lifesciences, Gene Therapy Research Institution Co., Ltd., and others
- Key AAV Vectors in Gene Therapy in Pipeline: GS010, Eladocagene exuparvovec, DTX401, AAV5-RPGR, Fidanacogene elaparvovec, AMT 061, Giroctocogene fitelparvovec, UX701, RGX-314, SPK-8011, SRP-9001, NFS-01, LYS-SAF302, GT005, MCO-010, HMI-102, DTX301, PBGM01, PBKR03, FLT180a, PBFT02, AT702, BBP-812, BBP-631, EGT-101, AT-GTX-501, AT-GTX-502, AAV2-hCHM, 4D-125, BMN 331, TSHA-118, SAR439483, BMN 307, ABO-102, CTx-PDE6b, RGX-121, RGX-111, AMT-130, TSHA-120, TSHA-101, AAV2/8-LSPhGAA, AAV2-GDNF, SRP-9003, ABO-101, SRP-9004, FBX-101, Isaralgagene civaparvovec (ST-920), (BAY-2599023), LB-001, SGT-001, rAAV2tYF-PR1.7-hCNGB3, AGTC-402, SPK-8016, PR001, PR006, 4D-310, LX1004, SPK-3006, GS030, ACTX 401, AAV- CNGA3, AAV-CNGB3, AT132, rAAVrh74.MCK.GALGT2, scAAV9.U7.ACCA, VTX 801, TAK 754, AskBio009, AT 845, SRP-6004, HMI-203, ADVM-022, AAV-hTERT, LX1001, RP-A501, AAV2-GDNF gene therapy, 4D 110, 4D-710, OCU400, LION-10, AAV gene therapies, 4D-150, JAG101, JAG201, AXV101, CTx-ABCA4, Optogenetic AAV gene therapy, Research programme: AAV ocular gene therapy, RGX-202, RGX-181, RGX-381, AMT-210, AMT-161, AMT-191, AMT-150, STRX-210, STRX-110, STRX-230, ABO-201, SRP-9005, SRP-9006, AAV gene therapy, Research programme: adeno associated virus based gene therapies, KT-A112, KT-A522, KT-A832, NFS-02, NFS- 04, NFS-05, NFS-10, P-OTC-101, BBP-815, BBP-818, DiNA-001, EGT-201, EGT-301, VG901, VG801, MMA-101, FLT210, FLT201, PR004, AAV-FIX, AAV-FVIII, AAV-AntiVEGF, TN-201, PKP2 PROGRAM, DWORF GENE THERAPY PROGRAM, SB-004, OCU410, ETF™ Gene Therapy, TSHA-102, LX2020, LX2021, LX2022, LX1020, LX1021, AMT-240, AMT-260, UX810, STRX-310, STRX-330, AAV-ATP7A Gene Therapy, SGT-003, TSHA-119, TSHA-104, TSHA-112, TSHA-106, TSHA-103, TSHA-105, TSHA-111-LAFORIN, TSHA-111-MALIN, Research programme: AAV based gene therapies, CTx-GBA1, CTx-FUS, Program: XLRS, Program: USH1B, KT-A281, KT-A252, KT-A261, AVB-202, TRACERᵀᴹ AAV Capsids, AAV gene therapies, AT GTX 701, LX2006, ARU-2801, Anc 80L65-arylsulfatase A, Anc 80L65-trastuzumab, ACTX 101, JAG301, NFS-07, NFS-09, NFS-11, NFS-12, NFS-13, STRX-220, STRX-240, vgAAV, AAV-SMN1, AAV-DMD, AAV.103, AAV.104, DINA-002, DINA-003, DINA-004, STRX-340, TSHA-113, TSHA-115, TSHA-114, TSHA-116, TSHA-117, TSHA-107, TSHA-108, TSHA-109, and others
To know more about AAV vectors in gene therapy, visit @ AAV Vector Technology
Table of Contents
| 1. | AAV Vectors in Gene Therapy Pipeline Report Introduction |
| 2. | AAV Vectors in Gene Therapy Pipeline Report Executive Summary |
| 3. | AAV Vectors in Gene Therapy Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | AAV Vectors in Gene Therapy Clinical Trial Therapeutics |
| 6. | AAV Vectors in Gene Therapy Pipeline: Late Stage Products (Pre-registration) |
| 7. | AAV Vectors in Gene Therapy Pipeline: Late Stage Products (Phase III) |
| 8. | AAV Vectors in Gene Therapy Pipeline: Mid Stage Products (Phase II) |
| 9. | AAV Vectors in Gene Therapy Pipeline: Early Stage Products (Phase I) |
| 10. | AAV Vectors in Gene Therapy Pipeline Therapeutics Assessment |
| 11. | Inactive Products in the AAV Vectors in Gene Therapy Pipeline |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products in the AAV Vectors in Gene Therapy Pipeline |
| 15. | Unmet Needs |
| 16. | Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on the AAV vectors in gene therapy pipeline therapeutics, reach out @ AAV Vector Gene Therapy
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