DelveInsight’s ‘AAV Vectors in Gene Therapy Pipeline Insight 2023‘ report provides comprehensive global coverage of pipeline AAV Vectors in Gene Therapy therapies 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.
For AAV Vectors in Gene Therapy emerging drugs, the AAV Vectors in Gene Therapy pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
Key Takeaways from the AAV Vectors in Gene Therapy Pipeline Report
- DelveInsight’s AAV Vectors in Gene Therapy Pipeline analysis depicts a robust space with 70+ active players working to develop 235+ pipeline therapies.
- The leading AAV Vectors in Gene Therapy companies include 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 their lead assets to improve the AAV Vectors in Gene Therapy treatment landscape.
- Key AAV Vectors in Gene Therapy in various stages of development include 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.
- 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 breakthroughs happening in the AAV Vectors in Gene Therapy pipeline landscape @ AAV Vectors in Gene Therapy Pipeline Outlook
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, with the ability to sustain long-term gene and protein expression after a single injection of the vector. AAV vectors are the most commonly utilized viral vectors for gene delivery in the treatment of a variety of human disorders. Recent developments in the development of therapeutically desirable AAV capsids, genome design optimization, and the application of innovative biotechnologies have all contributed considerably to the field’s expansion. Only a few AAV-based medicines have received regulatory approval in Europe and the United States, despite preclinical and clinical advances in AAV-mediated gene substitution, gene silencing, and gene editing.
Recombinant AAVs (rAAV) are currently employed to administer gene therapy in vivo. Glybera, the first gene therapy approved by the European Medicines Agency (EMA) in 2012 to treat lipoprotein lipase deficiency, was the result of revolutionary efforts and outcomes in gene therapy. After a few years, the US Food and Drug Administration (FDA) granted Luxturna regulatory approval. The approval of these medications marked a turning point for researchers working on successful gene therapy regimens for hereditary human illnesses.
Find out more about AAV Vectors in Gene Therapy @ New AAV Vectors in Gene Therapy
AAV Vectors in Gene Therapy Treatment Analysis: Drug Profile
GS010: Gensight Biologics
LUMEVOQ® (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform developed at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address mitochondrial defects using an AAV vector (Adeno-Associated Virus). The gene of interest is introduced into the cell and expressed, resulting in the production of a functional protein, which is subsequently shuttled to the mitochondria via specific nucleotidic sequences to restore the missing or defective mitochondrial function. The European Medicines Agency (EMA) approved “LUMEVOQ” as the invented name for GS010 (lenadogene nolparvovec) in October 2018.
DTX401: Ultragenyx Pharmaceutical
DTX401 is an experimental AAV8 gene therapy that is intended to provide stable expression and activity of G6Pase- under the control of the native promoter. DTX401 is given as a single intravenous infusion and has been proven in preclinical tests to enhance G6Pase activity and decrease hepatic glycogen levels, both of which are well-described biomarkers of disease progression. All nine patients in Phase I/II clinical investigation demonstrated a clinical response, with significant decreases in the requirement for cornstarch and improvements in glucose management and other metabolic parameters compared to baseline. The medication is now being tested in a Phase III clinical trial for the treatment of Glycogen storage disorder type I patients.
Key AAV Vectors in Gene Therapy Therapies and Companies
- GS010: Gensight Biologics
- DTX401: Ultragenyx Pharmaceutical
- AAV5-RPGR: MeiraGTx
- RGX-314: REGENXBIO
- SPK-8011: Spark Therapeutics
- NFS-01: Neurophth
- GT 005: Gyroscope Therapeutics
Learn more about the novel and emerging AAV Vectors in Gene Therapy @ AAV Vectors in Gene Therapy Clinical Trials
AAV Vectors in Gene Therapy Therapeutics Assessment
- By Product Type
-
- Monotherapy
- Combination Therapy
- By Stage
-
- Discovery
- Pre-Clinical
- Phase I
- Phase II
- Phase III
- Pre-registration
- By Route of Administration
-
- Intracerebral
- Intracerebroventricular
- Intracisternal
- Intramuscular
- Intraocular
- Intrapancreatic
- Intrastriatal
- Intrathecal
- Intravenous
- Intravitreous
- Introvitinreous
- Oral
- Parenteral
- Retinal
- Intra-arterial
- By Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
Scope of the AAV Vectors in Gene Therapy Pipeline Report
-
- Coverage: Global
- 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 Pipeline Therapies: 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. | Introduction |
| 2. | Executive Summary |
| 3. | AAV Vectors in Gene Therapy Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | AAV Vectors in Gene Therapy Pipeline Therapeutics |
| 6. | AAV Vectors in Gene Therapy Pipeline: Late Stage Products (Phase III) |
| 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. | Therapeutic Assessment |
| 11. | Inactive Products |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products |
| 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 in the pipeline, reach out @ AAV Vectors Gene Therapy
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