AAV vectors in gene therapy - Pipeline Insight, 2024

This “AAV vectors in gene therapy - Pipeline Insight, 2024 report provides comprehensive insights about 70+ companies and 235+ pipeline drugs in 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.

Geography Covered

• Global coverage

AAV vectors in gene therapy Understanding

AAV 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. Pre-clinical and clinical successes in AAV-mediated gene replacement, gene silencing, and gene editing have helped AAV to gain popularity as an ideal therapeutic vector, with few AAV-based therapeutics gaining regulatory approval in Europe and the United States. Nowadays, recombinant AAVs (r AAV) is being used for in vivo delivery of gene therapies. The ground-breaking effort and the outcomes of gene therapy led to the approval of Glybera, first approved gene therapy by the European Medicines Agency (EMA) to treat lipoprotein lipase deficiency in 2012. After a few years, Luxturna also received a regulatory nod from the US Food and Drug Administration (FDA). Approval of these therapies was a revolutionary achievement for researchers who have been working to develop successful gene therapy protocols for inherited humandisorders.

AAV vectors in gene therapy - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the AAV vectors in gene therapy pipeline landscape is provided which includes the disease overview and AAV vectors in gene therapy treatment guidelines. The assessment part of the report embraces, in depth AAV vectors in gene therapy commercial assessment and clinical assessment of the pipeline products under development. In the 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, AAV vectors in gene therapy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence AAV vectors in gene therapy R&D. The therapies under development are focused on novel approaches to treat/improve AAV vectors in gene therapy.

AAV vectors in gene therapy Emerging Drugs Chapters

This segment of the AAV vectors in gene therapy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

AAV vectors in gene therapy Emerging Drugs

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 October2018.

Valoctocogene roxaparvovec: Bio Marin PharmaceuticalValoctocogene 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 theFDA.

DTX401: Ultragenyx Pharmaceutical DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-a under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-a 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 typeI.

AAV5-RPGR: Meira GTx 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 inRPGRORF15.

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 STARstudy.

AMT 061: Uni Qure 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. ResearchandDevelopment.

RGX-314: REGENXBIORGX-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® 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 (r AAV2) 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/IIIclinicaltrial.

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.

AAV vectors in gene therapy: Therapeutic Assessment

This segment of the report provides insights about the different AAV vectors in gene therapy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in AAV vectors in gene therapy

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, Bio MarinPharmaceutical.

Phases

This 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

AAV vectors in gene therapy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

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

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

AAV vectors in gene therapy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses AAV vectors in gene therapy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging AAV vectors in gene therapy drugs.

AAV vectors in gene therapy Report Insights

• AAV vectors in gene therapy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

AAV vectors in gene therapy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing AAV vectors in gene therapy drugs?
• How many AAV vectors in gene therapy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of AAV vectors in gene therapy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the AAV vectors in gene therapy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for AAV vectors in gene therapy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Bio MarinPharmaceutical
• 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
• Bio MarinPharmaceutical
• 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
• Takeda
• 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
• Di NAQOR/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.

Key Products

• Valoctocogene roxaparvovec
• GS010
• Eladocagene exuparvovec
• DTX401
• AAV5-RPGR
• Fidanacogene elaparvovec
• Timrepigene emparvovec
• AMT 061
• Giroctocogene fitelparvovec
• UX701
• RGX-314
• BIIB112
• SPK-8011
• SRP-9001
• NFS-01
• LYS-SAF302
• GT005
• MCO-010
• HMI-102
• DTX301
• PBGM01
• PBKR03
• FLT180a
• PBFT02
• FLT190
• AT702
• BBP-812
• BBP-631
• EGT-101
• AXO-AAV-GM1
• AXO-AAV-GM2
• 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)
• DTX201 (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- AT751

• AT753
• 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
• TRACER?? AAV Capsids
• AAV gene therapies
• AT GTX 701
• LX2006
• ARU-2801
• Anc 80L65-arylsulfatase A
• Anc 80L65-trastuzumab
• ACTX 101
• GT0001X
• GT0002X
• GT0003X
• GT0004X
• GT0005X
• GT0006X
• GT0007X
• GT0008X
• GT0009X
• 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

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