DelveInsight’s Adeno-Associated Virus (AAV) Vectors in Gene Therapy Pipeline report provides in-depth analysis of the current clinical development landscape and growth prospects for Adeno-Associated Virus (AAV) Vectors in Gene Therapy.
Some of the Crucial Highlights from the AAV Vectors in Gene Therapy Pipeline Report
- AAV particles are made up of single-stranded DNA encased in a protein shell. Despite their basic structure, recombinant AAVs (rAAV) can play a critical role in nucleic acid delivery to cells.
- uniQure, Lysogene, Spark Therapeutics, MeiraGTx, Advernum Biotechnologies, Rocket Pharmaceuticals are among the major companies which are active in the AAV Vectors in Gene Therapy market.
- Etranacogene dezaparvovec is a pipeline AAV Vectors Gene Therapy which is in the Phase III of clinical trials while LYS-SAF302 pipeline AAV Vectors Gene Therapy is in the Phase II/III of development.
- Some pipeline AAV Vectors Gene Therapies such as AAV2-hCHM and AAV CNGA3 are in the Phase I/II stage of development.
- There are also some pipeline AAV Vectors Gene Therapies such as ADVM 022 and RP L401, which are still in the early stage of development i.e. Phase I of clinical trials.
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Adeno-Associated Virus Overview
Humans and certain other primate species are infected with Adeno-Associated Viruses (AAVs), which are tiny viruses. They are members of the Dependoparvovirus genus, which is part of the Parvoviridae family. AAV is not known to cause disease at this time. The viruses elicit only a minor immune response. AAV has a number of other characteristics that make it a good choice for generating viral vectors for gene therapy and isogenic human disease models.
AAV Vectors in Gene Therapy Pipeline Analysis: Drug Profile
AAV-CNGA3: MeiraGTx
Subretinal injection of AAV-CNGA3, a gene therapy treatment meant to restore cone function, is used to deliver AAV-CNGA3 to the cone receptors in the back of the eye. It was created with a synthetic promoter linked to high gene expression to accommodate for the higher amount of protein required to restore cone function in individuals with achromatopsia (ACHM) due to a CNGA3 gene mutation.
AAV Vectors in Gene Therapy Pipeline Therapies and Key Players
- Etranacogene dezaparvovec: uniQure
- LYS-SAF302: Lysogene
- AAV2-hCHM: Spark Therapeutics
- AAV CNGA3: MeiraGTx
- ADVM 022: Advernum Biotechnologies
- RP L401: Rocket Pharmaceuticals
Learn more about AAV @ AAV Vectors in Gene Therapy Major Companies
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
- Inhalation
- Intravenous
- Oral
- Subcutaneous
- By Molecule Type
- Small Molecule
- Monoclonal Antibody
- Proteins
- By Indications
- Solid Tumors
- Bladder Cancer
- Inactive Product Analysis
- Dormant
-
- Discontinued
Scope of the Report
Coverage: Global
AAV Vectors in Gene Therapy Key Players: uniQure, Lysogene, Spark Therapeutics, MeiraGTx, Advernum Biotechnologies, Rocket Pharmaceuticals, among others
AAV Vectors in Gene Therapy Pipeline Therapies: Etranacogene dezaparvovec, ADVM 022, LYS-SAF302, AAV2-hCHM, AAV CNGA3, RP L401, and others
Table of Contents
| 1. | Introduction |
| 2. | Executive Summary |
| 3. | Adeno-Associated Virus (AAV) Vectors in Gene Therapy: Overview |
| 4. | AAV Vectors in Gene Therapy Competitive Landscape – Active Drugs |
| 5. | AAV Vectors in Gene Therapy Pipeline Therapeutics |
| 6. | AAV Vectors in Gene Therapy Late Stage Products |
| 7. | AAV Vectors in Gene Therapy Mid Stage Products |
| 8. | AAV Vectors in Gene Therapy Early Stage Products |
| 9. | AAV Vectors in Gene Therapy Therapeutic Assessment |
| 10. | AAV Vectors in Gene Therapy Inactive Products |
| 11. | Appendix |
Know more of what’s covered in the AAV Vectors in Gene Therapy Pipeline Assessment Report
Key questions answered in the AAV Vectors in Gene Therapy Pipeline Report
- What are the current AAV Vectors in Gene Therapy treatment options?
- How many AAV Vectors in Gene Therapy developing therapies are in the early, mid, or late stages of development for AAV Vectors in Gene Therapy treatment?
- What are the reasons for AAV Vectors in Gene Therapy inactive and discontinued products?
- How many patents for developing therapeutics for the treatment of AAV Vectors in Gene Therapy have been awarded or are pending?
- What are the major designations for Adeno-Associated Virus (AAV) Vectors in Gene Therapy that have been granted?
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