AAV in Gene Therapy Pipeline Drugs Analysis Report (2024 Updates): FDA Approvals, Clinical Trials, Therapies, MOA, ROA by DelveInsight | Asklepios Biopharma, MeiraGTx, Gyroscope Therapeutics, Nanoscop

The AAV in Gene Therapy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

“AAV in Gene Therapy Pipeline Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the AAV in Gene Therapy Market.

Some of the key takeaways from the AAV in Gene Therapy Pipeline Report:

• Companies across the globe are diligently working toward developing novel AAV in Gene Therapy treatment therapies with a considerable amount of success over the years. AAV in Gene Therapy Key players such as – Asklepios Biopharmaceutical, MeiraGTx, Gyroscope Therapeutics, Nanoscope Therapeutics, Neurophth biological tech, Lysogene, Spark Therapeutics, and others, are developing therapies for the AAV in Gene Therapy treatment 

• AAV in Gene Therapy Emerging therapies such as – AAV2/8LSPhGAA, AAV CNGA3, GT 005, MCO-010, NFS-01, LYS-SAF302, SPK-9001, SPK-8011, and others are expected to have a significant impact on the AAV in Gene Therapy market in the coming years.   

• In January 2024, Akouos, Inc., a subsidiary of Eli Lilly and Company (NYSE: LLY), has released encouraging preliminary clinical findings from the Phase 1/2 AK-OTOF-101 study. These results show pharmacologic restoration of hearing within 30 days of AK-OTOF administration in the first participant, who had experienced profound hearing loss for over a decade. Further results, including initial data from a second participant receiving AK-OTOF, will be presented during the Late Breaking Presidential Symposium at the 2024 Association for Research in Otolaryngology (ARO) MidWinter Meeting.

• In December 2023, Neurogene has administered the first doses of its investigational adeno-associated virus (AAV) gene therapy, NGN-401, to the initial two female pediatric participants in a Phase I/II trial targeting Rett Syndrome. Rett Syndrome is a neurodevelopmental disorder lacking effective medical treatments. NGN-401 aims to boost therapeutic activity while avoiding toxicities related to excessive transgene expression.

AAV in Gene Therapy Overview

AAV stands for adeno-associated virus, a small, non-pathogenic virus that has gained significant attention in the field of gene therapy. AAV has become a popular vector for delivering therapeutic genes into target cells for the treatment of various genetic disorders and other diseases.

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Route of Administration

AAV 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

Molecule Type

Products have been categorized under various Molecule types, such as

• Monoclonal Antibody

• Peptides

• Polymer

• Small molecule

• Gene therapy

• Product Type

AAV in Gene Therapy Pipeline Therapeutics Assessment

• AAV in Gene Therapy Assessment by Product Type

• AAV in Gene Therapy By Stage and Product Type

• AAV in Gene Therapy Assessment by Route of Administration

• AAV in Gene Therapy By Stage and Route of Administration

• AAV in Gene Therapy Assessment by Molecule Type

• AAV in Gene Therapy by Stage and Molecule Type

DelveInsight’s AAV in Gene Therapy 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)

• Pre-clinical and Discovery stage candidates

• Discontinued & Inactive candidates

• Route of Administration

Further AAV in Gene Therapy product details are provided in the report. Download the AAV in Gene Therapy pipeline report to learn more about the emerging AAV in Gene Therapy therapies

Emerging AAV in Gene Therapy Drugs Under Different Phases of Clinical Development Include:

• AAV2/8LSPhGAA: Asklepios Biopharmaceutical

• AAV CNGA3: MeiraGTx

• GT 005: Gyroscope Therapeutics

• MCO-010: Nanoscope TherapeuticsXX

• NFS-01: Neurophth biological tech

• LYS-SAF302: Lysogene

• SPK-9001: Spark Therapeutics

• SPK-8011: Spark Therapeutics

AAV in Gene Therapy Pipeline Analysis:

The AAV in Gene Therapy pipeline report provides insights into 

• The report provides detailed insights about companies that are developing therapies for the treatment of AAV in Gene Therapy with aggregate therapies developed by each company for the same.

• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for AAV in Gene Therapy Treatment.

• AAV in Gene Therapy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• AAV in Gene Therapy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the AAV in Gene Therapy market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about AAV in Gene Therapy drugs and therapies

AAV in Gene Therapy Pipeline Market Drivers

• Increasing incidence of hematological and genetic disorders

• Favorable Research and Development Activities

• Increase in government support, ethical acceptance of gene therapy

AAV in Gene Therapy Pipeline Market Barriers

• Multiple technological challenges

• Unwanted immune responses

Scope of AAV in Gene Therapy Pipeline Drug Insight    

• Coverage: Global

• Key AAV in Gene Therapy Companies: Asklepios Biopharmaceutical, MeiraGTx, Gyroscope Therapeutics, Nanoscope Therapeutics, Neurophth biological tech, Lysogene, Spark Therapeutics, and others

• Key AAV in Gene Therapy Therapies: AAV2/8LSPhGAA, AAV CNGA3, GT 005, MCO-010, NFS-01, LYS-SAF302, SPK-9001, SPK-8011, and others

• AAV in Gene Therapy Therapeutic Assessment: AAV in Gene Therapy current marketed and AAV in Gene Therapy emerging therapies

• AAV in Gene Therapy Market Dynamics: AAV in Gene Therapy market drivers and AAV in Gene Therapy market barriers 

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Table of Contents

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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