Gene Therapy Competitive Landscape Drugs and Companies Insight Report, 2023 Updates: Analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments

Gene Therapy Overview

Gene therapy is defined as the treatment of disease by transfer of genetic material into cells. Gene therapy is understood as the capacity for gene improvement by means of the correction of altered (mutated) genes or site-specific modifications that have therapeutic treatment as target.

“Gene Therapy Competitive Landscape, 2023“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Gene Therapy Market.

The Gene Therapy Competitive Landscape report embraces in-depth commercial and clinical assessment of the Competitive Landscape 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.

Some of the key takeaways from the Gene Therapy Competitive Landscape Report:

• Companies across the globe are diligently working toward developing novel Gene Therapy treatment therapies with a considerable amount of success over the years. Gene Therapy Key players such as – Nervosave Therapeutics, Vivet Therapeutics, Homology Medicines, Duet Therapeutics, Rocket Pharmaceuticals, Nanoscope Therapeutics, Gyroscope Therapeutics, Adverum Biotechnologies, Dimension Therapeutics, Pfizer, HELIXMITH Co, Novartis, Spark Therapeutics, and others, are developing therapies for the Gene Therapy treatment 

• Gene Therapy Emerging therapies such as – NVO-101, VTX-802, HMI 103, DUET-01, RP-L102, MCO 010, GT005, ADVM-022, DTX401, Fordadistrogene movaparvovec, Engensis, Zolgensma, Voretigene neparvovec, and others are expected to have a significant impact on the Gene Therapy market in the coming years.  
• In April 2023, Pfizer will initiate long-term follow-up safety and efficacy study in participants with duchenne muscular dystrophy who have received Fordadistrogene Movaparvovec in a preceding clinical study. The purpose of this study is to understand the safety and effects of an experimental gene therapy called for dadistrogenemovaparvovec
• In April 2022, Pfizer announced that it will open its first US sites for a global Phase III trial assessing its for dadistrogenemovaparvovec (PF-06939926) in Duchenne muscular dystrophy (DMD) after the FDA lifted a clinical hold on the company’s investigational new drug (IND) application for the program, which was imposed in December 2021 after a patient died in a Phase Ib open-label study of the mini-dystrophin gene therapy candidate
• In April 2022, Novartis announced the US Food and Drug Administration (FDA) granted commercial licensure approval for its Durham, N.C. site, a multi-product gene therapy manufacturing facility
• In August 2022, The US Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
• In March 2022, REGENXBIO announced that the US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for RGX-202, which may entitle REGENXBIO to receive a priority review voucher which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred should a new BLA for RGX-202 be approved

Gene Therapy clinical assessment of products

The report comprises comparative clinical assessment of products by development stage, product type, and route of administration, molecule type.

• Gene Therapy Report Assessment
• Gene Therapy Company Analysis
• Gene Therapy Therapeutic Assessment
• Gene Therapy Pipeline Assessment
• Inactive Gene Therapy drugs assessment
• Gene Therapy Unmet Needs

DelveInsight’s Gene Therapy Report covers around 5+ 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

Some of the key companies in the Gene Therapy Therapeutics Market include:

Key companies developing therapies for Gene Therapy are – Abeona Therapeutics, Adaptimmune Therapeutics, Adverum Biotechnologies, Alnylam, Arrowhead Pharmaceuticals, Aspen Neuroscience, Astellas Gene Therapies, Atara Biotherapeutics, Atherys, Autolus Therapeutics, AVROBIO, Bit Bio, Bluebird Bio, Bristol-Myers Squibb, Cabaletta Bio, Capricor Therapeutics, Caribou Biosciences, Cellectis, Cellular Biomedicine Group, Celularity, CRISPR Therapeutics, Deep Genomics, Editas Medicine, eGenesis, Freeline Therapeutics, Gamida Cell, Generation Bio, Graphite Bio, Intellia Therapeutics, Krystal Biotech, MaxCyte, MeiraGTx, Mesoblast, Metagenomi, Mission Bio, NexImmune, NextCure, Novartis AG, Passage Bio, Pluristem Therapeutics, Regenxbio Inc., Roche Holding AG, Sangamo Therapeutics, Sarepta Therapeutics Inc., SQZ Biotechnologies, Vertex Pharmaceuticals, Verve Therapeutics, Vizgen, Voyager Therapeutics, and others

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

• NVO-101: Nervosave Therapeutics
• VTX-802: Vivet Therapeutics
• HMI 103: Homology Medicines
• DUET-01: Duet Therapeutics
• RP-L102: Rocket Pharmaceuticals
• MCO 010: Nanoscope Therapeutics
• GT005: Gyroscope Therapeutics
• ADVM-022: Adverum Biotechnologies
• DTX401: Dimension Therapeutics
• Fordadistrogene movaparvovec: Pfizer
• Engensis: HELIXMITH Co
• Zolgensma: Novartis
• Voretigene neparvovec: Spark Therapeutics

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Gene Therapy Competitive Landscape Analysis:

The Gene Therapy Competitive Landscape report provides insights into 

• The report provides detailed insights about companies that are developing therapies for the treatment of 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 Gene Therapy Treatment.
• Gene Therapy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• 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 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.

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

Gene Therapy Competitive Landscape Market Drivers

• Surge in research and development activities to develop gene therapies
• Technological advancements in genomic research

Gene Therapy Competitive Landscape Market Barriers

• High cost of gene therapies
• Lack of relevant procedures and aftercare

Scope of Gene Therapy Competitive Landscape Drug Insight    

• Coverage: Global
• Key Gene Therapy Companies: Nervosave Therapeutics, Vivet Therapeutics, Homology Medicines, Duet Therapeutics, Rocket Pharmaceuticals, Nanoscope Therapeutics, Gyroscope Therapeutics, Adverum Biotechnologies, Dimension Therapeutics, Pfizer, HELIXMITH Co, Novartis, Spark Therapeutics, and others
• Key Gene Therapy Therapies: NVO-101, VTX-802, HMI 103, DUET-01, RP-L102, MCO 010, GT005, ADVM-022, DTX401, Fordadistrogene movaparvovec, Engensis, Zolgensma, Voretigene neparvovec, and others
• Gene Therapy Therapeutic Assessment: Gene Therapy current marketed and Gene Therapy emerging therapies
• Gene Therapy Market Dynamics: Gene Therapy market drivers and 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.

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