The "Gene Therapy Market, 6th Edition: Industry Trends and Global Forecasts to 2035 - Therapeutic Area, Type of Vector, Type of Therapy, Type of Gene Delivery Method, Route of Administration, Geographical Region, Leading Market Players and Sales Forecast" report has been added to ResearchAndMarkets.com's offering.

The Global Gene Therapy Market is valued at USD 2.4 billion in 2024 growing at a CAGR of 19.25% during the forecast period 2024-2035.

A diverse range of disorders arising from congenital abnormalities and inherited genetic mutations, including autoimmune disorders, genetic disorders, neurological disorders and oncological disorders significantly impacts individuals' health. These disorders are consequences of certain inherited genetic anomalies that disrupt the normal functioning of genes and adversely affect the process of translation. The statistics presented by the World Health Organization (WHO) states that 10 out of every 1,000 individuals are affected by genetic disorders, contributing to 70 million people globally. In addition, more than 40% of infant mortality globally is associated with various genetic disorders. Therefore, to address such challenges, gene therapies have emerged as a revolutionary approach and have proven to be a promising modality in treating a wide array of diseases, focused on delivering targeted therapies directly to affected organs or cells.

The objective of gene therapy is to comprehend the underlying genetic cause of a disease, followed by either introduction of a healthy version of the mutated gene or by inactivation of the faulty / disease-causing genes in the body. It is worth noticing that in 2003, Gendicine (Sibiono GeneTech) became the world's first gene therapy to receive regulatory approval from the China FDA. In the past two decades, this has led to several breakthroughs related to the development of gene therapies. For instance, in December 2017, the USFDA approved Luxturna (Spark Therapeutics), the first gene therapy intended for the treatment of inherited retinal disorders. Additionally, in May 2019, the USFDA approved ZOLGENSMA (Novartis), the first gene therapy for the treatment of spinal muscular atrophy in pediatric patients. It is noteworthy that the therapy received multiple designations, namely breakthrough therapy, fast track, orphan drug and priority review designations resulting in the accelerated approval of the drug in the US. Further, in 2023, five gene therapies received the USFDA market approval for various therapeutic indications.

In addition, the month of April 2024 witnessed a significant increase in the number of active clinical trials (1,100 active clinical trials) for evaluating gene therapies across various phases of clinical studies. Moreover, three gene therapies have already received regulatory approval in 2024, and according to predictions made by USFDA in 2019, it is estimated that 10 to 20 would gain market access, each year, till 2025. It is noteworthy that the favorable results from ongoing clinical research initiatives have spurred investments from both government and private sectors to support the development of these therapeutic products. With the progressing domain, gene therapies are anticipated to be utilized for the treatment of more than 65.6 million patients suffering from a myriad of disease indications, by 2034.

The rapidly evolving gene therapy product portfolio tends to attract an increasing number of startups and big pharma players, marking their presence in the gene therapy market. At present, 345 gene therapy companies are involved in the development of various early and late-stage therapies worldwide. The recent years have observed a significant increase in the integration of novel technologies, such as gene modification, genome editing, genome sequencing and manipulation technologies (molecular / gene switch), in conjugation with advanced gene delivery methods. Additionally, next-generation delivery platforms, including nanoparticles and hybrid vector systems, have been proven to facilitate an effective, precise and safe delivery of gene-based therapeutics, thereby enabling more targeted treatment approaches. With the integration of advancements in technology and growing preference for single-dose effective therapeutics, the gene therapy market is poised to witness substantial market growth during the forecast period.

Key Benefits of Buying this Report

• The report offers market leaders and newcomers valuable insights into revenue estimations for both the overall market and its sub-segments.
• Stakeholders can utilize the report to enhance their understanding of the competitive landscape, allowing for improved business positioning and more effective go-to-market strategies.
• The report provides stakeholders with a pulse on the Gene Therapy Market, furnishing them with essential information on significant market drivers, barriers, opportunities, and challenges.

Leading Market Companies

• Amgen
• Artgen Biotech
• BioMarin Pharmaceutical
• bluebird bio
• CRISPR Therapeutics
• CSL Behring
• Ferring Pharmaceuticals
• Kolon TissueGene
• Krystal Biotech
• Novartis
• Orchard Therapeutics
• Pfizer
• PTC Therapeutics
• Sarepta Therapeutics
• Shanghai Sunway Biotech
• Sibiono GeneTech
• Spark Therapeutics

Key Topics Covered:

INTRODUCTION

• Evolution of Gene Therapies
• Classification of Gene Therapies
• Routes of Administration
• Mechanism of Action of Gene Therapies
• Advantages and Disadvantages of Gene Therapies
• Challenges associated with Gene Therapies
• Introduction to Genome Editing

GENE DELIVERY VECTORS

• Methods of Gene Transfer
• Viral Vectors for Genetically Modified Therapies
• Non-Viral Vectors for Genetically Modified Therapies

REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIOS

• Regulatory Guidelines in North America
• Regulatory Guidelines in Europe
• Regulatory Guidelines in Asia-Pacific
• Reimbursement Scenario

MARKET LANDSCAPE

• Marketed and Clinical Stage Gene Therapies: Market Landscape
• Preclinical and Discovery Stage Gene Therapies: Market Landscape

DEVELOPER LANDSCAPE

• Gene Therapy: Competitive Landscape

COMPANY PROFILES

Gene Therapy Developers in North America

• Amgen
• BioMarin Pharmaceutical
• bluebird bio
• CRISPR Therapeutics
• Kolon TissueGene
• Krystal Biotech
• Pfizer
• PTC Therapeutics
• Sarepta Therapeutics
• Spark Therapeutics

Gene Therapy Developers in Europe

• Artgen Biotech
• Ferring Pharmaceuticals
• Novartis
• Orchard Therapeutics

Gene Therapy Developers in Asia-Pacific and Rest of the World

• CSL Behring
• Shanghai Sunway Biotech
• Sibiono GeneTech

MARKETED GENE THERAPIES

• Gendicine (Shenzhen Sibiono GeneTech)
• Oncorine (Shanghai Sunway Biotech)
• Rexin-G (Epeius Biotechnologies)
• Neovasculgen (Human Stem Cells Institute)
• Imlygic (Amgen)
• Strimvelis (Orchard Therapeutics)
• LuxturnaT (Spark Therapeutics)
• ZolgensaT (Novartis)
• Collategene (AnGes)
• ZyntelgoT (bluebird bio)
• LibmeldyT (Orchard Therapeutics)

KEY COMMERCIALIZATION STRATEGIES

• Successful Drug Launch Strategy: ROOTS Framework
• Successful Drug Launch Strategy: Product Differentiation
• Commonly Adopted Commercialization Strategies based on Phase of Development
• List of Approved Gene Therapies
• Key Commercialization Strategies Adopted by Gene Therapy Developers
  • Strategies Adopted before Therapy Approval
    • Participation in Global Events
    • Collaborations with Stakeholders and Pharmaceutical Firms
    • Indication Expansion
  • Strategies adopted During / Post Therapy Approval
    • Geographical Expansion
    • Participation in Global Events
    • Patient Assistance Programs
    • Awareness through Product Websites
    • Collaboration with Stakeholders and Pharmaceutical Firms

LATE-STAGE GENE THERAPIES

• LUMEVOQ (GS010)
• OTL-103
• PTC-AADC
• BMN 270
• rAd-IFN/Syn3
• beti-cel
• eli-cel
• lovo-cel
• SRP-9001
• EB-101
• ProstAtak
• D-Fi
• CG0070
• Vigil-EWS
• Engensis
• VGX-3100
• INVOSSA (TG-C)
• VYJUVEKT
• PF-06939926
• PF06838435
• PF-07055480
• SPK-8011
• AMT-061
• VB-111
• Generx
• ADXS-HPV
• AGTC 501
• LYS-SAF302
• NFS-01
• AG0302-COVID-19
• RGX-314
• Hologene 5

PATENT ANALYSIS

• Gene Therapy: Patent Analysis
• Gene Editing Market: Patent Analysis
• Analysis by CPC Symbols
• Patent Benchmarking Analysis
• Patent Valuation Analysis
• Overall Intellectual Property Portfolio: Analysis by Type of Organization

MERGERS AND ACQUISITIONS

FUNDING AND INVESTMENTS

• Types of Funding
• Gene Therapy: Funding and Investment Analysis

CLINICAL TRIAL ANALYSIS

• Gene Therapy: Clinical Trial Analysis
• Analysis by Type of Sponsor / Collaborator
• Analysis by Prominent Treatment Sites
• Gene Therapy: Analysis of Enrolled Patient Population

COST PRICE ANALYSIS

START-UP VALUATION

BIG PHARMA INITIATIVES

Gene Therapy: List of Most Prominent Big Pharmaceutical Players

• Analysis by Therapeutic Area
• Analysis by Type of Vector Used
• Analysis by Type of Therapy
• Analysis by Type of Gene Delivery Method Used

Benchmarking Analysis of Key Parameters

• Spider Web Analysis: Pipeline Strength
• Spider Web Analysis: Mergers and Acquisitions
• Spider Web Analysis: Funding and Investments
• Spider Web Analysis: Clinical Trials
• Spider Web Analysis: Technologies
• Spider Web Analysis: Patents

Benchmarking Analysis of Big Pharmaceutical Players

• Spider Web Analysis: Novartis
• Spider Web Analysis: Takeda Pharmaceutical
• Spider Web Analysis: Roche
• Spider Web Analysis: Merck
• Spider Web Analysis: Pfizer
• Spider Web Analysis: Johnson & Johnson
• Spider Web Analysis: AbbVie
• Spider Web Analysis: Bristol Myers Squibb
• Spider Web Analysis: AstraZeneca
• Spider Web Analysis: Sanofi

DEMAND ANALYSIS

MARKET IMPACT ANALYSIS: DRIVERS, RESTRAINTS, OPPORTUNITIES AND CHALLENGES

GLOBAL GENE THERAPY MARKET

GENE THERAPIES MARKET, BY THERAPEUTIC AREA

GENE THERAPIES MARKET, BY TYPE OF VECTOR

GENE THERAPIES MARKET, BY TYPE OF THERAPY

GENE THERAPIES MARKET, BY TYPE OF GENE DELIVERY METHOD

GENE THERAPIES MARKET, BY ROUTE OF ADMINISTRATION

GENE THERAPIES MARKET, BY GEOGRAPHICAL REGIONS

GENE THERAPIES MARKET, BY LEADING PLAYERS

GENE THERAPIES MARKET, SALES FORECAST OF THERAPIES

• Adstiladrin
• BEQVEZT
• CasgevyT
• Elevidys
• Gendicine
• Hemgenix
• IMLYGIC
• Libmeldy
• Luxturna
• LYFGENIAT
• Neovasculgen
• Oncorine
• RoctavianT
• SKYSONA
• StrimvelisT
• UpstazaT
• VYJUVEKT
• Zolgensma
• ZyntegloT

Phase III Gene Therapies Market: Sales Forecast

• AAV-RPE65
• ABO-102 (UX111)
• AGTC 501 (rAAV2tYF-GRK1-RPGR)
• Cretostimogene Grenadenorepvec (CG0070)
• Dirloctocogene samoparvovec (SPK-8011)
• DTx-301
• DTx-401
• Generx (Ad5FGF-4)
• Giroctocogene fitelparvovec (SB-525 / PF-07055480)
• Hologene 5
• INVOSSAT (TG-C)
• LUMEVOQ
• NFS-01 (rAAV2-ND4)
• NTLA-2001
• Olvi-Vec (Olvimulogene nanivacirepvec)
• ProstAtak
• pz-cel (prademagene zamikeracel / EB-101)
• RGX-121
• RGX-314
• VGX-3100

GENE THERAPY MARKET: EMERGING TECHNOLOGIES

• Gene Editing Technologies
• Emerging Gene Editing Platforms
• Gene Therapy Regulation Technologies (Gene Switch Technology)
• Other Emerging Technologies
• Technology Platforms for Developing / Delivering Gene Therapies

VECTOR MANUFACTURING FOR GENE THERAPIES

• Viral Vector Manufacturing Process
  • Mode of Vector Production
  • Adherent and Suspension Cultures
  • Unit Processes and Multiple Parallel Processes
  • Cell Culture Systems for Production of Viral Vectors
  • Culture Media Specifications
• Bioprocessing of Viral Vectors
• Challenges Associated with Vector Manufacturing
• Contract Manufacturing Organizations Involved in Viral Vector Production

CASE STUDY: GENE THERAPY SUPPLY CHAIN

• Overview of Gene Therapy Supply Chain
• Implementation of Supply Chain Models
• Logistics in Gene Therapy
• Regulatory Supply Chain Across the Globe
• Challenges Associated with Gene Therapy Supply Chain
• Software Applications for Gene Therapy Supply Chain Management
• Recent Developments and Upcoming Trends

EXECUTIVE INSIGHTS

• Orchard Therapeutics
• Vivet Therapeutics
• Kubota Pharmaceutical
• Eyevensys
• AGTC
• LogicBio Therapeutics
• AAVogen
• Hemera Biosciences
• Myonexus Therapeutics
• Arthrogen
• PlasmidFactory
• Delphi Genetics
• Vigene Biosciences
• Gene Biotherapeutics
• Milo Biotechnology

For more information about this report visit https://www.researchandmarkets.com/r/r53zwp

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