The increasing occurrence of severe genetic disorders like spinal muscular atrophy, hemophilia, retinitis pigmentosa, and others, along with a rising number of pharmaceutical trials in gene therapy, advancements in gene editing tools, and other factors, are driving the demand for gene therapy. This is expected to contribute significantly to the overall growth of the gene therapy market from 2023 to 2028.
LAS VEGAS, Jan. 12, 2024 /PRNewswire/ — DelveInsight’s Gene Therapy Market Insights report provides the current and forecast market analysis, individual leading gene therapy companies’ market shares, challenges, gene therapy market drivers, barriers, trends, and key market gene therapy companies in the market.
Key Takeaways from the Gene Therapy Market Report
- As per DelveInsight estimates, North America is anticipated to dominate the global gene therapy market during the forecast period.
- Notable gene therapy companies such as Novartis AG, F. Hoffmann-La Roche Ltd., Ferring B.V., bluebird bio, Inc., Ultragenyx Pharmaceutical Inc., PTC Therapeutics, BioMarin, Biogen, Prevail Therapeutics, CSL, and several others, are currently operating in the gene therapy market.
- In January 2024, Vertex Pharmaceuticals Incorporated has disclosed that CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, has received Marketing Authorization from the Saudi Food and Drug Authority (SFDA) for addressing sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
- In January 2024, Precision BioSciences, Inc. has unveiled a pioneering alliance with TG Therapeutics, Inc., valued at $17.5 million. Under this partnership, TG Therapeutics secures an exclusive license to advance Azercabtagene Zapreleucel (azer-cel) for applications beyond cancer, specifically focusing on autoimmune diseases.
- In January 2024, Kriya Therapeutics, Inc., a major player in the biopharmaceutical industry, has unveiled an ambitious initiative to advance its groundbreaking gene therapy candidate to clinical trials by 2024. The company aims to have as many as five programs undergoing clinical testing by the conclusion of 2025, marking a significant transformation in the field of medicine.
- In January 2024, Bayer AG, in collaboration with Asklepios BioPharmaceutical, Inc., a gene therapy firm fully owned and autonomously managed under Bayer AG, declared the conclusion of the 18-month data gathering in the Phase Ib clinical study for AB-1005 (AAV2-GDNF). This investigational gene therapy aims to address Parkinson’s disease (PD) in patients.
- In January 2024, MeiraGTx, a company specializing in gene therapy, has disclosed a noteworthy financial decision. They are divesting their remaining financial stake in a collaborative eye-disease treatment with Johnson & Johnson. As part of the arrangement, MeiraGTx will receive an initial payment of $65 million, with the possibility of an extra $65 million in the following year. This strategic move involves relinquishing any future earnings from sales royalties associated with the eye-disease treatment.
- In January 2024, Genascence Corporation, a biotechnology company in the clinical stage that is transforming the approach to treating common musculoskeletal disorders through gene therapy, has declared the commencement of its Phase 1b clinical trial for GNSC-001, aimed at addressing knee osteoarthritis (OA) under the name DONATELLO. This study is currently accepting participants at ten clinical facilities across the United States, with an anticipated total enrollment of around 50 patients. The enrollment process is projected to conclude by the first quarter of 2024.
To read more about the latest highlights related to the gene therapy market, get a snapshot of the key highlights entailed in the Global Gene Therapy Market Report
Gene Therapy Overview
Gene therapy is a groundbreaking medical approach aimed at treating or preventing diseases by modifying the genetic material within a person’s cells. Unlike traditional treatments that focus on alleviating symptoms, gene therapy addresses the root cause of the ailment at the molecular level. The fundamental principle involves introducing, removing, or modifying specific genes to correct or replace faulty genetic information. This innovative technique holds immense promise for a wide array of genetic disorders, including inherited conditions and certain types of cancers. Researchers are actively exploring various delivery methods, such as viral vectors or CRISPR-Cas9 technology, to safely and effectively edit genes within target cells. While gene therapy has demonstrated remarkable success in some clinical trials, challenges remain, including ethical considerations, potential unforeseen consequences, and the need for rigorous safety assessments. Despite these challenges, gene therapy represents a transformative avenue in modern medicine, offering the potential to revolutionize the treatment of genetic diseases and pave the way for personalized, precision medicine.
Gene Therapy Market Insights
In 2022, North America held the largest portion of the gene therapy Market among all regions. This can be attributed to several key factors driving growth, including a growing patient population with genetic disorders, an increasing incidence of cancer, well-developed healthcare infrastructure, the presence of major industry players, and the swift regulatory approval process for new products. The region is expected to experience substantial growth in the field of gene therapy due to these influential factors.
In January 2019, the US Food and Drug Administration (FDA) issued a statement following an evaluation of the gene therapy pipeline and the clinical success rates of these products. The FDA projected that by 2025, it would approve 10 to 20 gene therapy products annually. Additionally, the FDA estimated that by 2020, they would receive 200 investigational new drug (INDs) applications each year. Consequently, the rising number of product development initiatives in the region is poised to expedite the expansion of the gene therapy market.
To know more about why North America is leading the market growth in the gene therapy market, get a snapshot of the Gene Therapy Market Outlook
The growth of the gene therapy market is primarily propelled by the increasing number of individuals affected by genetic disorders and the rising therapeutic significance of gene therapy in their treatment and control. According to the 2021 report from the World Federation of Hemophilia, there were approximately 347,026 reported cases of bleeding disorders globally in 2020. Specifically, in the same year, around 209,614 individuals were diagnosed with hemophilia, comprising 165,379 cases of hemophilia A, 33,076 cases of hemophilia B, and 11,159 cases of hemophilia with an unknown or unreported type. Notably, Hemgenix (etranacogene dezaparvovec) has received CE marking and approval from the US Food and Drug Administration as a gene therapy for treating adults with Hemophilia B, a congenital Factor IX deficiency.
The gene therapy market is anticipated to witness further expansion due to a rapid increase in the gene therapy pipeline. For instance, in January 2024, Bayer AG, in collaboration with Asklepios BioPharmaceutical, Inc., a gene therapy firm fully owned and autonomously managed under Bayer AG, declared the conclusion of the 18-month data gathering in the Phase Ib clinical study for AB-1005 (AAV2-GDNF). This investigational gene therapy aims to address Parkinson’s disease (PD) in patients.
Moreover, in January 2024, Genascence Corporation, a cutting-edge biotech firm transforming how common musculoskeletal diseases are treated through gene therapy, has launched the Phase 1b clinical trial of GNSC-001 for knee osteoarthritis (OA) treatment (named DONATELLO). This trial is currently recruiting patients at ten medical centers across the US, aiming for about 50 participants. Enrollments are projected to finish by the first quarter of 2024.
Nevertheless, the growth of the overall gene therapy market may face constraints due to ethical concerns associated with gene therapy diagnostics and the high costs associated with gene therapy treatments.
Get a sneak peek at the gene therapy market dynamics @ Gene Therapy Market Dynamics Analysis
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Report Metrics |
Details |
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Coverage |
Global |
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Study Period |
2020–2028 |
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Gene Therapy Market CAGR |
~19% |
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Key Gene Therapy Companies |
Novartis AG, F. Hoffmann-La Roche Ltd., Ferring B.V., bluebird bio, Inc., Ultragenyx Pharmaceutical Inc., PTC Therapeutics, BioMarin, Biogen, Prevail Therapeutics, CSL, among others |
Gene Therapy Market Assessment
- Gene Therapy Market Segmentation
- Gene Therapy Market Segmentation By Vector Type: Viral and Non-Viral
- Gene Therapy Market Segmentation By Delivery Type: Ex Vivo and In Vivo
- Gene Therapy Market Segmentation By Indication: Neurological, Oncological, Hematological, Ophthalmological, and Others
- Gene Therapy Market Segmentation By Geography: North America, Europe, Asia-Pacific, and Rest of World
- Porter’s Five Forces Analysis, Product Profiles, Case Studies, KOL’s Views, Analyst’s View
Which MedTech key players in the gene therapy market are set to emerge as the trendsetter explore @ Gene Therapy Companies
Table of Contents
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1 |
Report Introduction |
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2 |
Executive summary |
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3 |
Regulatory and Patent Analysis |
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4 |
Key Factors Analysis |
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5 |
Porter’s Five Forces Analysis |
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6 |
COVID-19 Impact Analysis on Gene Therapy Market |
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7 |
Gene Therapy Market Layout |
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8 |
Global Company Share Analysis – Key 3-5 Companies |
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9 |
Gene Therapy Market Company and Product Profiles |
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10 |
Project Approach |
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11 |
About DelveInsight |
Interested in knowing the gene therapy market by 2028? Click to get a snapshot of the Gene Therapy Market Trends
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SOURCE DelveInsight Business Research, LLP
