Key Takeaways:
· The Large B-cell lymphoma segment held the largest revenue share of 36.14% in 2023.
· The inherited retinal disease segment is expected to register a significant CAGR over the forecast period.
· The lentivirus dominated with a revenue share of 43.79% in 2023.
· The AAV segment is expected to grow at a highest CAGR over the forecast period.
· The intravenous segment dominated the market share in 2023 and is anticipated to grow at a higher CAGR over the forecast period.
· The others segment is expected to grow at a significant CAGR over the forecast period.
The Complete Study is Now Available for Immediate Access | Download the Sample Pages of this Report@ https://www.novaoneadvisor.com/report/sample/8455
Gene therapy is a medical field in which disease is prevented and cured by replacing an underlying genetic problem, which basically changes and replaces changed cell tissues with unhealthy cell tissues to treat diseases such as cancer. Its aim is to train the immune system of the body to help fight cancer-causing cells to protect healthy cells from the effects of cancer treatment.
The 4 main FDA-approved gene therapies are:
· Luxturna—It is a gene therapy for inherited retinal disorder.
· ROCTAVIAN—Gene therapy for Hemophilia A
· SKYSONA—A gene therapy for cerebral adrenoleukodystrophy (CALD)
· Zolgensma: It is a gene therapy for spinal muscular atrophy (SMA).
Apart from this, the U.S. Food and Drug Administration (FDA) has approved Teceltra, a gene therapy for the diagnosis of adults having unresectable or metastatic synovial sarcoma, those who have received chemotherapy on a prior basis for HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 antigen as determined by FDA-authorized companion diagnostic devices.
The recent government initiatives taken for gene therapy are that the U.S. Food and Drug Administration has granted NCATS both Rare Pediatric Diseases (PDA) designations and Orphan Drug Designations (ODD) for five major researched gene therapies. These therapies are for rare diseases that are examined by the Accelerating Medicines Partnership called “Bespoke Gene Therapy Consortium.”. Another crucial initiative taken by the same firm is that the FDA has recently released different documents for the manufacturing of gene therapy products and the design of clinical trials.
Fundamentally, cell and gene therapy are the borderlines of precision medicine that specifically target rare diseases and pathology too. So far, it has been found that cell and gene therapy has completely transformed patient care for sickle diseases like spinal muscular atrophy and certain cancers like leukemia and lymphoma, as well as inherited retinal disease. CGT is the fastest-growing field, as many minute-sized biotech firms present the capability for commercial success.
There are presently 4,000 gene, cell, and RNA therapies in the progress lineup with an 11% year-over-year rise specifically in Phase 1 programs. Also, there is a deal flow between small biotech firms and big pharmaceutical companies, which is rising and providing drug companies a variety of options to choose from and increasing their portfolios. With more than 4000 therapies in the line of progress, right from preclinical to pre-registration. With this, oncology and rare diseases are top focus areas in case of gene therapy progress with five targeted diseases such as:
· Myeloma
· Acute myelogenous leukemia
· Non-Hodgkin’s lymphoma
· B-cell lymphoma
· Ovarian Cancer
Artificial intelligence is drastically changing the field of gene therapy in terms of research and development by serving improved efficiency, accuracy, and safety. AL platforms can examine extensive genetic databases in order to discover new targets for particular gene therapy and improve molecular structures, giving researchers important information for the purpose of drug development. Overall, artificial intelligence in terms of gene therapy improves safety and efficacy for awaited off-target side effects, which optimizes delivery methods and enhances clinical trial patterns. The merging of Artificial Intelligence (AI) with Computer-Aided Drug Discovery (CADD) is necessary for the progress of gene therapy by making the drug discovery more effective and improving therapeutic design.
With respect to artificial intelligence, the Union Health Ministry’s Department of Health Research has been allocated an amplified 531 crore in the Union Budget, which will be totally invested in artificial intelligence (AI) in healthcare and newer therapies too.
Key Gene Therapy Insights:
· Gene therapy has spontaneously made advances in the diagnosis of neurodegenerative disorders such as Alzheimer's disease (AD), progressive supranuclear palsy, Huntington’s disease, Parkinson’s disease, ataxia, motor neuron disease, multiple system atrophy, etc. It has the capability to treat different neurologic conditions; it highlighted the urge of AAV capsid, regulation of transgene expression, and efficient animal tools too.
· Increasing prevalence of genetic disorders: The rising number of diagnosed genetic disorders and rare diseases is driving the demand for novel therapeutic approaches that focus on underlying causes rather than just alleviating symptoms.
· Favorable Regulatory Environment: Worldwide, regulatory agencies are hugely recognizing the capability of gene therapies, which offer streamlined approval processes and incentives to accelerate clinical development and market entry too.
· Future genetics screens are a valuable part of the molecular biology toolbox in order to target genes so as to identify new drugs and genes and these screens have gotten bigger and larger due to the constant progress and development in CRISPR-based technologies
U.S. Gene Therapy Market Trends:
· As published on 17 January 2025, The Lancet Respiratory Medicine is a phase ½ study provided by pivotal registrational data for the submission of a Biologics License Application to the U.S. Food and Drug Administration.
· Recent technological advancements in gene therapy have opened the door for clinical translation. Two specifically developed areas are viral vectors for gene delivery and the advent of CRISPR-Cas9 for precise gene editing. Viral vectors are utilized for pathogenic potential as vehicles for gene delivery as they have natural tendency to infect cell and integrate their genetic trails. On the other hand, CRISPR-Cas9 is a Nobel Prize-winning gene editing tool that enables accurate modification of DNA sequences.
· Gene therapy has pushed a compelling therapeutic approach for different neurodegenerative disorders. Various factors to enrich gene therapy to discover novel factors, recent curative targets, and the dependability of transgenic delivery paths.
· The Novel approaches new advancements in gene therapy in pharmaceuticals, immunotherapy, RNA Based therapeutics, cell-based therapies and targeted tumor therapies.
· Genprex, a clinical-stage gene therapy company, has a current market capitalization of USD XX million and has a cohesive licensing agreement with the University of Pittsburgh into a new arrangement for diabetes-related gene therapies.
U.S. Gene Therapy Market Report Scope
|
Report Attribute |
Details |
|
Market Size in 2024 |
USD 3.80 Billion |
|
Market Size by 2033 |
USD 18.50 Billion |
|
Growth Rate From 2024 to 2033 |
CAGR of 19.22% |
|
Base Year |
2023 |
|
Forecast Period |
2024 to 2033 |
|
Segments Covered |
Indication, route of administration, vector type |
|
Market Analysis (Terms Used) |
Value (US$ Million/Billion) or (Volume/Units) |
|
Report Coverage |
Revenue forecast, company ranking, competitive landscape, growth factors, and trends |
|
Key Companies Profiled |
Amgen Inc.; Novartis AG; F. Hoffmann-La Roche; Gilead Sciences, Inc.; bluebird bio, Inc.; Bristol-Myers Squibb Company; Legend Biotech.; BioMarin.; uniQure N.V.; Merck & Co.; Sarepta Therapeutics, Inc.; Krystal Biotech, Inc.; CRISPR Therapeutics. |
Segment Insights:
By Indication Insights:
Based on the indication type, this industry is divided into many types, such as large B-cell lymphoma, multiple myeloma, spinal muscular atrophy (SMA), acute lymphoblastic leukemia, melanoma (lesions), inherited retinal disease, beta-thalassemia major/SCD, and others. Among these, large B-cell lymphoma holds the largest income share in the year 2024. So Diffuse Large B-cell Lymphoma (DLBL) is the most common Hodgkin Lymphoma (NHL) subtype. It is calculated for about 30%-40% of all cases.
While the retinal disease segment is expected to have the fastest growing CAGR over the forecast period. There is a rise in research and development activities for the development of gene therapies in order to treat ocular diseases, including inherited retinal disease, which has driven the segment. In addition to this, Pfizer Inc. has officially announced that the U.S. Food and Drug Administration has accepted the Supplemental Biologics License Application in combination with lenalidomide and a rituximab product for the diagnosis of adult patients with relapsed or refractory large B-cell lymphoma.
By Vector Type Insights:
Based on vector type, the U.S. gene therapy market is divided into different types named retrovirus and gamma retrovirus, lentivirus, AAV, adenovirus, modified herpes simplex virus, and others too. The lentivirus dominated the market, holding a share X% in the year 2024. Basically, lentivirus is used in the laboratory in order to merge DNA into a host genome to manipulate target genes as well as gene expression. It serves many advantages over conventional gene vectors like retroviruses, such as the ability to provide constant and long-term gene expression, the capability to induce themselves to non-dividing cells, the possible generation of replication-adept lentiviruses during vector preparation, and many more.
The AVV segment with respect to vector type is the largest growing field holding the market share in the year 2024. This leads to growth in demand for clinical trials pertaining to the progress of ocular and orthopedic gene therapy treatment, which resulted in developed efficiency and efficacy both simultaneously. Adding more to that, rising clinical trials, research, and developments are further responsible for the growth of the segment over the forecast period.
For instance, after five continuous years of receiving Fabry disease gene therapies AVR-RD-01, kidney function in all five participants remains constant according to the Canadian FACT study.
Route of Administration Insights:
The intravenous segment has dominated the U.S. gene therapy market, holding the CAGR over the forecast period. Chiefly, intravenous administration includes a shorter diagnosis time period and a less invasive process as compared to alternative routes such as intramuscular or subcutaneous injections. This increases convenience and lessens the discomfort for patients, hence enhancing treatment adherence and overall patient satisfaction by giving priority to intravenous gene therapy products.
U.S. Gene Therapy Market Recent Developments:
· On 6 August 2024, Precigen, which is a pharmaceutical company that has expertise in the development of innovative gene and cell therapies so as to improve the lives of patients, declared strategic reprioritization of the company’s clinical portfolio and streamlining the required resources.
· On 24 January 2024, Nkure, a Bangalore-based therapeutics cell and gene therapy, is trying its best to solve this by gaining hands-on immunotherapy solutions, as they have the capability to offer off-the-shelf reliable alternatives in cancer care and diagnosis both.
· On 29 January 2024, CPC (Colder Products Company), part of Dover, and a rising manufacturer of connection technologies that is used in biopharmaceutical companies, declared the launch of its new MicroCNX, which is a Nano Series aseptic connector that is drafted to solve cell and gene therapy closed sterile processing.
Immediate Delivery is Available | Get Full Report Access@ https://www.novaoneadvisor.com/report/checkout/8455
Related Report
· Gene Therapy Market - https://www.precedenceresearch.com/gene-therapy-market
· Cell and Gene Therapy Market - https://www.precedenceresearch.com/cell-and-gene-therapy-market
· Cancer Gene Therapy Market - https://www.precedenceresearch.com/cancer-gene-therapy-market
· Plastic Compounding Market - https://www.precedenceresearch.com/plastic-compounding-market
Top Companies Operating in the Gene Therapy Market:
The top organizations working or providing Gene therapy in the market are listed below:
· Bluebird Co
· Caribou Biosciences
· Editas Medicine
· Intelia Therapeutics
· Novartis
· UniQure
· Arcturus Therapeutics
· JW Therapeutics
· Poseida Therapeutics
· Tessera Therapeutics.
· BioMarin Pharmaceutical
· MeiraGTx Hldgs
· Sarepta Therapeutics
· Beam Therapeutics
· Regenxbio
· Sangamo Therapeutics
· 4D Molecular Therapeutics
· Adverum BioTechnologies
· Asklepios Biopharmaceuticals, Inc
· Atara Biotherapeutics
· Scribe Therapeutics
Segments Covered in the Report
This report forecasts revenue growth at country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the U.S. Gene Therapy market.
By Indication
· Large B-cell Lymphoma
· Multiple Myeloma
· Spinal Muscular Atrophy (SMA)
· Acute Lymphoblastic Leukemia (ALL)
· Melanoma (lesions)
· Inherited Retinal Disease
· Beta-thalassemia Major/SCD
· Others
By Route Of Administration
· Intravenous
· Others
By Vector Type
· Lentivirus
· RetroVirus & Gamma RetroVirus
· AAV
· Modified Herpes Simplex Virus
· Adenovirus
· Others
Immediate Delivery Available | Buy This Premium Research https://www.novaoneadvisor.com/report/checkout/8455
USA: +1 804 441 9344
APAC: +61 485 981 310 or +91 87933 22019
Europe: +44 7383 092 044
Email: sales@novaoneadvisor.com
Web: https://www.novaoneadvisor.com/
You can place an order or ask any questions, please feel free to contact at sales@novaoneadvisor.com | +1 804 441 9344
