Gene Therapy for SCID Market Outlook (2024 to 2034)
The global gene therapy for SCID market is projected to grow at a robust CAGR and could reach up to US$ 2.0 billion by 2034.
Severe combined immunodeficiency (SCID) is a rare condition with very high mortality. The multiple mutations of genes affect the complete immune system of newborns, where the patient's immune system does not function up to the mark or is impaired. Newborns may appear healthy at the beginning but the defects of the immune system will progress and show secondary infections like meningitis and respiratory infections that give rise to diarrhea, inflammation of the skin, and other systemic diseases. SCID also come with a popular name called “bubble boy syndrome” and “living in the bubble”. In the early stages of treatment, doctors used to isolate the newborn patients completely until bone marrow transplantation is done, which was the ideal treatment method. It is regarded as one of the pediatric emergencies and requires immediate attention. As SCID results from multiple mutated genes, its diagnosis and management becomes difficult for healthcare professionals.
However, the current management of SCID is usually fast and accurate due to the development of advanced newborn screening modalities and other monitoring systems. Now a days the prognosis and diagnosis of SCID are possible in the first three months. SCID is treatable with hematopoietic stem cell transplantation and gene therapy along with conditioning regimen with chemotherapies and alternative therapies such as enzyme replacement therapy among others.
Historic Analysis (2019 to 2023) and Future (2024 to 2034) Pathway Analysis for the gene therapy for SCID market
Newborn screening started in the United States in 1961 and, after the advent of tandem mass spectrometry, this technology has been widely utilized in developed countries for the early diagnosis and treatment of phenylketonuria and other metabolic disorders. Nearly all countries have only recently added testing for a panel of additional genetic conditions into their NBS programs using DNA analysis. NBS (Newborn Screening) for SCID by the TREC assay was initiated in 2008 in Wisconsin and, since then, it has expanded to most of the states in the US, and in other countries such as Taiwan, Israel, New Zealand, Canada, Norway, Spain, etc. A 2019 paper published in Frontiers in Immunology estimated the incidence of SCID as 1 in 56,000 with a 92% survival rate post-treatment with gene therapy. SCID being a T-cell primary immunodeficiency (PID) most severe form, consists of a genetically characterized group of severe T-cell lymphopenia (TCL) diseases. The SCID infant screening program can also pick up other prominent forms of TCL (T-cell lymphopenia) such as 22q11 deletion, Down syndrome, and CHARGE syndrome, which occurs in 1 per 7,300 newborns. An article entitled "First Universal Newborn Screening Program for Severe Combined Immunodeficiency in Europe. Two-Years' Experience in Catalonia (Spain)” in 2019, noted that the TREC quantification utilized as a basis for NBS for SCID in Catalonia "does not only identify SCID patients but also other non-SCID lymphopenic conditions". These understanding of diseases and initiatives opened up pathways to novel treatment modalities from 2019 to 2023 through gene therapy.
- Short Term (2024 to 2028): Gene therapy for SCID has a promising future. Government and private organizations’ initiatives have spread awareness about newborn health. A new strategy that comes forward is making gene therapy available to newborn babies. Great Ormond Street Hospital Children's Charity, LifeArc, and Great Ormond Street Hospital have made a vision to address needs like those regarding regulatory challenges of new treatment drugs and to encourage hospitals to obtain licenses that provide better care access for patients. There has been an attempt to trial lentiviral gene therapy for ADA-Severe Combined Immuno-deficiency developed in collaboration with Great Ormond Street Hospital, the UCL Great Ormond Street Institute of Child Health, and UCLA. ADA-SCID is an ultra-rare disease where the development of an immune system is hindered. It is diagnosed every year in few children who become victims of serious infections. People with ADA-SCID have in them a faulty gene inhibiting the production of the necessary enzyme ADA to develop immune cells. Gene therapy replaces the faulty gene with a healthy gene to produce ADA to treat ADA-SCID. These factors are expected to boost the market growth significantly until the year 2027.
- Medium Term (2028 to 2030): In the medium term, the market will reach substantial growth due to the availability of medicines for the cure of SCID. Manufacturers will strategies in emerging geographies with their high-end product research & development activities. New reimbursement frameworks will tag along in the process to make the treatment and management of diseases affordable in underdeveloped markets.
- Long Term (2030 to 2034): Gene therapy in SCID has a lot to give and there has been immense development in the diagnosis and treatment of SCID. Researchers are focusing on how to understand immune reconstitution rather than focusing on B-cells, NK cells, and T- cells. The peculiar focus is on avoiding chemotherapy for better engraftment in the initial days of the newborn. This encourages market players to develop innovative treatment modalities and invest in studying the prognosis of disease leading to promising growth in the long term.
On the back of the aforementioned facts, the gene therapy for SCID market is anticipated to be valued around US$ 2 billion by the end of 2034, according to the Fact.MR, a market research and competitive intelligence provider.
Market Dynamics
What are the Factors Propelling Global Sales of Gene Therapy for SCID?
“Exhaustive Research and Development & New Screening Techniques to Enhance Treatment Modalities”
SCID is said to be a PID that affects the immune system of a newborn, and the body’s immune system does not work to fight even minor infections. As the research has been advancing, scientists have classified many molecular factors that can cause genetic alterations that result in SCID. Growing adoption rate of high-end screening modalities where SCID can be diagnosed within 3 months or even before the symptoms appear. Integration of SCID newborn screening based on T-cell receptor excision circle detection (TREC) into the respective NBS programs has shown a rise in the diagnosed patient pool of SCID as compared to the historic years. Affordable screening costs, raising awareness, and extending NBS applications will create market opportunities in the coming years.
Public health programs should continuously optimize screening programs for their stakeholders in pursuit of the most efficient screening strategy that balances high sensitivity while preventing high numbers of referrals. In 2005, the introduction of quantitative PCR for TREC detection in a large-scale population screening for SCID was reported. With suitable SCID detection methods, an understanding of the disease course, and a health problem that needs immediate attention are the screening criteria for detecting SCID proposed by Wilson and Jungner. The cost-effectiveness of NBS for SCID and robust knowledge for disease treatment and management will further drive the market growth.
What are the Challenges Encountered by Manufacturers of Gene Therapy for SCID?
“High Cost and Commercial Viability to Hamper Market Growth”
Gene therapy is indicative for several clinical conditions some of which are one in a million. Gene therapy, in the management and treatment of SCID, stands to save patients' lives however, the high costs taken to develop treatment do not make it seem like a promising investment for pharmaceutical companies with less market reach. Developing a new therapy, and completing all phases of clinical trials for regulatory approvals can be prohibitively expensive for the manufacturers. Considering, the lower count of diagnosed individuals and people opting for treatment and insurance coverage, it may be virtually impossible for leading manufacturers and or start-ups to profit from developing treatments for such rare genetic disorders. Some patients may just never be in a position to pay for these treatments.
Gene therapy is indeed a promising modality that works miraculously if personalized to each individual. For example, cells are removed from a patient, further addition of therapeutic genes, and lastly, the cells are transferred back to the person. Such an individualistic approach might be very powerful but prohibitively expensive. The costs are much higher than drugs made in quantity then can fast-track recovery of their development costs. If pharmaceutical companies decide that, a gene therapy treatment is unprofitable, manufacturers will come up with strategies that can be beneficial to the rich and the reach in underdeveloped countries will hamper the growth of the market in the future.
Country-wise Insights
Why is the Adoption of Gene Therapy for SCID High in the Europe?
“Rise in Clinical Trials and Advanced Healthcare Infrastructure to Show Significant Growth”
The rise in the number of clinical trials and gradually growing interest in corporate involvement in research and development activities will significantly influence gene therapy for SCID in the Europe. Gene therapy in oncology and other rare disorders has proven to show effective results in the management of diseases, furthermore, the gene therapy procedure has widely emerged in neurological, autoimmune, cardiovascular, musculoskeletal, and dermatological disorders. This creates market growth opportunities in the SCID market. This rise in clinical trials will encourage manufacturers to develop, and invest in research & development activities to show significant drug development in the coming years. With such increasing numbers of groundbreaking approvals, like the first CRISPR-based therapeutic approved for sale in the Europe, significant traction will likely be garnered going into 2024 and beyond.
Why is China a Lucrative Market for Gene Therapy for SCID?
“Government Initiatives Promoting New Policies to Avoid Product Similarity”
Chinese doctors were unaware of information on rare diseases; thus, researchers can often take years, even decades, to search for an accurate diagnosis for these rare diseases. In China, more than 20 million people in the country are estimated to be affected by these rare clinical conditions. Before 2018, China did not have any rare disease identification or treatment policies, but new initiatives taken in the last few years have shown remarkable improvements. The possibility of treating diseases once considered untreatable has helped to see gene therapy much deeper, thanks to clinical practices and methodologies that are just growing a lot faster these days. In China, there is no unified model regarding the screening and diagnosis of SCID. Recently, the diagnosis and treatment of subjects with IEI (Inborn Errors of Immunity) have significantly improved in mainland China through the development of genome sequencing, genetics, immunology, and respective treatment approaches. There still exists a large gap in either access or affordability for diagnostic facilities and precision treatments between different parts. Thus, the government paid more attention to the prevention, diagnosis, and treatment of rare diseases in recent years, and further development could be predicted in IEI in mainland China. This gap created an opportunity for the market to show promising growth in China.
Category-wise Insights
What are the Treatment Options for ADA-SCID and How Promising are they?
“Potential approach for Broader application of Gene Therapy and Regulatory Approvals to Boost Market Growth”
Strimvelis is developed for the treatment of Adenosine Deaminase Deficiency-Related Combined Immunodeficiency Syndrome more commonly known as ADA-SCID. This is the first ex vivo gene therapy that has been approved for the treatment of ADA-SCID and has been described as possibly being a cure. Strimvelis is performed by gene correction of a patient’s hematopoietic stem cells, which enables them to produce functional ADA because of the genetic modification and does not require enzyme replacement therapy or stem cell transplantation from other people. Early evidence of Strimvelis also indicates its long-term therapeutic effectiveness as a patient’s immune function recovery together with over a decade of survival was observed during clinical trials. Strimvelis is Recognized as a ground-breaking cure for ADA-SCID and its long-term success looks promising.
As for Libmeldy, a treatment developed for metachromatic leukodystrophy (MLD) treatment, however, It has also shown effects of improvement in treating SCID. It has been tested to be effective in treating other inherited immunodeficiency diseases that are related to SCID by correcting the gene in the stem cells. As it was mentioned not all gene therapy is applicable for SCID, however, the approach used in Libmeldy could be modified in order to treat certain forms of SCID. It shows that there is more to gene therapies implicating the immune system than what existed previously.
OTL-101 is an experimental work on the line of gene therapy solely for ADA-SCID. Created by Orchard Therapeutics it is based on the use of a lentiviral vector where a functional copy of the ADA gene is inserted into a patient’s stem cell. Clinical experience obtained along OTL-101’s phase 3 clinical trial has presented maximum patient recovery with complete immune restoration and overall survival rates. It is believed to be a therapeutic mode of treatment because it is to offer a cure for SCID since it directly targets its cause.
Competitive Landscape
Key players in the gene therapy for SCID market are Orchard Therapeutics plc., OSR and Telethon, Jasper Therapeutics, Inc., Graphite Bio, Inc., Mustang Bio Inc., University of California San Francisco, Genethon SA, Boston Children's Hospital, Shenzhen Geno-Immune Medical Institute, University College London, University of California Los Angeles, and The National Institute of Allergy and Infectious Diseases.
Companies devote a significant portion of their revenue to R&D to resolve problems and offer creative solutions to draw in new customers and stay competitive. Partnerships and collaboration in manufacturing and or distribution to meet industry-specific needs to compete with other players.
- In September 2023, Fondazione Telethon, a major Italian biomedical charity organization, and Orchard Therapeutics, a global gene therapy company today declared the successful transfer of the marketing authorization for Strimvelis, the gene therapy approved by the European Medicine Agency in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).
- In February 2021, Jasper Therapeutics, Inc., a biotechnology company developing blood stem cell therapies to cure a range of diseases, and Graphite Bio, Inc. a biotechnology company developing gene-editing therapies to treat or cure serious diseases, said that they will establish a research and clinical partnership for a treatment of X-SCID.
Fact.MR has provided detailed information about the price points of key manufacturers of gene therapy for SCIDs positioned across regions, sales growth, production capacity, and speculative technological expansion, in the recently published report.
