Published by Wanda Rich
Posted on March 19, 2025
Growing demand for research & development in SCID, raising awareness, and newborn screening (NBS) modalities in the management of SCID to boost the global market, States Fact.MR.
According to Fact.MR, a market research and competitive intelligence provider, the global gene therapy for SCID market is projected to grow at a robustCAGR and could reach US$ 2.0 billion by 2034.
The idea of gene therapy was proposed more than 50 years ago at the time molecular biology principles had been defined and the first disease−inducing genetic mutations were identified. However, it was not until nearly 30 years and several associated advances that it turned into a reality. European regulatory bodies have given a nod to one gene therapy product of primary immunodeficiency (PID) to cure ADA SCID, essentially, Strimvelis. Interestingly, SCID was the first disease known to be successfully treated through gene therapy even as it was the first and only indication of allogeneic HSC transplantation.
SCIDs are inherited immuno-deficiency diseases that lead to an early block in T-cell development with variable abnormalities of other lymphoid (or occasionally myeloid) lineages. γRV vector-based gene therapy for SCID X1 resulted in long-term correction of T cell defect solely. Therefore, the enhancement of mild myeloablation such as that done for gene therapy of ADA deficiency might favor the engraftment of the transduced HSCs, so that the differentiation of B-cells as well as NK cells would be enhanced. So far, fourteen kinds of SCID have been found. Most of the SCID types are inherited in a recessive manner. To name a few, are X-linked SCID, Adenosine Deaminase Deficiency SCID, IL7R Deficiency SCID, and RAG-1 AND RAG-2 Deficiency SCID among others.
Moreover, the LV vectors developed from HIV are tenfold more effective than γRV in transducing the HSCs. Therefore, the LV vectors, usage was anticipated to enhance the transduction of more HSCs as compared to the previous trials. This aforementioned understanding of the diseases and treatment modalities will show robust growth in the future.
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Key Takeaways from the Market Study:
“Partnership and Collaboration in Developing Disease Specific Gene Therapy to Gain Traction in the forecast period,” says a Fact.MR analyst.
Market Development:
An efficient and safe gene therapy approach has been identified for two SCIDs and WAS after 20 years. Gene therapy has shown very positive effects in the cure of SCID patients. This process includes introducing a viral vector into the patient’s HSCs where the faulty gene is inserted and further becomes part of the person’s DNA and subsequently, the corrected cells are brought back into the body. It has been applied successfully for the cure of X-linked SCID individuals and ADA-deficient SCID. However, some of the initial (first) attempts were not without problems like graft failures and other side effects that accompanied insertions of vectors including lymphoproliferation, and leukemias, particularly in X-SCID cases. To minimize these risks, some modifications have been implemented in the construction of retroviral vectors.
Pharmaceutical companies and non-profit agencies/organizations have been working together for gene editing therapy or cure of diseases. These collaborations and partnerships illustrate mutual commitment towards developing innovative therapeutic interventions that hold great promise in enhancing an individual’s quality of life with suitable treatment and screening modalities that are likely to benefit from gene replacement therapies.
FDA has been supporting manufacturers to produce or gain interest in orphan drugs like OTL-101 which is an investigational autologous ex vivo lentiviral hematopoietic stem cell-based gene therapy for AD-SCID. OTL-101 has been designated with breakthrough therapy in the rare disease field which is FDA and EMA regulated and has received a Rare Pediatric Disease Designation from the FDA.
Gene Therapy for SCID Market News:
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More Valuable Insights on Offer:
Fact.MR, in its new offering, presents an unbiased analysis of the global gene therapy for the SCID market, presenting historical data for 2019 to 2023 and forecast statistics for 2024 to 2034.
The segmentation of the Gene Therapy for SCID industry research is categorized based on various key factors. By type of SCID, the market is divided into X-Linked SCID, ADA-SCID, and other SCID types. Based on therapy type, it includes Gene Replacement Therapy, Gene Editing Therapy, and Stem Cell Gene Therapy. In terms of vector type, the industry is segmented into Viral Vectors and Non-Viral Vectors. The market is also classified by patient age group, covering Pediatric Patients and Adult Patients. Additionally, based on pricing models, the segmentation includes Single-dose treatments and Multi-dose treatments. Geographically, the market is divided into North America, Latin America, East Asia, South Asia & Pacific, Western Europe, Eastern Europe, and the Middle East & Africa.
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The global gene therapy market size is calculated at a value of US$ 10.34 billion in 2024. Increasing cases of rare diseases is contributing to the rising need for cell and gene therapies. Expanding at a CAGR of 14.1%, the market is forecasted to reach US$ 38.52 billion by the end of 2034.
The global gene delivery systems market currently accounts for a valuation of US$ 3 billion and is expected to reach US$ 4.1 billion by the end of 2027. Global demand for gene delivery systems is predicted to magnify at a CAGR of 6.5% from 2022 to 2027.
Global demand for CRISPR and Cas genes is anticipated to surge at a stupendous CAGR of 20% from 2023 to 2033. At present, the global CRISPR and Cas genes market is valued at US$ 2.6 billion and is thus expected to reach a market size of US$ 16 billion by the end of 2033.
The lentiviral vector market size is valued at US$ 170.7 million in 2024, and it is estimated to expand at an 11.5% CAGR, reaching a valuation of US$ 506.7 million by 2034.
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