High Investment and Government Initiatives to Boost Rare Diseases Treatment Modalities
Rockville, MD, Nov. 13, 2024 (GLOBE NEWSWIRE) — According to Fact.MR, a market research and competitive intelligence provider, the global gene therapy for SCID market is projected to grow at a robust CAGR 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 solely resulted in long-term T cell defect correction. 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.
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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.
Key Takeaways from the Market Study:
- The global gene therapy for SCID market is projected to grow at a robust CAGR and could reach US$ 2.0 billion by 2034
- Europe will hold the majority of market share in the future.
- Predominating market players include Orchard Therapeutics plc, Boston Children’s Hospital, and the University of California Los Angeles.
“Partnership and Collaboration in Developing Disease Specific Gene Therapy to Gain Traction in the forecast period,” says a Fact.MR analyst.
Leading Players Driving Innovation in the Gene Therapy for SCID Market:
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.
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.
- For instance, the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) where Telethon and Ospedale San Raffaele joined hands to develop Strimvelis gene therapy. It was then further advanced by GSK through the partnership that was initiated in the year 2010 between GSK, OSR, and Telethon. These collaborative strategies have further resulted in the approval of Strimvelis as well as a pipeline of more ex vivo gene therapies for treating diseases like MLD and WAS. That is why in the case of Libmeldy, a treatment option for the management of MLD in development, could be a telling example for looking for a practically applicable solution to the primary problem.
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.
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Gene Therapy for SCID Industry News:
- The successful transfer of the marketing authorisation for Strimvelis, the gene therapy approved by the European Medicine Agency in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID), was announced today in September 2023 by Orchard Therapeutics, a global gene therapy company, and Fondazione Telethon, a significant Italian biomedical charity organisation.
- A research and clinical partnership for the treatment of X-SCID was announced in February 2021 by Graphite Bio, Inc., a biotechnology company that develops gene-editing therapies to treat or cure serious diseases, and Jasper Therapeutics, Inc., a biotechnology company that develops blood stem cell therapies to cure a variety of diseases.
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.
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