DelveInsight’s “Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of the cell and gene therapies in rare disorders, historical and forecasted epidemiology as well as the Cell and Gene Therapies in Rare Disorders market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
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Key Takeaways from the Gene And Cell Therapies In Rare Disorder Market
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Gene And Cell Therapies In Rare Disorder Overview
Gene and cell therapies represent advanced medical treatments that offer potential cures or significant improvements for patients with rare disorders. Gene therapy involves modifying or manipulating the genes within an individual’s cells to treat or prevent disease. For rare disorders, which are often genetic in origin, gene therapy can provide a transformative approach by targeting the root cause of the disease.
Gene And Cell Therapies In Rare Disorder Epidemiology Segmentation
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Cell and Gene Therapies in Rare Disorders Marketed Drugs
ZOLGENSMA (onasemnogene abeparvovec-xioi), previously known as AVXS-101, is a proprietary gene therapy of Novartis designed for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
LUXTURNA (AAV2-hRPE65v2), known as voretigene neparvovec-rzyl, is a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. The drug was developed and commercialized in the US by Spark Therapeutics. In Europe, Novartis is currently marketing LUXTURNA as per a licensing agreement covering the development, registration, and commercialization rights of LUXTURNA in markets outside the US. It is indicated for the treatment of patients with vision loss due to Leber’s congenital amaurosis or retinitis pigmentosa inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations.
Cell and Gene Therapies in Rare Disorders Emerging Drugs
Amicus Therapeutics is developing adeno-associated virus serotype 9 AAV9-CLN3 (AAV9-CLN3) gene therapy for children with CLN3 Batten disease. The company is leading a Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9. Batten disease is caused by mutations in one of 14 different genes, designated CLN1 to CLN14. The mutated gene does not work properly, ultimately leading to disease. The idea behind gene therapy is to deliver a working version of the gene into a patient’s cells — conceptually, replacing the faulty gene.
FLT190 is a next-generation gene therapy that uses an AAV8 as a vehicle to deliver a healthy copy of the GLA gene in the hopes that it will induce the production of normal alpha-GAL A. In contrast to the regular infusions of enzyme replacement therapy (ERT), this gene therapy is designed to be given in a single dose. Preclinical data using a gene therapy platform for expressing the enzyme a-galactosidase A (GLA) has demonstrated safety and efficacy in various animal models.
RGX-121 is being developed as a novel, one-time treatment for MPS II, which is directly administered intra-cisternally into the CNS. It includes the NAV AAV9 vector encoding for human IDS (iduronate-2-sulfatase). Treatment with RGX-121 has been shown to restore IDS enzyme activity in animal models of MPS II to levels equivalent to or greater than those in non-affected animals. The extent of CNS correction in animal studies suggests that RGX-121 has the potential to be an important and suitable therapeutic option for MPS II patients.
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Gene And Cell Therapies In Rare Disorder Therapies and Companies
Cell and Gene Therapies in Rare Disorders Market Outlook
Approval of LIBMELDY, SKYSONA, HOLOCLAR, UPSTAZA, ROCTAVIAN, and other therapies has successfully paved regulatory pathways of other cell and gene therapies currently under development. The dynamics of the cell and gene therapies market are anticipated to change as companies across the globe are thoroughly working toward the development of new cell and gene therapy options to treat a wide array of indications such as hemophilia A and B, lysosomal storage disorder (Fabry, Pompe Disease, Danon Disease, MPS I, MPS II, MPS III), neurological disorders (Batten, Parkinson), musculoskeletal disorders (DMD, myotubular myopathy, LGMD), eye diseases (achromatopsia, choroideremia, limbal stem cell deficiency, retinitis pigmentosa, retinoschisis, age-related macular degeneration, Leber’s hereditary optic neuropathy), and other indications such as diabetic macular edema, inborn metabolism disorder (Wilson’s disease, Phenylketonuria, OTC deficiency/urea cycle disorders), dystrophic epidermolysis bullosa, gangliosidosis, and xerostomia.
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Scope of the Gene And Cell Therapies In Rare Disorder Market
Table of Content
1. Key Insights
2. Report Introduction
3. Key Highlights From the Report
4. Executive Summary of Cell and Gene Therapies in Rare Disorders
5. Key Events
6. Epidemiology and Market Forecast Methodology
7. Cell and Gene Therapies in Rare Disorders Market Overview at a Glance in the 7MM
8. Disease Background and Overview
9. Epidemiology and Patient Population
10. Marketed Drugs
11. Emerging Drugs
12. Cell and Gene Therapies in Rare Disorders: 7MM analysis
13. Unmet Needs
14. SWOT Analysis
15. KOL Views
16. Market Access and Reimbursement
17. Appendix
18. DelveInsight Capabilities
19. Disclaimer
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
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