Gene And Cell Therapies In Rare Disorder Market Size in the 7MM is anticipated to grow by 2032 | DelveInsight

DelveInsight's "Gene And Cell Therapies In Rare Disorder Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Gene And Cell Therapies In Rare Disorder (CRBSI), historical and forecasted epidemiology as well as the Gene And Cell Therapies In Rare Disorder (CRBSI) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Key Takeaways from the Gene And Cell Therapies In Rare Disorder Market Report

* The increase in Gene And Cell Therapies In Rare Disorder Market Size is a direct consequence of the increasing patient population and anticipated launch of emerging therapies in the 7MM.
* As per DelveInsight analysis, the Gene And Cell Therapies In Rare Disorder Market is anticipated to witness growth at a considerable CAGR.
* The total prevalent cases of selected indications for Cell and Gene Therapies in Rare Disorders in the 7MM comprised approximately 900,000 in 2022 and are projected to increase during the forecasted period
* Among all the rare indications selected for Cell and Therapies, the highest cases were for Retinitis Pigmentosa, followed by Limbal Stem Cell Deficiency in 2019.
* Also, in the 7MM, the total treated cases of indication-wise cell and gene therapies were approximately 500,000 in 2022, which is expected to grow during the forecast period, i.e., 2023-2032.
* The leading Gene And Cell Therapies In Rare Disorder Companies working in the market include Pfizer, Sangamo, Roche, Freeline Therapeutics, 4D Molecular Therapeutics, Astellas Gene Therapies, Actus Therapeutics, GenSight Biologics, Coave, Johnson & Johnson, MeiraGTx, GenSight Biologics, Nanoscope Therapeutics, Ocugen, jCyte, ReNeuron, REGENXBIO, Amicus, Sarepta, Capricor, Nippon Shinyaku, Brainstorm Cell Therapeutics, CRISPR Therapeutics/Vertex Pharmaceuticals, Editas Medicine, Krystal Biotech, Abeona Therapeutics, Castle Creek Biosciences, Holostem Terapie Avanzate S.r.l., RHEACELL, Ishin Pharma, Anterogen, Ultragenyx Pharmaceutical, and many others.
* Promising Gene And Cell Therapies In Rare Disorder Pipeline Therapies in the various stages of development include A166, and others.

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Gene And Cell Therapies In Rare Disorder Overview

Cell and gene therapies use genes and cells to treat disease. A gene is the unit of DNA that contains hereditary information passed down from generation to generation. All genes are called the genome; genes may contain information about visible traits, such as height or eye color. Many genes contain instructions for RNA or protein molecules that are not visible outside but perform important functions in the body's cells. Cells are the building blocks of plants and animals (including humans); they are small functional units that work together to form organs and tissues.

Gene And Cell Therapies In Rare Disorder Epidemiology Insights

The epidemiology section of Gene And Cell Therapies In Rare Disorder offers insights into both historical and current patient populations, as well as forecasted trends across seven major countries. This section aids in understanding the factors behind present and projected trends through analysis of various studies and input from key opinion leaders. Additionally, this portion of the market report provides information on the diagnosed patient pool, trends, and underlying assumptions.

* Total Gene And Cell Therapies In Rare Disorder Prevalent Cases of selected indications
* Total Gene And Cell Therapies In Rare Disorder Indication-wise eligible cases
* Gene And Cell Therapies In Rare Disorder Indication-wise treated cases

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Gene And Cell Therapies In Rare Disorder Marketed Drugs

* ZOLGENSMA (AVXS-101): Novartis/AveXis
* LUXTURNA: Spark Therapeutics (Roche)/Novartis

Gene And Cell Therapies In Rare Disorder Emerging Drugs

* Fidanacogene elaparvovec (SPK-9001/PF-06838435): Pfizer/Spark Therapeutics
* AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics
* FLT190: Freeline Therapeutics
* RGX-121: Regenxbio

Gene And Cell Therapies In Rare Disorder Drugs Market

The Gene And Cell Therapies In Rare Disorder Drugs Market is expected to witness substantial growth in the coming years as more targeted therapies receive regulatory approval and enter clinical practice. Additionally, ongoing research efforts aimed at better understanding the molecular mechanisms underlying Gene And Cell Therapies In Rare Disorder signaling in Gene And Cell Therapies In Rare Disorder are likely to uncover new therapeutic targets and further expand treatment options for patients.

Gene And Cell Therapies In Rare Disorder Treatment Market Landscape

The Gene And Cell Therapies In Rare Disorder treatment market landscape has witnessed significant advancements over the past years, with targeted therapies playing a crucial role in improving outcomes for patients. Among these targeted therapies, the emergence of Gene And Cell Therapies In Rare Disorder has garnered attention in the medical community, presenting both challenges and opportunities in treatment strategies.

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Gene And Cell Therapies In Rare Disorder 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 therapies 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.

Gene And Cell Therapies In Rare Disorder Drugs Uptake

The drug chapter of the Gene And Cell Therapies In Rare Disorder report provides a comprehensive analysis of both marketed drugs and late-stage pipeline drugs for this condition. It delves into the details of clinical trials, pharmacological actions, agreements, collaborations, approvals, patents, and advantages, and disadvantages of each drug, as well as the latest news and press releases related to Gene And Cell Therapies In Rare Disorder.

Major Gene And Cell Therapies In Rare Disorder Companies

Several Gene And Cell Therapies In Rare Disorder Companies working in the market include Pfizer, Sangamo, Roche, Freeline Therapeutics, 4D Molecular Therapeutics, Astellas Gene Therapies, Actus Therapeutics, GenSight Biologics, Coave, Johnson & Johnson, MeiraGTx, GenSight Biologics, Nanoscope Therapeutics, Ocugen, jCyte, ReNeuron, REGENXBIO, Amicus, Sarepta, Capricor, Nippon Shinyaku, Brainstorm Cell Therapeutics, CRISPR Therapeutics/Vertex Pharmaceuticals, Editas Medicine, Krystal Biotech, Abeona Therapeutics, Castle Creek Biosciences, Holostem Terapie Avanzate S.r.l., RHEACELL, Ishin Pharma, Anterogen, Ultragenyx Pharmaceutical, and many others.

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Scope of the Gene And Cell Therapies In Rare Disorder Market Research Report

* Coverage- 7MM

* Gene And Cell Therapies In Rare Disorder Companies- Pfizer, Sangamo, Roche, Freeline Therapeutics, 4D Molecular Therapeutics, Astellas Gene Therapies, Actus Therapeutics, GenSight Biologics, Coave, Johnson & Johnson, MeiraGTx, GenSight Biologics, Nanoscope Therapeutics, Ocugen, jCyte, ReNeuron, REGENXBIO, Amicus, Sarepta, Capricor, Nippon Shinyaku, Brainstorm Cell Therapeutics, CRISPR Therapeutics/Vertex Pharmaceuticals, Editas Medicine, Krystal Biotech, Abeona Therapeutics, Castle Creek Biosciences, Holostem Terapie Avanzate S.r.l., RHEACELL, Ishin Pharma, Anterogen, Ultragenyx Pharmaceutical, and many others.

* Gene And Cell Therapies In Rare Disorder Pipeline Therapies- A166
* Gene And Cell Therapies In Rare Disorder Market Dynamics: Gene And Cell Therapies In Rare Disorder Market Drivers and Barriers
* Gene And Cell Therapies In Rare Disorder Market Access and Reimbursement, Unmet Needs and Future Perspectives

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Table of Content

1. Key Insights

2. Executive Summary of Gene And Cell Therapies In Rare Disorder (CRBSI)

3. Competitive Intelligence Analysis for Gene And Cell Therapies In Rare Disorder (CRBSI)

4. Gene And Cell Therapies In Rare Disorder (CRBSI): Market Overview at a Glance

5. Gene And Cell Therapies In Rare Disorder (CRBSI): Disease Background and Overview

6. Patient Journey

7. Gene And Cell Therapies In Rare Disorder (CRBSI) Epidemiology and Patient Population

8. Treatment Algorithm, Current Treatment, and Medical Practices

9. Gene And Cell Therapies In Rare Disorder (CRBSI) Unmet Needs

10. Key Endpoints of Gene And Cell Therapies In Rare Disorder (CRBSI) Treatment

11. Gene And Cell Therapies In Rare Disorder (CRBSI) Marketed Products

12. Gene And Cell Therapies In Rare Disorder (CRBSI) Emerging Therapies

13. Gene And Cell Therapies In Rare Disorder (CRBSI): Seven Major Market Analysis

14. Attribute analysis

15. 7MM: Market Outlook

16. Access and Reimbursement Overview of Gene And Cell Therapies In Rare Disorder (CRBSI)

17. KOL Views

18. Market Drivers

19. Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

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