DelveInsight Business Research LLP

DelveInsight’s “Cell and Gene Therapies in Rare Disorders – Market Insights, Epidemiology and Market Forecast – 2032” 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.

Key Highlights Gene and Cell Therapies in the Rare Disorder  Market 

• Key Companies working in the Gene and Cell Therapies in Rare Disorder market are Pfizer, Roche, 4D Molecular Therapeutics, Coave Therapeutics, and many others 

• Key Therapies in Gene and Cell Therapies in Rare Disorders are ZOLGENSMA (AVXS-101), LUXTURNA, Fidanacogene elaparvovec , AT-GTX-502, and many others 

• The market size for the Gene and Cell Therapies in the Rare Disorder  market is USD 6 Billion by 2032 

• The CAGR for the Gene and Cell Therapies in the Rare Disorder market is 35%

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 therapy is the use of genetic material to treat genetic diseases. This may involve adding a wild-type copy of the gene (gene addition) or altering a gene with a mutation to the wild-type gene (gene editing). The treatment may occur outside the body (ex vivo) or inside the body (in vivo). Modified viruses or other vectors are used to get the gene into the genome inside the cells. Cell therapy uses cells from the patient themselves or a donor to treat diseases. Cells used for cell therapy are often stem cells, which can mature into specialized cells. Cells used for cell therapy may or may not be genetically altered. It is sometimes easier to remove cells from the body, treat them with gene therapy, and then place them back than treat the cells inside the body. This is the case for gene therapy for blood disorders; cell and gene therapies often go together. Cell and gene therapy technology is evolving rapidly for many different diseases. However, cell and gene therapies remain experimental medicines, and much more research is needed before many of these therapies are available to patients worldwide.

Gene and Cell Therapies in Rare Disorder Epidemiology Insights

• 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

• 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.

Click here to learn more about the Gene and Cell Therapies in Rare Disorder  Market Landscape

 The Report Covers the Gene and Cell Therapies in Rare Disorder Epidemiology Segmented by:

• Gene and Cell Therapies in Rare Disorder prevalent cases  

• Gene and Cell Therapies in Rare Disorder incident cases 

• Gene and Cell Therapies in Rare Disorder treatment cases 

• Gene and Cell Therapies in Rare Disorder diagnosed cases 

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, EGFR-Induced Skin Disorders  Market, 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.

In the past few years, the treatment landscape of many diseases has rapidly changed. Now companies are developing cell and gene therapies that will have a promising role in the future, especially for treating rare genetic diseases. The task of defining appropriate candidates for given gene therapy and cell therapy will need to await the enrollment and long-term follow-up of a sufficient number of study subjects to provide acceptable clarity about its safety and efficacy. To summarize, the outlook for cell and gene therapies is promising. Various clinical trials have been fairly positive in terms of safety and efficacy. The results of these studies encourage further investigation into multiple indications, and the current scenario also anticipates a positive shift in the market for the forecast period.

Key Companies Working in the Gene and Cell Therapies in Rare Disorder Market

• Pfizer: ZOLGENSMA (AVXS-101)

• Roche:  LUXTURNA

• 4D Molecular Therapeutics : Fidanacogene elaparvovec

• Coave Therapeutics: AT-GTX-502

And many others

 Gene and Cell Therapies in Rare Disorders Covered and Analyzed in the Report

• ZOLGENSMA (AVXS-101)

• LUXTURNA

• Fidanacogene elaparvovec

• AT-GTX-502

 And many others 

 Learn more about the Key Companies and Emerging Therapies in the Gene and Cell Therapies in Rare Disorder Market

 Table of Contents 

1.  Key Insights 

2.  Gene and Cell Therapies in Rare Disorders  Introduction 

3.  Executive Summary of Gene and Cell Therapies in Rare Disorder            

4.  Disease Background and Overview

5.  Epidemiology and patient population

6.  Gene and Cell Therapies in Rare Disorders Emerging Therapies

7.  Gene and Cell Therapies in Rare Disorders Market Outlook

8.  Market Access and Reimbursement of Therapies

9.  Market Drivers 

10.  Market Barriers 

11.  Appendix

12.  Report Methodology

13.  DelveInsight Capabilities

14.  Disclaimer

Learn about the detailed offerings of the report @ Gene and Cell Therapies in Rare Disorder  Market Outlook

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