The dynamics of the cell and gene therapies market in rare disorders are anticipated to change as companies across the globe are thoroughly working toward developing new therapeutic options to treat a wide array of indications.

LAS VEGAS, May 9, 2023 /PRNewswire/ --DelveInsight's Cell and Gene Therapies in Rare Disorders Market Insights report includes a comprehensive understanding of current treatment practices, emerging cell and gene therapies for various rare disorders, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Takeaways from the Cell and Gene Therapies in Rare Disorders Market Report

Discover which therapies are expected to grab the major cell and gene therapies in rare disorders market share @ Cell and Gene Therapies in Rare Disorders Market Report

Cell and Gene Therapies in Rare Disorders Overview

Cell and gene therapies use genes and cells to treat disease. A gene is a unit of DNA containing genetic information passed down from generation to generation. The genome comprises all genes; genes may contain information on observable features such as height or eye color. Many genes contain instructions for RNA or protein molecules that are not visible from the outside but serve crucial tasks 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 generate organs and tissues. Cell and gene therapy technology is quickly evolving for many different diseases. However, cell and gene treatments are still experimental drugs, and much more study is required before many of these therapies are available to patients worldwide.

Cell and Gene Therapies in Rare Disorders Epidemiology Segmentation

DelveInsight estimates that there were approximately 900K prevalent cases of selected indications for cell and gene therapies in rare disorders in the 7MM in 2022.

As per our analysis, the highest prevalent cases from the selected indications for cell and gene therapies in rare disorders were for Retinitis Pigmentosa in the United States, whereas the least cases were reported for Hunter Syndrome in 2022.

The cell and gene therapies in rare disorders market reportproffers epidemiological analysis for the study period 20192032 in the 7MM segmented into:

Cell and Gene Therapies in Rare Disorders Market Insights

Numerous cell and gene therapies for rare diseases are currently approved in the 7MM, including retinitis pigmentosa (LUXTURNA), beta-thalassemia (ZYNTEGLO), epidermolysis bullosa (JACE), limbal stem cell deficiency (OCURAL), and many others. As per Delveinsight analysis, the total cell and gene therapies in rare disorders market size was around USD 1.5 billion in 2022. According to predictions, the United States will have the largest cell and gene therapies in rare disorders market. ZOLGENSMA produced the highest revenue of roughly USD 1 billion among the 7MM in 2022, while ROCTAVIAN is predicted to take the highest market share by 2032. ROCTAVIAN has received conditional approval in Europe for the treatment of severe hemophilia A. In addition to this approval, BioMarin Pharmaceutical is working to approve the drug in the United States, with a PDUFA target action date of June 30, 2023.

Hemophilia A is predicted to produce the most revenue among the selected indications by 2032, owing to the precedence of existing high treatment cost and expected high cost for emerging therapies along with significant residual unmet need. Gene treatments for Hemophilia A are estimated to earn around USD 6 billion in sales revenue by 2032 in the 7MM. The field of cell and gene therapies for rare indications is expected to rapidly expand in the coming years, as an increasing number of companies submit investigational new drug applications for these treatments each year, along with rising regulatory approval in the United States and Europe. In terms of manufacturing aspects of cell and gene therapies, there will be more competition for contract manufacturing businesses and pharmaceutical/biotechnology firms. The competition for contract manufacturing organization's production capacity will intensify as more companies enter the cell and gene therapy market, possibly driving up manufacturing costs. To ensure pharmaceutical/biotechnology firms can compete or obtain an advantage over competitors, companies may need to invest in manufacturing technologies or acquire companies with manufacturing expertise.

To know more about cell and gene therapies in rare disorders treatment guidelines, visit @ Cell and Gene Therapy Insights

Emerging Cell and Gene Therapies for Hemophilia A and Key Companies

Emerging Cell and Gene Therapies for Hemophilia B and Key Companies

Emerging Cell and Gene Therapies for Fabry Disease and Key Companies

Emerging Cell and Gene Therapies for Pompe Disease and Key Companies

Emerging Cell and Gene Therapies for Leber Hereditary Optic Neuropathy and Key Companies

Emerging Cell and Gene Therapies for Retinitis Pigmentosa and Key Companies

Emerging Cell and Gene Therapies for Hunter Syndrome and Key Companies

Emerging Cell and Gene Therapies for Batten Disease and Key Companies

Emerging Cell and Gene Therapies for Duchenne Muscular Dystrophy (DMD) and Key Companies

Emerging Cell and Gene Therapies for Amyotrophic Lateral Sclerosis (ALS) and Key Companies

Emerging Cell and Gene Therapies for Beta Thalassemia and Sickle Cell Anemia and Key Companies

Emerging Cell and Gene Therapies for Dystrophic Epidermolysis Bullosa and Key Companies

Emerging Cell and Gene Therapies for Ornithine Transcarbamylase Deficiency and Key Companies

Emerging Cell and Gene Therapies for Sanfilippo Syndrome Type A and Key Companies

Emerging Cell and Gene Therapies for Glycogen Storage Disease Type IA and Key Companies

Learn more about the FDA-approved cell and gene therapies for rare disorders @ Approved Cell and Gene Therapies in Rare Disorders Treatment

Cell and Gene Therapies in Rare Disorders Market Dynamics

The cell and gene therapies in rare disorders market is predicted to grow positively due to an increase in the approval of a growing number of gene therapies and their ease of adoption following approval, the ability to treat a wide range of conditions, an increase in the number of cases, an expected one-time dosing approach, and curative treatment options.

The approval of LIBMELDY, SKYSONA, HOLOCLAR, UPSTAZA, ROCTAVIAN, and other medicines has successfully created regulatory channels for the development of further cell and gene therapies. Companies around the world are working hard to develop new cell and gene therapies options to treat a wide range 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), eye diseases (achromatopsia, 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.

Many diseases' treatment landscapes have drastically evolved in the last few years. Companies are now developing cell and gene therapies that will play an important role in the future, particularly in the treatment of rare genetic disorders. The process of defining ideal candidates for given gene therapy and cell therapy will have to wait for the enrolment and long-term follow-up of a sufficient number of study subjects to provide satisfactory clarity regarding its safety and efficacy. In conclusion, the future of cell and gene therapy looks optimistic. Several clinical trials have yielded favorable results in terms of safety and efficacy. The findings of these studies motivate additional research into many indications, and the current scenario predicts a positive shift in the cell and gene therapies in rare disorders market for the forecast period.

However, several factors may impede the growth of cell and gene therapies in rare disorders market in the coming years. Despite advances since the enactment of the Orphan Drug Act, people in the United States with rare diseases continue to face challenges to diagnosis, care, and treatment. Moreover, in Europe, a possible crisis with gene treatments for rare diseases is developing, and few companies have withdrawn the drug after failing to get reimbursement in the EU.

Report Metrics

Details

Study Period

20192032

Coverage

7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]

Base Year

2019

Cell and Gene Therapies in Rare Disorders Market CAGR

35.8%

Cell and Gene Therapies in Rare Disorders Market Size in 2022

USD 1.5 Billion

Key Cell and Gene Therapies in Rare Disorders Companies

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

Key Pipeline Cell and Gene Therapies in Rare Disorders Therapies

Giroctocogene fitelparvovec, Dirloctocogene samoparvovec, Fidanacogene elaparvovec, Verbrinacogene setparvovec (FLT-180a), FLT190, Isaralgagene civaparvovec (ST-920), 4D-310, SPK-3006, AT845, ACTUS-101, LUMEVOQ (lenadogene nolparvovec), CTx-PDE6b, Botaretigene sparoparvovec, ATGC-501 (laruparetigene zosaparvovec), GS030, MCO-010 (sonpiretigene isteparvovec), 4D-125, OCU400, jCell, RGX-121, AT-GTX-502 (scAAV9.P546.CLN3), PF-06939926, SRP-9001, CAP-1002, NurOwn (MSC-NTF cells), Exagamglogene autotemcel, EDIT-301, BIVV003, VYJUVEK (beremagene geperpavec), EB-101, D-Fi (dabocemagene autoficel), RV-LAMB3-transduced epidermal stem cells, Allogeneic ABCB5-positive Stem Cells, ISN001, ALLO-ASC-SHEET, DTX301, UX111 (ABO-102), DTX401 (AAV8G6PC), and others

Scope of the Cell and Gene Therapies in Rare Disorders Market Report

Discover more about cell and gene therapies for rare disorders in development @ Cell and Gene Therapy Clinical Trials

Table of Contents

1

Cell and Gene Therapies in Rare Disorders Market Key Insights

2

Cell and Gene Therapies in Rare Disorders Market Report Introduction

3

Cell and Gene Therapies in Rare Disorders Market Key Highlights from Report

4

Executive Summary of Cell and Gene Therapies in Rare Disorders

5

Key Events: Cell and Gene Therapies in Rare Disorders

6

Cell and Gene Therapies in Rare Disorders 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 of Cell and Gene Therapies in Rare Disorders

9

Epidemiology and Patient Population

10

Marketed Cell and Gene Therapies in Rare Disorders

10.1

Key Competitors in Cell and Gene Therapies in Rare Disorders

10.2

Hemophilia A

10.2.1

ROCTAVIAN (valoctocogene roxaparvovec): BioMarin Pharmaceutical

10.3

Hemophilia B

10.3.1

HEMGENIX (etranacogene dezaparvovec): CSL Behring/uniQure

10.4

Retinitis Pigmentosa

10.4.1

LUXTURNA: Sparks Therapeutics (a company of Roche)/ Novartis

10.5

Spinal Muscular Atrophy (SMA)

10.5.1

ZOLGENSMA (onasemnogene abeparvovec-xioi): Novartis (AveXis)

10.6

Metachromatic Leukodystrophy (MLD)

10.6.1

LIBMELDY (atidarsagene autotemcel): Orchard Therapeutics

10.7

Limbal Stem Cell Deficiency

10.7.1

HOLOCLAR (Autologous human corneal epithelial cells containing stem cells): Holostem Terapie Avanzate S.r.l.

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