Gene And Cell Therapies In Rare Disorders Market Size in the US is ~USD 600 million in 2022, estimates DelveInsight

"The Cell and Gene Therapies in Rare Disorders market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period (2023-2032). Furthermore, launching various multiple- Gene And Cell Therapies In Rare Disorders pipeline products will significantly revolutionize the Gene And Cell Therapies In Rare Disorders market dynamics"

The cell and gene therapies in rare disorders market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM cell and gene therapies in rare disorders market size from 2019 to 2032. The report also covers current cell and gene therapies in rare disorders treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.

Key Takeaways from the Cell and Gene Therapies in Rare Disorders Market Research Report
• The Gene And Cell Therapies In Rare Disorders market is expected to grow with a significant high rate and mainly driven by increased R&D related to number of gene therapies and readily adoption on approval, ability to treat broad array of conditions, increase in number of cases, easy dosing approach and curative treatment options.
• According to the Spinal muscular atrophy (SMA) [A European Policy and Access Tracker], SMA is a rare, genetic, neuromuscular disease with an estimated incidence in Europe ranging from 1 in 3,900-16,000 live births.
• The leading Gene And Cell Therapies In Rare Disorders Companies include Pfizer/Sangamo Therapeutics, Roche (Spark Therapeutics), Pfizer/Spark Therapeutics, Freeline Therapeutics, , 4D Molecular Therapeutics, Astellas Gene Therapies, Actus Therapeutics (Acquired by Bayer), GenSight Biologics, Coave Therapeutics, Johnson & Johnson/ MeiraGTx, Applied Genetic Technologies Corporation, GenSight Biologics, Nanoscope Therapeutics, 4D Molecular Therapeutic, 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
• Promising Gene And Cell Therapies In Rare Disorders Pipeline Therapies include LUXTURNA (voretigene neparvovec-rzyl), ZOLGENSMA (onasemnogene abeparvovec-xioi), CARVYKTI (ciltacabtagene autoleucel), Fidanacogene elaparvovec, Giroctocogene fitelparvovec, OTL-103, OAV101, TVI-Brain-1, AV-GBM-1, ABCB5+ MSCs, CAP-1002, and others.

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Gene And Cell Therapies In Rare Disorders 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 Disorders Epidemiology Segmentation in the 7MM
• Total Gene And Cell Therapies In Rare Disorders Prevalent cases of selected indications
• Total Gene And Cell Therapies In Rare Disorders Indication-wise eligible cases
• Gene And Cell Therapies In Rare Disorders Indication-wise treated cases

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Recent Developmental Activities in the Gene And Cell Therapies In Rare Disorders Treatment Landscape
• In February 2022, FDA approved CARVYKTI (ciltacabtagene autoleucel) for the treatment of patients with relapsed or refractory multiple myeloma after 4 or more prior lines of therapy including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. CARVYKTI is Janssen's first cell therapy. The chimeric antigen receptor T-cell (CAR-T) therapy features 2 B-cell maturation antigen (BCMA)-targeting single domain antibodies. Cilta-cel's safety information includes a boxed warning regarding a number of syndromes. The most common adverse events consist of pyrexia, CRS, and others.
• In December 2022, Pfizer announced positive top-line results from the Phase III BENEGENE-2 study (NCT03861273). The study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion versus prophylaxis regimen with Factor IX (FIX), administered as part of usual care. It has been granted breakthrough, regenerative medicines advance therapy (RMAT), and orphan drug designations from the FDA, as well as PRIME and ODD from the EMA. Pfizer will discuss these data with regulatory authorities in early 2023. Fidanacogene elaparvovec, previously SPK-9001 or PF-06838435, is a novel, investigational vector that contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity human coagulation FIX gene. The goal of this gene therapy for people living with hemophilia B, once treated, is that they will be able to produce FIX via this one-time treatment rather than having to regularly receive exogenous FIX.
• In September 2022, Pfizer and Sangamo Therapeutics announced that the Phase III AFFINE (NCT04370054) study evaluating giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe hemophilia A, has re-opened recruitment. Trial sites will begin to resume enrollment this month, with dosing expected to resume in October. All trial sites are anticipated to be active by the end of 2022 and a pivotal readout is expected in the first half of 2024. Giroctocogene fitelparvovec (SB-525/PF-07055480), comprises a recombinant AAV6 encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. It is being developed as part of a collaboration agreement for the global development and commercialization of gene therapies for hemophilia A between Sangamo and Pfizer. In late 2019, Sangamo transferred the manufacturing technology and the Investigational New Drug (IND) application to Pfizer. The FDA has granted Orphan Drug, Fast Track, and RMAT designations to giroctocogene fitelparvovec, which also received Orphan Medicinal Product designation from the EMA. Pfizer and partner Sangamo Therapeutics voluntarily paused the trial of giroctocogene fitelparvovec to adjust protocol after finding the therapy may raise blood clot risk. The delay was disclosed in November 2021, when the FDA slapped a clinical hold on the study because of "factor VIII levels greater than 150% in some study participants," according to a May 3 earnings call.

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 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, OTC deficiency/urea cycle disorders), dystrophic epidermolysis bullosa, gangliosidosis, and xerostomia.

To know more about Gene And Cell Therapies In Rare Disorders Treatment options, visit @ Gene And Cell Therapies In Rare Disorders Drugs- https://www.delveinsight.com/sample-request/gene-and-cell-therapies-in-rare-disorder-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Gene And Cell Therapies In Rare Disorders Marketed Drugs
• ZOLGENSMA (AVXS-101): Novartis/AveXis
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: Spark Therapeutics (Roche)/Novartis
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.

Gene And Cell Therapies In Rare Disorders Emerging Drugs

• Fidanacogene elaparvovec (SPK-9001/PF-06838435): Pfizer/Spark Therapeutics
Fidanacogene elaparvovec, previously SPK-9001 or PF-06838435, is a novel, investigational bio-engineered AAV vector utilizing a high-activity F9 transgene for hemophilia B or factor IX deficiency.
Currently, SPK-9001 is under Phase III (NCT03861273) clinical trial study wherein the study will evaluate the efficacy and safety of SPK-9001 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The study's main objectives are to compare the annualized bleeding rate [ABR] of the gene therapy to routine prophylaxis from the lead-in study and to evaluate its impact on the participant's Factor IX circulating activity [FIX:C]. This study includes 55 participants, with an estimated completion date of May 2027. The company anticipated the pivotal trial readout in the first quarter of 2023.
Pfizer anticipated to discuss BENEGENE-2 data with regulatory authorities in early 2023 and additional key data at a scientific conference in early 2023.

• AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics
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.
In May 2020, the US FDA granted fast-track designation to AT-GTX-502 for Batten disease caused by mutations in the CLN3 gene.

• FLT190: Freeline Therapeutics
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: Regenxbio
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.

Learn more about the Gene And Cell Therapies In Rare Disorders Pipeline Therapies in clinical trials @ Gene And Cell Therapies In Rare Disorders Market Landscape- https://www.delveinsight.com/sample-request/gene-and-cell-therapies-in-rare-disorder-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Gene And Cell Therapies In Rare Disorders Market Report
• Coverage- 7MM
• Study Period-2019-2032
• Gene And Cell Therapies In Rare Disorders Companies- Pfizer/Sangamo Therapeutics, Roche (Spark Therapeutics), Pfizer/Spark Therapeutics, Freeline Therapeutics, , 4D Molecular Therapeutics, Astellas Gene Therapies, Actus Therapeutics (Acquired by Bayer), GenSight Biologics, Coave Therapeutics, Johnson & Johnson/ MeiraGTx, Applied Genetic Technologies Corporation, GenSight Biologics, Nanoscope Therapeutics, 4D Molecular Therapeutic, 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
• Gene And Cell Therapies In Rare Disorders Pipeline Therapies- LUXTURNA (voretigene neparvovec-rzyl), ZOLGENSMA (onasemnogene abeparvovec-xioi), CARVYKTI (ciltacabtagene autoleucel), Fidanacogene elaparvovec, Giroctocogene fitelparvovec, OTL-103, OAV101, TVI-Brain-1, AV-GBM-1, ABCB5+ MSCs, CAP-1002, and others.
• Gene And Cell Therapies In Rare Disorders Market Dynamics: Gene And Cell Therapies In Rare Disorders Market Drivers and Barriers

Discover more about Gene And Cell Therapies In Rare Disorders Drugs in development @ Gene And Cell Therapies In Rare Disorders Ongoing Clinical Trials Analysis- https://www.delveinsight.com/sample-request/gene-and-cell-therapies-in-rare-disorder-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

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. Epidemiology and Patient Population
9. Marketed Drugs
10. Emerging Drugs
11. Cell and Gene Therapies in Rare Disorders: 7MM analysis
12. Unmet Needs
13. SWOT Analysis
14. KOL Views
15. Market Access and Reimbursement
16. Appendix
17. DelveInsight Capabilities
18. Disclaimer
19. About DelveInsight

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