{"id":837787,"date":"2026-09-24T17:17:03","date_gmt":"2026-09-24T17:17:03","guid":{"rendered":"https:\/\/www.abnewswire.com\/pressreleases\/?p=837787"},"modified":"2026-09-24T17:17:03","modified_gmt":"2026-09-24T17:17:03","slug":"cell-and-gene-therapies-in-rare-disorders-market-to-expand-as-regulatory-approvals-and-pipeline-innovation-accelerate-adoption-delveinsight","status":"publish","type":"post","link":"https:\/\/www.abnewswire.com\/pressreleases\/cell-and-gene-therapies-in-rare-disorders-market-to-expand-as-regulatory-approvals-and-pipeline-innovation-accelerate-adoption-delveinsight_837787.html","title":{"rendered":"Cell and Gene Therapies in Rare Disorders Market to Expand as Regulatory Approvals and Pipeline Innovation Accelerate Adoption | DelveInsight"},"content":{"rendered":"<div style=\"float:right;  width:250px; padding:8px 10px 10px 10px;\"><a href=\"https:\/\/www.abnewswire.com\/upload\/2026\/09\/1790247963.jpg\" style=\"border:none !important;\" target=\"_blank\" rel=\"nofollow\" ><img loading=\"lazy\" decoding=\"async\" class=\"alignnone size-medium wp-image-29\" title=\"Cell and Gene Therapies in Rare Disorders Market to Expand as Regulatory Approvals and Pipeline Innovation Accelerate Adoption | DelveInsight\" src=\"https:\/\/www.abnewswire.com\/upload\/2026\/09\/1790247963.jpg\" alt=\"Cell and Gene Therapies in Rare Disorders Market to Expand as Regulatory Approvals and Pipeline Innovation Accelerate Adoption | DelveInsight\" width=\"225\" height=\"118\" \/><\/a><\/div>\n<div style=\"font-style:italic; padding:8px 0px;\">Cell and Gene Therapies in Rare Disorders market is undergoing significant transformation as advances in genetic medicine, increasing clinical development activity, regulatory approvals, &#038; the expansion of one-time therapeutic approaches create new opportunities across rare disease treatment. According to DelveInsight\u2019s the total 7MM market size for cell and gene therapies in rare disorders was approximately USD 2.0 billion in 2023 &#038; is expected to grow significantly through the forecast period.<\/div>\n<p style=\"text-align: justify;\"><strong>(Albany, New York) &ndash; September 24, 2026<\/strong> &ndash; The <strong>Cell and Gene Therapies in Rare Disorders market<\/strong> is undergoing significant transformation as advances in genetic medicine, increasing clinical development activity, regulatory approvals, and the expansion of one-time therapeutic approaches create new opportunities across rare disease treatment. According to DelveInsight&rsquo;s latest report, <strong>&ldquo;Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast &ndash; 2036,&rdquo;<\/strong> the total <strong>7MM market size for cell and gene therapies in rare disorders was approximately USD 2.0 billion in 2023<\/strong> and is expected to grow significantly through the forecast period as more companies advance investigational therapies and regulatory approvals increase in the United States and Europe.<\/p>\n<p style=\"text-align: justify;\">The report covers the <strong>United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan<\/strong>, providing historical and forecasted market and epidemiology analysis. The study period extends from <strong>2020 to 2034<\/strong>, with analysis of current treatment practices, emerging therapies, market shares, epidemiology, treatment algorithms, unmet needs, drug uptake, competitive dynamics, market access, reimbursement, and pipeline development.<\/p>\n<p style=\"text-align: justify;\">The United States accounted for approximately <strong>USD 800 million of the market in 2023<\/strong> and is projected to increase over the forecast period. Among the selected indications, <strong>Hemophilia A is expected to generate the highest revenue in the US by 2034, followed by Retinitis Pigmentosa<\/strong>. In Japan, Hemophilia A is also expected to generate the highest revenue among the selected indications by 2034, followed by Limbal Stem Cell Deficiency.<\/p>\n<p style=\"text-align: justify;\"><strong>Key Takeaways<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>The <strong>7MM Cell and Gene Therapies in Rare Disorders market was approximately USD 2.0 billion in 2023<\/strong>.<\/li>\n<li>The market is expected to grow significantly through <strong>2034<\/strong>, supported by increasing regulatory approvals and pipeline development.<\/li>\n<li>The <strong>United States market was approximately USD 800 million in 2023<\/strong>.<\/li>\n<li>The total prevalent population of selected indications for cell and gene therapies in rare disorders across the <strong>7MM was approximately 930,200 cases in 2023<\/strong>.<\/li>\n<li>Approximately <strong>504,000 treated cases<\/strong> of the selected indications were reported across the 7MM in 2023.<\/li>\n<li><strong>Retinitis Pigmentosa<\/strong> represented the largest prevalent population among the selected indications in 2023, followed by <strong>Limbal Stem Cell Deficiency<\/strong>.<\/li>\n<li>In the US, <strong>ZOLGENSMA (onasemnogene abeparvovec-xioi)<\/strong> represented the largest market size among therapies in 2023.<\/li>\n<li>By 2034, <strong>Hemophilia A<\/strong> is expected to generate the highest revenue among the selected indications in the US.<\/li>\n<li>Marketed therapies include <strong>ZOLGENSMA, LUXTURNA, ROCTAVIAN, HEMGENIX, LIBMELDY, HOLOCLAR, UPSTAZA, and SKYSONA<\/strong>, among others.<\/li>\n<li>Emerging therapies include <strong>AT-GTX-502, FLT190, RGX-121<\/strong>, and multiple other gene and cell therapy candidates across rare diseases.<\/li>\n<li>The competitive landscape includes <strong>Pfizer, Roche, Spark Therapeutics, Freeline Therapeutics, REGENXBIO, Amicus Therapeutics, Sarepta Therapeutics, Ultragenyx Pharmaceutical, Krystal Biotech, Abeona Therapeutics<\/strong>, and others.<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>Keen to know more about the Cell and Gene Therapies in Rare Disorders market and its emerging pipeline? <\/strong>Request a sample copy of DelveInsight&rsquo;s <strong>Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast &ndash; 2036<\/strong> report:<\/p>\n<p style=\"text-align: justify;\"><a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/gene-and-cell-therapies-in-rare-disorder-market?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=kspr\"><strong>https:\/\/www.delveinsight.com\/sample-request\/gene-and-cell-therapies-in-rare-disorder-market<\/strong><\/a><\/p>\n<p style=\"text-align: justify;\"><strong>Key Factors Driving the Cell and Gene Therapies in Rare Disorders Market<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Increasing Regulatory Approvals<\/strong><\/p>\n<p style=\"text-align: justify;\">Regulatory approvals of cell and gene therapies have established important precedents for the broader rare disease therapeutic landscape. DelveInsight highlights the approval of therapies such as <strong>LIBMELDY, SKYSONA, HOLOCLAR, UPSTAZA, and ROCTAVIAN<\/strong> as developments that have helped establish regulatory pathways for other cell and gene therapies under development.<\/p>\n<p style=\"text-align: justify;\">These approvals demonstrate the increasing regulatory acceptance of advanced therapeutic modalities and provide development precedents for companies working on new rare disease indications.<\/p>\n<p style=\"text-align: justify;\"><strong>Expansion of Clinical Development<\/strong><\/p>\n<p style=\"text-align: justify;\">Cell and gene therapy technologies are being investigated across a broad range of rare diseases, including <strong>hemophilia, lysosomal storage disorders, neurological diseases, inherited retinal diseases, muscular disorders, metabolic disorders, and skin diseases<\/strong>.<\/p>\n<p style=\"text-align: justify;\">Companies are advancing therapies through different stages of clinical development, creating a growing pipeline of potentially transformative treatment options.<\/p>\n<p style=\"text-align: justify;\"><strong>Growing Adoption of One-Time Treatment Approaches<\/strong><\/p>\n<p style=\"text-align: justify;\">Many gene therapies are designed to provide a therapeutic effect through a single administration rather than requiring repeated conventional treatments. This approach is particularly relevant in rare genetic disorders where the underlying disease mechanism is associated with a defective or missing gene.<\/p>\n<p style=\"text-align: justify;\">For example, <strong>ZOLGENSMA<\/strong> is designed as a one-time gene therapy for pediatric patients with SMA, while <strong>LUXTURNA<\/strong> is a one-time gene therapy for eligible patients with RPE65 mutation-associated retinal disease.<\/p>\n<p style=\"text-align: justify;\"><strong>Increasing Investment in Manufacturing Capabilities<\/strong><\/p>\n<p style=\"text-align: justify;\">The growing number of gene therapy programs is increasing demand for specialized manufacturing capabilities. DelveInsight notes that competition for contract manufacturing organization production capacity is expected to intensify as more companies enter the gene therapy market.<\/p>\n<p style=\"text-align: justify;\">This environment could increase manufacturing costs and encourage pharmaceutical and biotechnology companies to invest in manufacturing technologies or acquire organizations with specialized manufacturing expertise.<\/p>\n<p style=\"text-align: justify;\"><strong>Significant Residual Unmet Needs<\/strong><\/p>\n<p style=\"text-align: justify;\">Although several cell and gene therapies have reached the market, substantial unmet needs remain across rare diseases. DelveInsight highlights continued opportunities to improve therapeutic efficacy, safety, accessibility, diagnosis, manufacturing, and long-term disease management.<\/p>\n<p style=\"text-align: justify;\">The report also emphasizes that additional clinical evidence and long-term follow-up are required to better establish the safety and efficacy of many emerging therapies.<\/p>\n<p style=\"text-align: justify;\"><strong>Competitive Landscape<\/strong><\/p>\n<p style=\"text-align: justify;\">The Cell and Gene Therapies in Rare Disorders market includes a broad group of pharmaceutical, biotechnology, and specialized gene therapy companies.<\/p>\n<p style=\"text-align: justify;\">Key companies identified by DelveInsight include:<\/p>\n<ul style=\"text-align: justify;\">\n<li><strong>Pfizer\/Sangamo Therapeutics<\/strong><\/li>\n<li><strong>Roche\/Spark Therapeutics<\/strong><\/li>\n<li><strong>Freeline Therapeutics<\/strong><\/li>\n<li><strong>4D Molecular Therapeutics<\/strong><\/li>\n<li><strong>Astellas Gene Therapies<\/strong><\/li>\n<li><strong>GenSight Biologics<\/strong><\/li>\n<li><strong>Coave Therapeutics<\/strong><\/li>\n<li><strong>Johnson &amp; Johnson\/MeiraGTx<\/strong><\/li>\n<li><strong>Nanoscope Therapeutics<\/strong><\/li>\n<li><strong>REGENXBIO<\/strong><\/li>\n<li><strong>Amicus Therapeutics<\/strong><\/li>\n<li><strong>Sarepta Therapeutics<\/strong><\/li>\n<li><strong>CRISPR Therapeutics\/Vertex Pharmaceuticals<\/strong><\/li>\n<li><strong>Editas Medicine<\/strong><\/li>\n<li><strong>Krystal Biotech<\/strong><\/li>\n<li><strong>Abeona Therapeutics<\/strong><\/li>\n<li><strong>Castle Creek Biosciences<\/strong><\/li>\n<li><strong>Holostem Terapie Avanzate<\/strong><\/li>\n<li><strong>RHEACELL<\/strong><\/li>\n<li><strong>Ultragenyx Pharmaceutical<\/strong><\/li>\n<li><strong>Capricor Therapeutics\/Nippon Shinyaku<\/strong><\/li>\n<li><strong>Brainstorm Cell Therapeutics<\/strong><\/li>\n<li><strong>Ocugen<\/strong><\/li>\n<li><strong>jCyte<\/strong><\/li>\n<li><strong>Anterogen<\/strong><\/li>\n<li><strong>Ishin Pharma<\/strong><\/li>\n<\/ul>\n<p style=\"text-align: justify;\">These companies are developing or commercializing therapies across multiple rare disease indications and therapeutic modalities.<\/p>\n<p style=\"text-align: justify;\"><strong>Marketed Cell and Gene Therapies<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>ZOLGENSMA &ndash; Novartis\/AveXis<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>ZOLGENSMA (onasemnogene abeparvovec-xioi)<\/strong> is a gene therapy developed for pediatric patients younger than 2 years with spinal muscular atrophy associated with biallelic mutations in the <strong>SMN1 gene<\/strong>.<\/p>\n<p style=\"text-align: justify;\">The US FDA approved ZOLGENSMA in May 2019. The European Commission subsequently granted conditional approval in May 2020 for specified patients with 5q SMA.<\/p>\n<p style=\"text-align: justify;\"><strong>LUXTURNA &ndash; Spark Therapeutics\/Roche and Novartis<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>LUXTURNA (voretigene neparvovec-rzyl)<\/strong> is a one-time gene therapy for patients with vision loss associated with confirmed biallelic <strong>RPE65 mutations<\/strong> and sufficient viable retinal cells.<\/p>\n<p style=\"text-align: justify;\">The FDA approved LUXTURNA in December 2017, followed by European Commission approval in November 2018.<\/p>\n<p style=\"text-align: justify;\"><strong>ROCTAVIAN &ndash; BioMarin Pharmaceutical<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>ROCTAVIAN (valoctocogene roxaparvovec)<\/strong> is a gene therapy for Hemophilia A and is among the marketed therapies assessed within the report. DelveInsight identifies Hemophilia A as a major revenue-generating indication within the future rare disease cell and gene therapy landscape.<\/p>\n<p style=\"text-align: justify;\"><strong>HEMGENIX &ndash; CSL Behring\/uniQure<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>HEMGENIX (etranacogene dezaparvovec)<\/strong> is a gene therapy included in DelveInsight&#8217;s analysis of marketed cell and gene therapies for <strong>Hemophilia B<\/strong>.<\/p>\n<p style=\"text-align: justify;\"><strong>LIBMELDY &ndash; Orchard Therapeutics<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>LIBMELDY (atidarsagene autotemcel)<\/strong> is a cell and gene therapy for <strong>metachromatic leukodystrophy (MLD)<\/strong> and forms part of the marketed rare disease cell and gene therapy landscape evaluated by DelveInsight.<\/p>\n<p style=\"text-align: justify;\"><strong>HOLOCLAR &ndash; Holostem Terapie Avanzate<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>HOLOCLAR<\/strong> is a cell therapy used in the treatment of <strong>limbal stem cell deficiency<\/strong>, another indication included within DelveInsight&#8217;s selected rare disease cell and gene therapy markets.<\/p>\n<p style=\"text-align: justify;\"><strong>Emerging Cell and Gene Therapies<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>AT-GTX-502 &ndash; Amicus Therapeutics<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>AT-GTX-502 (scAAV9.P546.CLN3)<\/strong> is an AAV9-based gene therapy being developed for children with <strong>CLN3 Batten disease<\/strong>.<\/p>\n<p style=\"text-align: justify;\">The therapy is being evaluated in a Phase I\/IIa gene transfer clinical trial. The US FDA granted Fast Track designation to AT-GTX-502 in May 2020.<\/p>\n<p style=\"text-align: justify;\"><strong>FLT190 &ndash; Freeline Therapeutics<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>FLT190<\/strong> is an AAV8-based gene therapy candidate designed to deliver a functional copy of the <strong>GLA gene<\/strong> with the goal of restoring production of alpha-galactosidase A.<\/p>\n<p style=\"text-align: justify;\">The therapy is being investigated as a potential single-dose approach for <strong>Fabry disease<\/strong>, offering an alternative to repeated enzyme replacement therapy.<\/p>\n<p style=\"text-align: justify;\"><strong>RGX-121 &ndash; REGENXBIO<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>RGX-121<\/strong> is being developed as a one-time treatment for <strong>Mucopolysaccharidosis Type II (MPS II)<\/strong>. It uses the NAV AAV9 vector to deliver the human <strong>IDS gene<\/strong> directly into the central nervous system.<\/p>\n<p style=\"text-align: justify;\">REGENXBIO has been developing a pivotal program for RGX-121 and has planned regulatory development using the FDA accelerated approval pathway.<\/p>\n<p style=\"text-align: justify;\"><strong>Recent Developments in the Cell and Gene Therapies in Rare Disorders Market<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li><strong>May 2025:<\/strong> VGXI, Inc., a CDMO specializing in plasmid DNA manufacturing for gene therapies and vaccines, announced the successful FDA inspection of its GMP facility, supporting the approval of a client&#8217;s Biologics License Application.<\/li>\n<li>Regulatory approvals of <strong>LIBMELDY, SKYSONA, HOLOCLAR, UPSTAZA, ROCTAVIAN<\/strong>, and other therapies have helped establish regulatory pathways for additional cell and gene therapy programs.<\/li>\n<li>Multiple companies continue to advance cell and gene therapies for <strong>hemophilia A and B, lysosomal storage disorders, neurological disorders, inherited retinal diseases, muscular disorders, metabolic diseases, and other rare conditions<\/strong>.<\/li>\n<li>Increasing demand for specialized manufacturing is creating greater competition for CDMO capacity as the number of gene therapy developers increases.<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>What are Cell and Gene Therapies in Rare Disorders?<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Cell and gene therapies in rare disorders<\/strong> are advanced therapeutic approaches designed to address the underlying mechanisms of rare genetic or acquired diseases.<\/p>\n<p style=\"text-align: justify;\"><strong>Gene therapy<\/strong> delivers genetic material into a patient&#8217;s cells to correct, replace, or compensate for defective genes. Depending on the therapeutic strategy, gene therapy can involve <strong>gene addition, gene editing, or other genetic modifications<\/strong> and may be administered either <strong>in vivo<\/strong> or <strong>ex vivo<\/strong>.<\/p>\n<p style=\"text-align: justify;\"><strong>Cell therapy<\/strong> involves the use of living cells, including stem cells or immune cells, which may be collected from the patient or a donor, modified or expanded outside the body when necessary, and subsequently administered to repair, replace, or restore damaged tissues or biological functions.<\/p>\n<p style=\"text-align: justify;\">Gene and cell therapies frequently overlap, particularly when patient-derived cells are genetically modified outside the body and subsequently returned to the patient.<\/p>\n<p style=\"text-align: justify;\"><strong>Current Treatment Landscape<\/strong><\/p>\n<p style=\"text-align: justify;\">The treatment landscape varies substantially by rare disease and therapeutic mechanism. Cell and gene therapies are being evaluated as alternatives or complements to conventional treatments such as enzyme replacement therapy, protein replacement therapy, repeated infusions, and other supportive approaches.<\/p>\n<p style=\"text-align: justify;\">The current marketed landscape assessed by DelveInsight includes therapies across:<\/p>\n<ul style=\"text-align: justify;\">\n<li><strong>Hemophilia A<\/strong><\/li>\n<li><strong>Hemophilia B<\/strong><\/li>\n<li><strong>Retinitis Pigmentosa<\/strong><\/li>\n<li><strong>Spinal Muscular Atrophy<\/strong><\/li>\n<li><strong>Metachromatic Leukodystrophy<\/strong><\/li>\n<li><strong>Limbal Stem Cell Deficiency<\/strong><\/li>\n<li><strong>Adenosine Deaminase Severe Combined Immunodeficiency<\/strong><\/li>\n<li><strong>Cerebral Adrenoleukodystrophy<\/strong><\/li>\n<li>Other rare disorders<\/li>\n<\/ul>\n<p style=\"text-align: justify;\">The report evaluates the treatment algorithm, clinical development, regulatory milestones, safety and efficacy, pharmacological mechanisms, advantages and limitations, and product profiles of marketed and emerging therapies.<\/p>\n<p style=\"text-align: justify;\"><strong>Epidemiology<\/strong><\/p>\n<p style=\"text-align: justify;\">DelveInsight&#8217;s patient-based epidemiology model covers selected rare disease indications for which cell and gene therapies are marketed or under development.<\/p>\n<p style=\"text-align: justify;\">The <strong>total prevalent population of selected indications across the 7MM was approximately 930,200 cases in 2023<\/strong> and is projected to increase during the forecast period. <strong>Retinitis Pigmentosa<\/strong> accounted for the highest number of cases among the selected indications, followed by <strong>Limbal Stem Cell Deficiency<\/strong>.<\/p>\n<p style=\"text-align: justify;\">The total number of <strong>indication-wise treated cases was approximately 504,000 in 2023<\/strong> and is expected to increase during 2024&ndash;2034.<\/p>\n<p style=\"text-align: justify;\">The epidemiology analysis covers:<\/p>\n<ul style=\"text-align: justify;\">\n<li>Total prevalent cases of selected indications<\/li>\n<li>Indication-wise eligible cases<\/li>\n<li>Indication-wise treated cases<\/li>\n<li>Historical epidemiology<\/li>\n<li>Forecast epidemiology<\/li>\n<li>Patient populations across the US, EU4, UK, and Japan<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>Unmet Needs in Cell and Gene Therapies for Rare Disorders<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Long-Term Safety and Efficacy<\/strong><\/p>\n<p style=\"text-align: justify;\">Many cell and gene therapies are relatively new modalities, making long-term follow-up important for understanding durability, safety, immunogenicity, and potential delayed adverse events.<\/p>\n<p style=\"text-align: justify;\"><strong>High Development and Manufacturing Costs<\/strong><\/p>\n<p style=\"text-align: justify;\">Gene therapy research, development, and production costs can be considerably higher than those associated with traditional biologics. DelveInsight notes that gene therapy-related costs can range from approximately <strong>USD 500,000 to USD 1 million<\/strong>, contributing to high treatment prices.<\/p>\n<p style=\"text-align: justify;\"><strong>Manufacturing Capacity<\/strong><\/p>\n<p style=\"text-align: justify;\">As more therapies progress through clinical development, competition for specialized manufacturing capacity is increasing. This creates potential challenges involving production scale, quality control, vector manufacturing, and supply availability.<\/p>\n<p style=\"text-align: justify;\"><strong>Patient Identification and Eligibility<\/strong><\/p>\n<p style=\"text-align: justify;\">Because these therapies frequently target highly specific genetic or clinical characteristics, accurate diagnosis and identification of eligible patients remain important for successful treatment.<\/p>\n<p style=\"text-align: justify;\"><strong>Access and Reimbursement<\/strong><\/p>\n<p style=\"text-align: justify;\">The high upfront cost of one-time therapies creates challenges for conventional reimbursement models. DelveInsight highlights the need for innovative payment approaches and collaboration among pharmaceutical companies, private payers, and government stakeholders.<\/p>\n<p style=\"text-align: justify;\"><strong>Market Access and Reimbursement<\/strong><\/p>\n<p style=\"text-align: justify;\">The high cost of cell and gene therapies represents a major consideration for healthcare systems.<\/p>\n<p style=\"text-align: justify;\">Unlike conventional medicines that may generate recurring treatment costs, many gene therapies are administered as one-time treatments. This creates a substantial upfront financial burden for payers, despite the potential for durable therapeutic effects.<\/p>\n<p style=\"text-align: justify;\">DelveInsight notes that the US payment environment remains under-equipped to fully address the financial consequences associated with these therapies, creating a need for new business models and collaborations among manufacturers, payers, and government stakeholders.<\/p>\n<p style=\"text-align: justify;\">For example, <strong>LUXTURNA<\/strong> was launched in Germany in April 2019 at a manufacturer price of approximately <strong>EUR 345,000 per eye per patient<\/strong>. NICE recommended LUXTURNA within its marketing authorization under a commercial arrangement, while Germany&#8217;s G-BA provided a &ldquo;considerable added benefit&rdquo; assessment.<\/p>\n<p style=\"text-align: justify;\"><strong>Scope of the Cell and Gene Therapies in Rare Disorders Market Report<\/strong><\/p>\n<p style=\"text-align: justify;\">The DelveInsight report provides a comprehensive assessment of the cell and gene therapy landscape across the <strong>7MM<\/strong>:<\/p>\n<p style=\"text-align: justify;\"><strong>Geographies: <\/strong>United States, Germany, France, Italy, Spain, United Kingdom, and Japan<\/p>\n<p style=\"text-align: justify;\"><strong>Study Period:<\/strong> 2020&ndash;2034 <strong>Market Base Year:<\/strong> 2023 for the key market figures presented on the current report page <strong>7MM Market Size in 2023:<\/strong> Approximately USD 2.0 billion <strong>US Market Size in 2023:<\/strong> Approximately USD 800 million <strong>7MM Selected-Indication Prevalent Cases in 2023:<\/strong> Approximately 930,200 <strong>7MM Treated Cases in 2023:<\/strong> Approximately 504,000<\/p>\n<p style=\"text-align: justify;\">The report evaluates market size by <strong>indication and therapy<\/strong>, epidemiology, treatment algorithms, marketed drugs, emerging therapies, pipeline activity, drug uptake, competitive landscape, unmet needs, SWOT analysis, KOL insights, market access, reimbursement, collaborations, licensing, mergers and acquisitions, and patent activity.<\/p>\n<p style=\"text-align: justify;\"><strong>To Know More About the Cell and Gene Therapies in Rare Disorders Market<\/strong><\/p>\n<p style=\"text-align: justify;\"><a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/gene-and-cell-therapies-in-rare-disorder-market?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=kspr\">Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast &ndash; 2036<\/a><\/p>\n<p style=\"text-align: justify;\"><strong>Table of Contents<\/strong><\/p>\n<ol style=\"text-align: justify;\">\n<li>Key Insights<\/li>\n<li>Report Introduction<\/li>\n<li>Key Highlights From the Report<\/li>\n<li>Executive Summary of Cell and Gene Therapies in Rare Disorders<\/li>\n<li>Key Events<\/li>\n<li>Epidemiology and Market Forecast Methodology<\/li>\n<li>Cell and Gene Therapies in Rare Disorders Market Overview in the 7MM<\/li>\n<li>Disease Background and Overview<\/li>\n<li>Epidemiology and Patient Population<\/li>\n<li>Marketed Drugs<\/li>\n<li>Emerging Therapies<\/li>\n<li>Market Forecast<\/li>\n<li>Unmet Needs<\/li>\n<li>SWOT Analysis<\/li>\n<li>Key Opinion Leader Views<\/li>\n<li>Market Access and Reimbursement<\/li>\n<li>Appendix<\/li>\n<li>Report Methodology<\/li>\n<li>DelveInsight Capabilities<\/li>\n<li>Disclaimer<\/li>\n<li>About DelveInsight<\/li>\n<\/ol>\n<p style=\"text-align: justify;\"><strong>About DelveInsight<\/strong><\/p>\n<p style=\"text-align: justify;\">DelveInsight is a leading market research and consulting firm specializing in the life sciences and healthcare industries. Founded in 2014, the company provides comprehensive market intelligence, epidemiology, and insights across pharmaceuticals, biotechnology, medical devices, and emerging therapies. DelveInsight helps healthcare organizations make informed strategic decisions through data-driven research and industry expertise.<\/p>\n<p style=\"text-align: justify;\">&nbsp;<\/p>\n<p><span style='font-size:18px !important;'>Media Contact<\/span><br \/><strong>Company Name:<\/strong> <a href=\"https:\/\/www.abnewswire.com\/companyname\/delveinsight.com_75707.html\" rel=\"nofollow\">DelveInsight Business Research LLP<\/a><br \/><strong>Contact Person:<\/strong> Kirti Sharma<br \/><strong>Email:<\/strong> <a href=\"https:\/\/www.abnewswire.com\/email_contact_us.php?pr=cell-and-gene-therapies-in-rare-disorders-market-to-expand-as-regulatory-approvals-and-pipeline-innovation-accelerate-adoption-delveinsight\" rel=\"nofollow\">Send Email<\/a><br \/><strong>Phone:<\/strong> +14699457679<br \/><strong>Address:<\/strong>304 S. Jones Blvd #2432  <br \/><strong>City:<\/strong> Las Vegas<br \/><strong>State:<\/strong> Nevada<br \/><strong>Country:<\/strong> United States<br \/><strong>Website:<\/strong> <a href=\"https:\/\/www.delveinsight.com\/consulting\/due-diligence-services\" target=\"_blank\" rel=\"nofollow\">https:\/\/www.delveinsight.com\/consulting\/due-diligence-services<\/a><\/p>\n<p><img decoding=\"async\" src=\"https:\/\/www.abnewswire.com\/press_stat.php?pr=cell-and-gene-therapies-in-rare-disorders-market-to-expand-as-regulatory-approvals-and-pipeline-innovation-accelerate-adoption-delveinsight\" alt=\"\" width=\"1px\" height=\"1px\" \/><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Cell and Gene Therapies in Rare Disorders market is undergoing significant transformation as advances in genetic medicine, increasing clinical development activity, regulatory approvals, &#038; the expansion of one-time therapeutic approaches create new opportunities across rare disease treatment. According to DelveInsight\u2019s &hellip; <a href=\"https:\/\/www.abnewswire.com\/pressreleases\/cell-and-gene-therapies-in-rare-disorders-market-to-expand-as-regulatory-approvals-and-pipeline-innovation-accelerate-adoption-delveinsight_837787.html\">Continue reading <span class=\"meta-nav\">&rarr;<\/span><\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[435,405,406,420,404],"tags":[],"class_list":["post-837787","post","type-post","status-publish","format-standard","hentry","category-Europe","category-Health-Medicine","category-Pharmaceuticals-Biotech","category-Science","category-US"],"_links":{"self":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts\/837787","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/comments?post=837787"}],"version-history":[{"count":0,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts\/837787\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/media?parent=837787"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/categories?post=837787"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/tags?post=837787"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}