{"id":563361,"date":"2026-04-06T00:00:00","date_gmt":"2026-04-06T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/dlsfor0001-2025-biopharma-gene-therapy-landscape-forecast-disease-landscape-forecast-g7\/"},"modified":"2026-04-14T10:10:19","modified_gmt":"2026-04-14T10:10:19","slug":"dlsfor0001-2026-biopharma-gene-therapy-landscape-forecast-disease-landscape-forecast-g7","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/dlsfor0001-2026-biopharma-gene-therapy-landscape-forecast-disease-landscape-forecast-g7\/","title":{"rendered":"Gene Therapy &#8211; Landscape &#038; Forecast &#8211; Disease Landscape &#038; Forecast (G7)"},"content":{"rendered":"<p>Approximately 80% of rare diseases have a genetic etiology. Gene therapies have the potential to transform the treatment of rare diseases and, in some cases, provide a functional cure. The pipeline for gene therapies is burgeoning, as large pharmaceutical companies (e.g., Johnson &#038; Johnson Innovative Medicine, Roche) assume increasing stakes in this arena. However, these innovative treatments must come with equally innovative programs to optimize access in a global healthcare market that is still grappling with how to fairly assess these therapies\u2019 value and absorb their high up-front costs. In this content, we explore the clinical and commercial potential of select gene therapies for rare disorders such as spinal muscular atrophy (<abbr title=\"spinal muscular atrophy\">SMA<\/abbr>), Duchenne muscular dystrophy (<abbr title=\"Duchenne muscular dystrophy\">DMD<\/abbr>), cerebral adrenoleukodystrophy (<abbr title=\"cerebral adrenoleukodystrophy\">CALD<\/abbr>), beta thalassemia, and inherited retinal dystrophies such as retinitis pigmentosa.<\/p>\n<p><strong>Questions answered<\/strong><\/p>\n<ul class=\"round-bullets\">\n<li>What are the sizes of the eligible populations for gene therapies in key indications in the G7?<\/li>\n<li>What are the main advantages and disadvantages of marketed gene therapies, and where do clinicians want to see improvements?<\/li>\n<li>Given that gene therapies have the potential to be single-administration cures, how will the treatment-eligible population change over time in indications served by a gene therapy?<\/li>\n<li>What are the key drivers of and barriers to the adoption of gene therapies?<\/li>\n<li>What is the expected uptake of the first CRISPR-based gene-editing therapy (Casgevy) approved for beta thalassemia?<\/li>\n<li>What is the commercial potential of emerging gene therapies in key indications (e.g., <abbr title=\"Duchenne muscular dystrophy\">DMD<\/abbr>, Leber hereditary optic neuropathy, <abbr title=\"cerebral adrenoleukodystrophy\">CALD<\/abbr>)?<\/li>\n<\/ul>\n<p><strong>Content highlights<\/strong><\/p>\n<p>Geography: United States, <abbr title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr>, Japan<\/p>\n<p>Primary research: More than 28 country-specific interviews with thought-leading ophthalmologists, neurologists, and hematologists, supported by survey data collected for this and other Clarivate research<\/p>\n<p>Epidemiology: Diagnosed prevalent and\/or incident patient populations eligible for gene therapies in the covered indications<\/p>\n<p>Forecast: 10-year, annualized, drug-level sales and patient share of key gene therapies through 2034, segmented by geography and key indication<\/p>\n<p>Drug treatments: Coverage of key current and emerging gene therapies in select indications across therapy areas<\/p>\n<p><strong>Product description<\/strong><\/p>\n<p>Disease Landscape &#038; Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research.<\/p>\n<p><strong>Key features<\/strong><\/p>\n<p>Disease Landscape &#038; Forecast will be updated throughout the year to provide timely insights and analyses as material indication-specific news and events unfold.<\/p>\n","protected":false},"template":"","class_list":["post-563361","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-date-2609"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/563361","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report"}],"about":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/types\/report"}],"version-history":[{"count":2,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/563361\/revisions"}],"predecessor-version":[{"id":575220,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/563361\/revisions\/575220"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=563361"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}