{"id":673686,"date":"2023-10-25T10:04:02","date_gmt":"2023-10-25T10:04:02","guid":{"rendered":"https:\/\/www.abnewswire.com\/pressreleases\/?p=673686"},"modified":"2023-10-25T10:04:02","modified_gmt":"2023-10-25T10:04:02","slug":"pompe-disease-pipeline-report-2023-treatment-drugs-therapies-fda-approvals-and-companies-by-delveinsight-spark-therapeutics-amicus-therapeutics-asklepios-biopharmaceutical-audentes-therapeutic","status":"publish","type":"post","link":"https:\/\/www.abnewswire.com\/pressreleases\/pompe-disease-pipeline-report-2023-treatment-drugs-therapies-fda-approvals-and-companies-by-delveinsight-spark-therapeutics-amicus-therapeutics-asklepios-biopharmaceutical-audentes-therapeutic_673686.html","title":{"rendered":"Pompe Disease Pipeline Report 2023: Treatment Drugs, Therapies, FDA Approvals and Companies by DelveInsight | Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutic"},"content":{"rendered":"<div style=\"float:right; width:250px; padding:8px 10px 10px 10px;\">\n<div><a href=\"https:\/\/www.abnewswire.com\/uploads\/1698206993.jpeg\" style=\"border:none !important;\" target=\"_blank\" rel=\"nofollow\" ><img loading=\"lazy\" decoding=\"async\" class=\"alignnone size-medium wp-image-29\" title=\"Pompe Disease Pipeline Report 2023: Treatment Drugs, Therapies, FDA Approvals and Companies by DelveInsight | Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutic\" src=\"https:\/\/www.abnewswire.com\/uploads\/1698206993.jpeg\" alt=\"Pompe Disease Pipeline Report 2023: Treatment Drugs, Therapies, FDA Approvals and Companies by DelveInsight | Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutic\" width=\"225\" height=\"225\" style=\"padding:0px 0px 10px 10px; border:0 solid !important;\" \/><\/a><\/div>\n<div class=\"quotes\">\n<div>Pompe Disease Pipeline<\/div>\n<\/div>\n<\/div>\n<p style=\"text-align: justify;\">(Albany, USA) DelveInsight&rsquo;s,&nbsp;<strong>&ldquo;Pompe Disease Pipeline Insights, 2023,&rdquo;&nbsp;<\/strong>report provides comprehensive insights about&nbsp;<strong>15+ companies and 20+ pipeline drugs<\/strong>&nbsp;in Pompe Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.<\/p>\n<p style=\"text-align: justify;\"><strong>&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp; <\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Request a sample report of Pompe Disease @ <a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/report-store\/pompe-disease-pipeline-insight?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=apr\">Pompe Disease Pipeline Report<\/a><\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Some of the key highlights from the&nbsp;Pompe Disease Pipeline&nbsp;report:<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>The Pompe Disease Pipeline report offers a rich analysis of&nbsp;<strong>17+&nbsp;<\/strong>key players and&nbsp;<strong>17+&nbsp;<\/strong>key therapies.<\/li>\n<li>Pompe Disease pipeline comprises therapies in different stages of the clinical phase such as include<strong>&nbsp;Cipaglucosidase alfa, SPK-3006, ACTUS 101, AT845, RP A501, AOC-Pompe disease, AVR RD 03, GYS1 Program, ETV-GAA,&nbsp;<\/strong>and many others expected to enter the Pompe Disease market in the coming years.<\/li>\n<li>Key companies strengthening the Pompe Disease Pipeline are&nbsp;<strong>Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics, Novartis AG, Fresenius Kabi AG, Akorn Incorporated, Teva Pharmaceutical, Mylan N.V, Johnsons &amp; Johnsons, F. Hoffman-La Roche Ltd., Trinity Biotech plc, and others. <\/strong><\/li>\n<li>In May 2021, Amicus Therapeutics announced the completion of a successful Type B Pre-Biologics License Application (BLA) meeting with the US Food and Drug Administration (FDA) for AT-GAA (cipaglucosidase alfa co-administered with miglustat), for the treatment of Pompe disease.<\/li>\n<li>In March 2021, Maze Therapeutics revealed its first three lead therapeutic candidates in the company&rsquo;s wholly owned pipeline. One of the candidates includes an oral therapy targeting GYS1 for the treatment of Pompe disease, enabled by Maze&rsquo;s COMPASS platform. The company expects to start clinical trials in the first half of 2022.<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Pompe Disease Overview<\/strong><\/p>\n<p style=\"text-align: justify;\">Pompe Disease is a rare genetic disease that is a result of a build-up of a complex sugar, glycogen in the cells of the body. It exhibits variable rates of progression and different ages of onset. The cause of the disease is a deficiency of an enzyme called acid alfa glucosidase (GAA), which breaks downs complex sugars in the body.<\/p>\n<p style=\"text-align: justify;\">&nbsp;<\/p>\n<p style=\"text-align: justify;\"><strong>Pompe Disease Pipeline Drugs<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>Cipaglucosidase alfa &#8211; Amicus Therapeutics<\/li>\n<li>SPK-3006 &#8211; Spark Therapeutics<\/li>\n<li>AT845 &#8211; Audentes Therapeutics<\/li>\n<li>ACTUS 101 &#8211; Asklepios Biopharmaceutical<\/li>\n<li>RP A501 &#8211; Rocket Pharmaceuticals<\/li>\n<li>AVR-RD-03 &#8211; AVROBIO<\/li>\n<li>AOC-Pompe disease &#8211; Avidity Biosciences<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Request for Sample Report@ <a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/pompe-disease-pipeline-insight?utm_source=%20abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=apr\">https:\/\/www.delveinsight.com\/sample-request\/pompe-disease-pipeline-insight<\/a><\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Pompe Disease Therapeutic Assessment<\/strong><\/p>\n<p style=\"text-align: justify;\">The Pompe Disease Pipeline report lays down complete insights into active Pompe Disease pipeline therapies segmented by Stage, Product Type, Route of Administration, Molecule Type, Target, and Mechanism of Action.<\/p>\n<p style=\"text-align: justify;\"><strong>By Product Type<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>Mono<\/li>\n<li>Combination<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>By Stage<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>Discovery<\/li>\n<li>Pre-clinical<\/li>\n<li>IND<\/li>\n<li>Phase I<\/li>\n<li>Phase II<\/li>\n<li>Phase III<\/li>\n<li>Pre-registration<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Request for Sample Report@&nbsp;<a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/report-store\/pompe-disease-pipeline-insight?utm_source=abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=apr\">Pompe Disease Clinical Trials<\/a><\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>By Molecule Type<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>Gene therapies<\/li>\n<\/ul>\n<p style=\"text-align: justify;\">Recombinant fusion proteins<\/p>\n<ul style=\"text-align: justify;\">\n<li>Small interfering RNA<\/li>\n<li>Cell therapies<\/li>\n<li>Monoclonal antibodies<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>By Route of Administration<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>Intravenous<\/li>\n<li>Parenteral<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>By Mechanism of Action<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>Alpha-glucosidase replacements<\/li>\n<li>Glycogen synthase kinase modulators<\/li>\n<li>RNA interference<\/li>\n<li>Gene transference<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>By Targets<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li>Alpha-glucosidase<\/li>\n<li>Gene replacement<\/li>\n<li>Glycogen synthase kinase<\/li>\n<\/ul>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Request for Sample Report@ <a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/pompe-disease-pipeline-insight?utm_source=%20abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=apr\">https:\/\/www.delveinsight.com\/sample-request\/pompe-disease-pipeline-insight<\/a><\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Scope of the&nbsp;Pompe Disease Pipeline&nbsp;Report<\/strong><\/p>\n<ul style=\"text-align: justify;\">\n<li><strong>Coverage<\/strong>: Global<\/li>\n<li><strong>Key Pompe Disease Companies:<\/strong> Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics, Novartis AG, Fresenius Kabi AG, Akorn Incorporated, Teva Pharmaceutical, Mylan N.V, Johnsons &amp; Johnsons, F. Hoffman-La Roche Ltd., Trinity Biotech plc, and others.<\/li>\n<li><strong>Key Pompe Disease Pipeline Therapies<\/strong>: Cipaglucosidase alfa, SPK-3006, ACTUS 101, AT845, RP A501, AIM vectors, AVR RD 03, GYS1 Program, ETV-GAA, Research programme: glycogen storage disease type II therapy, AOC-Pompe disease, and others.<\/li>\n<\/ul>\n<p style=\"text-align: justify;\">&nbsp;<\/p>\n<p style=\"text-align: justify;\"><strong>Visit to know more of what&rsquo;s covered @ Pompe Disease Emerging Therapies, Treatments and Ongoing Clinical Trials -@ <a rel=\"nofollow\" href=\"https:\/\/www.delveinsight.com\/sample-request\/pompe-disease-pipeline-insight?utm_source=%20abnewswire&amp;utm_medium=pressrelease&amp;utm_campaign=apr\">https:\/\/www.delveinsight.com\/sample-request\/pompe-disease-pipeline-insight<\/a><\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>&nbsp;<\/strong><\/p>\n<p style=\"text-align: justify;\"><strong>Table of Contents<\/strong><\/p>\n<ol style=\"text-align: justify;\" start=\"1\">\n<li>Report Introduction<\/li>\n<li>Pompe Disease Disease Overview<\/li>\n<li>Pompe Disease Pipeline Outlook<\/li>\n<li>Comparative Analysis<\/li>\n<li>Pompe Disease Therapeutic Products in Clinical Stage<\/li>\n<li>Pompe Disease Late Stage Products (Phase III)<\/li>\n<li>Pompe Disease Mid Stage Products (Phase II)<\/li>\n<li>Pompe Disease Early Stage Products (Phase I)<\/li>\n<li>Pompe Disease Therapeutic Products in Non-clinical Stage<\/li>\n<li>Pompe Disease Preclinical and Discovery Stage Products<\/li>\n<li>Pompe Disease &ndash; DelveInsight&rsquo;s Analytical Perspective<\/li>\n<li>In-depth Commercial Assessment<\/li>\n<li>Pompe Disease Collaboration Deals<\/li>\n<li>Pompe Disease Therapeutics Pipeline Analysis<\/li>\n<li>Inactive Pompe Disease Pipeline Products<\/li>\n<li>Pompe Disease Companies<\/li>\n<li>Pompe Disease Unmet Needs<\/li>\n<li>Pompe Disease Market Drivers and Barriers<\/li>\n<li>Pompe Disease- Future Perspectives and Conclusion<\/li>\n<li>Appendix<\/li>\n<li>Report Methodology<\/li>\n<li>Pompe Disease Consulting Services<\/li>\n<li>Disclaimer<\/li>\n<li>About DelveInsight<\/li>\n<\/ol>\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_.html\" rel=\"nofollow\">DelveInsight Business Research LLP<\/a><br \/><strong>Contact Person:<\/strong> Ankit Nigam<br \/><strong>Email:<\/strong> <a href=\"https:\/\/www.abnewswire.com\/email_contact_us.php?pr=pompe-disease-pipeline-report-2023-treatment-drugs-therapies-fda-approvals-and-companies-by-delveinsight-spark-therapeutics-amicus-therapeutics-asklepios-biopharmaceutical-audentes-therapeutic\" rel=\"nofollow\">Send Email<\/a><br \/><strong>Phone:<\/strong> +19193216187<br \/><strong>Address:<\/strong>304 S. Jones Blvd #2432  <br \/><strong>City:<\/strong> Albany<br \/><strong>State:<\/strong> New York<br \/><strong>Country:<\/strong> United States<br \/><strong>Website:<\/strong> <a href=\"https:\/\/www.delveinsight.com\/consulting\" target=\"_blank\" rel=\"nofollow\">https:\/\/www.delveinsight.com\/consulting<\/a><\/p>\n<p><object type=\"text\/html\" data=\"https:\/\/www.delveinsight.com\/consulting\" style=\"width:900px; height:400px;\"><\/object><img decoding=\"async\" src=\"https:\/\/www.abnewswire.com\/press_stat.php?pr=pompe-disease-pipeline-report-2023-treatment-drugs-therapies-fda-approvals-and-companies-by-delveinsight-spark-therapeutics-amicus-therapeutics-asklepios-biopharmaceutical-audentes-therapeutic\" alt=\"\" width=\"1px\" height=\"1px\" \/><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Pompe Disease Pipeline (Albany, USA) DelveInsight&rsquo;s,&nbsp;&ldquo;Pompe Disease Pipeline Insights, 2023,&rdquo;&nbsp;report provides comprehensive insights about&nbsp;15+ companies and 20+ pipeline drugs&nbsp;in Pompe Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics &hellip; <a href=\"https:\/\/www.abnewswire.com\/pressreleases\/pompe-disease-pipeline-report-2023-treatment-drugs-therapies-fda-approvals-and-companies-by-delveinsight-spark-therapeutics-amicus-therapeutics-asklepios-biopharmaceutical-audentes-therapeutic_673686.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":[401,405,417,406,423],"tags":[],"class_list":["post-673686","post","type-post","status-publish","format-standard","hentry","category-Business","category-Health-Medicine","category-Marketing-Sales","category-Pharmaceuticals-Biotech","category-Professional-Services"],"_links":{"self":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts\/673686","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=673686"}],"version-history":[{"count":0,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/posts\/673686\/revisions"}],"wp:attachment":[{"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/media?parent=673686"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/categories?post=673686"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.abnewswire.com\/pressreleases\/wp-json\/wp\/v2\/tags?post=673686"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}