{"id":19969,"date":"2025-03-17T11:28:47","date_gmt":"2025-03-17T11:28:47","guid":{"rendered":"https:\/\/www.hotelsalepage.com\/feed\/cision-pr-newswire\/rona-therapeutics-receives-ind-clearance-from-fda-to-initiate-phase-2-study-of-rn0361-an-apoc3-targeted-sirna-for-management-of-hypertriglyceridemia\/"},"modified":"2025-03-17T11:28:47","modified_gmt":"2025-03-17T11:28:47","slug":"rona-therapeutics-receives-ind-clearance-from-fda-to-initiate-phase-2-study-of-rn0361-an-apoc3-targeted-sirna-for-management-of-hypertriglyceridemia","status":"publish","type":"post","link":"https:\/\/thaipropertynews.com\/feeds\/?p=19969","title":{"rendered":"Rona Therapeutics Receives IND Clearance from FDA to Initiate Phase 2 Study of RN0361: an APOC3 Targeted siRNA for Management of Hypertriglyceridemia"},"content":{"rendered":"<p><span class=\"legendSpanClass\"><span class=\"xn-location\">SHANGHAI<\/span><\/span>, <span class=\"legendSpanClass\"><span class=\"xn-chron\">March 17, 2025<\/span><\/span> \/PRNewswire\/ &#8212;\u00a0Rona Therapeutics Inc., a global leader in RNA-targeted therapeutics, announced the clearance of an Investigational New Drug (IND) by the U.S. Food and Drug Administration (FDA) for\u00a0RN0361-an APOC3 targeted siRNA\u00a0(small interfering RNA) therapeutic for severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS) treatment. The company plans to advance RN0361 development in HTG patients based on a favorable safety profile and sustained triglyceride (TG) reduction in a 6-month single dose phase 1 clinical trial. The upcoming randomized, double-blind, placebo controlled, multicenter global Ph2 study will evaluate RN0361 efficacy, safety and duration in hypertriglyceridemia patient population for nine-month follow-up period.<\/p>\n<p>RN0361 is a potential best-in-class APOC3 target\u00a0siRNA\u00a0utilizes Rona&#8217;s proprietary siRNA conjugate and oligo chemistry platform\u00a0GAIA to enable robust and durable hepatocyte-specific silencing via subcutaneous administration. The therapy achieves durable gene silencing through optimized chemical modifications, significantly silencing APOC3 mRNA and reducing the expression of APOC3 protein.<\/p>\n<p>&#8220;This IND submission underscores our commitment to advance transformative RNAi therapies for patients with severe lipid disorders,&#8221; said\u00a0<b><span class=\"xn-person\">Stella Shi<\/span>, CEO and Founder of Rona Therapeutics<\/b>. &#8220;RN0361&#8217;s Phase I data validate its potential as a best-in-class siRNA with durable efficacy and patient-friendly dosing. We look forward to collaborating with global investigators and agency to accelerate its development.&#8221;<\/p>\n<p><b>Dr. <span class=\"xn-person\">Alex M. DePaoli<\/span>, CMO and EVP of translational medicine<\/b>, was excited to share: &#8220;This Phase 2 study of RN0361 in dysmetabolic patients with hypertriglyceridemia will built on the evidence for RN0361 as an important therapeutic for multiple populations with significant hypertriglyceridemia and its pathologic consequences.&#8221; He added, &#8220;The remarkable potency and durability of triglyceride reduction already seen in the First in Human single dose study offers patients a potential powerful tool in the management of a substantial unmet need.&#8221;<\/p>\n<p><b>About Rona Therapeutics <\/b><\/p>\n<p>Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of metabolic and neurological diseases. Rona Therapeutics is always committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH. In addition, Rona Therapeutics is unlocking the potential of extra-hepatic delivery for neurological disorders and adipose\/muscle delivery or various metabolic syndromes.<\/p>","protected":false},"excerpt":{"rendered":"<p><!-- wp:html --><\/p>\n<p><span class=\"legendSpanClass\"><span class=\"xn-location\">SHANGHAI<\/span><\/span>, <span class=\"legendSpanClass\"><span class=\"xn-chron\">March 17, 2025<\/span><\/span> \/PRNewswire\/ &#8212;\u00a0Rona Therapeutics Inc., a global leader in RNA-targeted therapeutics, announced the clearance of an Investigational New Drug (IND) by the U.S. Food and Drug Administration (FDA) for\u00a0RN0361-an APOC3 targeted siRNA\u00a0(small interfering RNA) therapeutic for severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS) treatment. The company plans to advance RN0361 development in HTG patients based on a favorable safety profile and sustained triglyceride (TG) reduction in a 6-month single dose phase 1 clinical trial. The upcoming randomized, double-blind, placebo controlled, multicenter global Ph2 study will evaluate RN0361 efficacy, safety and duration in hypertriglyceridemia patient population for nine-month follow-up period.<\/p>\n<p>RN0361 is a potential best-in-class APOC3 target\u00a0siRNA\u00a0utilizes Rona&#8217;s proprietary siRNA conjugate and oligo chemistry platform\u00a0GAIA to enable robust and durable hepatocyte-specific silencing via subcutaneous administration. The therapy achieves durable gene silencing through optimized chemical modifications, significantly silencing APOC3 mRNA and reducing the expression of APOC3 protein.<\/p>\n<p>&#8220;This IND submission underscores our commitment to advance transformative RNAi therapies for patients with severe lipid disorders,&#8221; said\u00a0<b><span class=\"xn-person\">Stella Shi<\/span>, CEO and Founder of Rona Therapeutics<\/b>. &#8220;RN0361&#8217;s Phase I data validate its potential as a best-in-class siRNA with durable efficacy and patient-friendly dosing. We look forward to collaborating with global investigators and agency to accelerate its development.&#8221;<\/p>\n<p><b>Dr. <span class=\"xn-person\">Alex M. DePaoli<\/span>, CMO and EVP of translational medicine<\/b>, was excited to share: &#8220;This Phase 2 study of RN0361 in dysmetabolic patients with hypertriglyceridemia will built on the evidence for RN0361 as an important therapeutic for multiple populations with significant hypertriglyceridemia and its pathologic consequences.&#8221; He added, &#8220;The remarkable potency and durability of triglyceride reduction already seen in the First in Human single dose study offers patients a potential powerful tool in the management of a substantial unmet need.&#8221;<\/p>\n<p><b>About Rona Therapeutics <\/b><\/p>\n<p>Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of metabolic and neurological diseases. Rona Therapeutics is always committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH. In addition, Rona Therapeutics is unlocking the potential of extra-hepatic delivery for neurological disorders and adipose\/muscle delivery or various metabolic syndromes.<\/p>\n<p><!-- \/wp:html --><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"rop_custom_images_group":[],"rop_custom_messages_group":[],"rop_publish_now":"initial","rop_publish_now_accounts":[],"rop_publish_now_history":[],"rop_publish_now_status":"pending","footnotes":""},"categories":[5,7],"tags":[],"class_list":["post-19969","post","type-post","status-publish","format-standard","hentry","category-cision-pr-newswire","category-cision-pr-newswire-en"],"_links":{"self":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/posts\/19969","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=19969"}],"version-history":[{"count":0,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/posts\/19969\/revisions"}],"wp:attachment":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=19969"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=19969"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=19969"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}