{"id":8898,"date":"2026-10-30T14:28:56","date_gmt":"2026-10-30T11:28:56","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8898"},"modified":"2026-10-30T14:35:21","modified_gmt":"2026-10-30T11:35:21","slug":"pepr-124-rt001-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/pepr-124-rt001-duchenne\/","title":{"rendered":"PEPR-124 (RT001): Duchenne Kas Distrofisi \u0130\u00e7in Yapay Zeka Taraf\u0131ndan Ke\u015ffedilen, Mutasyondan Ba\u011f\u0131ms\u0131z Bir \u0130la\u00e7 Aday\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>PEPR-124 (RT001 olarak da adland\u0131r\u0131l\u0131r), Hindistan&#039;\u0131n Bengaluru \u015fehrinde bulunan bir giri\u015fim \u015firketi olan Peptris Technologies taraf\u0131ndan geli\u015ftirilen ve \u00f6zellikle Duchenne Musk\u00fcler Distrofisi (DMD) hastal\u0131\u011f\u0131n\u0131 hedefleyen, yapay zeka taraf\u0131ndan ke\u015ffedilmi\u015f bir ila\u00e7 aday\u0131d\u0131r.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">2019 y\u0131l\u0131nda kurulan ve C-CAMP&#039;te faaliyet g\u00f6steren Peptris, nadir hastal\u0131klar, kanser ve iltihaplanma gibi eskiden &quot;ila\u00e7la tedavi edilemez&quot; hastal\u0131klar i\u00e7in optimize edilmi\u015f molek\u00fcller tasarlamak amac\u0131yla yapay zeka ve \u00fcretken kimyay\u0131 kullan\u0131yor. Hindistan&#039;\u0131n d\u00fc\u015f\u00fck maliyetli ekosisteminden yararlanarak tam \u00f6l\u00e7ekli bir ila\u00e7 ke\u015fif \u015firketine d\u00f6n\u00fc\u015fmek i\u00e7in RDI Fonu&#039;ndan ald\u0131\u011f\u0131 86 crore rupilik (yakla\u015f\u0131k 9.047.200 ABD dolar\u0131) yeni bir hibe ile desteklenen Peptris, bu yeniden tasarlanm\u0131\u015f varl\u0131\u011f\u0131 k\u00fcresel olarak ba\u015far\u0131yla lisanslad\u0131. Kurucu ortak ve CEO Venkatasubramanian Narayanan&#039;\u0131n (Venkat) vurgulad\u0131\u011f\u0131 gibi, &quot;Yapay zeka i\u015fleri h\u0131zland\u0131r\u0131yor, ancak bilim hala yol g\u00f6steriyor.&quot; Bu geli\u015fme, daha \u00f6nce son derece s\u0131n\u0131rl\u0131 tedavi se\u00e7enekleriyle kar\u015f\u0131 kar\u015f\u0131ya kalan Hindistan&#039;daki tahmini 400.000 DMD hastas\u0131 i\u00e7in hayati bir at\u0131l\u0131m anlam\u0131na geliyor.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#discovery-and-structural-overview\">PEPR-124: Ke\u015fif ve Yap\u0131sal Genel Bak\u0131\u015f<\/a><\/li><li><a href=\"#mechanism-of-action-mutation-agnostic-utrophin-upregulation\">Etki Mekanizmas\u0131: Mutasyondan Ba\u011f\u0131ms\u0131z Utrofin Art\u0131\u015f\u0131<\/a><\/li><li><a href=\"#preclinical-efficacy-and-safety-data\">Klinik \u00d6ncesi Etkinlik ve G\u00fcvenlik Verileri<\/a><\/li><li><a href=\"#commercialization-strategic-licensing-and-regulatory-status\">Ticari Hale Getirme, Stratejik Lisanslama ve D\u00fczenleyici Durum<\/a><\/li><li><a href=\"#future-outlook-the-hybrid-clinical-path\">Gelece\u011fe Bak\u0131\u015f: Hibrit Klinik Yol<\/a><\/li><li><a href=\"#references\">Referanslar<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"discovery-and-structural-overview\" class=\"wp-block-heading\">PEPR-124: Ke\u015fif ve Yap\u0131sal Genel Bak\u0131\u015f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>PEPR-124, ba\u015flang\u0131\u00e7ta Peptris Technologies taraf\u0131ndan, kapsaml\u0131 kimyasal alanlar\u0131 haritalamak ve i\u015flevsel k\u00fc\u00e7\u00fck molek\u00fclleri izole etmek amac\u0131yla C-CAMP&#039;te kendi geli\u015ftirdi\u011fi hesaplama platformu (pepAI) kullan\u0131larak ke\u015ffedilmi\u015ftir.<\/strong> Son d\u00f6nemde ald\u0131\u011f\u0131 86 crore rupilik Ar-Ge Fonu hibesiyle desteklenen \u015firket, Hindistan&#039;daki d\u00fc\u015f\u00fck i\u015fletme maliyetlerinden yararlanarak, tedavisi zor hastal\u0131klar i\u00e7in kal\u0131c\u0131 ve eri\u015filebilir \u00e7\u00f6z\u00fcmler geli\u015ftirmek amac\u0131yla tam \u00f6l\u00e7ekli bir ila\u00e7 ke\u015fif firmas\u0131na d\u00f6n\u00fc\u015fmek i\u00e7in aktif olarak geni\u015fliyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Y\u00fcksek translasyonel riskler ta\u015f\u0131yan yeni sentezlenmi\u015f bile\u015fiklerin aksine, PEPR-124 yeniden ama\u00e7land\u0131r\u0131lm\u0131\u015f bir varl\u0131kt\u0131r. Bu stratejik tan\u0131mlama, temel molek\u00fcl\u00fcn yerle\u015fik g\u00fcvenlik profillerine sahip oldu\u011fu anlam\u0131na gelir; bu da tipik on y\u0131ll\u0131k preklinik do\u011frulama s\u00fcrecini \u00f6nemli \u00f6l\u00e7\u00fcde k\u0131salt\u0131r ve geli\u015ftirme s\u0131ras\u0131nda toksisite veya karars\u0131zl\u0131k kaynakl\u0131 ba\u015far\u0131s\u0131zl\u0131klar\u0131 azalt\u0131r.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"mechanism-of-action-mutation-agnostic-utrophin-upregulation\" class=\"wp-block-heading\">Etki Mekanizmas\u0131: Mutasyondan Ba\u011f\u0131ms\u0131z Utrofin Art\u0131\u015f\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMD, kas h\u00fccresi zarlar\u0131n\u0131 stabilize eden kritik bir protein olan distrofinin i\u015flevsel \u00fcretimini engelleyen genetik hatalardan kaynaklanan, y\u0131k\u0131c\u0131 ve ilerleyici bir n\u00f6rom\u00fcsk\u00fcler bozukluktur. Standart gen d\u00fczeltme teknikleri (ekson atlama gibi) s\u0131n\u0131rl\u0131d\u0131r \u00e7\u00fcnk\u00fc belirli genetik varyantlar\u0131 hedef al\u0131rlar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>PEPR-124, mutasyondan ba\u011f\u0131ms\u0131z bir tedavi yakla\u015f\u0131m\u0131 uygulayarak bu k\u0131s\u0131tlamay\u0131 a\u015fmaktad\u0131r:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Hedef Protein:<\/strong> Bu \u00e7al\u0131\u015fma, embriyonik geli\u015fimde distrofin ile i\u015flevsel olarak \u00f6zde\u015f olan yap\u0131sal bir analog protein olan utrofinin yukar\u0131 d\u00fczenlenmesine odaklanmaktad\u0131r.<\/li>\n\n\n\n<li><strong>H\u00fccresel Telafi:<\/strong> Yerel \u00fctrofin seviyelerinde kal\u0131c\u0131 bir art\u0131\u015f sa\u011flayarak, eksik olan distrofin yap\u0131s\u0131n\u0131 telafi eder ve \u00e7ocu\u011fun genetik delesyon diziliminden ba\u011f\u0131ms\u0131z olarak kas liflerini mekanik gerilime kar\u015f\u0131 korur.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"preclinical-efficacy-and-safety-data\" class=\"wp-block-heading\">Klinik \u00d6ncesi Etkinlik ve G\u00fcvenlik Verileri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Kas Distrofisi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131 da dahil olmak \u00fczere b\u00fcy\u00fck bilimsel sempozyumlarda sunulan preklinik \u00e7al\u0131\u015fmalar, \u00f6nemli terap\u00f6tik belirte\u00e7ler ortaya koymaktad\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>In Vitro Testler:<\/strong> PEPR-124, hem C2C12 miyoblastlar\u0131nda hem de do\u011frudan insan DMD hastalar\u0131ndan elde edilen insan kaynakl\u0131 pluripotent k\u00f6k h\u00fccrelerde (hIPSC&#039;ler) doza ba\u011f\u0131ml\u0131 \u00fctrofin art\u0131\u015f\u0131n\u0131 ba\u015far\u0131yla sa\u011flam\u0131\u015ft\u0131r.<\/li>\n\n\n\n<li><strong>Canl\u0131da Kas Fonksiyonu:<\/strong> 28 ve 90 g\u00fcnl\u00fck aral\u0131klarla de\u011ferlendirilen \u015fiddetli D2-Mdx fare modellerinde (25-100 mg\/kg dozlar\u0131nda), bile\u015fik genel kas g\u00fcc\u00fcnde, kavrama g\u00fcc\u00fcnde, as\u0131l\u0131 kalma gecikme testlerinde ve genel ko\u015fu band\u0131 performans\u0131nda \u00f6nemli iyile\u015fmeler sa\u011flam\u0131\u015ft\u0131r.<\/li>\n\n\n\n<li><strong>Kombinasyon Sinerjileri:<\/strong> Standart tedavi kortikosteroidi deflazacort ile birlikte uyguland\u0131\u011f\u0131nda, PEPR-124 \u00fcst\u00fcn yap\u0131sal kas korumas\u0131, geli\u015fmi\u015f as\u0131lma dayan\u0131kl\u0131l\u0131\u011f\u0131 ve serum kreatin kinaz d\u00fczeyinde (devam eden kas y\u0131k\u0131m\u0131 i\u00e7in alt\u0131n standart bir kan biyobelirteci) belirgin bir azalma sa\u011flad\u0131.<\/li>\n\n\n\n<li><strong>G\u00fcvenlik Profili:<\/strong> Daha da \u00f6nemlisi, PEPR-124, v\u00fccut a\u011f\u0131rl\u0131\u011f\u0131 art\u0131\u015f\u0131 veya metabolik toksisite a\u00e7\u0131s\u0131ndan herhangi bir olumsuz olay g\u00f6stermedi ve uzun s\u00fcreli kortikosteroid monoterapisine k\u0131yasla olumlu bir g\u00fcvenlik marj\u0131 sundu.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"commercialization-strategic-licensing-and-regulatory-status\" class=\"wp-block-heading\">Ticari Hale Getirme, Stratejik Lisanslama ve D\u00fczenleyici Durum<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Uluslararas\u0131 klinik geli\u015ftirmeyi h\u0131zland\u0131rmak amac\u0131yla Peptris Technologies, onayl\u0131 ila\u00e7lar\u0131n optimizasyonunda uzmanla\u015fm\u0131\u015f ABD merkezli \u00f6zel bir ila\u00e7 giri\u015fim \u015firketi olan Revio Therapeutics ile k\u00fcresel bir lisans anla\u015fmas\u0131 imzalad\u0131.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Piyasa Tahsisleri:<\/strong> Revio Therapeutics, k\u00fcresel \u00e7apta m\u00fcnhas\u0131r geli\u015ftirme ve ticarile\u015ftirme haklar\u0131na sahipken, Peptris ise \u00f6zellikle BRICS pazarlar\u0131 i\u00e7in ticari haklar\u0131n\u0131 elinde bulundurmaktad\u0131r.<\/li>\n\n\n\n<li><strong>FDA Yetim \u0130la\u00e7 Tan\u0131m\u0131:<\/strong> Bu varl\u0131k, ABD FDA&#039;nin RT001\/PEPR-124&#039;e Yetim \u0130la\u00e7 Stat\u00fcs\u00fc (ODD) vermesiyle hayati bir d\u00fczenleyici d\u00f6n\u00fcm noktas\u0131na ula\u015ft\u0131. Bu d\u00fczenleyici stat\u00fc, geli\u015ftirme te\u015fvikleri, klinik testler i\u00e7in vergi indirimleri, PDUFA \u00fccretlerinden muafiyet ve potansiyel onay sonras\u0131 pazar m\u00fcnhas\u0131rl\u0131\u011f\u0131 sa\u011flamaktad\u0131r.<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"future-outlook-the-hybrid-clinical-path\" class=\"wp-block-heading\">Gelece\u011fe Bak\u0131\u015f: Hibrit Klinik Yol<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>2026 y\u0131l\u0131n\u0131n sonlar\u0131na do\u011fru PEPR-124, Faz 2&#039;ye haz\u0131r bir varl\u0131k olarak s\u0131n\u0131fland\u0131r\u0131lm\u0131\u015ft\u0131r. Revio Therapeutics, FDA&#039;nin d\u00fczenleyici 505(b)(2) hibrit yolundan yararlanmaktad\u0131r.<\/strong> Bu, sponsorun temel k\u00fc\u00e7\u00fck molek\u00fcl i\u00e7in mevcut g\u00fcvenlik verilerine ba\u015fvurmas\u0131na olanak tan\u0131yarak, sa\u011fl\u0131kl\u0131 g\u00f6n\u00fcll\u00fclerde gereksiz Faz 1 g\u00fcvenlik denemelerine olan ihtiyac\u0131 ortadan kald\u0131r\u0131r. Revio, DMD tan\u0131s\u0131 konmu\u015f \u00e7ocuklarda yap\u0131lacak olan Faz 2 klinik denemelerinin yap\u0131s\u0131n\u0131 kesinle\u015ftirmek i\u00e7in ABD FDA ile \u00f6n-IND (Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7) toplant\u0131lar\u0131 haz\u0131rl\u0131yor.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"references\" class=\"wp-block-heading\">Referanslar<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Revio Therapeutics. <em>\u201cRevio, Kas Distrofisi \u0130\u00e7in RT001 (PEPR124) Lisans\u0131n\u0131 Almak \u00dczere Peptris ile Ortakl\u0131k Kurdu.\u201d<\/em> <a href=\"https:\/\/www.reviotx.com\/blog\/revio-partners-with-peptris\" target=\"_blank\" rel=\"noopener\">Revio Terapi Blogu<\/a>.<\/li>\n\n\n\n<li>Senin Hikayen. <em>\u201cPeptris, yapay zekay\u0131 kullan\u0131yor ve Duchenne kas distrofisi aday\u0131 i\u00e7in lisans veriyor; 86 crore rupilik Ar-Ge hibesi kazan\u0131yor.\u201d<\/em> <a href=\"https:\/\/yourstory.com\/2026\/08\/peptris-ai-drug-discovery\" target=\"_blank\" rel=\"noopener\">YourStory Teknoloji Medya<\/a>.<\/li>\n\n\n\n<li>Clival Haberleri. <em>\u201cPeptris, Hindistan&#039;da Yapay Zeka ile Ke\u015ffedilen \u0130lk \u0130lac\u0131n Lisans\u0131n\u0131 Duchenne Kas Distrofisi \u0130\u00e7in Revio Therapeutics&#039;e Verdi.\u201d<\/em> <a href=\"https:\/\/clival.com\/news\/peptris-licenses-indias-first-ai-discovered-drug-to-revio-therapeutics\" target=\"_blank\" rel=\"noopener\">Clival Biyoteknoloji Pazarlar\u0131<\/a>.<\/li>\n\n\n\n<li>Revio Therapeutics. <em>\u201cRT001 D\u00fczenleyici D\u00f6n\u00fcm Noktas\u0131: FDA, Duchenne Kas Distrofisi Program\u0131na Yetim \u0130la\u00e7 Stat\u00fcs\u00fc Kazand\u0131rd\u0131.\u201d<\/em> <a href=\"https:\/\/www.reviotx.com\/blog\/%20rt001-regulatory-milestone\" target=\"_blank\" rel=\"noopener\">Revio Therapeutics Haberleri<\/a>.<\/li>\n\n\n\n<li>Kas Distrofisi Derne\u011fi (MDA). <em>\u201cPEPR124 (RT001): Duchenne Musk\u00fcler Distrofisi i\u00e7in mutasyondan ba\u011f\u0131ms\u0131z ve g\u00fcvenli bir tedavi olarak yeniden tasarlanm\u0131\u015f k\u00fc\u00e7\u00fck molek\u00fcll\u00fc bir ila\u00e7.\u201d<\/em> <a href=\"https:\/\/www.mdaconference.org\/abstract-library\/pepr124-rt001-a-repurposed-small-molecule-drug-as-a-mutation-agnostic-and-safe-therapeutic-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener\">MDA Konferans \u00d6zet K\u00fct\u00fcphanesi<\/a>.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>PEPR-124 (also designated as RT001) is an AI-discovered drug candidate developed by Peptris Technologies, a Bengaluru startup based in India, specifically targeting Duchenne Muscular Dystrophy (DMD). Founded in 2019 and operating from C-CAMP, Peptris uses artificial intelligence and generative chemistry to design optimized molecules for once &#8220;undruggable&#8221; diseases like rare illnesses, cancer, and inflammation. Supported [&hellip;]<\/p>\n","protected":false},"author":3,"featured_media":8901,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[83,937,938,939,940,925],"class_list":["post-8898","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-india","tag-pepr-124","tag-peptris-technologies","tag-revio-therapeutics","tag-rt001","tag-utrophin"],"subtitle":"Yapay zek\u00e2 taraf\u0131ndan ke\u015ffedilen bir ila\u00e7, Duchenne kas distrofisine yeni bir yakla\u015f\u0131m sunabilir mi? Peptris Technologies taraf\u0131ndan geli\u015ftirilen PEPR-124 (RT001), altta yatan mutasyondan ba\u011f\u0131ms\u0131z olarak utrofin \u00fcretimini art\u0131rmay\u0131 hedefliyor. Klinik \u00f6ncesi bulgular\u0131n\u0131 ve geli\u015ftirme potansiyelini ke\u015ffedin.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8898","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8898"}],"version-history":[{"count":4,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8898\/revisions"}],"predecessor-version":[{"id":8905,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8898\/revisions\/8905"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8901"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8898"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8898"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8898"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}