REGENXBIO Inc. yakazivisa mamwe mashoko akanaka enguva pfupi kubva kuChikamu II ALTITUDE ™ muedzo weRGX-314 wekurapa chirwere cheshuga retinopathy (DR) pasina centre-involved diabetic macular edema (CI-DME) uchishandisa mu-office suprachoroidal delivery. Iyo data iri kuiswa paAngiogenesis, Exudation, uye Degeneration 2022 musangano naMichael A. Klufas, MD, Retina Service, Wills Eye Hospital, Mubatsiri Purofesa weOphthalmology, Thomas Jefferson University. RGX-314 iri kuongororwa seyaigona imwe-nguva gene therapy yekurapa kwekunyorova kwakabatana nezera remacular degeneration uye DR.
Kudzidza Dhizaini uye Chengetedzo Yekuvandudza kubva Phase II ALTITUDE Muedzo weRGX-314 weKurapwa kweDR Uchishandisa Suprachoroidal Delivery.
ALTITUDE inzvimbo yakawanda, yakazaruka-label, randomized, controlled dose-escalation trial inoongorora kushanda, kuchengeteka uye kushivirira kwe suprachoroidal kusununguka kweRGX-314 uchishandisa SCS Microinjector® kune varwere vane chirwere cheDR chechirwere chechirwere cheshuga chepakati kana chakanyanya. retinopathy (NPDR) kana mild proliferative diabetesic retinopathy (PDR). Varwere makumi maviri muCohort 1 vakagadziriswa kuti vagamuchire RGX-314 pamwero we 2.5 × 1011 genomic makopi paziso (GC / ziso) maererano nekucherechedza kutarisa pachiyero che3: 1. Cohort 2 ichabatanidza varwere ve20 randomized kuti vagamuchire RGX-314 pamwero wekuwedzera we5 × 1011 GC / ziso rinopesana nekutarisa kutarisa pachiyero che3: 1. Cohort 3 yakagadzirirwa kuongorora RGX-314 pachiyero chedosi seCohort 2 muvarwere ve20 vari neutralizing antibody (NAb) yakanaka. Kunyoresa kuri kuenderera mberi muCohorts 2 uye 3. Varwere vari mumuedzo uyu havagamuchire prophylactic immune suppressive corticosteroid therapy isati yasvika kana mushure mekutonga kweRGX-314.
Kubva munaJanuary 18, 2022, RGX-314 yakanzi yakanyatsoshivirirwa muCohort 1. Zviitiko zviviri zvakakomba zvakashata zvakataurwa muvarwere vaviri, izvo zvose zvakanga zvisingafungidzirwi zvine chokuita nemishonga. Pakati pevarwere muCohort 1 dosed neRGX-314, hapana kuzvimba kwemukati kwakaonekwa. Sezvakambotaurwa, mumwe murwere akawana nyoro nyaya ye episcleritis yakagadziriswa ne topical corticosteroids. Kurapwa kwakajairika kwemaziso kwakamuka zviitiko zvakashata muziso rekudzidza kuburikidza nemwedzi mitanhatu zvaisaonekwa zvine chekuita nezvinodhaka uye zvaive zvakanyanya zvinyoro. Izvi zvaisanganisira conjunctival hemorrhage uye conjunctival hyperemia.
Pfupiso yeData yeCohort 1 paMwedzi Mitanhatu
Pamwedzi mitanhatu, yevarwere gumi nevashanu vakapihwa RGX-15 muCohort 314, varwere vanomwe (1%) vakaratidza nhanho mbiri kana kuvandudzwa kubva padanho rekutanga paKurapa Kwekutanga Kwechirwere cheshuga Retinopathy Chidzidzo-Diabetic Retinopathy Severity Scale (DRSS), the Chidzidzo chekutanga chekupedzisira, zvichienzaniswa ne zero yevarwere vashanu (47%) muboka rekutarisa rekutarisa. Mumwe murwere (0%) akadzvinyirirwa neRGX-7 anoenderera mberi nekuratidza nhanho ina. Iro muzana yevarwere veCohort 314 vakaiswa neRGX-1 vachiwana zvishoma-matanho maviri ekuvandudza pamwedzi mitanhatu muRGX-314 maziso akabatwa (314%) yakawedzera kubva pane zvakambotaurwa zvemwedzi mitatu (47%). Kuvandudzwa kwematanho maviri muDRSS kwakagamuchirwa seyakakosha magumo neFDA kune DR kiriniki miedzo.
Muvarwere vanomwe vaiva neNPDR (DR severity level 47-53) pakutanga, 57% yevarwere vakaratidza nhanho mbiri kana hukuru huri nani kubva kune yekutanga DRSS pamwedzi mitanhatu mushure mekutonga kweRGX-314. Muvarwere vasere vaiva nePDR (DR severity level ≥ 61) pakutanga, 38% yevarwere vakaratidza nhanho mbiri kana hukuru huri nani pamwedzi mitanhatu mushure mekutonga kweRGX-314.
Mwedzi mitanhatu mushure mekutonga kweRGX-314, varwere veCohort 1 vakaratidza kugadzikana kunoreva kuchinja muBCVA ye + 0.3 tsamba kana ichienzaniswa nekutanga, nepo varwere vashanu vari mukutarisa kwekutarisa ruoko vakaratidza kugadzikana kunoreva kuchinja muBCVA ye -2.0 mavara akaenzaniswa nekutanga.
