{"id":45657,"date":"2026-01-28T00:04:34","date_gmt":"2026-01-27T17:04:34","guid":{"rendered":"https:\/\/saigonbiz.com\/vn\/skyhawk-therapeutics-cong-bo-ket-qua-nghien-cuu-tam-thoi-tren-benh-nhan-sau-9-thang-tien-hanh-thu-nghiem-lam-sang-giai-doan-1-thuoc-sky-0515-de-dieu-tri-benh-huntington\/"},"modified":"2026-01-28T00:04:34","modified_gmt":"2026-01-27T17:04:34","slug":"skyhawk-therapeutics-cong-bo-ket-qua-nghien-cuu-tam-thoi-tren-benh-nhan-sau-9-thang-tien-hanh-thu-nghiem-lam-sang-giai-doan-1-thuoc-sky-0515-de-dieu-tri-benh-huntington","status":"publish","type":"post","link":"https:\/\/saigonbiz.com\/vn\/skyhawk-therapeutics-cong-bo-ket-qua-nghien-cuu-tam-thoi-tren-benh-nhan-sau-9-thang-tien-hanh-thu-nghiem-lam-sang-giai-doan-1-thuoc-sky-0515-de-dieu-tri-benh-huntington\/","title":{"rendered":"Skyhawk Therapeutics C\u00f4ng B\u1ed1 K\u1ebft Qu\u1ea3 Nghi\u00ean C\u1ee9u T\u1ea1m Th\u1eddi tr\u00ean B\u1ec7nh Nh\u00e2n sau 9 Th\u00e1ng ti\u1ebfn h\u00e0nh Th\u1eed Nghi\u1ec7m L\u00e2m S\u00e0ng Giai \u0110o\u1ea1n 1 thu\u1ed1c SKY-0515 \u0111\u1ec3 \u0110i\u1ec1u Tr\u1ecb b\u1ec7nh Huntington"},"content":{"rendered":"
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Sau 9 th\u00e1ng, d\u1eef li\u1ec7u cho th\u1ea5y b\u1ec7nh nh\u00e2n s\u1eed d\u1ee5ng thu\u1ed1c n\u00e0y c\u00f3 m\u1ee9c c\u1ea3i thi\u1ec7n trung b\u00ecnh \u0111\u1ea1t +0,64 \u0111i\u1ec3m (theo Thang \u0110i\u1ec3m \u0110\u00e1nh Gi\u00e1 B\u1ec7nh Huntington Th\u1ed1ng Nh\u1ea5t T\u1ed5ng H\u1ee3p – cUHDRS) so v\u1edbi m\u1ee9c ban \u0111\u1ea7u, <\/b>trong khi theo di\u1ec5n bi\u1ebfn t\u1ef1 nhi\u00ean c\u1ee7a b\u1ec7nh, t\u00ecnh tr\u1ea1ng c\u1ee7a b\u1ec7nh nh\u00e2n c\u00f3 tri\u1ec7u ch\u1ee9ng th\u01b0\u1eddng chuy\u1ec3n bi\u1ebfn x\u1ea5u \u0111i -0,73 \u0111i\u1ec3m (theo thang cUHDRS) trong c\u00f9ng kho\u1ea3ng th\u1eddi gian 9 th\u00e1ng, d\u1ef1a tr\u00ean ph\u01b0\u01a1ng ph\u00e1p hi\u1ec7u ch\u1ec9nh b\u1eb1ng tr\u1ecdng s\u1ed1 \u0111i\u1ec3m xu h\u01b0\u1edbng.<\/i><\/p>\n

Skyhawk c\u0169ng th\u00f4ng b\u00e1o m\u1edf r\u1ed9ng quy m\u00f4 Giai \u0110o\u1ea1n 2\/3 c\u1ee7a th\u1eed nghi\u1ec7m FALCON-HD cho thu\u1ed1c SKY-0515 tr\u00ean to\u00e0n th\u1ebf gi\u1edbi. Hi\u1ec7n t\u1ea1i, \u0111\u00e3 c\u00f3 h\u01a1n 90 b\u1ec7nh nh\u00e2n d\u00f9ng thu\u1ed1c c\u1ee7a Skyhawk.<\/i><\/p>\n

BOSTON<\/span>, ng\u00e0y 27 th\u00e1ng M\u1ed9t n\u0103m 2026<\/span> \/PRNewswire\/ —\u00a0Skyhawk Therapeutics, Inc.<\/a>, m\u1ed9t c\u00f4ng ty c\u00f4ng ngh\u1ec7 sinh h\u1ecdc giai \u0111o\u1ea1n l\u00e2m s\u00e0ng, chuy\u00ean ph\u00e1t tri\u1ec3n c\u00e1c li\u1ec7u ph\u00e1p ph\u00e2n t\u1eed nh\u1ecf ti\u00ean ti\u1ebfn \u0111\u1ec3 \u0111i\u1ec1u ch\u1ec9nh c\u00e1c m\u1ee5c ti\u00eau RNA quan tr\u1ecdng, h\u00f4m nay \u0111\u00e3 c\u00f4ng b\u1ed1 k\u1ebft qu\u1ea3 t\u00edch c\u1ef1c t\u1eeb ph\u00e2n t\u00edch t\u1ea1m th\u1eddi sau 9 th\u00e1ng \u0111\u1ed1i v\u1edbi ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111ang trong giai \u0111o\u1ea1n th\u1eed nghi\u1ec7m c\u1ee7a C\u00f4ng Ty cho b\u1ec7nh Huntington (HD) b\u1eb1ng SKY-0515.<\/p>\n

\"Skyhawk<\/a>
Skyhawk Therapeutics, Inc.<\/span> <\/div>\n

Vi\u1ec7c \u0111i\u1ec1u tr\u1ecb b\u1eb1ng SKY-0515 l\u00e0m gi\u1ea3m 62% l\u01b0\u1ee3ng protein mHTT trong m\u00e1u v\u1edbi li\u1ec1u 9mg (ph\u1ee5 thu\u1ed9c li\u1ec1u) v\u00e0 gi\u1ea3m 26% l\u01b0\u1ee3ng mRNA PMS1 (ph\u1ee5 thu\u1ed9c li\u1ec1u). PMS1 l\u00e0 y\u1ebfu t\u1ed1 then ch\u1ed1t th\u00fac \u0111\u1ea9y s\u1ef1 m\u1edf r\u1ed9ng l\u1eb7p \u0111o\u1ea1n CAG trong t\u1ebf b\u00e0o soma v\u00e0 s\u1ef1 ti\u1ebfn tri\u1ec3n b\u1ec7nh l\u00fd c\u1ee7a HD. SKY-0515 c\u0169ng \u0111\u00e3 ch\u1ee9ng minh kh\u1ea3 n\u0103ng ti\u1ebfp c\u1eadn tuy\u1ec7t v\u1eddi v\u1edbi h\u1ec7 th\u1ea7n kinh trung \u01b0\u01a1ng v\u00e0\u00a0nh\u00ecn chung l\u00e0 an to\u00e0n v\u00e0 c\u00f3 \u0111\u1ed9 dung n\u1ea1p t\u1ed1t.<\/p>\n

\u1ede c\u00e1c m\u1ed1c th\u1eddi gian 3, 6 v\u00e0 9 th\u00e1ng, nh\u1eefng ng\u01b0\u1eddi d\u00f9ng SKY-0515 thu\u1ed9c nh\u00f3m b\u1ec7nh nh\u00e2n Ph\u1ea7n C c\u1ee7a th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng Giai \u0110o\u1ea1n 1 thu\u1ed1c SKY-0515 \u0111\u00e3 c\u00f3 m\u1ee9c c\u1ea3i thi\u1ec7n trung b\u00ecnh (theo Thang \u0110i\u1ec3m \u0110\u00e1nh Gi\u00e1 B\u1ec7nh Huntington Th\u1ed1ng Nh\u1ea5t T\u1ed5ng H\u1ee3p – cUHDRS) so v\u1edbi m\u1ee9c ban \u0111\u1ea7u. \u1ede m\u1ed1c 9 th\u00e1ng, k\u1ebft qu\u1ea3 ph\u00e2n t\u00edch t\u1ed5ng h\u1ee3p cho th\u1ea5y m\u1ee9c c\u1ea3i thi\u1ec7n \u0111\u1ea1t +0,64 \u0111i\u1ec3m, trong khi theo di\u1ec5n bi\u1ebfn b\u1ec7nh t\u1ef1 nhi\u00ean, t\u00ecnh tr\u1ea1ng c\u1ee7a b\u1ec7nh nh\u00e2n c\u00f3 tri\u1ec7u ch\u1ee9ng th\u01b0\u1eddng chuy\u1ec3n bi\u1ebfn x\u1ea5u \u0111i (-0,73 \u0111i\u1ec3m) sau 9 th\u00e1ng theo thang cUHDRS, d\u1ef1a tr\u00ean ph\u01b0\u01a1ng ph\u00e1p hi\u1ec7u ch\u1ec9nh b\u1eb1ng tr\u1ecdng s\u1ed1 \u0111i\u1ec3m xu h\u01b0\u1edbng s\u1eed d\u1ee5ng d\u1eef li\u1ec7u t\u1eeb Enroll-HD v\u00e0 TRACK-HD.<\/p>\n

“T\u00f4i r\u1ea5t ph\u1ea5n kh\u1edfi tr\u01b0\u1edbc nh\u1eefng d\u1eef li\u1ec7u v\u1ec1 \u0111\u1ed9 an to\u00e0n\u00a0v\u00e0\u00a0hi\u1ec7u qu\u1ea3\u00a0ban \u0111\u1ea7u c\u1ee7a thu\u1ed1c SKY-0515 tr\u00ean b\u1ec7nh nh\u00e2n t\u1eeb th\u1eed nghi\u1ec7m Giai \u0110o\u1ea1n 1 Ph\u1ea7n C. K\u1ebft qu\u1ea3 n\u00e0y cho th\u1ea5y s\u1ef1 c\u1ea3i thi\u1ec7n r\u00f5 r\u1ec7t (theo thang cUHDRS) so v\u1edbi chuy\u1ec3n bi\u1ebfn ng\u00e0y c\u00e0ng x\u1ea5u \u0111i theo di\u1ec5n bi\u1ebfn b\u1ec7nh t\u1ef1 nhi\u00ean\u00a0t\u1ea1i c\u00e1c m\u1ed1c ph\u00e2n t\u00edch \u0111\u01b0\u1ee3c x\u00e1c \u0111\u1ecbnh tr\u01b0\u1edbc \u1edf th\u00e1ng th\u1ee9 3, th\u1ee9 6 v\u00e0 th\u1ee9 9”, Gi\u00e1o S\u01b0 Th\u1ea7n Kinh H\u1ecdc Ed Wild t\u1ea1i \u0110\u1ea1i H\u1ecdc College London cho bi\u1ebft. “SKY-0515 \u0111ang l\u00e0 lo\u1ea1i thu\u1ed1c c\u00f3 kh\u1ea3 n\u0103ng l\u00e0m gi\u1ea3m protein mHTT m\u1ea1nh nh\u1ea5t\u00a0so v\u1edbi b\u1ea5t k\u1ef3 li\u1ec7u ph\u00e1p n\u00e0o t\u1eebng \u0111\u01b0\u1ee3c th\u1eed nghi\u1ec7m tr\u00ean c\u01a1 th\u1ec3 ng\u01b0\u1eddi cho \u0111\u1ebfn nay. D\u1eef li\u1ec7u l\u00e2m s\u00e0ng v\u00e0 ch\u1ec9 d\u1ea5u sinh h\u1ecdc c\u0169ng cho th\u1ea5y thu\u1ed1c \u0111\u01b0\u1ee3c dung n\u1ea1p t\u1ed1t \u1edf t\u1ea5t c\u1ea3 c\u00e1c li\u1ec1u th\u1eed nghi\u1ec7m. SKY-0515 c\u00f3 kh\u1ea3 n\u0103ng l\u00e0m gi\u1ea3m\u00a0c\u1ea3 mHTT v\u00e0 PMS1, \u0111\u00e2y l\u00e0 “\u0111\u00f2n t\u1ea5n c\u00f4ng k\u00e9p” m\u1ea1nh m\u1ebd t\u00e1c \u0111\u1ed9ng tr\u1ef1c ti\u1ebfp v\u00e0o hai c\u01a1 ch\u1ebf g\u00e2y b\u1ec7nh c\u1ed1t l\u00f5i c\u1ee7a b\u1ec7nh Huntington. Nh\u1eefng k\u1ebft qu\u1ea3 t\u1eeb th\u1eed nghi\u1ec7m nh\u00e3n m\u1edf n\u00e0y (d\u1ef1 ki\u1ebfn s\u1ebd \u0111\u01b0\u1ee3c x\u00e1c nh\u1eadn trong th\u1eed nghi\u1ec7m FALCON-HD c\u00f3 \u0111\u1ed1i ch\u1ee9ng gi\u1ea3 d\u01b0\u1ee3c \u0111ang \u0111\u01b0\u1ee3c tri\u1ec3n khai) m\u1edf ra s\u1ef1 k\u1ef3 v\u1ecdng v\u1ec1\u00a0m\u1ed9t t\u00e1c \u0111\u1ed9ng c\u00f3 \u00fd ngh\u0129a \u0111\u1ed1i v\u1edbi nh\u1eefng ng\u01b0\u1eddi m\u1eafc b\u1ec7nh HD tr\u00ean to\u00e0n th\u1ebf gi\u1edbi \u2013 \u0111\u1ed1i v\u1edbi h\u1ecd, ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb gi\u1ea3m huntingtin \u0111\u01b0\u1eddng u\u1ed1ng nh\u01b0 SKY-0515 s\u1ebd th\u1ef1c s\u1ef1 mang t\u00ednh \u0111\u1ed9t ph\u00e1.”<\/p>\n

\u00d4ng Sergey Paushkin, Tr\u01b0\u1edfng b\u1ed9 ph\u1eadn Nghi\u00ean C\u1ee9u v\u00e0 Ph\u00e1t Tri\u1ec3n t\u1ea1i Skyhawk Therapeutics, cho bi\u1ebft: “M\u1ee5c ti\u00eau c\u1ee7a ch\u00fang t\u00f4i trong nghi\u00ean c\u1ee9u Giai \u0110o\u1ea1n 1 l\u00e0 x\u00e1c \u0111\u1ecbnh \u0111\u1ed9 an to\u00e0n v\u00e0 kh\u1ea3 n\u0103ng t\u00e1c \u0111\u1ed9ng \u0111\u1ebfn c\u00e1c ch\u1ec9 d\u1ea5u sinh h\u1ecdc. K\u1ebft qu\u1ea3 ph\u00e2n t\u00edch d\u1eef li\u1ec7u t\u1ea1m th\u1eddi sau 9 th\u00e1ng cho th\u1ea5y ph\u1ea3n \u1ee9ng ch\u1ec9 d\u1ea5u sinh h\u1ecdc t\u00edch c\u1ef1c v\u00e0 \u1ed5n \u0111\u1ecbnh c\u1ee7a SKY-0515, v\u00e0 s\u1ef1 c\u1ea3i thi\u1ec7n \u1edf \u0111i\u1ec3m cu\u1ed1i ti\u1ec1m n\u0103ng (cUHDRS) so v\u1edbi t\u00ecnh tr\u1ea1ng b\u1ec7nh x\u1ea5u \u0111i (theo \u0111i\u1ec3m cUHDRS) do di\u1ec5n bi\u1ebfn b\u1ec7nh t\u1ef1 nhi\u00ean \u2013 \u0111\u00e3 kh\u1eb3ng \u0111\u1ecbnh ti\u1ec1m n\u0103ng c\u1ee7a SKY-0515, m\u1ed9t li\u1ec7u ph\u00e1p \u0111i\u1ec1u tr\u1ecb h\u1ee9a h\u1eb9n s\u1ebd l\u00e0m thay \u0111\u1ed5i di\u1ec5n bi\u1ebfn b\u1ec7nh t\u1ed1t nh\u1ea5t d\u00e0nh cho b\u1ec7nh Huntington. Nh\u1eefng d\u1eef li\u1ec7u t\u1ea1m th\u1eddi n\u00e0y \u0111\u00e1nh d\u1ea5u m\u1ed9t c\u1ed9t m\u1ed1c quan tr\u1ecdng \u0111\u1ed1i v\u1edbi SKY-0515 v\u00e0 kh\u1eb3ng \u0111\u1ecbnh n\u0103ng l\u1ef1c c\u1ee7a n\u1ec1n t\u1ea3ng Skyhawk trong vi\u1ec7c cung c\u1ea5p c\u00e1c thu\u1ed1c ph\u00e2n t\u1eed nh\u1ecf ti\u00ean phong cho c\u00e1c b\u1ec7nh hi\u1ec3m ngh\u00e8o m\u00e0 hi\u1ec7n ch\u01b0a c\u00f3 li\u1ec7u ph\u00e1p \u0111i\u1ec1u tr\u1ecb t\u1eadn g\u1ed1c n\u00e0o \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t.”<\/p>\n

B\u1ec7nh Huntington l\u00e0 m\u1ed9t ch\u1ee9ng r\u1ed1i lo\u1ea1n tho\u00e1i h\u00f3a th\u1ea7n kinh hi\u1ebfm g\u1eb7p, c\u00f3 t\u00ednh di truy\u1ec1n v\u00e0 cu\u1ed1i c\u00f9ng d\u1eabn \u0111\u1ebfn t\u1eed vong. T\u1ea1i Hoa K\u1ef3, hi\u1ec7n c\u00f3 h\u01a1n 40.000 b\u1ec7nh nh\u00e2n c\u00f3 tri\u1ec7u ch\u1ee9ng v\u00e0 \u01b0\u1edbc t\u00ednh c\u00f3 h\u00e0ng tr\u0103m ngh\u00ecn ng\u01b0\u1eddi tr\u00ean to\u00e0n th\u1ebf gi\u1edbi \u0111ang ph\u1ea3i chung s\u1ed1ng v\u1edbi c\u0103n b\u1ec7nh n\u00e0y. Hi\u1ec7n t\u1ea1i ch\u01b0a c\u00f3 ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb n\u00e0o \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t c\u00f3 kh\u1ea3 n\u0103ng l\u00e0m ch\u1eadm ho\u1eb7c ng\u0103n ch\u1eb7n s\u1ef1 ti\u1ebfn tri\u1ec3n c\u1ee7a b\u1ec7nh. SKY-0515 l\u00e0 m\u1ed9t lo\u1ea1i thu\u1ed1c ph\u00e2n t\u1eed nh\u1ecf d\u00f9ng \u0111\u01b0\u1eddng u\u1ed1ng, c\u00f3 kh\u1ea3 n\u0103ng \u0111i\u1ec1u ch\u1ec9nh RNA. \u0110\u00e2y l\u00e0 s\u1ea3n ph\u1ea9m \u0111ang trong giai \u0111o\u1ea1n nghi\u00ean c\u1ee9u, \u0111\u01b0\u1ee3c ph\u00e1t tri\u1ec3n th\u00f4ng qua n\u1ec1n t\u1ea3ng \u0111i\u1ec1u bi\u1ebfn RNA m\u1edbi c\u1ee7a c\u00f4ng ty – SKYSTAR\u00ae<\/sup>. SKY-0515 c\u00f3 t\u00e1c d\u1ee5ng \u0111i\u1ec1u tr\u1ecb gi\u00fap l\u00e0m gi\u1ea3m c\u1ea3 protein HTT v\u00e0 protein PMS1. PMS1 c\u0169ng l\u00e0 m\u1ed9t y\u1ebfu t\u1ed1 quan tr\u1ecdng th\u00fac \u0111\u1ea9y s\u1ef1 m\u1edf r\u1ed9ng l\u1eb7p \u0111o\u1ea1n CAG trong t\u1ebf b\u00e0o soma v\u00e0 s\u1ef1 ti\u1ebfn tri\u1ec3n b\u1ec7nh l\u00fd c\u1ee7a HD. Do \u0111\u00f3, vi\u1ec7c t\u00e1c \u0111\u1ed9ng v\u00e0o PMS1 s\u1ebd t\u1ed1i \u01b0u h\u00f3a hi\u1ec7u qu\u1ea3 c\u1ee7a vi\u1ec7c gi\u1ea3m HTT \u0111\u1ed9t bi\u1ebfn.<\/p>\n

H\u00f4m nay, Skyhawk c\u0169ng th\u00f4ng b\u00e1o m\u1edf r\u1ed9ng quy m\u00f4 th\u1eed nghi\u1ec7m FALCON-HD Giai \u0110o\u1ea1n 2\/3 cho SKY-0515 tr\u00ean to\u00e0n th\u1ebf gi\u1edbi. Th\u1eed nghi\u1ec7m n\u00e0y \u0111\u00e3 \u0111\u01b0\u1ee3c tri\u1ec3n khai t\u1ea1i 12 \u0111\u1ecba \u0111i\u1ec3m nghi\u00ean c\u1ee9u \u1edf \u00dac v\u00e0 New Zealand. Hi\u1ec7n t\u1ea1i, \u0111\u00e3 c\u00f3 h\u01a1n 90 b\u1ec7nh nh\u00e2n d\u00f9ng thu\u1ed1c c\u1ee7a Skyhawk.<\/i>\u00a0<\/p>\n

SKY-0515 l\u00e0 lo\u1ea1i thu\u1ed1c \u0111\u1ea7u ti\u00ean c\u1ee7a Skyhawk \u0111\u01b0\u1ee3c \u0111\u01b0a v\u00e0o th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng.<\/p>\n

Skyhawk d\u1ef1 ki\u1ebfn s\u1ebd \u0111\u01b0a th\u00eam nhi\u1ec1u lo\u1ea1i thu\u1ed1c ph\u00e2n t\u1eed nh\u1ecf kh\u00e1c v\u00e0o th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng \u0111\u1ebfn cu\u1ed1i n\u0103m 2027. C\u00e1c lo\u1ea1i thu\u1ed1c n\u00e0y \u0111i\u1ec1u tr\u1ecb c\u00e1c b\u1ec7nh th\u1ea7n kinh hi\u1ebfm g\u1eb7p m\u00e0 hi\u1ec7n v\u1eabn ch\u01b0a c\u00f3 li\u1ec7u ph\u00e1p \u0111i\u1ec1u tr\u1ecb t\u1eadn g\u1ed1c n\u00e0o \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t.<\/p>\n

Gi\u1edbi thi\u1ec7u v\u1ec1 Nghi\u00ean C\u1ee9u L\u00e2m S\u00e0ng Giai \u0110o\u1ea1n 1 c\u1ee7a thu\u1ed1c SKY-0515\u00a0
<\/b>Th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng Giai \u0110o\u1ea1n 1 c\u1ee7a SKY-0515 l\u00e0 th\u1eed nghi\u1ec7m tr\u00ean ng\u01b0\u1eddi \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c thi\u1ebft k\u1ebf \u0111\u1ec3 \u0111\u00e1nh gi\u00e1 \u0111\u1ed9 an to\u00e0n, kh\u1ea3 n\u0103ng dung n\u1ea1p, d\u01b0\u1ee3c \u0111\u1ed9ng h\u1ecdc v\u00e0 d\u01b0\u1ee3c l\u1ef1c h\u1ecdc c\u1ee7a SKY-0515 \u1edf c\u00e1c t\u00ecnh nguy\u1ec7n vi\u00ean kh\u1ecfe m\u1ea1nh v\u00e0 nh\u1eefng ng\u01b0\u1eddi m\u1eafc b\u1ec7nh Huntington (HD) giai \u0111o\u1ea1n \u0111\u1ea7u. Th\u1eed nghi\u1ec7m \u0111\u01b0\u1ee3c chia th\u00e0nh ba ph\u1ea7n. Ph\u1ea7n A v\u00e0 B \u0111\u00e1nh gi\u00e1 SKY-0515 tr\u00ean c\u00e1c t\u00ecnh nguy\u1ec7n vi\u00ean kh\u1ecfe m\u1ea1nh. Ph\u1ea7n C l\u00e0 m\u1ed9t nghi\u00ean c\u1ee9u thi\u1ebft k\u1ebf song song, m\u00f9 \u0111\u00f4i, c\u00f3 \u0111\u1ed1i ch\u1ee9ng gi\u1ea3 d\u01b0\u1ee3c, v\u1edbi hai m\u1ee9c li\u1ec1u SKY-0515 v\u00e0 gi\u1ea3 d\u01b0\u1ee3c, d\u00f9ng cho nh\u1eefng ng\u01b0\u1eddi m\u1eafc HD giai \u0111o\u1ea1n \u0111\u1ea7u (HD-ISS Giai \u0110o\u1ea1n 1, 2 ho\u1eb7c Giai \u0110o\u1ea1n 3 nh\u1eb9) trong 84 ng\u00e0y. Sau \u0111\u00f3 l\u00e0 giai \u0111o\u1ea1n m\u1edf r\u1ed9ng \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb t\u00edch c\u1ef1c trong 12 th\u00e1ng, khi \u0111\u00f3 t\u1ea5t c\u1ea3 ng\u01b0\u1eddi tham gia s\u1ebd d\u00f9ng SKY-0515 li\u1ec1u th\u1ea5p ho\u1eb7c li\u1ec1u cao m\u00e0 kh\u00f4ng bi\u1ebft m\u1ee9c li\u1ec1u m\u00ecnh \u0111ang d\u00f9ng. M\u1ee5c ti\u00eau c\u1ee7a nghi\u00ean c\u1ee9u bao g\u1ed3m \u0111\u00e1nh gi\u00e1 protein HTT \u0111\u1ed9t bi\u1ebfn v\u00e0 mRNA PMS1. Nh\u1eefng b\u1ec7nh nh\u00e2n \u0111\u1ea7u ti\u00ean \u0111\u00e3 d\u00f9ng thu\u1ed1c SKY-0515 trong Ph\u1ea7n C c\u1ee7a nghi\u00ean c\u1ee9u v\u00e0o th\u00e1ng M\u1ed9t n\u0103m 2025. Vi\u1ec7c tuy\u1ec3n ch\u1ecdn b\u1ec7nh nh\u00e2n tham gia Giai \u0110o\u1ea1n 1C c\u1ee7a th\u1eed nghi\u1ec7m SKY-0515 hi\u1ec7n \u0111\u00e3 ho\u00e0n t\u1ea5t.<\/p>\n

Gi\u1edbi thi\u1ec7u v\u1ec1 Nghi\u00ean C\u1ee9u L\u00e2m S\u00e0ng FALCON-HD Giai \u0110o\u1ea1n 2\/3 c\u1ee7a thu\u1ed1c SKY-0515\u00a0
<\/b>FALCON-HD<\/b>\u00a0(NCT06873334<\/a>) l\u00e0 m\u1ed9t nghi\u00ean c\u1ee9u Giai \u0110o\u1ea1n 2\/3 ng\u1eabu nhi\u00ean, m\u00f9 \u0111\u00f4i, c\u00f3 \u0111\u1ed1i ch\u1ee9ng gi\u1ea3 d\u01b0\u1ee3c, s\u1eed d\u1ee5ng nhi\u1ec1u m\u1ee9c li\u1ec1u \u0111\u1ec3 \u0111\u00e1nh gi\u00e1 d\u01b0\u1ee3c \u0111\u1ed9ng h\u1ecdc, \u0111\u1ed9 an to\u00e0n v\u00e0 hi\u1ec7u qu\u1ea3 c\u1ee7a thu\u1ed1c SKY-0515 tr\u00ean 120 ng\u01b0\u1eddi tham gia m\u1eafc b\u1ec7nh HD Giai \u0110o\u1ea1n 2 v\u00e0 \u0111\u1ea7u Giai \u0110o\u1ea1n 3 t\u1ea1i 12 \u0111\u1ecba \u0111i\u1ec3m nghi\u00ean c\u1ee9u \u1edf \u00dac\u00a0v\u00e0 New Zealand, v\u00e0 400 ng\u01b0\u1eddi tham gia m\u1eafc b\u1ec7nh HD Giai \u0110o\u1ea1n 2 v\u00e0 \u0111\u1ea7u Giai \u0110o\u1ea1n 3 t\u1ea1i h\u01a1n 40 \u0111\u1ecba \u0111i\u1ec3m nghi\u00ean c\u1ee9u tr\u00ean to\u00e0n th\u1ebf gi\u1edbi. C\u00e1c b\u1ec7nh nh\u00e2n \u0111\u1ee7 \u0111i\u1ec1u ki\u1ec7n s\u1ebd d\u00f9ng SKY-0515 \u0111\u01b0\u1eddng u\u1ed1ng m\u1ed7i ng\u00e0y m\u1ed9t l\u1ea7n \u1edf m\u1ed9t trong ba m\u1ee9c li\u1ec1u kh\u00e1c nhau, ho\u1eb7c d\u00f9ng gi\u1ea3 d\u01b0\u1ee3c. Th\u1eddi gian \u0111i\u1ec1u tr\u1ecb k\u00e9o d\u00e0i \u00edt nh\u1ea5t 12 th\u00e1ng. Th\u1eed nghi\u1ec7m n\u00e0y nh\u1eb1m m\u1ee5c \u0111\u00edch \u0111\u00e1nh gi\u00e1 ti\u1ec1m n\u0103ng c\u1ee7a SKY-0515 trong vi\u1ec7c \u0111i\u1ec1u ch\u1ec9nh qu\u00e1 tr\u00ecnh gh\u00e9p n\u1ed1i RNA, t\u1eeb \u0111\u00f3 gi\u1ea3m l\u01b0\u1ee3ng protein mHTT v\u00e0 PMS1 \u2013 nh\u1eefng y\u1ebfu t\u1ed1 c\u00f3 li\u00ean quan tr\u1ef1c ti\u1ebfp \u0111\u1ebfn s\u1ef1 ti\u1ebfn tri\u1ec3n b\u1ec7nh l\u00fd c\u1ee7a b\u1ec7nh\u00a0Huntington. \u0110\u1ec3 bi\u1ebft th\u00eam th\u00f4ng tin v\u1ec1 FALCON-HD, bao g\u1ed3m c\u00e1c \u0111\u1ecba \u0111i\u1ec3m tham gia nghi\u00ean c\u1ee9u v\u00e0 ti\u00eau ch\u00ed \u0111\u1ee7 \u0111i\u1ec1u ki\u1ec7n, vui l\u00f2ng truy c\u1eadp ClinicalTrials.gov<\/a>\u00a0v\u00e0\u00a0www.FALCON-HD.com<\/a>.<\/p>\n

Gi\u1edbi thi\u1ec7u v\u1ec1 Skyhawk Therapeutics
<\/b>Skyhawk Therapeutics l\u00e0 m\u1ed9t c\u00f4ng ty c\u00f4ng ngh\u1ec7 sinh h\u1ecdc giai \u0111o\u1ea1n l\u00e2m s\u00e0ng, s\u1eed d\u1ee5ng n\u1ec1n t\u1ea3ng \u0111\u1ed9c quy\u1ec1n SKYSTAR\u00ae<\/sup> \u0111\u1ec3 nghi\u00ean c\u1ee9u v\u00e0 ph\u00e1t tri\u1ec3n c\u00e1c li\u1ec7u ph\u00e1p ph\u00e2n t\u1eed nh\u1ecf c\u00f3 kh\u1ea3 n\u0103ng \u0111i\u1ec1u bi\u1ebfn RNA, gi\u00fap \u0111i\u1ec1u tr\u1ecb nh\u1eefng b\u1ec7nh kh\u00f3 ch\u1eefa nh\u1ea5t tr\u00ean th\u1ebf gi\u1edbi. \u0110\u1ec3 bi\u1ebft th\u00eam th\u00f4ng tin, h\u00e3y truy c\u1eadp www.skyhawktx.com<\/a>.<\/p>\n

Ng\u01b0\u1eddi li\u00ean h\u1ec7 c\u1ee7a Skyhawk
<\/b>Maura McCarthy
Tr\u01b0\u1edfng b\u1ed9 ph\u1eadn Ph\u00e1t Tri\u1ec3n Doanh Nghi\u1ec7p
maura@skyhawktx.com<\/a><\/p>\n

\u1ea2nh – https:\/\/mma.prnasia.com\/media2\/2870056\/Skyhawk_Therapeutics_9_MONTH_DATA_Graph.jpg?p=medium600<\/a>
Logo – https:\/\/mma.prnasia.com\/media2\/710814\/Skyhawk_Therapeutics_Logo.jpg?p=medium600<\/a><\/p>\n<\/div>\n","protected":false},"excerpt":{"rendered":"

Sau 9 th\u00e1ng, d\u1eef li\u1ec7u cho th\u1ea5y b\u1ec7nh nh\u00e2n s\u1eed d\u1ee5ng thu\u1ed1c n\u00e0y c\u00f3 m\u1ee9c c\u1ea3i thi\u1ec7n trung b\u00ecnh \u0111\u1ea1t +0,64 \u0111i\u1ec3m (theo Thang \u0110i\u1ec3m \u0110\u00e1nh Gi\u00e1 B\u1ec7nh Huntington Th\u1ed1ng Nh\u1ea5t T\u1ed5ng H\u1ee3p – cUHDRS) so v\u1edbi m\u1ee9c ban \u0111\u1ea7u, trong khi theo di\u1ec5n bi\u1ebfn t\u1ef1 nhi\u00ean c\u1ee7a b\u1ec7nh, t\u00ecnh tr\u1ea1ng c\u1ee7a b\u1ec7nh nh\u00e2n c\u00f3 […]\n","protected":false},"author":2604,"featured_media":50415,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"om_disable_all_campaigns":false,"_monsterinsights_skip_tracking":false,"footnotes":""},"categories":[2293],"tags":[],"class_list":["post-45657","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-pr-newswire"],"yoast_head":"\nSkyhawk Therapeutics C\u00f4ng B\u1ed1 K\u1ebft Qu\u1ea3 Nghi\u00ean 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