Hoth Therapeutics, Inc. i aso nei na fa'asalalauina se fa'afouga tau atina'e mo lana tala fou o le kanesa, HT-KIT. O le auala fou a Hoth, lea e faʻaaogaina ai se oligonucleotide antisense chemically-stable e faʻatatau i le proto-oncogene KIT e ala i le faʻaogaina o tusitusiga KIT mRNA, e mafai ona avea o se KIT-targeted therapeutic naʻo ia, poʻo le tuʻufaʻatasia ma sui e faʻatatau i le KIT faʻailoga, i le togafitiga o KIT-faiga leaga.

E ala i se maliliega su'esu'e fa'asaienisi lagolago ma North Carolina State University, na fa'aogaina ai e le 'au le HT-KIT mRNA fa'asolo fa'avaa-shifting i luga o le mast cell leukemia sela i vitro ma maua ai le fa'aaliga o le porotini KIT, fa'ailo ma galuega na fa'aitiitia. Togafitiga fa'atasi ma le HT-KIT e puipuia ai le tupu a'e o le kanesa ma fa'atupu ai le oti i luga ole 72 itula. I se fa'ata'ita'iga o isumu o le mast cell leukemia, na fa'aitiitia le tuputupu a'e o le tuma ma le fa'aulufaleina o isi totoga ma fa'atupula'ia le oti o le tumo ina ua fa'aoso e le HT-KIT le c-KIT mRNA.

Ua faila e Hoth le tele o talosaga pateni e puipuia ai lenei IP i le lalolagi atoa.

“Fa'atasi ai ma le matou vaila'au HT-KIT, o lo'o matou fa'ate'aina se fa'ailoga autu o le kanesa o lo'o a'afia i le tele o kanesa fa'alavelave, e pei o le mastocytosis, mast cell leukemia, gastrointestinal stromal tumors ma acute myeloid leukemia. O la matou auala e aloese ai mai mailei e fesoʻotaʻi ma suiga ole KIT e ala ile tulituliloaina o le mRNA. O loʻo faʻagasolo le matou isi taamilosaga o suʻesuʻega faʻapitoa ma matou te fiafia e faʻaogaina faʻaiʻuga mo la matou fonotaga a le Pre-IND ma le FDA i se taimi mulimuli ane o lenei tausaga, "o le tala lea a Robb Knie, Ofisa Sili o Pulega o Hoth.

O le HT-KIT, o se vaega mole mole fou, na filifilia e avea o se Orphan Drug mo le togafitiga o le mastocytosis i le amataga o le 2022. HT-KIT Hoth ua faʻamaeʻaina lelei le gaosiga o le vailaʻau HT-KIT i le galulue faʻatasi ma WuXi STA.

O le FDA Orphan Drug Designation ua tu'uina atu i togafitiga su'esu'e e fa'atatau i fa'ama'i fa'afoma'i seasea po'o tulaga e a'afia ai le itiiti ifo i le 200,000 tagata i le Iunaite Setete. O le tulaga o fualaau oona matuaoti e maua ai faamanuiaga i le au atia'e fualaau faasaina, e aofia ai le fesoasoani i le faagasologa o le atinaeina o fualaau faasaina, aitalafu o lafoga mo tau falemai, tuusaunoaga mai nisi o totogifuapauina a le FDA ma le fitu tausaga o le mae'a ona faamaonia le maketiina.

OA MEA E AVEA MAI LENEI TUSI:

Hoth’s innovative approach, which employs a chemically-stable antisense oligonucleotide to target the proto-oncogene KIT through by frameshifting KIT mRNA transcripts, has potential as a KIT-targeted therapeutic alone, or in combination with agents that target KIT signaling, in the treatment of KIT-associated malignancies.
Through a sponsored scientific research agreement with North Carolina State University, the team used the HT-KIT mRNA frame-shifting approach on mast cell leukemia cells in vitro and found that KIT protein expression, signaling and function were reduced.
HT-KIT, a new molecular entity, was designated as an Orphan Drug for the treatment of mastocytosis earlier in 2022.