Adnkronos (Adnkronos) – I panuitia e Italfarmaco Spa me JCR Pharmaceuticals i tēnei rā tētahi whakaaetanga raihana motuhake mō te whakawhanaketanga me te hokohoko o JR-141 i roto i te Hononga o Amerika, i Ūropi, me Amerika Latina (ngā "Whenua Raihana"), i raro i te whakaaetanga a te ture. E ai ki tētahi kōrero, ko te JR-141 he rongoā whakakapinga whākōkī e whakawhiti ana i te arai toto-roro (BBB) kei roto i te aromatawai Wāhanga 3 (NCT04573023) hei rongoā mō te mate Hunter (e mōhiotia ana ko te mucopolysaccharidosis momo II, MPS II rānei).
E whai ana a JCR me Italfarmaco ki te whiwhi whakaaetanga ture mō te JR-141 mai i te U.S. Food and Drug Administration (FDA), te European Medicines Agency (EMA), te UK's Medicines and Healthcare Products Regulatory Agency (MHARA), me te Brazilian Health Regulatory Agency (ANVISA). Kua whakaaetia kētia te ngota ngota, ā, kua hokohokohia ki Hapani mai i te tau 2021 hei pabinafusp alfa.
Whai muri i ngā whakaaetanga ture mai i te FDA, EMA, MHRA, me ANVISA, ka hokohoko me te tohatoha a Italfarmaco i te rongoā ki ngā rohe kua whakamanahia, ko JCR ia ka pupuri i te kawenga mō te hanga o JR-141 ina whakaaetia. Ka whiwhi a JCR i ngā utunga o mua, ngā utu tohu, me ngā utu rangatira, hei tāpiri atu ki ngā moni whiwhi e pā ana ki te tuku o te rongoā.
Ko te JR-141 – ngā taipitopito o te tuhipoka – he pūmua hanumi recombinant o te whakatipuranga e whai ake nei, he paturopi e whawhai ana ki te kaiwhakawhiwhi transferrin tangata me te iduronate-2-sulfatase, he whākōkī kāore i te kitea, kāore rānei e mahi tika ana i roto i ngā tāngata e pāngia ana e te mate Hunter.
I whakawhanakehia a JR-141 mā te whakamahi i te J-Brain Cargo, te hangarau whakauru BBB motuhake a JCR. Kua hangaia a J-Brain Cargo hei tuku i te whākōkī rongoā mā roto i te BBB kia tae tika atu ki te roro, hei aro atu hoki ki ngā tohu somatic me neuronopathic o te mate, ka taea ai te heke haere o te hinengaro. Ka whakawhānui ake tēnei whakaaetanga i te whanaungatanga i waenga i a JCR me Italfarmaco, i uru atu ki tētahi whakaaetanga raihana motuhake i te Hakihea 2025 mō te hokohoko o Givinostat i Hapani, he maimoatanga mō te Duchenne muscular dystrophy, me tētahi whakaaetanga hononga rautaki mō ngā maimoatanga mate onge.
"E harikoa ana mātou ki te whakauru atu ki tēnei whakaaetanga rautaki me Italfarmaco," te kī a Hiroyuki Sonoda, Ph.D., te Perehitini me te Āpiha Pūtaiao Matua o JCR, "me te mahi tahi me tētahi hoa hokohoko o te ao i a mātou e mahi ana ki tā mātou whāinga kia wātea te JR-141 ki te hunga whai mate Hunter puta noa i te ao. He tohungatanga tō Italfarmaco ki te whakawhanake me te hokohoko i ngā maimoatanga puta noa i te ao, ā, e tumanako ana mātou ki te mahi tahi me rātou kia wātea te JR-141 ki ngā tūroro i waho o Hapani i te wā e taea ai. Mā tēnei whakaaetanga ka whakapakari ake i tā mātou whakapau kaha ki tā mātou hononga ki a Italfarmaco, me te whakawhanake i ngā maimoatanga mō ngā mate onge me ngā mate ira mō ngā tūroro puta noa i te ao."
"Ko tēnei mahi tahi," te kī a Francesco Di Marco, te Tumu Whakahaere Matua o Italfarmaco Group, "he tohu nui tēnei i roto i te whanaungatanga i waenganui i a Italfarmaco me JCR Pharmaceuticals, ā, e whakaata ana i te whakapau kaha ki te whakawhanake i ngā maimoatanga auaha mō te hunga e pāngia ana e ngā mate onge me ngā mate ira. Mā te whakakotahi i te tohungatanga matua o JCR i roto i ngā hangarau ārai toto-roro me te tohungatanga o Italfarmaco o te ao ki te whakawhanake me te hokohoko i ngā maimoatanga mō ngā mate onge, kei te hanga mātou i tētahi turanga pakari hei whakatere i te auahatanga hei painga mō ngā hapori tūroro e tino hiahia ana." "I runga i te angitu o te mahi tahi i waenganui i a Italfarmaco me JCR i roto i te mate uaua o Duchenne, ka whakapakari ake tēnei whakaaetanga i tā mātou hononga mahi tahi me te whakakaha i tā mātou wawata kia noho hei kamupene ārahi i roto i te mara o ngā mate onge me ngā mate ira," te kī a Antonio Nardi, te Tumuaki Tuarua me te Upoko Pakihi me te Whakawhanaketanga Putea o Italfarmaco. "I te taha o tō mātou hoa mahi, ka haere tonu tā mātou whakapau kaha ki te whakarato i ngā maimoatanga auaha ki ngā tūroro me ngā whānau e tino hiahia ana ki a rātou."
Ko te utunga ā-mua i raro i tēnei whakaaetanga, e ai ki te kōrero, kua whakaurua ki roto i te aratohu whiwhinga moni whakakotahi a JCR mō te tau pūtea e mutu ana i te 31 o Poutū-te-rangi, 2027. Kua whakawhanakehia e JCR Pharmaceuticals tētahi hangarau motuhake e kaha ana ki te whiti i te arai toto-roro (BBB), J-Brain Cargo, hei tuku i ngā rongoā koiora ki roto i te pūnaha io matua (CNS). Ko te rongoā tuatahi i whakawhanakehia i runga i tēnei hangarau ko te pabinafusp alfa, i whakaaetia i Hapani mō te rongoā i te mate Hunter, he mate rokiroki lysosomal. Me J-Brain Cargo, e whakapau kaha ana a JCR ki te aro atu ki ngā wero haumanu kāore anō kia whakatauhia o ēnei mate mā te tuku i te whākōkī ki roto i te tinana me te roro.
Ko te mate Hunter (mucopolysaccharidosis momo II, MPS II rānei) he mate rokiroki lysosomal recessive X-linked i puta mai i te korenga o te iduronate-2-sulfatase, he whākōkī e wawahi ana i ngā warowaihā matatini e kiia nei ko te glycosaminoglycans (GAGs, e mōhiotia ana ko ngā mucopolysaccharides) i roto i te tinana. Ko te mate Hunter, e pāngia ana e te 2.000–3.000 tāngata puta noa i te ao (e ai ki te rangahau a JCR), ka puta he whānuitanga o ngā tohu tinana me ngā tohu pūnaha io. Ko te paerewa tiaki o nāianei mō te mate Hunter ko te rongoā whakakapinga whākōkī, kāore e aro ki ngā tohu e pā ana ki te ngoikore o te pūnaha io matua i roto i tēnei mate lysosomal.
-
pukapuka
webinfo@adnkronos.com (Mohio Tukutuku)