Ua hoʻolaha ʻo Sionna Therapeutics i kēia lā i ka hoʻomaka ʻana o ka hui a me ka pani ʻana o kahi kālā $ 111 miliona Series B. Ua alakaʻi ʻia ka pōʻai e OrbiMed me ke komo ʻana mai nā kālā i ʻōlelo ʻia e T. Rowe Price Associates, Inc., Q Healthcare Holdings, LLC., kahi ʻoihana piha piha o QIA, ke kālā waiwai nui o Qatar, a me nā mea hoʻopukapuka mua me RA Capital, TPG's. ʻO ka Rise Fund, Atlas Venture, a me ka Cystic Fibrosis Foundation. Ua hoʻonui ʻo Zionna ma kahi o $150 miliona a hiki i kēia lā.
Ke holomua nei ʻo Sionna i ka pipeline o nā molekole liʻiliʻi mua i hoʻolālā ʻia e hoʻihoʻi piha i ka hana o ka protein cystic fibrosis transmembrane conductance regulator (CFTR) i hemahema i ka CF, ma o ka hoʻopaʻa ʻana i ka domain nucleotide-binding mua o CFTR (NBD1). ʻO ke kumu nui o ka CF ka genetic mutation ΔF508 e pili ana i ka NBD1 paʻa a me ka hana CFTR.
I loko o nā hiʻohiʻona CF in vitro wānana, ua hōʻike ʻo Sionna's NBD1-target molekula liʻiliʻi, i hui pū ʻia me nā modulators hoʻohui ʻē aʻe, i ka hiki ke hoʻomaʻamaʻa maʻamau i ka pelu ʻana, ka makua, a me ke kūpaʻa o ka protein CFTR i hoʻopili ʻia e ka ΔF508 genetic mutation. ʻAe kēia i ke kālepa kūpono o CFTR i ka ʻili o ke kelepona a me ka hoʻoponopono maʻamau o ke kahe o nā ion a me ka wai. Ma ka hoʻihoʻi piha ʻana i ka hana CFTR no nā poʻe maʻi me ka ΔF508 genetic mutation i nā pae i ʻike ʻia i loko o nā cell o ka poʻe me ka CF ʻole, hiki i ka pipeline o Sionna ke hāʻawi i ka maikaʻi maikaʻi loa i ka papa a me ka pono lapaʻau maikaʻi loa no ka poʻe me CF.
"ʻO NBD1 kahi pahuhopu kaulana a noiʻi ʻia, akā ua manaʻo ʻia ʻaʻole hiki ke hoʻopaʻa ʻia a hiki i kēia manawa. Ma muli o kā mākou hoʻoikaika ʻana a hoʻomau i ka holomua ma NBD1, ʻike mākou i ka hiki ke hoʻomaʻamaʻa i ka hana CFTR i ka hapa nui o nā poʻe me CF, "wahi a Mike Cloonan, Pelekikena a Luna Nui o Sionna Therapeutics. "ʻO kā mākou misionari ma Sionna ka hoʻomaikaʻi nui ʻana i ke olakino a me ka maikaʻi o ke ola no ka poʻe e hoʻomau nei i ka ʻeha mai nā hopena lōʻihi o ke ola a me ke kaumaha o ka noho ʻana me CF. Me kēia kālā, kā mākou hui ikaika, a me kā mākou hui ʻike a akamai, hauʻoli mākou e hoʻolauna iā Sionna a ke kālele nei mākou i ka hoʻonui ʻana i nā pūhui mua mai kā mākou pipeline ʻokoʻa i loko o ke keʻena.
ʻO ka CF he maʻi kino koʻikoʻi, hiki ke make ma muli o ka hoʻololi ʻana i ka gene CFTR e alakaʻi i ke kūkulu ʻana o ka mucus i loko o ka māmā a me nā ala ea, ka hana pancreatic ʻino a me nā hana ʻino ʻē aʻe e hiki ke loaʻa i kahi hopena koʻikoʻi i ke olakino a me ke ola. manaʻolana. Aia ma luna o 100,000 poʻe e noho ana me CF a puni ka honua, ii, he 90 pakeneka o lākou i loaʻa ka genetic mutation ΔF508 i loaʻa i loko o ka domain NBD1 o CFTR. Hoʻomaka kēia hoʻololi i ka NBD1 e wehe i ka mahana o ke kino a hōʻino i ka hana CFTR. ʻOiai ka loaʻa ʻana o nā lāʻau lapaʻau i ʻae ʻia i kēia manawa a me ka holomua nui i hana ʻia ma nā pahuhopu ʻē aʻe i loko o CFTR, ʻo ka hapa nui o ka poʻe me ΔF508 ʻaʻole i hoʻokō i ka hana CFTR piha. Pono ʻo NBD1 e hoʻomaʻamaʻa i ka hana o ka CFTR a me ka hana ʻana o ke kino olakino, kahe manuahi i nā ala ea, ʻōnaehana digestive, a me nā mea ʻē aʻe.
He aha e lawe ʻia mai kēia ʻatikala:
- CF is a serious, potentially fatal genetic disease caused by a mutation in the CFTR gene that leads to a buildup of mucus in the lungs and airways, impaired pancreatic function and other organ dysfunction that can have a significant and often severe impact on health and life expectancy.
- In clinically predictive in vitro CF models, Sionna’s NBD1-targeted small molecules, in combination with other complementary modulators, have demonstrated the potential to normalize folding, maturation, and stability of the CFTR protein affected by the ΔF508 genetic mutation.
- By fully restoring CFTR function for patients with the ΔF508 genetic mutation to levels seen in cells of people without CF, Sionna’s pipeline has the potential to deliver best-in-class efficacy and optimal clinical benefit for people with CF.
