Krys­tal Biotech dis­closed promis­ing ear­ly find­ings for a ‘mu­ta­tion-ag­nos­tic’ treat­ment for cys­tic fi­bro­sis, and it wants to head di­rect­ly in­to a piv­otal tri­al for the gene ther­a­py.

Ver­tex Phar­ma­ceu­ti­cal­s' cys­tic fi­bro­sis treat­ments have been game-chang­ing for most pa­tients with the dis­ease. But around 30,000 pa­tients are ei­ther in­el­i­gi­ble for those ther­a­pies due to the mu­ta­tion of the dis­ease they have, or they don't see the full ben­e­fits of the treat­ment, ac­cord­ing to Krys­tal’s pre­sen­ta­tion Thurs­day. That trans­lates to a $2 bil­lion-plus mar­ket, the biotech said.

Krys­tal’s cys­tic fi­bro­sis gene ther­a­py is re­dos­able and us­es a mod­i­fied her­pes virus to de­liv­er genes that ex­press the full, na­tive CFTR pro­tein, which is de­fec­tive in pa­tients with the dis­ease. Known as KB407, the treat­ment is in­haled via a neb­u­liz­er.

An­oth­er biotech has latched on­to the Chi­na-to-US mod­el, putting mon­ey be­hind a Chi­na as­set that will be ad­vanced by an Amer­i­can start­up.

Pa­lo Al­to-based AirNex­is Ther­a­peu­tics will get $200 mil­lion from in­vestors to ad­vance a drug for chron­ic ob­struc­tive pul­monary dis­ease from Hais­co Phar­ma­ceu­ti­cal Group.

The drug, AN01/HSK39004, is a dual PDE3/4 in­hibitor in Phase 2 test­ing in Chi­na. Hais­co will keep the rights there and al­so in Hong Kong, Macau and Tai­wan. It could com­pete against sim­i­lar drugs at Mer­ck and GSK in the dis­ease.

The in­vest­ment is be­ing backed by Fra­zier Life Sci­ences, Or­biMed, Life Sci­ences at Gold­man Sachs Al­ter­na­tives, SR One, Lon­gi­tude Cap­i­tal and Ena­vate Sci­ences.

Hais­co will al­so get a 19.9% stake in AirNex­is, and $40 mil­lion in up­front cash. AirNex­is could al­so end up pay­ing as much as $955 mil­lion in fu­ture mile­stone pay­ments.

Two gi­ants and long­time col­lab­o­ra­tors in the mR­NA world are part­ner­ing for an ear­ly-stage ven­ture fund.

BioN­Tech and the Uni­ver­si­ty of Penn­syl­va­nia are join­ing up for a $50 mil­lion ini­tia­tive that will in­vest in star­tups found­ed on sci­ence out of the uni­ver­si­ty.

The fund will be known as the Penn-BioN­Tech In­no­v­a­tive Ther­a­peu­tics Seed Fund, or PxB Fund. Os­age Uni­ver­si­ty Part­ners, which is a ven­ture cap­i­tal firm based in Philadel­phia that in­vests in star­tups from uni­ver­si­ties, will be man­ag­ing the fund.

“Penn and BioN­Tech have had a long­stand­ing part­ner­ship," the uni­ver­si­ty's chief in­no­va­tion of­fi­cer John Swart­ley told End­points News. "One of the very im­por­tant out­comes of that has been the Covid-19 vac­cines and the mR­NA com­po­nents of those, which were al­so No­bel Prize-win­ning tech­nolo­gies that were de­vel­oped here at the Uni­ver­si­ty of Penn­syl­va­nia."

Roche has signed an­oth­er li­cens­ing deal with its Chi­na-based part­ner MediLink, this time fo­cused on an an­ti­body-drug con­ju­gate for sol­id tu­mors that tar­gets B7-H3.

The Swiss drug­mak­er will ob­tain ex­clu­sive rights to MediLink’s drug, called YL201, out­side of Ch