Last April, Chi­nese reg­u­la­tors ap­proved Be­lief Bio­Med’s gene ther­a­py for the bleed­ing dis­or­der he­mo­phil­ia B, set­ting up a chal­lenge to one of the prici­est med­i­cines.

The treat­ment marked the first he­mo­phil­ia gene ther­a­py in Chi­na to be de­vel­oped and man­u­fac­tured en­tire­ly by a do­mes­tic com­pa­ny. Be­lief Bio­Med priced it at $350,000, one-tenth the cost of a ri­val ther­a­py that sells for $3.5 mil­lion in the US.

In the year since, it's be­come clear that Be­lief Bio­Med’s ap­proval is a bell­wether: In a glob­al race to de­vel­op cheap­er and po­ten­tial­ly bet­ter al­ter­na­tives to the world’s 10 most ex­pen­sive med­i­cines, Chi­na has pulled ahead.

An End­points News analy­sis of clin­i­cal tri­al data­bas­es shows that Chi­na ac­counts for 48 of the 77 pro­grams tar­get­ing this group of ul­tra-ex­pen­sive gene ther­a­pies. Chi­na's to­tal is more than dou­ble the num­ber in the US and sev­en times as many as Eu­rope.

Tenaya Ther­a­peu­tics, a heart health biotech with three clin­i­cal-stage med­i­cines, is gain­ing a well-known part­ner to val­i­date its ge­net­ic tar­get plat­form.

RNAi whiz Al­ny­lam Phar­ma­ceu­ti­cals is send­ing a small $10 mil­lion up­front check to Tenaya for ac­cess to new hu­man ge­net­ic tar­gets that could serve as the ba­sis for fu­ture car­dio­vas­cu­lar treat­ments. If the col­lab­o­ra­tion goes smooth­ly, and all 15 gene tar­gets are act­ed up­on, then Tenaya could gain up to $1.13 bil­lion across de­vel­op­ment, reg­u­la­to­ry and sales mile­stones, ac­cord­ing to the Thurs­day pact.

The move marks a key part­ner­ship for Tenaya, which has cut back on its work­force over the past few years, with lay­offs in 2025 and 2024 to con­serve cash. The Bay Area biotech then se­cured more than $100 mil­lion from two pub­lic of­fer­ings last year as it pre­pares for po­ten­tial late-stage de­vel­op­ment in the cost­ly gene ther­a­py are­na.

Mer­ck KGaA has trimmed its R&D pipeline af­ter a strate­gic re­view.

The Ger­man phar­ma has re­moved stud­ies of two drugs it picked up from the Spring­Works Ther­a­peu­tics buy­out. As it stands, there are no oth­er on­go­ing clin­i­cal tri­als from that pur­chase, al­though it could still have pre­clin­i­cal as­sets from Spring­Works that are yet to en­ter tri­als.

Mer­ck KGaA has al­so cut M9466 (al­so known as HRS-1167), a PARP1 in­hibitor that it li­censed from Hen­grui in Oc­to­ber 2023.

Mer­ck KGaA's full-year earn­ings pre­sen­ta­tion no longer lists Og­siveo (nirogace­s­tat) as un­der in­ves­ti­ga­tion for ovar­i­an gran­u­losa cell tu­mors. The Phase 2 tri­al is list­ed as com­plet­ed on Clin­i­cal­Tri­als.gov, with a 53-pa­tient en­roll­ment tar­get. In late 2024, Spring­Works said that ini­tial da­ta would be re­port­ed in the first half of last year.

The mid-stage tri­al of Pep­Gen’s on­ly pipeline prod­uct, a po­ten­tial ther­a­py for a rare ge­net­ic mus­cle dis­or­der, has been placed on par­tial clin­i­cal hold by the FDA.

The agency raised ques­tions about the pro­duc­t's pre­vi­ous­ly sub­mit­ted pre­clin­i­cal phar­ma­col­o­gy and tox­i­col­o­gy stud­ies, the com­pa­ny said af­ter mar­ket close on Wednes­day. An­a­lysts at Stifel of­fered more con­text in a March 4 note, writ­ing that the FDA’s con­cerns are “specif­i­cal­ly re­lat­ed to blood pres­sure de­creas­es in mice that were ob­served in a sub-chron­ic tox­i­col­o­gy study.”

This has not been seen in pri­mates or hu­mans, the an­a­lysts wrote. The FDA had the mouse da­ta since mid-2024, they added, and it is like­ly that Pep­Gen’s re­quest to amend its Phase 2 tri­al pro­to­col to in­clude US sites “might have spurred the agency to re­vis­it the da­ta,” the Stifel an­a­lysts wrote.