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7 Re­al-World Ways RWE Is Trans­form­ing Health­care
spotlight
top stories
1. Lundbeck CEO talks drug pricing, protecting biotech and how Europe needs to 'step up'
2. Study suggests non-coding RNA, long considered genomic dark matter, has potential in anti-inflammation
3. China is going after the world's most expensive drugs: Endpoints Signal
4. BioNTech co-founders to exit company and start a new one
5. Vertex’s kidney drug clears a critical Phase 3 test. Does it still have Humira-like potential?
6. FDA widens use of leucovorin based on external controls, no new trial data
more stories
 
Alexis Kramer
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The FDA is once again reviewing Capricor’s cell therapy for Duchenne muscular dystrophy after rejecting the treatment last summer. Capricor has since submitted positive data from a Phase 3 trial, and a review decision is now expected in August.

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Alexis Kramer
Editor, Endpoints News
Charl van Zyl, Lundbeck CEO
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by Zachary Brennan

With scant da­ta, HHS Sec­re­tary Robert F. Kennedy Jr. said last month that Eu­ro­peans were al­ready pay­ing high­er drug prices thanks to more than a dozen se­cret agree­ments the White House forged with the bio­phar­ma in­dus­try.

But the ef­fects of the so-called "most fa­vored na­tion" deals, which cut cer­tain prices un­der Med­ic­aid to lev­els sim­i­lar to some Eu­ro­pean coun­tries, aren't be­ing felt across the At­lantic quite yet, Charl van Zyl, CEO of the Copen­hagen-based phar­ma com­pa­ny Lund­beck, told End­points News in an in­ter­view Tues­day.

While not­ing that Lund­beck won't be forg­ing its own MFN deal with the White House, he said the com­pa­ny could end up par­tic­i­pat­ing in one of the CMS pi­lots — GLOBE and GUARD.

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PhD student Janice Pang (L) and University of Toronto biologist Omar Khan (R) created synthetic lncRNA molecules (Tim Fraser, KITE Studio)
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by Ryan Cross

In the lat­est ad­di­tion to the grow­ing cor­nu­copia of ideas for how to turn RNAs in­to med­i­cines, sci­en­tists are now de­vel­op­ing syn­thet­ic ver­sions of a his­tor­i­cal­ly over­looked mol­e­cule that could un­lock a new class of drugs.

These mol­e­cules, known as long non-cod­ing RNAs, are im­por­tant for con­trol­ling how genes are reg­u­lat­ed. They out­num­ber the more well-stud­ied pro­tein-cod­ing genes near­ly two-to-one, yet there's been com­par­a­tive­ly lit­tle re­search on them.

That dis­crep­an­cy has al­ways both­ered Omar Khan, an RNA bi­ol­o­gist at the Uni­ver­si­ty of Toron­to. Now he has shown that three lncR­NAs pre­vi­ous­ly known to con­trol im­mune sys­tem genes can re­duce in­flam­ma­tion when ad­min­is­tered to hu­man cells and in mice.

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Drug Discovery Day 2026
AI-driven drug discovery is already reshaping pipelines. But algorithms alone won’t get a drug to the clinic. Join us for a free virtual program — then continue the conversation at an in-person only fireside chat and happy hour in Boston. Reserve your spot.
3
by Tom Randall, Jared Whitlock

Gene ther­a­pies were sup­posed to be com­pe­ti­tion-proof. They are sci­en­tif­i­cal­ly com­plex, re­quire be­spoke man­u­fac­tur­ing, of­ten go af­ter small pa­tient pop­u­la­tions and can cost mil­lions per dose.

But that as­sump­tion is be­ing test­ed by a wave of com­peti­tors be­ing de­vel­oped in Chi­na — with prices start­ing at a frac­tion of what's cur­rent­ly avail­able in the US.

An End­points News analy­sis looked at the world's 10 most ex­pen­sive gene ther­a­pies and found 77 com­peti­tors in de­vel­op­ment around the world. Of those, 48 are from Chi­na — more than dou­ble the num­ber un­der de­vel­op­ment in the US, and 7 times what's be­ing worked on in Eu­rope.


The de­vel­op­ment of these gene ther­a­pies is the lat­est piece of ev­i­dence that Chi­na is com­pet­ing high­er and high­er on the spec­trum of in­no­v­a­tive sci­ence.

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4
by Elizabeth Cairns

Uğur Şahin and Özlem Türe­ci are set to leave BioN­Tech by the end of the year and es