Why did Novo pull heart failure paperwork for Wegovy?
Hey, humans. Today, we talk about the decades-long pursuit to get MDMA approved as a medicine for PTSD, in advance of Lykos Therapeutics’ Sunday PDUFA date. Also, we see a little girl’s ultra-rare disease improve after she was given a genetic medicine designed just for her, and more.
The need-to-know this morning
- Merck acquired an experimental bispecific antibody from China-based Curon Biopharmaceutical for $700 million upfront, plus an additional $600 million in potential milestone payments. The drug, called CN201, is being studied in blood cancers, but Merck said it sees the potential to target B-cell-associated autoimmune diseases.
- Avidity Biosciences said its RNA-based drug produced 25% of normal dystrophin in patients with Duchenne muscular dystrophy caused by a mutation in exon 44.
How a bespoke gene therapy worked
Susannah Rosen, a little girl with a rare and degenerative neurologic disease, received a bespoke treatment about two years ago. Doctors injected her with stretches of genetic code targeting a gene that caused her symptoms into the 8-year-old’s spine, where it could then make its way to her brain. A couple months later, she was able to stand on her own for the first time in four years.
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