With US rights already taken by Acadia, perhaps a European-based pharma?
Roche is certainly a leader in rare disease, has a collaboration with PTC, the company mentioned in the lead post in this thread and is also a majority holder of the large Japanese pharma, Chugai.
Then there's Novartis and Sanofi, of course, which both have major rare disease divisions as well as presence in Asia.
Astra Zeneca perhaps, which exploded its rare disease portfolio with its US$39bn acquisition of Alexion in 2020 - now known as Alexion Astra Zeneca Rare Disease.
Or perhaps UCB, which is based in Belgium, where trofinetide is manufactured. UCB is keen on expanding in rare disease, especially neurological, and has purchased two rare disease companies in the US$2bn range (Ra Pharmaceuticals and Zogenix) in the last couple of years.
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