In $900M+ deal, rare dis­ease biotechs team up on RNA ther­a­pies for CNS de­vel­op­men­tal dis­or­ders

Two biotechs that have built their rep­u­ta­tions in rare dis­ease re­search signed a new col­lab­o­ra­tion Mon­day morn­ing aim­ing to de­vel­op three new RNA-based ther­a­pies. And the deal could be worth near­ly $1 bil­lion in biobucks if every mile­stone is met.

Aca­dia Phar­ma­ceu­ti­cals and Stoke Ther­a­peu­tics will team up on the treat­ments in rare ge­net­ic neu­rode­vel­op­men­tal dis­eases of the CNS, the com­pa­nies an­nounced. Up first will be the re­cent­ly dis­cov­ered SYN­GAP1 syn­drome, fol­lowed by Rett syn­drome (MECP2) and an undis­closed tar­get the biotechs say is of mu­tu­al in­ter­est.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER