Per­lara col­lab­o­rates with Har­vard/Un­di­ag­nosed Dis­eases Net­work; Verona shares soar on pos­i­tive CF da­ta

→ Per­lara, the rare dis­ease com­pa­ny found­ed by Twit­ter’s fa­vorite biotech rebel Ethan Perl­stein, an­nounced two sci­en­tif­ic dis­cov­ery col­lab­o­ra­tions Fri­day with Har­vard Med­ical School and the Un­di­ag­nosed Dis­eases Net­work. The re­search part­ner­ships are for two rare mono­genic neu­rode­vel­op­men­tal dis­or­ders. One is caused by a mu­ta­tion in GNA01 and the oth­er caused by a mu­ta­tion in RPS6KA3 (Cof­fin-Lowry Syn­drome). Per­lara plans to de­vel­op and val­i­date ne­ma­tode and fly “pa­tient avatars” of path­o­gen­ic GNA01 and RPS6KA3 mu­ta­tions for use in high-through­put phe­no­typ­ic drug screens. Perl­stein, CEO of the Per­lara, is best known in the bio­phar­ma world through his out­spo­ken Twit­ter pres­ence, where he’s laid bare the dif­fi­cul­ty of rais­ing cap­i­tal as a young com­pa­ny work­ing on “dis­eases thought too rare to at­tract the in­ter­est of phar­ma­ceu­ti­cal com­pa­nies.” He’s been called rad­i­cal for his trans­paren­cy, and a rebel for chal­leng­ing fundrais­ing norms on a pub­lic stage. In lieu of sig­nif­i­cant ven­ture cap­i­tal funds, Perl­stein has in­stead re­lied heav­i­ly on part­ner­ships with pa­tient groups since the com­pa­ny’s 2014 in­cep­tion. Last year, how­ev­er, the com­pa­ny did raise a $7.4M Se­ries A.

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