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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