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Orphan Star Therapeutics Announces Initial Financing and Corporate Update

NEW YORK, Sept. 25, 2018 /PRNewswire/ -- Orphan Star Therapeutics, LLC, a biopharmaceutical company focused on developing innovative therapies for rare diseases, today announced it has successfully secured a commitment of $20 million from a commercial pharmaceutical partner.

With this funding, Orphan Star plans to advance its early-stage pipeline of innovative candidates for several rare diseases, including Canavan Disease, a devastating rare genetic neurodegenerative disease that is usually fatal within the first decade of life.  Additionally, Orphan Star intends to complement its early-stage portfolio with promising clinical-stage drug candidates through building strategic partnerships within industry and academia.

"We are extremely pleased with this development," said Dr. Nick France, Founder & CEO of Orphan Star.  "Securing this funding allows us to focus our efforts on building a cutting-edge pipeline of therapeutic options for patients with enormous unmet medical needs. It will also allow us to continue to build a highly experienced, energetic, and capable team to take these drugs through clinical development and into the healthcare ecosystem globally."

Orphan Star additionally announced it has strengthened its team through the appointment of key hires, including Adam Simpson, PhD, as Head of Business Development, and Katie St. Ledger, as Senior Director of Clinical Research and Development.  Orphan Star also announced the appointment of Scientific Advisory Board Members Professor Paola Leone, PhD, a leading expert in Canavan disease and gene therapy for rare neurodegenerative disorders, and Prof. Dr. Alex Veldman, a leading academic, innovative drug developer and pediatrician.

Dr. Simpson has joined Orphan Star from Dermavant Sciences, where he was integral in the early growth of the company while leading business development and medical affairs functions.  Previously, Dr. Simpson was a member of the Roivant Sciences business development team, where he led the sourcing and acquisition of drug candidates across the therapeutic landscape.  Dr. Simpson holds a B.A. in Chemistry with highest honors and highest distinction from the University of North Carolina at Chapel Hill, and a Ph.D. in Molecular Biophysics and Biochemistry from Yale University where he was an HHMI scholar.

Katie St. Ledger MS, an expert in strategic clinical drug development and approvals, joins the company from Axovant Sciences where she led several clinical development programs.  Prior to Axovant she was a Clinical Development Scientific Director at CSL Behring and a key leader in the clinical development group where she was instrumental in obtaining orphan drug designations and regulatory approvals of several rare disease drugs in multiple territories including the US and Europe.

About Orphan Star Therapeutics
Orphan Star Therapeutics, a biopharmaceutical company focused on the development of innovative therapies for rare genetic diseases, is currently building a portfolio of early-stage drug candidates for rare neurometabolic diseases.  Our mission is to bring hope to life for patients suffering with serious rare disorders by expediting the development of novel, targeted treatments that address diseases at their source.

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Last Updated: 27-Sep-2018