"We are excited to expand our gene therapy efforts to include targets in rare, inherited retinal diseases, for which there is tremendous unmet need," said
PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us on www.ptcbio.com and follow us on
Odylia Therapeutics is a 501(c)(3) nonprofit corporation working to bring treatment for rare eye diseases from the lab into phase I/II/III clinical trials using AAV technology and other gene delivery techniques. Odylia was established in 2017 with support from Massachusetts Eye and Ear and the Usher 2020 Foundation, a charitable organization focused on finding cures for rare eye diseases. There are more than 250 genetic forms of Inherited Retinal Disease (IRD) that lead to blindness. Most are extremely rare, affecting only one in a million people worldwide. Today, there is pre-clinical proof-of-concept for treatments for more than 30 of these diseases, with more being discovered every day. Odylia Therapeutics was co-founded by Scott Dorfman of Usher 2020 Foundation, and Luk Vandenberghe, Ph.D., Eric Pierce, M.D., Ph.D., Joan Miller, M.D., and John Fernandez, M.S. of Massachusetts Eye and Ear. For more information, please visit Odylia.org
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Massachusetts Eye and Ear
+1 (617) 573-4385
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