On September 29, 2021 Castle Creek Biosciences, Inc., a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options, reported a research collaboration with Mayo Clinic to advance discovery and pre-clinical development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta (OI) and classical Ehlers-Danlos syndrome (EDS), which are rare genetic connective tissue disorders that currently have no treatments approved by the U.S. Food & Drug Administration (FDA) (Press release, Mayo Clinic, SEP 29, 2021, View Source [SID1234590494]). The research will be led by principal investigator David R. Deyle, M.D., a board-certified medical geneticist with the department of medical genetics at Mayo Clinic and a leader in the field of connective tissue disorders.
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"We are honored to be working with Dr. Deyle and his highly regarded research team at Mayo Clinic to identify and evaluate gene therapy candidates that hold promise for treating debilitating, rare connective tissue disorders with high unmet medical needs," said Matthew Gantz, president and chief executive officer of Castle Creek Biosciences. "We expect this initiative will be the first of multiple Castle Creek strategic collaborations with leading medical research institutions that have the potential to expand our innovative gene therapy discoveries for rare diseases and offer hope to underserved patient communities impacted by these devastating conditions."
Osteogenesis imperfecta, also known as brittle bone disease, is caused by genetic mutations that affect the synthesis of Type I collagen and can lead to fragile bones, scoliosis, short stature, dental disorders, and laxity of skin ligaments. OI is estimated to affect one in 6,600 people in the U.S. and may be diagnosed at any age. Classical EDS results from genetic mutations affecting synthesis of Type V collagen and is associated with skin hyperextensibility and fragility, hypotonia, joint instability, chronic pain, and fragile blood vessels. Vascular and pulmonary complications have also been reported. EDS is estimated to affect one in 20,000 people in the U.S.
"Castle Creek is leveraging its proven expertise and experience in rare diseases and late-stage clinical development of cell and gene therapies to establish strategic collaborations with world-class research organizations for studying early-stage novel treatments to address critical, unmet medical challenges of patients suffering from rare genetic conditions," said Jeff Aronin, founder and chairman of Castle Creek Biosciences, and founder, chairman and chief executive officer of Paragon Biosciences, LLC. "We commend Matthew and his leadership team for their strategic insight and guidance that have long-term potential to fuel discovery and advancement of innovative gene therapy candidates and enhance the depth of Castle Creek’s pipeline."
For this research collaboration, Castle Creek will contribute its proficiency in rare diseases and gene therapy development and has licensed intellectual property related to OI and classical EDS from Mayo Clinic. Following completion of the discovery through pre-clinical development phases at Mayo Clinic, Castle Creek anticipates moving into clinical development of selected gene therapy candidates at its in-house, commercial-scale current good manufacturing practices (cGMP) manufacturing facility located in Exton, Pa.