Castle Creek Collaborates with Mayo Clinic for Rare Connective Tissue Disorder Gene Therapy

Castle Creek Biosciences entered into a research collaboration with Mayo Clinic to advance discovery and preclinical development of its experimental gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome, which are rare genetic connective tissue disorders that currently have no treatments approved in the United States.

Photo: Matthew Gantz, president and CEO of Castle Creek Biosciences

The research will be led by principal investigator David Deyle, a board-certified medical geneticist with the department of medical genetics at Mayo Clinic and a leader in the field of connective tissue disorders.

“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,” said Matthew Gantz, president and CEO of Castle Creek Biosciences.

Osteogenesis imperfecta (OI), 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 United States and may be diagnosed at any age.

Classical Ehlers-Danlos syndrome (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 United States.

Under the 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 preclinical development phases at Mayo Clinic, Castle Creek anticipates moving into clinical development of selected gene therapy candidates at its in-house, commercial-scale manufacturing facility.

Author: Rare Daily Staff