Castle Creek Biosciences Collaborates with Mayo Clinic to Expand Gene Therapy Platform

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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, has formed a new research collaboration with Mayo Clinic.  The partnership was forged under the leadership of Castle Creek President and CEO, Mathew Gantz, and direction from Founder and Chairman Jeff Aronin

Leveraging the clinical development expertise of cellular and genetic technology developed by Castle Creek, Mayo Clinic principal investigator, David R. Deyle, M.D., a board-certified geneticist and nationally recognized medical expert in connective tissue disorders, will focus on two devastating genetic diseases that alter the synthesis of collagen, namely osteogenesis imperfecta and classical Ehlers-Danlos syndrome.  Dr. Deyle will direct the discovery and pre-clinical development phases.

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.

In a press release, Gantz said

After completion of the discovery and pre-clinical phases, clinical development of selected gene therapy agents will be relocated Castle Creeks’ its in-house, commercial-scale manufacturing facility in Exton, Pennsylvania.

Osteogenesis imperfecta, also known as brittle bone disease, is a condition that results from genetic mutations in genes encoding Type I collagen.  The disorder can present with symptoms that include fracture-prone bones, scoliosis (a lateral curvature of the spine), diminished height, fragile teeth, and weakened skin ligaments.

Classical Ehlers-Danlos syndrome, another connective tissue disorder, occurs from genetic mutations that affects synthesis of Type V collagen.  Affected individuals may display abnormal scarring, hyperelastic skin, decreased muscle tone (hypotonia), hypermobile joints, chronic pain, and weak blood vessels.

Castle Creek’s Technology and Clinical Trials

Castle Creek’s approach to personalized biologics is distinctive based on its proprietary, autologous fibroblast technology platform. Through the extraction of fibroblast cells from a patient’s own skin, the company can create localized treatments that are compatible with the unique biology of each patient and have the potential to address the underlying cause of disease.

The company is already conducting clinical trials evaluating targeted gene therapy agents.  One is a Phase 3 trial assessing the efficacy of a gene therapy product for recessive dystrophic epidermolysis bullosa. Another is a Phase 1/2 clinical trial assessing a gene therapy product for moderate to severe localized scleroderma.

Recessive dystrophic epidermolysis bullosa is a rare, genetic skin disorder that results in highly sensitive skin susceptible to severe blisters and abrasions from ordinary exposure to irritants and friction.  Localized scleroderma is an uncommon chronic connective tissue disorder characterized by inflammation and hardening of the skin due to uncontrolled collagen buildup. 

Aronin commended Gantz 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.”  He praised the Castle Creek team for their ability “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.”

Castle Creek is just one portfolio company of many under Paragon Biosciences, LLC, a business founded and chaired by Jeff Aronin, who also serves as its CEO.  Paragon has established a portfolio of seven companies, including CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, and Skyline Biosciences and continues to generate a steady stream of companies though its Paragon Innovation Labs, having invested along with its partners more than $1.3 billion since 2017.

All these biology-based companies are focused on the advancement of biotechnology, optimized protein design, and cell and gene therapy to solve some of the world’s greatest challenges.  Aronin has more than 30 years of experience in life sciences, bringing needed treatments to markets with several FDA approvals under his belt. 

As a philanthropic endeavor, Aronin founded and serves as chairman for MATTER, a nonprofit organization that supports biotechnology start-ups and entrepreneurs, bringing together more than 200 healthcare companies at any one time, securing more than 60 corporate sponsors, and conducting more than $1.7 billion in deals since 2015.  A proponent of education, Paragon’s founder also frequently lectures at the University of Chicago’s Booth School of Business and Northwestern University’s Kellogg School of Business.

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