CiRC Biosciences, a portfolio company of Paragon Biosciences, announced a milestone in March 2021 for a potential therapy to treat retinal disorders, including retinitis pigmentosa (RP) and geographic atrophy age-related macular degeneration (Dry AMD).
CiRC’s technology was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of RP. (Orphan diseases refer to any conditions that affect less than 200,000 people in the U.S.)
CEO Jeff Aronin of Paragon Biosciences views the achievement as an important addition to Paragon’s existing cell and gene therapy platforms and how CiRC may be able to help people that suffer from these life-impacting retinal conditions.
CiRC Biosciences is currently advancing pre-clinical development of chemically induced photoreceptor-like cells (CiPCs) for vision restoration in adults with RP. CiPCs have shown to partially restore pupil reflex and visual function in a mouse model of RP.
RP affects about 1 in every 4,000 people. The disorder is a breakdown of the retina that typically affects night and peripheral vision in its early stages. Without effective therapies to treat RP, it can eventually lead to blindness.
The company is also looking at how the therapy can be applied to other retinal disorders, including Dry AMD. Dry AMD can lead to a loss of acute vision before progressing to legal blindness or even total blindness. Its symptoms are often mistaken for general signs of aging, which can make the condition more difficult to treat if it’s not caught early.
The FDA has stringent requirements for granting any designations to potential new therapies. Sai Chavala M.D., the co-founder and acting chief scientific officer of CiRC Biosciences, was encouraged by the designation for a potential treatment of RP. He remarked, “The FDA’s orphan drug designation provides CiRC Biosciences with the opportunity to efficiently advance this technology through research and development stages to help make meaningful strides for this devastating disease.”
The Work Ahead
Jeff Aronin has more than three decades of experience in life sciences. This announcement for orphan drug designation aligns with Aronin’s focus of addressing conditions with high unmet needs.
Both Aronin and Dr. Chavala are passionate about the exploration of these advanced cell therapies, with the ultimate goal of treating retinal diseases. The new discoveries with this therapy are making it possible to rethink how scientists study cell therapy.
Considering there are currently no FDA-approved treatments to restore vision loss for patients with RP, CiRC may become the breakthrough solution. “Utilizing CiRC Biosciences’ cell therapy to potentially restore vision in those living with vision loss caused by retinopathies and macular degeneration advances our mission to find solutions to some of the world’s greatest challenges,” said Aronin.
Sai Chavala Honored with the Ludwig von Sallmann Clinician-Scientist Award
In addition to the FDA designation, the Dr. Chavala recently celebrated a personal milestone for his career. In June, he was awarded the 2021 Ludwig von Sallmann Clinician-Scientist Award from the Association for Research in Vision and Ophthalmology (ARVO) Foundation for Eye Research. This accomplishment was due to his work in the field of ophthalmology as a young clinician-scientist.
Dr. Chavala said, “I am honored to receive this recognition by the ARVO Foundation for Eye Research and excited CiRC Biosciences will continue to develop the technology for retinitis pigmentosa and geographic atrophy age-related macular degeneration.”
When Jeff Aronin examines life sciences, he looks at people living with conditions with no effective treatments and how biology can help uncover new treatments and potential cures for these conditions. Supporting the innovative cell therapy work of Dr. Chavala is one example of this.
CiRC is one of several portfolio companies under Paragon Biosciences, which focuses on three key areas of innovation: cell and gene therapy, adaptive biology and advanced biotechnology.