Syncona Acquires AGTC, Bolstering Gene Therapy Efforts for Inherited Retinal Diseases

Syncona Acquires AGTC, Bolstering Gene Therapy Efforts for Inherited Retinal Diseases

For individuals and families navigating the challenges of inherited retinal diseases (IRDs), news of advancements in gene therapy research and development offers a beacon of hope. In a significant move within the biotech landscape, Syncona, a leading healthcare company creator, has acquired Applied Genetic Technologies Corporation (AGTC), a clinical-stage biotechnology company focused on developing gene therapies for rare and debilitating illnesses, with an initial focus on IRDs. This acquisition, announced on October 24, 2022, is particularly relevant to the IRD community as it aims to accelerate the development of potential new treatments.

AGTC's primary focus has been on developing adeno-associated virus (AAV)-based gene therapies for ophthalmic conditions, including X-linked retinitis pigmentosa (XLRP) and achromatopsia. The acquisition by Syncona is intended to advance these retinal gene therapy programs. Syncona has a history of investing in and building gene therapy companies, particularly in the ocular space, having previously been involved with Nightstar Therapeutics and Gyroscope Therapeutics, both of which were later acquired.

Key Details of the Acquisition

The acquisition involved a newly established portfolio company of Syncona acquiring all outstanding shares of AGTC. The initial payment was $0.34 per share in cash, totaling approximately $23.5 million. Additionally, there is a potential for up to an extra $50.0 million in payments, or up to $0.73 per share, through contingent value rights (CVRs), which would be realized if AGTC's lead program meets specific development milestones.

AGTC's lead candidate, AGTC-501 (laruparetigene zosaparvovec), is an AAV gene therapy for X-linked retinitis pigmentosa (XLRP), a rare genetic ocular disease that causes progressive vision loss. This program has received Orphan Drug Designation from the U.S. Food & Drug Administration (FDA) and equivalent classification in the European Union. Syncona stated its intention to help drive this treatment through late-stage clinical development, leveraging its expertise in retinal gene therapy.

What This Means for Patients and Families

This acquisition signifies a continued commitment to addressing inherited retinal diseases through gene therapy. For patients and families affected by conditions like XLRP and achromatopsia, the backing of a company like Syncona could provide stability and resources to push promising therapies forward. The goal is to accelerate the development of AGTC-501, potentially bringing a much-needed treatment closer to those living with XLRP.

Syncona's Chief Investment Officer, Chris Hollowood, stated that the company believes it can apply its model and expertise to drive strong returns and deliver significant impact for patients. The transaction aimed to ensure that AGTC's retinal gene therapy programs could continue to advance, especially given previous funding challenges faced by AGTC.

Looking Ahead

As of December 5, 2022, the acquisition was successfully completed, and AGTC became an indirect, wholly-owned subsidiary of Syncona Limited. This integration is expected to combine AGTC's scientific innovation with Syncona's strategic investment and development capabilities, with the shared goal of bringing new gene therapies to patients with IRDs. The focus remains on the continued clinical development of AGTC's programs, particularly AGTC-501 for XLRP, with the hope of positively impacting the lives of those affected by these challenging conditions.