Applied Genetic Technologies Corp. (NASDAQ: AGTC), a gene therapy company based in Gainesville, Florida, has announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its gene therapy product candidate to treat X-linked retinitis pigmentosa (XLRP). The company plans to conduct a Phase I/II clinical trial to evaluate the safety and efficacy of its proprietary gene therapy for XLRP caused by mutations in the RPGR gene. According to the company, pre-clinical results of the experimental treatment demonstrated the capability to slow the loss of visual function in dogs with mutations in the RPGR gene.
X-linked retinitis pigmentosa is a genetic disorder that causes a progressive deterioration in vision, beginning with night blindness, in mostly young males, followed by progressive constriction of the field of vision. Patients are expected to progress to legal blindness by the fourth decade of life. Of the broad range of photoreceptor dystrophies, the X-linked forms of RP are understood to be one of the most common causes of severe vision loss. In over 70% of XLRP cases, mutations in the retinitis pigmentosa GTPase regulator gene (RPGR) are responsible. Within the RPGR gene itself, a mutational hot spot in one of the exons accounts for an estimated 22–60% of cases recorded. The RPGR gene codes for a photoreceptor ciliary protein, necessary for correct cilia function in the photoreceptors.
AGTC, based in Gainseville, Florida was founded in 2001 by leading AAV scientists at the University of Florida. The company secured rights to a broad portfolio of IP assets focused on the use of AAV vectors for gene therapy applications. The company claims to have licenses to over 40 US and foreign patents and, prior to its IPO in April 2014, had raised over $45M in venture financing. At IPO, the company raised just over $50 million on the US NASDAQ, selling 4.2 million shares at $12 per share valuing the company at $160.7 million. Today the company is valued at approximately $84M. Commenting on the regulatory milestone Sue Washer, President and CEO of AGTC stated, “”[W]e are deeply committed to advancing therapies to address the unmet needs of patients with rare inherited retinal disorders, and filing our fifth IND in this field represents a significant milestone that may ultimately improve the lives of individuals affected by XLRP. Gene therapy clinical trials continue to rapidly progress, making this an exciting time for AGTC, the clinical community and patients affected by ocular conditions with no currently available treatments.”