GAINESVILLE, Fla., and CAMBRIDGE, Mass., (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, today announced the formation of a Patient Advisory Council to build on its focus of incorporating the patient and caregiver voice into the Company’s culture and clinical and pre-clinical programs.
Engagement with patients and caregivers has provided AGTC with substantive information that has guided its clinical trial design, enhanced its understanding of retinal disorders, and inspired its patient centric culture. The Council, spearheaded by Jill Dolgin, PharmD, Head of Patient Advocacy at AGTC, is comprised of individuals with inherited retinal diseases (IRDs) and members from the global community of organizations that represent them.
“Patient engagement will continue to be a critical success factor for our programs moving forward, and we are very pleased to formalize the establishment of this distinguished group of patient experts, especially as we move into the next phase of development for our X-linked retinitis pigmentosa gene therapy candidate,” said Sue Washer, President and CEO of AGTC.
In July, AGTC announced next steps in the clinical development of the Company’s potential treatment of XLRP caused by mutations in the RPGR gene following receipt of written feedback from the U.S. Food and Drug Administration. AGTC is expanding the ongoing Phase 1/2 trial to dose additional patients in two masked dosing arms to collect additional functional data. In parallel, a planned Phase 2/3 trial, which is expected to begin in Q1 2021, will be designed to evaluate sustained efficacy across multiple measures of potential benefit in patients with XLRP.
“We are delighted to be a part of this advisory council established by AGTC to lend our voice and collective experience that spans more than five decades in the search for treatments to address blindness and vision loss,” said Brian Mansfield, PhD, Executive Vice President of Research and Interim Chief Scientific Officer at the Foundation Fighting Blindness. “Having the patient’s perspective at the center of clinical drug development is a crucial component for addressing the unmet needs of patients within the inherited retinal diseases community.”
About AGTC
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Company’s most advanced clinical programs leverage its best-in-class investigational technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.
About X-linked Retinitis Pigmentosa (XLRP)
XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. AGTC was granted U.S. Food and Drug Administration (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.
