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Thursday 13 Aug 2020 , 5:38 am

AGTC Formalizes Patient Advisory Council to Provide Insights and Guidance on Patient Perspectives for Pipeline Therapies

Patient Advisory Council to include advocates from the inherited retinal diseases global community. New Patient Advisory Council to focus initially on Company’s clinical stage programs for X-linked retinitis pigmentosa (XLRP).
By SIN Bureau
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Applied Genetic Technologies Corporation, 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.”




Neha Mule

Neha writes articles on sectors including medicine, food, materials, and science & technology. A qualified statistician, she has the ability to observe and analyze the trends in global markets and write compelling articles that help CXOs in decision making. She is a bookworm and loves to read fiction, lifestyle, science and technology. Neha comes with 6 years of experience in content writing and editing that involves blog writing, preparation of study materials and OERs.

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