July 14, 2026 —
Lyora Therapeutics launched with seed funding and a leadership team focused on advancing genetic medicines for inherited retinal diseases with no currently available treatments.
The Providence, Rhode Island-based biotech is developing a portfolio of biologically validated, locally delivered, “one-and-done” therapies designed to target the root causes of hereditary retinal conditions that can cause vision loss beginning in childhood. Lyora has acquired advanced preclinical assets and plans to optimize them before rapidly advancing into clinical development.
The company’s lead program, LYA-101, is being developed for retinitis pigmentosa caused by pathogenic variants in the PRPF31 gene. The therapy is designed to augment PRPF31 expression, with the goal of reversing symptoms and addressing the genetic driver of disease.
Lyora’s second program, LYA-102, is being developed for Usher syndrome type 2 and uses optimized CRISPR gene editing technology to address mutations in exon 13 of the USH2A gene. The program may also have potential relevance for hearing loss, reflecting the multisensory nature of Usher syndrome.
Editas Medicine has granted Lyora an exclusive option to license certain rights applicable to USH2A gene editing. Lyora said its pipeline programs are supported by existing preclinical proof-of-concept data and are designed as durable, one-time therapies that may stop disease progression and, in some cases, improve vision.
The company has raised $2.5 million in pre-seed funding and plans to submit an Investigational New Drug application for LYA-101 within the next 18 months, with LYA-102 expected to follow closely behind.
Lyora is led by Pam Stetkiewicz, PhD, who has experience advancing gene editing programs from research into clinical development and previously held senior roles at Editas Medicine, Arbor Biotechnologies, Novartis, and Flagship Pioneering. The company also appointed Rob Aboud as Chief Business Officer and Chris Wilson, PhD, as Senior Vice President and Head of Research.
Lyora was co-founded by Luk Vandenberghe, PhD, Associate Director of the Ocular Genomics Institute at Massachusetts Eye and Ear and Harvard Medical School, and Eric Pierce, MD, PhD, Director of the Ocular Genomics Institute and Chatlos Professor of Ophthalmology at Harvard Medical School. Both bring deep expertise in inherited retinal disease, ocular gene therapy, and clinical translation.
The launch adds another company to the growing retinal genetic medicine field, where local delivery, validated disease biology, and one-time treatment strategies have already supported clinical and regulatory progress. If successful, Lyora’s programs could offer new therapeutic options for patients with PRPF31-associated retinitis pigmentosa, USH2A-associated Usher syndrome, and other inherited retinal diseases with significant unmet need.
