Rhode Island-based Lyora Therapeutics has emerged from stealth with USD 2.5 million in pre-seed financing and a two-program pipeline of genetic medicines targeting inherited retinal diseases for which no approved treatments currently exist. The company's investor base was not disclosed.
Lyora's scientific foundation rests on co-founders Luk Vandenberghe, PhD, who serves as board chair and is Associate Director of the Ocular Genomics Institute at Massachusetts Eye and Ear Infirmary and Harvard Medical School, and Eric Pierce, MD, PhD, Director of the Ocular Genomics Institute and Chatlos Professor of Ophthalmology at Harvard Medical School. Vandenberghe has previously co-founded Odylia Therapeutics, GenSight Biologics, and Akouos, giving Lyora a leadership profile with direct precedent in ocular gene therapy development and commercialization.
Pam Stetkiewicz, PhD, has been appointed CEO, bringing prior experience as Vice President of Program and Alliance Management at Editas Medicine and COO at Arbor Biotechnologies, as well as scientific roles at Novartis and Flagship Pioneering. Rob Aboud, JD, MSc, joins as Chief Business Officer, having previously co-founded Affinia Therapeutics and held business development roles at GSK. Chris Wilson, PhD, joins as SVP Head of Research, with prior positions at Novartis, Editas Medicine, and Stylus Medicine.
The lead program, LYA-101, targets retinitis pigmentosa driven by pathogenic variants in the PRPF31 gene, using a gene therapy approach designed to augment PRPF31 expression and thereby reverse disease-associated symptoms. The second program, LYA-102, applies an optimized CRISPR editing strategy to correct mutations in exon 13 of the USH2A gene in patients with Usher Syndrome Type 2, with the company noting potential applicability to hearing loss as well. Both programs are described as locally delivered and intended to produce durable, single-administration effects. Lyora has secured an exclusive option from Editas Medicine to license rights applicable to USH2A gene editing for LYA-102. The company states that pre-clinical proof-of-concept data support both programs, and it plans to submit an investigational new drug application for LYA-101 within 18 months, with LYA-102 to follow.