Lyora Therapeutics Launches With $2.5M Seed Funding to Advance Genetic Medicines for Inherited Retinal Diseases
核心洞察
Lyora Therapeutics (搜索) launched with $2.5 million in pre-seed funding to develop one-and-done genetic medicines targeting inherited retinal diseases that currently lack treatments.
The lead program, LYA-101 (搜索), targets retinitis pigmentosa (搜索) caused by PRPF31 (搜索) mutations, with an IND filing planned within 18 months.
A second program, LYA-102 (搜索), uses optimized CRISPR technology to address USH2A (搜索) mutations in Usher Syndrome Type 2 (搜索), with an exclusive option licensed from Editas Medicine.
Lyora Therapeutics (搜索), a biotechnology company focused on developing genetic medicines for inherited retinal diseases, launched today with $2.5 million in pre-seed funding and an experienced leadership team assembled to propel its pipeline of biologically validated therapies toward the clinic. The Providence, R.I.-based company has acquired advanced pre-clinical assets and will tailor them using optimized technology before rapidly advancing them into clinical development.
"Lyora Therapeutics (搜索) is on a mission to help patients impacted by hereditary conditions that cause vision loss beginning as early as childhood, by creating medicines that intervene at the root cause of disease," said Pam Stetkiewicz, PhD, CEO of Lyora. "We are building on the successful pathways of approved therapies for ophthalmologic conditions with our suite of one-and-done treatments that are delivered locally and remain durable for life."
Pipeline Programs Target Unmet Needs in Retinal Disease
Lyora's lead program, LYA-101 (搜索), is designed for the treatment of retinitis pigmentosa (搜索) caused by pathogenic variants in the PRPF31 (搜索) gene. The therapeutic approach aims to reverse symptoms in patients by augmenting expression of PRPF31. The company plans to submit an Investigational New Drug (IND) application for LYA-101 within the next 18 months.
The second program, LYA-102 (搜索), targets Usher Syndrome Type 2 (搜索) and employs an optimized CRISPR technology to address mutations in exon 13 of the USH2A (搜索) gene. Notably, this program may also translate into a therapeutic option for hearing loss. Editas Medicine has granted Lyora an exclusive option to license certain rights applicable to USH2A gene editing.
Both pipeline products are supported by existing pre-clinical proof of concept data demonstrating that these one-and-done therapies have the potential to stop disease progression and, in some cases, improve vision.
Leadership and Scientific Expertise
Lyora has appointed Pam Stetkiewicz, PhD, as its Chief Executive Officer. Stetkiewicz brings deep domain expertise managing gene editing programs from research into the clinic, having previously served as Vice President of Program and Alliance Management at Editas Medicine and COO at Arbor Biotechnologies. Her career also includes scientific leadership roles at Novartis and Flagship Pioneering.
Rob Aboud, JD, MSc, joins as Chief Business Officer, bringing business development and transactions experience from GSK and Affinia Therapeutics, where he was CLO & Head of IP. Chris Wilson, PhD, has been appointed SVP Head of Research, with senior scientific experience at Novartis, Editas Medicine, and Stylus Medicine.
The company's scientific foundation rests on the expertise of its co-founders: Luk Vandenberghe, PhD, 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 the Chatlos Professor of Ophthalmology at Harvard Medical School. Pierce is described as a world-renowned expert with deep clinical experience who has participated in multiple clinical trials of genetic therapies for inherited retinal diseases.
"Lyora's strength comes in the form of biologically validated science, an experienced, focused team and a vision to push the boundaries of genetic-based approaches beyond what we've seen in recent drug development," said Vandenberghe, who serves as Chair of the Board of Directors. "Our approach and superior delivery method have the capability to address complex retinal diseases with no currently existing treatment, and we are enthusiastic about the possibility that Lyora can make a near-term meaningful impact to patients."
The company's therapeutic strategy leverages de-risked and validated biology, with medicines delivered locally and designed to remain durable for life, offering a potential paradigm shift for patients with inherited retinal diseases who currently have no available treatment options.