PolyActiva and RareSight Partner to Develop First-in-Class Treatments for Rare Pediatric Retinal Diseases
核心洞察
PolyActiva (搜索) and RareSight (搜索) announced a strategic collaboration to develop breakthrough therapies for rare pediatric retinal diseases using PolyActiva's PREZIA drug delivery platform technology.
The partnership aims to create first-in-class, sustained-release ocular implants for inherited retinal diseases (搜索) that currently have no approved drug treatments.
The collaboration expands PolyActiva (搜索)'s clinical portfolio beyond glaucoma (搜索), with the potential to address up to 6.8 million people worldwide affected by inherited retinal disorders.
PolyActiva (搜索), a clinical-stage biopharmaceutical company, and RareSight (搜索), Inc., an ophthalmology-focused life sciences company, have announced a strategic collaboration to develop first-in-class therapies for rare pediatric retinal diseases that currently have no approved drug treatments. The partnership leverages PolyActiva's proprietary PREZIA drug delivery platform technology and RareSight's expertise in inherited retinal diseases (搜索) (IRDs).
Novel Drug Delivery Approach for Pediatric Patients
The research collaboration will utilize PolyActiva (搜索)'s PREZIA platform to create first-of-its-kind, new chemical entity (NCE)-eligible pro-drug candidates for people affected by blinding eye conditions. The treatment approach involves intravitreal administration to deliver sustained-release, zero-order treatment directly to the retina (搜索), requiring no patient administration—a significant benefit for children and their caregivers.
"This collaboration represents an important step in PolyActiva (搜索)'s growth as we expand our proprietary platform into new therapeutic areas," said Jerry St. Peter, CEO and Board Director of PolyActiva. "Partnering with RareSight (搜索) allows us to apply our PREZIA technology beyond glaucoma (搜索) to address the urgent unmet needs of children living with inherited retinal disorders."
Addressing Critical Unmet Medical Need
Inherited retinal diseases (搜索) are rare genetic disorders that cause progressive vision loss and blindness, with symptoms often beginning in childhood. These conditions collectively affect up to 6.8 million people worldwide, yet there are no approved drug treatments for these severe and progressive genetic ophthalmic conditions.
Carmen Caricchio, CEO and Founder of RareSight (搜索), emphasized the collaboration's potential impact: "By combining RareSight's therapeutic discovery and development expertise with PolyActiva (搜索)'s novel drug delivery platform, this collaboration represents a major step toward transforming how inherited eye diseases are treated. Together, we are advancing a new class of long-acting pharmacologic therapies to address early-onset vision loss, with the potential to change a child's life and bring hope to generations to come."
Clinical Perspective and Current Portfolio
Dr. Sandeep Grover, a Medical Retina (搜索) and Inherited Retinal Disease Specialist and Professor of Ophthalmology based in Jacksonville, Florida, highlighted the clinical significance: "Children with inherited retinal diseases (搜索) face lifelong visual challenges with no approved drug therapies. As a pioneer of the earliest research in this field and caring for kids and their families for more than two decades, I am encouraged by novel therapeutic strategies that support our goal of helping patients maintain vision, function, and independence as long as possible."
The collaboration expands PolyActiva (搜索)'s clinical-stage ophthalmic portfolio, which includes PA5108, a new chemical entity and biodegradable ocular micro-implant designed for sustained intraocular pressure control for up to six months. PolyActiva's Phase 2b U.S. trial is currently underway in approximately 75 patients across 12 sites to evaluate PA5108's safety, tolerability, and durability in controlling intraocular pressure over time.
PREZIA Platform Technology
PolyActiva (搜索)'s proprietary PREZIA drug delivery platform distinguishes itself from traditional polymer matrix or nanoparticle-based systems that rely on passive diffusion. Instead, PREZIA uses covalent bonding to attach therapeutic agents to a polymer backbone, enabling precise, consistent, and fully customizable drug release over periods ranging from one week to over one year.
The platform's biodegradable design eliminates residual buildup and supports repeat dosing. PREZIA-based therapies can be formulated as rod-shaped implants or injectable gels and are compatible with both single-agent and combination therapies for a broad range of ocular conditions. The technology allows for new patent protection over existing drugs when delivered with this platform.
By broadening its proprietary pro-drug technology into new therapeutic domains, including pediatric rare retinal diseases, both companies have the potential to develop the first-ever drug treatments for underserved patient populations.