AviadoBio Secures $413M Option Deal for UGX-202 Optogenetics Gene Therapy to Restore Vision in Retinitis Pigmentosa
核心洞察
AviadoBio (搜索) has entered an exclusive option and license agreement worth up to $413 million with UgeneX Therapeutics (搜索) for UGX-202, an investigational AAV-based gene therapy targeting retinitis pigmentosa (搜索).
UGX-202 employs optogenetics technology to deliver light-sensitive proteins to retinal cells (搜索), reprogramming preserved cells to function as photoreceptors (搜索) and restore vision regardless of underlying genetic cause.
The therapy addresses a significant unmet medical need for over 1.5 million people worldwide living with retinitis pigmentosa (搜索), a progressive inherited retinal disease that leads to blindness.
London-based AviadoBio (搜索) has announced an exclusive option and license agreement with Shanghai-based UgeneX Therapeutics (搜索) for UGX-202, an investigational AAV-based gene therapy designed to restore vision in patients with retinitis pigmentosa (搜索) (RP). The deal, valued at up to $413 million, represents a significant investment in next-generation optogenetics technology for treating inherited retinal diseases (搜索).
Novel Optogenetics Approach Targets Vision Restoration
UGX-202 represents a breakthrough approach in treating retinal degeneration through visual optogenetics. The therapy delivers a light-sensitive protein to cells in the retina, addressing the fundamental problem in many retinal diseases where patients experience a loss of photoreceptors (搜索) – the cells responsible for detecting light and converting it into visual signals for the brain.
The innovative mechanism works by reprogramming preserved cells in the retina to take on the function of lost photoreceptors (搜索), thus re-establishing vision irrespective of the underlying genetic cause. According to the companies, the UGX-202 capsid, regulatory sequences, and opsin transgene have all been optimized for potential best-in-class performance.
Addressing Significant Unmet Medical Need
Retinitis pigmentosa (搜索) affects over 1.5 million people worldwide, representing a substantial unmet medical need for new treatments. RP is a group of progressive inherited retinal diseases (搜索) characterized by the primary degeneration of rod photoreceptors (搜索), followed by the loss of cone photoreceptors. It serves as a leading cause of visual disability and blindness.
The disease is typically diagnosed in children and young adults, though the average age of diagnosis is approximately 35.1 years. Progressive symptoms include night blindness, reduction in peripheral and central vision commonly called "tunnel vision," and reduced ability to see details and colors. The progression of RP varies widely between individuals, though many patients are legally blind by 40-50 years of age.
Clinical Development and Commercial Terms
UGX-202 is currently being studied in patients with retinitis pigmentosa (搜索) in an investigator-initiated study. The therapy is delivered as a one-time intravitreal injection and has the potential to treat RP as well as multiple other inherited retinal diseases (搜索) and macular degeneration (搜索). A second undisclosed indication is set to enter clinical development by year-end.
Under the agreement terms, AviadoBio (搜索) will have the option to receive a worldwide exclusive license outside of Greater China for development and commercialization rights to UGX-202 in RP and other indications. If the option is exercised, UgeneX will be eligible to receive upfront payments, research and development milestones, and sales milestone payments of up to $413 million, as well as royalties on net sales.
Strategic Partnership Expansion
"We are thrilled to expand our portfolio of clinic-stage assets that span neurodegenerative conditions from the eye to the brain, united by our core expertise in targeted delivery, potent genetic payloads, and rapid translation to now include RP and other retinal diseases," said Lisa Deschamps, CEO of AviadoBio (搜索). "Optogenetics is an exciting field with clinical precedent and clear opportunity for next-generation approaches, like UGX-202, to improve vision for people with retinal disease."
Dr. Wu Kai, President of UgeneX, expressed enthusiasm about the partnership: "We are delighted to partner with AviadoBio (搜索) to jointly promote the clinical application and global collaboration of ophthalmic gene therapy. This partnership is a significant milestone for our company toward entering the global market and also demonstrates the R&D capabilities and global potential of our pipeline."
The agreement adds to the growing trend of Western biotechnology companies partnering with Chinese firms to access novel therapeutic approaches and expand their development pipelines in specialized therapeutic areas.