U.K. biotech Ikarovec has teamed up with VectorBuilder to work on an eye disease gene therapy that could be administered in a doctor’s office.
Ikarovec’s IKAR-003 is currently in preclinical development for intermediate age-related macular degeneration (AMD). The one-time injectable therapy is designed to prevent the progression of this eye disease by offering both neuroprotection and complement modulation.
This morning’s agreement will enable VectorBuilder’s AAV capsid technology to create a version of IKAR-003 that can be “delivered to the eye via the minimally invasive intravitreal route in a doctor’s office,” according to the Jan. 6 release.
The fledgling partnership is still at an early stage. For now, the technology will be evaluated before the companies fully commit. But, assuming the deal is finalized, it is expected to be worth more than $1 billion.
There are currently no approved treatments for intermediate AMD, which can progress to more severe sight-threatening conditions like geographic atrophy or wet AMD.
VectorBuilder, which was founded by the University of Chicago’s Bruce Lahn, Ph.D., out-licenses gene delivery intellectual property and provides gene-drug contract development and manufacturing services. The company has already been working with Ikarovec for “several years,” according to today’s release.
“By utilizing our AI-powered DeepCap platform that integrates rational design with ultradeep search of AAV sequence space, we were able to engineer best-in-class ocular capsids,” Lahn said in a statement.
“In non-human primate studies, our intravitreally administered capsids can target wide areas of the retina and successfully transduce virtually all cells of the macula, demonstrating broader and more robust transduction than current clinical intravitreal capsids,” Lahn added.
“Partnering with VectorBuilder on IKAR-003 increases the value of Ikarovec’s pipeline by creating an office-based intravitreally delivered product, ideal for large-scale adoption in the prevention of disease progression setting,” Ikarovec CEO Thomas Ciulla, M.D., said in the release.
“We have assessed other intravitreal capsid options and are confident that VectorBuilder’s technology will provide superior efficacy,” Ciulla added.
Ikarovec has another dual-pathway eye disease gene therapy in development in the form of IKAR-001, which the Norfolk, England-based biotech is hoping to take into the clinic later this year.
The past year has seen a flurry of news for eye-related gene therapies. While Johnson & Johnson’s $130 million bet on an X-linked retinitis pigmentosa candidate suffered a phase 3 setback, the likes of Opus Genetics, MeiraGTx, Beacon Therapeutics and 4D Molecular Therapeutics have seen better news in the clinic.
Meanwhile, Eli Lilly secured an eye disease therapy from MeiraGTx and bought Adverum Biotechnologies outright.