R100 is an adeno-associated virus (AAV) vector invented by 4DMT for intravitreal delivery.
Astellas Pharma Inc. announced a license agreement under which Astellas gains rights to utilize the intravitreal retinotropic R100* vector invented by 4DMT for 1 genetic target implicated in rare monogenic ophthalmic diseases, with options to add up to two additional targets implicated in rare monogenic ophthalmic diseases after paying additional option exercise fees.
According to the companies, R100 is an adeno-associated virus (AAV) vector invented by 4DMT for intravitreal delivery. It has the ability to penetrate the internal limiting membrane barrier and to efficiently transduce the entire retina, resulting in robust transgene expression within retinal cells. All three 4DMT clinical-stage ophthalmic product candidates utilize the R100 vector, including 4D-150 for wet age-related macular degeneration and diabetic macular edema.
The companies noted that under the terms of the agreement, 4DMT will provide its proprietary R100 vector technology to Astellas to deliver Astellas’ unique genetic payloads for the treatment of rare monogenic diseases.
According to the agreement, Astellas will conduct all subsequent research, development, manufacturing, and commercialization activities. 4DMT will receive $20 million upfront, and potential future option fees and milestones of up to $942.5 million including potential near-term development milestones of $15 million for the initial target. In addition, 4DMT is entitled to receive mid-single-digit to double-digit, sub-teen royalties on net sales of all licensed products.
David Kirn, MD, co-founder and CEO of 4DMT, said the collaboration with Astellas, a leader in AAV gene therapy, continues to validate R100 for routine intravitreal low-dose delivery of genetic payloads for the treatment of retinal diseases.
“With over 70 patients dosed to date with R100-based product candidates in wet age-related macular degeneration and rare ophthalmic diseases, this collaboration also demonstrates the modularity of the Therapeutic Vector Evolution platform resulting in efficient design and development of new intravitreal products,” he said in the news release. “4DMT retains rights to large market non-hereditary ophthalmic diseases.”
Moreover, Adam Pearson, chief strategy officer at Astellas said in the release his company has “a strong commitment to developing novel treatments for ophthalmic diseases, and have positioned Blindness & Regeneration as one of the Primary Focuses of our R&D strategy.”
“Staying at the forefront of gene therapy technology is a key part of our strategy,” Pearson concluded in the news release. “We believe that this collaboration will bring synergies between the two companies' cutting-edge research, and will ultimately lead to the development of new therapeutics for patients with ophthalmic diseases at high risk of blindness.”