Astellas Pharma has said it will be gaining the rights to use one of 4D Molecular Therapeutics’ (4DMT) gene therapy vectors for at least one rare eye disease.
Under the terms of the licensing agreement, the US genetic medicines company will receive an upfront payment of $20m in exchange for its proprietary R100 vector technology and will also be eligible for future option fees and milestones of up to $942.5m plus certain royalties.
R100 is an adeno-associated virus vector invented by 4DMT for intravitreal delivery. According to the companies, 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”.
Astellas will use the vector initially for one genetic target implicated in rare monogenic ophthalmic diseases and will have the option to add up to two more targets after paying additional option exercise fees.
The Japanese drugmaker will be responsible for all research, development, manufacturing, and commercialisation activities for the resulting programmes.
David Kirn, co-founder and chief executive officer of 4DMT, outlined that over 70 patients have been dosed with R100-based product candidates in wet age-related macular degeneration and rare ophthalmic diseases.
“This 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,” he said.
Adam Pearson, chief strategy officer at Astellas, said: “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.”
The deal comes just less than a month after Astellas and Cullgen entered into a research collaboration and exclusive option agreement aimed at advancing targeted protein degraders.
The partnership, which is worth up to $1.9bn, will utilise Cullgen’s uSMITE targeted protein degradation platform, featuring novel E3 ligands, alongside Astellas’ drug discovery capabilities.
The two companies will jointly identify clinical development candidates, after which Astellas will have an exclusive option to licence the rights for further development and commercialisation of any discovered compounds.