Astellas and 4D Molecular Therapeutics enter license agreement for rare ophthalmic targets

Betsy Goodfellow | July 11, 2023 | News story | Research and Development 4D Molecular Therapeutics, AAV vectors, Astellas Pharma, Opthalmology, ophthalmology 

Astellas Pharma and 4D Molecular Therapeutics (4DMT) have announced a license agreement under which Astellas has the right to use the intravitreal retinotropic R100 vector invented by 4DMT for one genetic target related to rare monogenic ophthalmic disease, with the option to investigate two additional implicated targets on the provision of additional option exercise fees.

R100, an adeno-associated virus (AAV) vector, was invented by 4DMT for intravitreal delivery and can penetrate the internal limiting membrane barrier to efficiently transduce the entire retina which results in robust transgene expression within retinal cells.

Under the terms of this agreement, 4DMT will provide its R100 vector technology to Astellas in order to deliver Astellas’ unique genetic payloads for the treatment of rare monogenic diseases. Astellas will conduct any subsequent research, development, manufacturing and commercialisation activities, while 4DMT will receive an upfront payment of $20m along with potential future milestone payments of up to $942.5m.

David Kirn, MD, co-founder and CEO of 4DMT, commented: “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. 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. 4DMT retains rights to large market non-hereditary ophthalmic diseases.”

Adam Pearson, chief strategy officer at Astellas, added: “At Astellas, we have a strong commitment to developing novel treatments for ophthalmic diseases, and have positioned Blindness and Regeneration as one of the Primary Focuses of our research and development strategy. Staying at the forefront of gene therapy technology is a key part of our strategy. We believe that this collaboration will ultimately lead to the development of new therapeutics for patients with ophthalmic diseases at high risk of blindness.” Betsy Goodfellow

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