RARE Daily

Astellas Licenses 4DMT’s Vector for Rare Ophthalmic Targets

July 11, 2023

Rare Daily Staff

Astellas Pharma and 4D Molecular Therapeutics entered a license agreement under which Astellas will gain rights to utilize 4DMT’s intravitreal retinotropic R100 vector for one genetic target implicated in rare monogenic ophthalmic disease.

Astellas will also have the option to add up to two additional targets implicated in rare monogenic ophthalmic diseases after paying additional option exercise fees.

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.

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. 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.

“This collaboration with Astellas continues to validate R100 for routine intravitreal low dose delivery of genetic payloads for the treatment of retinal diseases,” said David Kirn, co-founder and CEO of 4DMT. “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.”

Photo: David Kirn, co-founder and CEO of 4DMT

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