Astellas Pharma has entered an agreement for licensing 4D Molecular Therapeutics’ (4DMT) R100 vector for a genetic target associated with rare monogenic ophthalmic ailments.

Retinotropic R100 is an adeno-associated virus (AAV) vector intended to be administered intravitreally.

Astellas holds options to include up to two further targets linked to rare monogenic ophthalmic ailments, contingent on the payment of further option exercise fees.

R100 has the potential to cross the internal limiting membrane barrier and successfully transduce the whole retina. This approach leads to transgene expression in retinal cells. 

All three clinical-stage ophthalmic products of 4DMT leverage the R100 vector. 

Astellas will make an upfront payment of $20m to 4DMT. 

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4DMT is eligible to receive option fees and milestone payments totalling $942.5m.

This payment includes $15m near-term development milestone fees for the first target. 

Astellas will also make royalty payments to 4DMT on net sales of products that are licensed under the agreement.

Research, development, production and marketing will be carried out by Astellas following licensing.

4DMT co-founder and CEO David Kirn stated: “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 the efficient design and development of new intravitreal products.”

The latest development comes after the US Food and Drug Administration granted priority review for Astellas’ biologics licence application for zolbetuximab for gastric cancer.