4DMT inks potential $962 million gene therapy licensing deal

Gene therapy company 4D Molecular Therapeutics (NASDAQ: FDMT) has signed a licensing agreement with pharma giant Astellas worth a potential $962 million. Astellas has licensed the rights to use 4DMT’s intravitreal retinotropic R100 vector for one genetic target implicated in rare monogenic ophthalmic diseases, with options to add up to two additional targets.

Under the terms of the agreement, Astellas will use 4DMT’s proprietary R100 vector technology to deliver unique genetic payloads for the treatment of rare monogenic diseases. 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.

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. 4DMT has three clinical-stage ophthalmic product candidates that utilize the R100 vector for indications including wet age-related macular degeneration and diabetic macular edema.

“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,” said Dr David Kirn, 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.”

“At Astellas, we have 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,” said Adam Pearson, Chief Strategy Officer at Astellas. “Staying at the forefront of gene therapy technology is a key part of our strategy. 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.”

Photograph: Freepik