Astellas licenses AAV vector from 4DMT for rare eye disease gene therapy

Astellas will pay 4D Molecular Therapeutics $20 million upfront to use its viral vector for a rare monogenic eye disease in a deal worth up to $962 million, the companies announced Monday evening.

Astellas is not disclosing what rare eye disease it plans to target with the adeno-associated virus, or AAV, vector. But it is not one that 4D Molecular Therapeutics, known as 4DMT, is working on already, according to the companies. Astellas also has the option to use the vector for two more rare monogenic diseases down the line.

The vector Astellas is licensing, known as R100, is the one that 4DMT has used for its eye disease gene therapy programs, which include wet AMD, diabetic macular edema, X-linked retinitis pigmentosa, choroideremia, and geographic atrophy.

Morten Sogaard

Astellas and 4DMT both pointed to the fact that the vector has been tested in humans as a major driver for the licensing deal. “There are other companies also moving forward with vectors that are starting to look quite good as well,” Astellas’ division head of gene therapy research Morten Sogaard said. “I think the differentiator for us, at this point in time, is that they’ve been to the clinic.”

In data released in April on its wet AMD gene therapy candidate, 4DMT said that four of five patients in the high-dose group were taken off their regular anti-VEGF injections, and that 14 of 15 saw no inflammation — a side effect seen with other gene therapies delivered to the eye.

With previous eye gene therapies, such as Roche/Spark Therapeutics’ Luxturna, doctors do surgery to deliver the treatment, significantly complicating the procedure, according to 4DMT CEO David Kirn. “We do a simple outpatient intravitreal injection just like it’s done for Eylea, Lucentis, thousands of thousands of times every day,” he said, referring to Regeneron and Roche’s wet AMD drugs.

In addition to the $20 million upfront, Astellas could pay 4DMT $15 million in early-stage milestones, and possibly $927.5 million more down the line, with “mid-single digit to double-digit, sub-teen” royalties if a product with 4DMT’s vector makes it to market.

Earlier this year Astellas paid $5.9 billion to buy Iveric Bio and its late-stage drug for geographic atrophy. The FDA is expected to make its decision on Iveric’s drug, which is a complement inhibitor, by Aug. 19.

therapeutics
gene therapy
fda
research
buy