Astellas will pay 4D Molecular Therapeutics (4DMT) US$20 million upfront to use its viral vector for a rare monogenic eye disease in a deal worth up to $962 million.
In a media release issued by the companies, Astellas does not disclose what rare eye disease it plans to target with the adeno-associated virus (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 pay to use the vector for two more rare monogenic diseases in the future.
The vector, known as R100, is the one that 4DMT has used for its eye disease gene therapy programs, which include wet AMD, diabetic macular oedema, X-linked retinitis pigmentosa, choroideremia, and geographic atrophy.
“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, Co-Founder and Chief Executive Officer of 4DMT.
We believe that this collaboration will bring synergies between the two companies’ cutting-edge research
“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.”
He said 4DMT retains rights to large market non-hereditary ophthalmic diseases.
Astellas’ Chief Strategy Officer, Adam Pearson, said the company had positioned blindness and regeneration as one of its primary focus points.
“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.”
Reference
- Astellas, Astellas and 4D Molecular Therapeutics (4DMT) wnter into license agreement to use 4DMT’s proprietary intravitreal R100 vector for rare ophthalmic targets (news release), 11 July 2023. Available at: astellas.com/en/news/28136com/en/news/28136 [accessed 14 July 2023].
