Astellas and 4D Molecular Therapeutics Join Forces to Combat Rare Ophthalmic Diseases with Intravitreal R100 Vector.

Astellas Pharma Inc. and 4D Molecular Therapeutics, Inc. have reached an exciting new agreement – Astellas has been granted access to the intravitreal retinotropic R100* vector developed by 4DMT to target one genetic cause of rare monogenic ophthalmic disease, with the potential to explore two additional targets after paying additional fees. This collaboration could bring life-changing breakthroughs to patients suffering from these diseases.

4DMT’s revolutionary R100 adeno-associated virus (AAV) vector lifts the barrier to gene therapy for ocular disorders, allowing transgene expression right within patient’s retinal cells. Three 4DMT clinical-stage ophthalmic product candidates are developed with the R100 vector, including 4D-150 for wet age-related macular degeneration and diabetic macular edema – enabling hope for patients and the development of more effective treatments.

4DMT has concluded an agreement with Astellas to use its revolutionary R100 vector technology to develop treatments for rare monogenic diseases. Astellas will be responsible for all subsequent research, development, manufacturing, and commercialization activities.

4DMT will be compensated with US$20 million upfront, and the potential to receive option fees and milestones of up to US$942.5 million, including $15 million for the first target. 4DMT also stands to gain mid-single to double-digit, sub-teen royalties on net sales of all products generated from the collaboration.

4DMT is thrilled to announce its collaboration with Astellas, a leader in AAV gene therapy. The collaboration serves to further validate R100, an intravitreal low dose delivery of genetic payloads for treating retinal diseases.

To date, over 70 patients have been dosed with R100-based product candidates in wet age-related macular degeneration and rare ophthalmic diseases, showcasing the modularity of its Therapeutic Vector Evolution platform. Additionally, 4DMT retains rights for large market non-hereditary ophthalmic diseases. This exciting partnership is yet another example of 4DMT’s longstanding commitment to advanced treatments for eye disorders.

At Astellas, we are dedicated to creating groundbreaking treatments for eye diseases and have made Blindness & Regeneration a priority focus in our R&D efforts. We are committed to staying ahead of the curve in gene therapy technology, and we believe that this collaboration between the two companies will be highly beneficial, allowing us to work together to generate new therapies and treatments for those suffering from ophthalmic diseases with a high risk of blindness.

About Astellas

Astellas Pharma Inc. is on the cutting edge of healthcare innovation, with a Focus Area Approach to creating new drugs that address some of today’s most pressing unmet needs. Led by extensive Biological and Modality knowledge, Astellas is extending beyond their traditional Rx focus to develop Rx+® healthcare solutions combining the best of their expertise with the latest technology from external partners. It all adds up to Astellas turning groundbreaking science into real VALUE for the patients who need it the most.

About 4DMT

4DMT combines Nobel Prize-winning directed evolution technology with synthetic AAV capsid-derived sequences to create a powerful and proprietary invention platform for genetic medicines. Our clinical-stage and preclinical product candidates are specifically targeted to treat a variety of large market and rare diseases, including wet AMD, DME, cystic fibrosis, Fabry disease cardiomyopathy, XLRP, and choroideremia.

We strive for our products to provide high-efficiency transduction of diseased cells, low-dose administration, and reduced immunogenicity with potential to be delivered through clinically routine and minimally invasive methods.

Additionally, we are currently developing preclinical product candidates such as 4D-175 for geographic atrophy and 4D-725 for AATLD. By using 4DMT’s novel Therapeutic Vector Evolution platform, genetic medicines can finally fulfill their potential in providing safe and effective treatments.

We are currently exploring the exciting possibilities of our five product candidates — 4D-150, 4D-710, 4D-310, 4D-125, and 4D-110 — as treatments for various therapeutic uses. Although they have yet to be approved by the US FDA or any other regulatory authority, we are hopeful that these promising treatments could make a real difference in people’s lives.

4D Molecular Therapeutics™ (4DMT™) is revolutionizing the way therapies are developed, utilizing its proprietary Therapeutic Vector Evolution™ technology. Forging the future of molecular medicine, 4DMT is boldly branded with its unforgettable logo.

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