AbbVie (NYSE:ABBV) will work with the gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) to develop serious eye diseases with few treatment options. The partnership will unite AbbVie’s development and commercialization capabilities with Capsida’s adeno-associated virus (AAV) engineering platform. AbbVie will also help explore therapeutic cargo approaches. The companies plan on identifying and advancing three programs.
The collaboration will center around Capsida’s AAV engineering platform and manufacturing capabilities to develop novel ophthalmological therapies.
Under the terms of the agreement, AbbVie will pay Capsida an upfront payment of $70 million. In addition, AbbVie could also make potential equity investments in the expanded agreement.
Capsida is also eligible for up to $595 million in option fees and R&D milestones if the company reaches commercial milestones. It could also receive mid-to-high single-digit royalty payments assuming the collaboration yields viable drugs.
In 2021, the two companies announced they were developing neurodegenerative disease treatments.
Capsida Biotherapeutics’ approach to gene therapy is based on capsid engineering. Unlike many gene therapies that use naturally occurring wild-type viruses, Capsida’s proprietary AAV engineering platform allows for more targeted delivery of genetic material to specific cells. As a result, this approach can overcome some of the limitations of traditional gene therapy.
“The problem with a lot of [wild-type] viruses is twofold,” said Peter Anastasiou in an interview with Drug Discovery & Development at the J.P. Morgan Healthcare Conference. “One, in brain disorders, you don’t get very good penetration across the blood-brain barrier.” The other issue is that such viruses tend to accumulate in the liver, resulting in toxicity and safety issues, he said.
Capsida aims to sidestep those problems by engineering the virus’s surface to optimize the amino acid sequences there. The company can thus encourage tropism — or getting to specific targets and organs. Additionally, the engineering approach can discourage gene therapy from ending up in non-target organs such as the liver.