AbbVie, Regenxbio in macular degeneration focused gene therapy deal

By Jane Byrne

- Last updated on GMT

© GettyImages/Marcelo Ricardo Daros
© GettyImages/Marcelo Ricardo Daros

Related tags AAV platform vector Gene therapy Macular degeneration AbbVie

AbbVie is aligning with Regenxbio to develop and commercialize a potential one-time gene therapy for the treatment of chronic retinal diseases.

The partners will look to develop and commercialize RGX-314, a gene therapy candidate for the treatment of eye diseases such as wet age-related macular degeneration (wet AMD), and diabetic retinopathy (DR).

Regenxbio said it wants to leverage AbbVie's global developmental and commercial infrastructure within eye care with its own in-house capabilities - its expertise in AAV gene therapy clinical development and knowledge of manufacturing and production - in the development of the therapy.

The Maryland, US headquartered biotech is set to get an upfront payment of $370m and could also potentially receive up to $1.38bn in milestone payments.

Regenxbio, as per the terms of the deal, will be responsible for the completion of the ongoing studies of RGX-314. But both companies will share the costs of additional trials of RGX-314, according to the partners.

AbbVie will lead the clinical development and commercialization of RGX-314 globally.

The companies said they will equally split the profit from net sales of RGX-314 in the US, and AbbVie would pay Regenxbio tiered royalties on net sales of RGX-314 outside the country.

AAV platform

Regenxbio's NAV technology platform consists of exclusive rights to more than 100 novel adeno-associated virus (AAV) vectors.

The biotech recently outlined how it expects to begin utilizing its new headquarters in Rockville, Maryland in the first half of 2021, and its new cGMP facility, which is expected to allow for production of NAV vectors at scales up to 2,000 liters using the company’s platform suspension cell culture process, is on track to be operational, starting in the first half of 2022.

The firm’s third-party manufacturing network includes Fujifilm Diosynth Biotechnologies​ and Advanced Bioscience Labs.  

Gene therapy targeting VEGF pathway

Wet AMD, noted the companies, is a significant cause of vision loss in the US, Europe and Japan, with up to 2 million people living with wet AMD in those regions alone. 

RGX-314 is currently in a pivotal trial for patients with wet AMD utilizing subretinal delivery. The therapy consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 reportedly inhibits the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

Current anti-VEGF therapies require repetitive and inconvenient intraocular injections, typically ranging from every four to eight weeks in frequency, to maintain efficacy. Due to a variety of factors, including inconvenience and discomfort associated with frequent injections in the eye, patient compliance is a significant concern with anti-VEGF therapies, according to Regenxbio. It added that this is where RGX-314 differs - it is a single treatment, with the potential for long-lasting results.  

Big players entering AMD gene therapy space 

Other big players are actively exploring the use of gene therapy to treat AMD. 

December last year saw Janssen Pharmaceuticals acquire the rights​ to Hemera Biosciences’ investigational gene therapy HMR59, which has been designed to help preserve vision in patients with severe AMD.

Massachusetts-based Hemera, which was founded in 2010 to focus on using gene therapy for patients with AMD, has been investigating HMR59 for both dry and wet forms of the disease.

Gene therapy for late-stage eye diseases is ‘largely unchartered’, noted Mathai Mammen, global head of Janssen R&D, back then. "Through this acquisition, we are blazing the trail to bring innovative solutions to patients who are losing their vision," ​he added.

And Novartis acquired Vedere Bio ​in October last year, giving the company a new platform for AAV-based delivery of gene therapies and an optogenetics program for the treatment and prevention of vision loss and blindness. 

The technologies it acquired include light-sensing proteins that can be delivered to cells in the retina; and AAV delivery vectors that enable treatment via intravitreal injection. These will be able to help patients with photoreceptor death, including inherited retinal dystrophies. Novartis said then that the technology could also prove to have uses for treating  a 'dry form' of AMD, geographic atrophy, which affects more than 5 million people globally. 

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