AbbVie Helps to Advance Gene Therapy for Chronic Retinal Diseases

AbbVie and Regenxbio recently partnered to develop and commercialize RGX-314, a potential one-time gene therapy to treat various chronic retinal diseases. 

Researchers are currently evaluating RGX-314 in patients with wet age-related macular degeneration (wet AMD) in a pivotal trial utilizing subretinal delivery and in patients with wet AMD and diabetic retinopathy (DR) in two separate Phase 2 trials using in-office suprachoroidal delivery. 

Under the collaboration, Regenxbio will be responsible for completing the ongoing trials, while AbbVie and Regenxbio will collaborate and share costs on additional gene therapy trials.

Additional trials include the already planned second trial evaluating subretinal delivery in wet AMD patients and future trials. 

AbbVie will also lead the clinical development and commercialization of RGX-314 globally. Regenxbio will participate in commercialization efforts as part of the commercialization plan.

“We are committed to finding solutions for patients living with difficult-to-treat retinal diseases and to helping preserve and protect our patients from visual impairment and devastating vision loss,” Tom Hudson, MD, senior vice president of research & development and chief scientific officer at AbbVie, said in the announcement.

“In collaboration with REGENXBIO, we aim to make a remarkable impact for the millions of patients suffering from vision loss associated with retinal diseases,” Hudson continued. 

AbbVie will pay Regenxbio a $370 million upfront payment with the potential for Regenxbio to receive up to $1.38 billion in additional development, regulatory, and commercial milestones. But the companies will share equally in profits from net sales of RGX-314 in the US.

Additionally, Regenxbio will lead the manufacturing of RGX-314 for clinical development and commercial US supply. And AbbVie will lead the manufacturing of the gene therapy for commercial supply outside the US.

The companies expect the transaction to close by the end of 2021.

“We expect to leverage AbbVie’s global developmental and commercial infrastructure within eye care with our expertise in AAV gene therapy clinical development and deep in-house knowledge of manufacturing and production to continue the development of RGX-314,” said Kenneth T. Mills, president and chief executive officer of REGENXBIO.

Wet AMD causes loss of vision due to new, leaky blood vessel formation in the retina. Nearly two million people currently live with wet AMD in the US, Europe, and Japan alone. 

In March 2020, Oregon Health & Science University held the first-ever gene therapy clinical trial, Brilliance, to address blindness-causing gene mutation, LCA10. 

The procedure used the gene-editing tool CRISPR to edit human genes, also known as in vivo gene editing. Previously, gene-editing methods have edited genetic material after it was removed from the human body.