REGENXBIO Inc.

Company Overview

Regenxbio Inc. is a biotechnology company. The company focuses on developing gene therapies to treat genetic disorders, with the main goal of producing therapeutic proteins or antibodies that can significantly impact various diseases.

Regenxbio investigational gene therapies rely on adeno-associated virus (AAV) vectors from its proprietary NAV Technology Platform. These AAV vectors are chosen for their safety and do not cause immune responses or diseases, making them ideal for delivering gene therapies. NAV Technology Platform includes exclusive rights to a wide range of AAV vectors, such as AAV7, AAV8, AAV9, and over 100 other novel AAV vectors. These vectors form the basis of the company’s ongoing clinical programs, and the company continues to expand its platform and pipeline of potential AAV vector-based gene therapies through continuous research and development efforts, collectively known as AAV Therapeutics.

The company’s investigational AAV Therapeutics pipeline targets various diseases, focusing on retinal, neuromuscular, and neurodegenerative disorders. Critical therapies include RGX-314 for age-related macular degeneration (wet AMD) and diabetic retinopathy (DR), RGX-202 for Duchenne muscular dystrophy (Duchenne), RGX-121, RGX-111, and RGX-181 for Mucopolysaccharidosis type II, Mucopolysaccharidosis type I, and late infantile neuronal ceroid lipofuscinosis type II, respectively, and RGX-381 for the ocular manifestations of CLN2 disease.

The company’s revenue primarily comes from license and royalty revenue. A significant portion of the total revenues in recent years has come from key customers such as Novartis Gene Therapies and AbbVie.

Financial Highlights (FY 2024)

Company's Business Segments

• Zolgensma Royalties : This segment includes Commercial sales of Zolgensma, Royalties received from the third party clients.
• Other License and Royalty Revenue : This segment includes other commercial sales of Licensees, Collaborators products.