REGENXBIO Inc. (NASDAQ: RGNX)

REGENXBIO Inc., a prominent player in the biotechnology industry, is dedicated to enhancing lives through the curative possibilities of gene therapy. The company's innovative gene therapies are designed to deliver functional genes to address genetic defects in cells, enabling the production of therapeutic proteins or antibodies that can impact disease. REGENXBIO's proprietary gene delivery platform, known as the NAV Technology Platform, utilizes adeno-associated virus (AAV) vectors to deliver genes to cells, a method that has been proven to be safe and effective in gene therapy.

The company's pipeline of investigational AAV Therapeutics is concentrated on three key areas: retinal diseases, neuromuscular diseases, and neurodegenerative diseases. One of its most advanced programs is ABBV-RGX-314, a potential one-time treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). This investigational AAV Therapeutic uses a NAV AAV8 vector to deliver a gene for a monoclonal antibody fragment designed to inhibit vascular endothelial growth factor (VEGF) activity, which is responsible for the formation of new blood vessels and retinal fluid accumulation.

REGENXBIO is currently conducting two pivotal trials, ATMOSPHERE and ASCENT, for the treatment of wet AMD using ABBV-RGX-314 delivered subretinally. The company has also initiated a Phase II bridging study to evaluate the pharmacodynamics, safety, and efficacy of ABBV-RGX-314 delivered subretinally. Additionally, REGENXBIO is evaluating the efficacy, safety, and tolerability of suprachoroidal delivery of ABBV-RGX-314 in the AAVIATE trial for the treatment of wet AMD.

The NAV Technology Platform has enabled the development of a broad pipeline of investigational AAV Therapeutics, including RGX-202, which is being developed for the treatment of Duchenne muscular dystrophy (DMD). This rare disease is caused by mutations in the gene responsible for making dystrophin, a protein of central importance for muscle function. REGENXBIO's investigational AAV Therapeutic, RGX-202, uses a NAV AAV9 vector to deliver a functional copy of the dystrophin gene to muscle cells, which has been shown to be safe and effective in preclinical studies.

REGENXBIO's manufacturing capabilities are designed to support the development and commercialization of its investigational AAV Therapeutics. The company has developed a proprietary, high-yielding manufacturing process platform, known as NAVXpress, which can be applied across multiple AAV Therapeutics. REGENXBIO has also developed a comprehensive set of analytical methods to assess quality and characterize the product.

The company's delivery device expertise includes the development of image-guided device delivery of AAV Therapeutics into the cerebrospinal fluid to target the brain and central nervous system for neurodegenerative diseases. REGENXBIO has also led the development of two different types of delivery devices for AAV Therapeutics into the eye for targeting the retina of patients with retinal diseases.

REGENXBIO has collaborated with several leading biotechnology and pharmaceutical companies, including AbbVie, Novartis, and GSK, to develop and commercialize AAV Therapeutics. The company has also formed partnerships with academic institutions, such as the University of Pennsylvania and the University of Minnesota, to develop and commercialize AAV Therapeutics.

In terms of competition, REGENXBIO faces intense competition from other companies developing gene therapies, including 4D Molecular Therapeutics, Adverum Biotechnologies, Amicus Therapeutics, BioMarin Pharmaceutical, Homology Medicines, MeiraGTx Limited, Novartis AG, Passage Bio, Inc., PTC Therapeutics, Inc., Roche, Sanofi, Sarepta Therapeutics, Inc., Solid Biosciences, Inc., Taysha Gene Therapies, Inc., and uniQure N.V. Additionally, the company faces competition from other methods for modifying genes and regulating gene expression.

REGENXBIO's customers are primarily biotechnology and pharmaceutical companies that have licensed its NAV Technology Platform for the development of gene therapies. The company's revenues are generated from license and royalty fees paid by its customers. In 2023, one customer, Novartis Gene Therapies, accounted for approximately 95% of the company's total revenues.

The company's investigational gene therapies are currently in the clinical stage, involving the administration of its gene therapy candidates to healthy volunteers or patients under the supervision of qualified investigators. Clinical studies are conducted under protocols detailing the objectives of the clinical study, dosing procedures, subject selection and exclusion criteria, and the parameters to be used to monitor subject safety, including stopping rules.