Regeneron is a biotechnology company developing advanced medical treatments. Their work includes a focus on gene therapy, an innovative approach that aims to address diseases at their genetic roots. This field seeks to modify or introduce genetic material to prevent or treat various conditions, highlighting the potential for new therapeutic strategies.
What is Gene Therapy?
Gene therapy is a medical strategy designed to treat or prevent disease by altering genetic material within a patient’s cells. This can involve introducing a healthy gene to compensate for a faulty one, inactivating a disease-causing gene, or introducing a new gene to fight a disease. The goal is to target the underlying genetic cause rather than just symptoms.
To deliver genetic material into cells, scientists often employ vectors, typically modified viruses. These viruses are engineered to be harmless and serve as vehicles, carrying genetic instructions into specific cells. Once delivered, the genetic material can direct the cell to produce a missing protein, block a harmful protein, or perform another therapeutic function.
Regeneron’s Distinctive Gene Therapy Methods
Regeneron employs several methods in its gene therapy research. A primary strategy involves adeno-associated virus (AAV) vectors, non-disease-causing viruses modified to deliver genetic payloads to target cells. These AAVs carry specific DNA sequences, such as a functional gene or gene editing components, into the body. Regeneron enhances AAV specificity by creating antibody-retargeted AAVs, allowing precise delivery to particular cell types or tissues beyond natural AAV targets. This minimizes delivery to unintended organs, like the liver, potentially reducing toxicity and improving efficacy.
The company also integrates CRISPR technologies into its gene therapy platforms for targeted gene insertion and editing. Combining AAV delivery with CRISPR tools aims to improve the durability of genetic medicines. Regeneron applies its proprietary VelociGene® mouse model platform to test and validate antibody targeting, ensuring precise delivery to target tissues. This allows exploration of diverse therapeutic applications by combining novel payloads, viral vectors, and antibodies. Regeneron’s extensive genetics expertise, supported by the Regeneron Genetics Center (RGC™), helps identify suitable genetic targets for these advanced therapies.
Targeted Diseases and Clinical Applications
Regeneron’s gene therapy research focuses on serious diseases with a clear genetic basis. A prominent area is hearing loss, especially rare forms caused by specific gene mutations. The company is developing DB-OTO, an AAV-based gene therapy for children with hearing loss linked to otoferlin gene mutations. This therapy involves injecting benign viruses carrying a functional OTOF gene copy directly into the cochlea, similar to cochlear implantation.
Beyond hearing loss, Regeneron is exploring gene therapy for other conditions, including neurological diseases, cardiometabolic disorders, lysosomal storage disorders, and eye diseases. For instance, they are investigating siRNA (small interfering RNA) technology to address neurological conditions such as Alzheimer’s disease, Huntington’s disease, and amyotrophic lateral sclerosis. In eye diseases like age-related macular degeneration, diabetic retinopathy, and glaucoma, Regeneron aims to restore vision by leveraging genetics-based approaches, including siRNA technologies to reduce dosing frequency. Their research also extends to musculoskeletal disorders, where gene therapy may offer solutions for inherited conditions like myasthenia gravis.
Overview of Approved Therapies and Pipeline
Regeneron’s current portfolio includes 14 approved medicines in the U.S. and other countries, primarily protein therapeutics, and does not yet include an approved gene therapy. The company has a robust pipeline with over 30 programs in clinical development, 18 in late-stage trials across approximately 60 countries. These investigational medicines reflect a broad range of therapeutic approaches, including gene therapy, gene editing, and gene silencing.
The most advanced gene therapy candidate in Regeneron’s pipeline is DB-OTO, currently in Phase 1/2 clinical trials for hearing deficit due to otoferlin gene variants. Recent CHORD trial data showed notable hearing improvements in 10 of 11 children treated with DB-OTO, with some achieving nearly normal hearing. This demonstrates promising early efficacy. Regeneron is engaged with regulatory authorities, including the FDA, which has granted DB-OTO orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations.