Ensuring Our Gene Therapy is Accessible
Developing investigational gene therapies is time consuming and expensive. Correcting a gene is one of the most complicated and specialized fields in medicine.
As a result, few companies and even fewer capital sources are willing to focus their efforts on developing cures like APR-2020. Access to novel gene therapies, particularly for rare pediatric diseases, is often hindered by both availability and expense.
Given their low incidence rates, locating a specialized physician with the necessary expertise and experience with the disease can be challenging. When available, these experts are rarely located near the patient who may have resource constraints or be too sick to travel.
Apriligen is committed to supporting rare pediatric disease research and the development of gene therapies using lentiviral vectors with the goal of ensuring patients who need access to cutting-edge medicines for rare, genetic illnesses, such as APR-2020, can receive treatment.
More Cures, More Birthdays
Apriligen’s goal is to have APR-2020’s success serve as a first step to serially develop and release gene therapies by leveraging APR-2020’s technology platform (i.e. the vector/plasmid construct and gene sequencing expertise), regulatory pathway and the aggregated safety and efficacy data.
We believe that our novel approach to autologous cellular gene therapy is scalable and has many significant advantages over donor matched cell transplants, including removing the need for immunosuppression and reducing the amount of toxic, pre-treatment chemotherapy administered.
Given the slow development of novel treatments and orphan drugs for rare pediatric diseases and the potential benefits from our autologous cellular gene therapy platform, we see the potential for APR-2020-style therapy to treat, and potentially cure, many conditions caused by faulty or non-functioning genes.
DBAS is considered an ultra-rare pediatric and orphan disease. APR-2020, Apriligen’s lead therapeutic candidate has been granted Orphan Drug, Rare Pediatric Disease, Fast Track and Rare Disease Evidence Principles (RDEP) designations by the US Food and Drug Administration (FDA) and Orphan Drug Designation by European Medicines Agency (EMA).