After decades of research and investment, revolutionary treatments for rare diseases are increasing every year, with more promising therapies on the horizon. As scientific understanding of biomarkers and disease activity increases – along with advancements in AI – bold new approaches have become viable for advancing drug discovery and development.

One company, BERG, a Boston-based biotech, has embraced an industry-disrupting approach that harnesses the power of AI, coupled with patient biology, to accelerate clinical identification and pursuit of promising therapeutic targets to treat disease.

Back to Biology™

BERG’s proprietary Interrogative Biology® platform is the engine that drives the company’s discovery and innovation. By taking a biology-driven approach to mapping disease, researchers at BERG can decode and compare vast biological samples to identify and leverage biomarkers as a window into disease – eliminating much of the ‘guess and check’ approach from drug discovery and development.

“We're convinced that patient biology is the key to unlocking insights that will catalyze drug discovery for some of our most challenging and debilitating diseases,” said Niven R. Narain, Ph.D., Co-Founder, President & Chief Executive Officer of BERG. “Through the identification of predictive and prognostic biomarkers of disease activity, we can more effectively personalize medicine, while advancing development programs faster and more cost-effectively.”

The Pain Is Real

Despite recent advancements, many rare conditions still lack approved treatment options. One such condition, Epidermolysis Bullosa (EB), often referred to as the ‘worst disease you’ve never heard of,’ is a rare, pervasive and debilitating connective tissue disorder for which there is currently no FDA-approved treatment or cure.

“As an advocate and father of a child with EB, I’m excited to be working in partnership with BERG to bring hope to the 25,000 patients and nearly 200 children born each year with this terrible disease,” said Brett Kopelan, Executive Director of debra of America, the only U.S. nonprofit providing all-inclusive support to the EB community.

Encouraging News

During his tenure as Director of Cutaneous Oncology Research at the University of Miami’s Miller School of Medicine, Dr. Narain identified that coenzyme Q10 (CoQ10), or ubidecarenone, could be utilized in a range of dermatological and cancer-fighting applications – including as a potential topical wound treatment for patients suffering from EB.

Now one of BERG’s leading product candidates, BPM 31510 (ubidecarenone) has received two orphan drug designations in 2018 from the U.S. Food and Drug Administration (FDA) for the treatment of EB and pancreatic cancer, respectively.

“We’re very encouraged by the results of our ongoing research of BPM 31510 and are committed to delivering breakthrough therapies to make a difference in the lives of these patients,” explained Dr. Narain.

For BERG, EB is just the first target, with future studies being planned for expanded research into other rare diseases, including Ewing’s Sarcoma and mitochondrial diseases.