Genetic diseases affect 25 million Americans and countless more across the globe. Because genetic medicine is still in its adolescence, the space contains vast unmet needs and many grievous conditions. These diseases are also often targetable via precision medicine ñ one can characterize the source of the disease, define the mechanism, and drug the molecular pathophysiology at its source.
BridgeBio Pharma is dedicated to finding and efficiently developing novel, genetically targeted therapies. Led by experienced scientists, drug developers and investors, BridgeBio combines deep research and development expertise with a corporate model that efficiently distributes central resources across multiple subsidiary companies, each of which develops a specific drug. The company has a portfolio of 17 targeted genetic drugs that run from preclinical to late-stage trials with the underlying premise that a more efficient drug development ecosystem is in patients' best interests. These programs span multiple therapeutic areas including oncology, dermatology, mitochondrial disease, and cardiology. The fruits of this model are apparent in the work being done at subsidiary Eidos Therapeutics for patients with Transthyretin (TTR) Amyloidosis.
TTR Amyloidosis is a rare, devastating disease without an FDA-approved therapy which presents in two forms - a cardiomyopathy leading to progressive heart failure, and a polyneuropathy resulting in degeneration of sensory and motor function. Current treatment options for each focus upon symptom management and do not address the underlying source or mechanism of disease.
TTR amyloidosis is brought on by the genetically-driven destabilization of a common blood borne tetramer (TTR), resulting in its disassociation into monomers, which then aggregate into amyloid fibrils and deposit in tissues where they cause disease. Eidos Therapeutics is developing a drug candidate, AG10, to treat this amyloidosis by stabilizing the TTR tetramer in the blood, halting the disease's progression. AG10, conceived to mimic a saving mutation seen in nature that stabilizes TTR, has shown promising efficacy thus far, and will enter Phase II trials this spring.
Neil Kumar, Ph.D., is the chief executive officer of Eidos Therapeutics and its parent company, BridgeBio Pharma. Prior to founding BridgeBio, he was a principal at Third Rock Ventures, a healthcare venture firm, where he supported and managed various portfolio companies in addition to focusing on new company formation and due diligence. He also held the role of vice president, business development and operations for MyoKardia, a precision medicine company working on targeted therapies for the treatment of rare cardiovascular diseases. Before joining Third Rock, Dr. Kumar served as an associate principal at McKinsey & Company, where he developed strategies for pharmaceutical and medical device companies and helped lead McKinsey's personalized medicine efforts. Previously, Dr. Kumar was involved in the formation of a gene chip startup and was a technical consultant for AstraZeneca's pathway signaling group.
Dr. Kumar is the author of several peer-reviewed papers in the fields of oncology and systems biology. He holds B.S. and M.S. degrees in chemical engineering from Stanford University and received his Ph.D. in chemical engineering from the Massachusetts Institute of Technology.