At Rallybio, we are building a portfolio of product candidates that target devastating rare disorders with significant morbidity and mortality. Each product candidate must also meet the following criteria:
Devastating rare disorder with significant morbidity and mortality
Potential for transformative clinical and functional impact
Clear understanding of disease pathophysiology
Targets a therapeutic area outside of oncology and infectious disease
Engineered proteins, antibodies, small molecules
Disease with significant unmet need that impact >3 patients per million
Late discovery to early clinical stage
Our passion is bringing life-transforming therapies to patients suffering from devastating and rare disorders. It is estimated that 25 to 30 million Americans are affected by rare disorders, as are 6 – 8 percent of the European population, according to the European Organization for Rare Diseases (EURORDIS). A rare disorder, also referred to as an orphan disease, is defined as a condition that affects fewer than 200,000 people in the United States, or 1 person per 2,000 population in Europe. There may be as many as 7,000 distinct rare disorders.
Unfortunately, significant unmet need exists for the vast majority of patients suffering from these rare disorders. While researchers have made considerable progress in recent years in figuring out ways to diagnose, treat and even prevent a variety of rare disorders, much more remains to be done.
Our passion, along with our collective track record of success in developing and delivering transformative therapies for patients with rare disorders, including diagnosing and defining diseases, designing of novel endpoints, and developing innovative clinical development programs, will enable us to identify and accelerate the development of life-transforming treatment options for patients suffering from these horrible rare and ultra-rare disorders.