At Rallybio, we apply four pillars for transforming devastating diseases. As we look to evaluate assets for development, we seek to license assets where we are confident that (1) the biology of the asset is well understood, (2) the asset will impact the pathology, (3) the asset is capable of addressing the clinical symptoms, and (4) the asset can ultimately translate into a drug that delivers transformative quality of life and/or patient survival.
Our small team of determined mentally-agile team members will continue to search for and assess assets using the following robust clinical and commercial filters that will allow us to identify and acquire a portfolio of high-quality small molecule and protein-based assets in the late discovery to early clinical stages of drug development.
Biological and Pathological Assessment
- Clear biological or genetic rationale
- Devastating disease with transformative potential
- Antibody, protein, or small molecule
Pre-clinical and Clinical DVPMT Assessment
- Tractable pre-clinical pathway
- Potential for early POC path for rapid pivot to registration
Registration and Business Assessment
- Epidemiology over 3 per million – ultra rare to orphan
- Commercially viable
- Possibility of Orphan exclusivity
It is estimated that rare diseases affect an estimated 25 to 30 million Americans and the European Organization for Rare Diseases (EURORDIS) estimates that six to eight percent of the population of the European Union is affected by one. A rare disease, also sometimes called an orphan disease, is generally considered to be a disease that affects fewer than 200,000 people in the United States at any given time. While EURORDIS estimates that there are as many as 5,000 to 7,000 distinct rare diseases, the National Human Genome Institute claims that there are more than 6,800 rare diseases.
Unfortunately, only about 400 rare diseases have therapies and about eighty percent have a genetic component according to Rare Genomics Institute. This unmet need has not been addressed for the vast majority of patients suffering from these rare diseases. While researchers have made considerable progress in recent years in figuring out ways to diagnose, treat and even prevent a variety of rare diseases, much more remains to be done because there are no treatments for the vast majority of rare diseases.
Large and mid-size research companies are moving out of or deprioritizing their efforts in ultra-rare and rare diseases for a variety of reasons, but primarily on the concern that patient access to and reimbursement for these medicines won’t be maintained. The cost to develop a medicine for a rare disease is often the same as a non-rare disease and the risks impacting the return on investment for the research efforts absent access and reimbursement are not insignificant.
These factors create an opportunity to discover and develop new therapies for small and mid-size companies that can create significant value due to their relative size. Our approach is to leverage the ability to support big pharma growth aspirations with an infrastructure cost base that minimizes the risk to the return on investment.
Our collective track record of success, extensive knowledge of bioinformatics, high-quality science capabilities, and most importantly our passion, will enable us to discover and develop drugs for patients suffering from these horrible rare and ultra-rare diseases.