RUBY Study

A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects with Severe Sickle Cell Disease (RUBY Trial)

Study Purpose

The purpose of this study is evaluating the safety and effectiveness of a single intravenous (IV) infusion of a new investigational therapy (EDIT-301) for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. The study will look to evaluate the safety, efficacy and tolerability of treatment with EDIT-301 in reducing and/or resolving the number of severe Vaso Occlusive Events (VOEs). A VOEs, you may have also heard them referred to as a pain crisis, is defined as occurring when the flow of blood is blocked to an area because the sickle-shaped cells have become stuck in the blood vessel. This blockage can cause severe pain or damage to the affected tissue. Pain can occur anywhere but most often occurs in the bones of the arms, joints, legs, chest, and spine. Painful VOE’s remain the most common reason for presenting to the Emergency Department and hospitalization in patients with SCD.

EDIT-301 is a new investigational therapy which uses the patients’ own stem cells, modifies them by using CRISPR technology, and infuses them back into the patient to treat SCD. Investigational means that the study medicine being tested has not been approved by the United States Food and Drug Administration (FDA), European Medicines Agency (EMA), or any other regulatory authority. This is a First-In-Human research study, which means that the study medicine has not been used in people before.

Who Can Participate

The study is planned to involve 40 adult patients, aged 18 to 50 years, of which approximately 2 adults will be enrolled at University Hospitals, who have severe SCD and who are eligible for HSCT, but who do not have an available human leukocyte antigens (HLA)-matched related donor. Further eligibility will be evaluated by the doctor in charge of the study.

• STUDY20211538
• NCT04853576