Ranicel found effective in transfusions dependent beta thalassemia in phase 1/2 clinical trial

Renizgamglogene autogedtemcel (reni-cel), formerly known as EDIT-301 has demonstrated promising effect in the treatment of transfusion-dependent beta-thalassemia (TDT) in phase 1/2 clinical trial.

Reni-cel was well-tolerated and demonstrated an early and robust increase of total hemoglobin and fetal hemoglobin (HbF) without transfusions for up to 12.8 months in EdiTHAL trial as announced by Editas Medicine, Inc.

In the EdiTHAL trial to date, reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all patients (N=7). Following treatment with reni-cel, all EdiTHAL patients had early and robust increase of total hemoglobin (Hb) and fetal hemoglobin (HbF) and remain transfusion-free at last follow-up for a range of 4.1 to 12.8 months (N=7). All patients in the EdiTHAL trial underwent 1.0 apheresis and mobilization cycle.

“We continue to make significant progress in the development of reni-cel for transfusion-dependent beta thalassemia. In these new data at EHA, we demonstrate that all patients experienced early increases in fetal hemoglobin, maintained hemoglobin levels above the transfusion threshold and are free from red blood cell transfusions following reni-cel infusion. These data further support our belief that reni-cel has the potential to be a clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients,” said Baisong Mei, M.D., Ph.D., Chief Medical Officer, Editas Medicine. “I would like to thank the participants, their families and caregivers, clinicians, and colleagues at collaborating institutions that contribute to the EdiTHAL trial.”

“The preliminary safety and efficacy results from the EdiTHAL trial demonstrate this investigational medicine has been well-tolerated and shows promising efficacy for patients. Reni-cel has the potential to be a functional cure for people living with transfusion-dependent beta thalassemia, and we look forward to continuing to evaluate its effectiveness for these patients,” said Haydar Frangoul, M.D., M.S., Medical Director, Pediatric Hematology and Oncology, Sarah Cannon Research Institute and HCA Healthcare’s TriStar Centennial Children’s Hospital.

Efficacy of reni-cel in Patients with Transfusion-dependent Beta Thalassemia

In the EdiTHAL trial, patients demonstrated early and robust total Hb and HbF increases, with total Hb rising above the transfusion independence threshold of 9.0 g/dL. For patients with ≥6 months follow-up, the mean (standard deviation; SD) total Hb and HbF concentrations at month 6 were 12.1 g/dL (1.3 g/dL) and 10.9 g/dL (1.5 g/dL) (n=6), respectively, which were sustained at or above the transfusion threshold from 6 months through subsequent follow-ups.

All patients (N=7) have been transfusion free for a range of 4.1 to 12.8 months after receiving the last red blood cell transfusion at 0.5–2.2 months post-reni-cel infusion.

All patients in the EdiTHAL trial showed sustained high levels of editing in the HBG1 and HBG2 promoter regions.

Safety of reni-cel in Patients with Transfusion-dependent Beta Thalassemia

Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated EdiTHAL trial patients (N=7).

After reni-cel infusion, all patients (N=7) demonstrated successful neutrophil and platelet engraftment. Neutrophil engraftment occurred at a median of 23 days (min: 16 days, max: 30 days), and platelet engraftment occurred at a median of 38 days (min: 24 days, max: 49 days).

No serious adverse events (SAEs) related to reni-cel treatment in the EdiTHAL trial have been reported.

EHA Presentations

In addition to the EdiTHAL poster presentation, Editas will also present data from the RUBY clinical trial of reni-cel for the treatment of severe sickle cell disease in an oral presentation on Saturday, June 15.

EdiTHAL Poster Presentation Details:

Title: Reni-cel, the first AsCas12a gene-edited cell therapy, shows promising preliminary results in key clinical outcomes in transfusion-dependent beta thalassemia patients treated in the EdiThal trial

Presenting Author: Haydar Frangoul, M.D., M.S., Medical Director, Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy at TriStar Centennial, Nashville, TN, United States

Date/Time: Friday, June 14, 2024, 6:00 – 7:00 p.m. CEST / Noon – 1:00 p.m. EDT

Location: Hall 7, IFEMA MADRID Recinto Ferial (Fairgrounds)

Session: Poster Session

RUBY Oral Presentation Details:

Title: Reni-cel, the first AsCas12a gene-edited cell therapy, led to hemoglobin normalization and increased fetal hemoglobin in severe sickle cell disease patients in an interim analysis of the RUBY trial

Presenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s, Cleveland, OH, United States

Date/Time: Saturday, June 15, 2024, 11:30 a.m. – 12:45 p.m. CEST/ 5:30 – 6:45 a.m. EDT

Location: Hall Velasquez, IFEMA MADRID Recinto Ferial (Fairgrounds)

Session: s425 Gene therapy, cellular immunotherapy and vaccination – Clinical

The abstracts can be accessed on the EHA website and the presentations can be accessed on the Editas Medicine website in the posters and presentations section.

Reni-cel is currently being investigated in a clinical study in patients with severe sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta thalassemia (EDITHAL trial, NCT05444894). In addition to the clinical data update from the RUBY trial and the EdiTHAL trial at EHA, the Company will present a further clinical update from both trials by year-end 2024.