EDITAS MEDICINE INC (EDIT)

NASDAQ — Healthcare: Manufacturing, Biotechnology: Biological Products (No Disgnostic Substances)

www.editasmedicine.com

Weekly Summary (2025-05-12)

Highlights

  • In vivo proof of concept data for a potentially first-in-class treatment for an undisclosed liver target presented at ASGCT.
  • Maximal liver editing of the target gene (~70%) achieved with robust target protein upregulation (>80% reduction in disease biomarker).
  • Significant progress towards clinical development with upcoming presentations scheduled.

Catalysts

  • Oral presentation on May 21, 2025, at TIDES USA 2025 regarding the undisclosed liver target.

Outlook

Editas Medicine is making significant strides in gene editing for liver diseases, with promising data that could lead to transformative treatments.

Upcoming Events

May 23, 2025, 4:00:00 AM UTC

EDIT-101 LCA10 Phase 1/2 trial completion expected

Editas Medicine is expected to complete its Phase 1/2 clinical trial of EDIT-101 for Leber Congenital Amaurosis 10 (LCA10) on May 23, 2025. The open-label, single ascending dose study (NCT03872479) is evaluating the safety and efficacy of EDIT-101, a CRISPR-based gene editing therapy targeting the CEP290 gene mutation in up to 34 adult and pediatric participants. LCA10 is a rare inherited retinal disorder causing vision loss. Trial completion may inform future regulatory and development decisions for EDIT-101.

trial completion
Aug 1, 2025, 4:00:00 AM UTC

Expected Trial Completion for EDIT-301 Study in Severe Sickle Cell Disease

Editas Medicine's RUBY Phase 1/2 trial (NCT04853576) evaluating EDIT-301, a gene-edited autologous hematopoietic stem cell therapy for severe sickle cell disease, is expected to complete on August 1, 2025. The study assesses the safety and efficacy of a single dose of EDIT-301 in patients aged 12 to 50 with severe sickle cell disease. EDIT-301 is a CRISPR-based cell therapy designed to increase fetal hemoglobin and reduce sickling of red blood cells. The trial's outcome may impact Editas Medicine's clinical pipeline and future regulatory submissions.

trial completion
Dec 1, 2025, 5:00:00 AM UTC

EDIT-301 Phase 1/2 Trial Completion in Beta Thalassemia

Editas Medicine expects to complete its Phase 1/2 clinical trial of EDIT-301 for transfusion-dependent beta thalassemia by 1 December 2025. The study evaluates the safety, tolerability, and efficacy of a single dose of EDIT-301, a CRISPR-edited autologous hematopoietic stem cell therapy, in adults aged 18-35. EDIT-301 is being developed as a potential one-time treatment for beta thalassemia, a rare inherited blood disorder. The trial is not recruiting new participants and has an enrollment of nine.

trial completion
Dec 31, 2025, 5:00:00 AM UTC

Presentation of In Vivo HSC Preclinical Data

Editas Medicine plans to present further in vivo HSC preclinical data by year-end 2025, showcasing advancements in gene editing.

clinical readout
Aug 1, 2040, 5:00:00 AM UTC

Expected Trial Completion for EDIT-301 Long-Term Follow-Up Study

Editas Medicine is conducting a long-term follow-up study (NCT06363760) to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease or transfusion-dependent β-thalassemia who previously received the gene-editing therapy in parent trials. The study, which began on June 17, 2024, is expected to complete on August 1, 2040. EDIT-301 is an investigational CRISPR-based gene-editing therapy targeting hemoglobinopathies. The study will monitor 54 participants for long-term outcomes.

trial completion