Clinical Stage Therapeutic Area
Laber Congenital AmaurosisSickle Cell Disease
Laber Congenital Amaurosis 10
Leber congenital amaurosis is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. People with this disorder typically have severe visual impairment beginning in infancy.Gene Defect: Mutation in the CEP290 gene lead to degeneration in ocular photoreceptor cells that are critical for vision. The disease is inherited in an autosomal dominant pattern.
Treatment Approach: EDIT-101 is an investigational therapy (in vivo) designed to remove the CEP290 mutation and correct the disorder.
Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disease have atypical hemoglobin molecules called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape.
Gene Defect:
Mutations in the HBB gene cause sickle cell disease that codes for the beta-chain of the hemoglobin. The disease is inherited in an autosomal recessive manner.EDIT-301 is an ex-vivo investigational gene therapy that utilizes CRISPR to edit the beta-globin locus and directly increase fetal hemoglobin. In autologous hematopoietic stem cells (CD 34+ cells) from the patients are isolated, are used for editing the HBB gene, and infused back to the patients
Clinical Stage: IND (End of 2020)
Therapeutic Platform: CRISPR gene editing with 2 distinct CRISPR nucleases: Cas9 & Cas12a with AAV based delivery system
Overview of Pipeline
*Refer to the company website for a detailed portfolio