Editas test CRISPR to treat beta-thalassemia & sickle cell disease
Category: #health  | By Nikita Chaurasia  | Date: 2019-06-17 |
  • shareshare_icon
  • Twitter
  • Facebook
  • LinkedIn

Editas test CRISPR to treat beta-thalassemia & sickle cell disease

Editas Medicine Inc., a U.S.-based pharmaceutical company recently announced results from trial of two different CRISPR-Cas9 genome editing strategies, one targeting the beta-globin locus and the other targeting the erythroid enhancer, for treatment of beta-thalassemia & sickle cell disease. The data was presented by the company at the 24th Congress of the European Hematology Association in Amsterdam, report sources.

For the record, Editas Medicine Inc., is a leading genome editing pharmaceutical company, that focus on potential of CRISPR-Cas9 & Cas12a genome editing strategies in treatment of patients with serious diseases. The company aims to develop, manufacture & commercialize durable, transformative precision genomic medicines for wide-ranging class of diseases.

Charles Albright, Chief Scientific Officer, Editas Medicine, was reported to say that the pre-clinical results validating cells edited at the beta-globin locus repopulated all lineages of the blood system including RBC precursors & the high percentage of fetal hemoglobin expression, showed potential to treat beta-thalassemia & sick cell disease. Editing at the site continues to meet the company’s pre-clinical goals for making the medicine for durable induction of fetal hemoglobin & maintaining normal hematopoietic stem cell function, Albright added.

In this study, NBSGW mice (supports engraftment of human hematopoietic stem cells without irradiation) were reportedly given an infusion of human CD34+ cells which were edited either at beta-globin locus or the BCL11Ae. In vivo-derived erythroid cells from BCL11Ae and edited CD34+ hematopoietic stem cells had increased non-productive indels and reduced total indels, as compared to other lineages that have been tested (a phenomenon not observed with beta-globin locus editing). Also, further optimization of guide RNA & nuclease combinations led to fetal hemoglobin expression of approximately 40% in the beta-globin, locus-edited erythroid cells.

Based on the data, the pharmaceutical company has initiated IND-enabling activities for an experimental CRISPR medicine, EDIT-301. The drug is designed to durably treat beta-thalassemia & sickle cell disease by editing the beta-globin locus, cite sources.

Source credits: http://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-presents-pre-clinical-data-treatment-sickle-cell

  • shareshare_icon
  • Twitter
  • Facebook
  • LinkedIn


About Author

Nikita Chaurasia     twitter

Nikita Chaurasia

Having always been daft at wordplay, Nikita Chaurasia, post the completion of post-graduation, commenced her journey into the content generation cosmos. Endowed with a professional MBA degree in Advertising and Public Relations, Nikita strives to integrate her creativ...

Read More..

More News By Nikita Chaurasia

Shell trades off Russian retail business to native oil producer Lukoil

Shell trades off Russian retail business to native oil producer Lukoil

By Nikita Chaurasia

Energy giant Shell plans on selling more than 400 petrol stations of its Russian subsidiary Shell Neft to Lukoil, the second largest oil producer in the country after state backed Rosneft. As several Western oil and gas firms seek to get rid of th...

Mitsubishi, Siemens sign an MoU to cooperate in SiC traction technology

Mitsubishi, Siemens sign an MoU to cooperate in SiC traction technology

By Nikita Chaurasia

Mitsubishi Electric Europe and Siemens Mobility have recently signed a memorandum of understanding (MoU) to work together in silicon carbide (SiC) traction power technology. As per reports, JR Central’s next-generation N700S Shinkansen train...

Legato opens 80 new jobs in its Limerick Research & Development hub

Legato opens 80 new jobs in its Limerick Research & Development hub

By Nikita Chaurasia

The global capability center of a leading U.S. health care insurance company, Legato Health Technologies is planning to build an additional 80 posts at its Limerick facility. It would bring the total number of staff to 200, which the fir...