Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…
Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The…
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The…
Therapeutic Candidate or Device Haploidentical (half-match) T cell depleted blood stem cell transplant with a low-toxic conditioning regimen Indication Adult patients with severe sickle cell disease who are excluded from…
Therapeutic Candidate or Device forward-oriented globin-expressing vector CD34+ HSCs Indication Severe Sickle Cell Disease (SCD) Therapeutic Mechanism Successful gene modified HSC generate RBC with effective repaired hemoglobin in 30% of…
Therapeutic Candidate or Device Blood stem cells collected from individuals with sickle cell disease will have the sickle gene corrected and then given back to the same individual. Indication Sickle…
Therapeutic Candidate or Device Autologous blood stem cells edited to correct the sickle cell disease mutation to be given back to the patient as an autologous stem cell transplant Indication…
Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to “sickle” under conditions of low oxygen, occurs with a frequency…
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