The U.S. Food and Drug Administration placed a clinical hold on the investigational new drug (IND) application for CTX001, an experimental therapy for the treatment of sickle cell disease, according to a statement by CRISPR Therapeutics and Vertex Pharmaceuticals. The hold on the clinical development of CTX001 will…
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As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
Loss of an important part of the antioxidant cell defense system decreases production of hemoglobin in the blood and worsens spleen damage and inflammation in a mouse model of sickle cell disease (SCD), researchers found. Findings were reported in the study, “Loss of Nrf2 function exacerbates…
A significant proportion of children with sickle cell disease (SCD) are not receiving key recommended vaccines on time or at all, according to a new study examining pneumococcal and meningococcal vaccinations among children in Michigan. The study, “Pneumococcal and Meningococcal Vaccination among Michigan Children with Sickle Cell Disease,”…
Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
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Mutation Associated with Sickle Cell Disease Corrected in Patient’s Stem Cells, MaxCyte Reports
Global biotechnology company MaxCyte announced the successful correction of a genetic mutation associated with sickle cell disease (SCD) in a patient’s hematopoietic stem cells (HSCs), the type of cells that give rise to blood cells. The mutation was corrected in the hemoglobin gene using MaxCyte’s non-viral cell engineering…
Sickle cell trait (SCT) is not associated with an increased incidence of stroke, a study in African-American patients suggests. The study, “Association of Sickle Cell Trait With Ischemic Stroke Among African Americans: A Meta-analysis,” was published in the journal JAMA Neurology. Sickle cell trait occurs in…
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…
The U.S. Food and Drug Administration recently accepted an investigational new drug (IND) application submitted by Bioverativ and Sangamo Therapeutics for BIVV003, a gene-edited cell therapy candidate for the treatment of sickle cell disease. BIVV003 uses a nonviral approach that uses zinc finger nuclease (ZFN) gene-editing technology to…
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