Author Archives: Patricia Inacio PhD

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

Note: This story was updated June 16, 2021, to clarify that the HGB-207 and HGB-212 clinical trials were testing betibeglogene autotemcel, a gene therapy that uses the same viral vector as LentiGlobin.  A patient diagnosed initially with the blood cancer myelodysplastic syndrome (MDS) after receiving LentiGlobin, Bluebird Bio’s investigational…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Adding Cell Source’s special class of immune cell, Veto T-cells, to blood stem cells from an unrelated donor led to a successful transplant and to the production of normal red blood cells in a mouse model of sickle cell disease (SCD), the company announced. The preclinical Cell Source-sponsored…

A recent manufacturing agreement between Aruvant Sciences and Lonza will advance the development of ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD). Under the agreement, Lonza will be responsible for producing Aruvant’s gene therapy for an upcoming pivotal clinical trial. The company already has…

A clinical trial will assess a combination of MGTA-145 plus plerixafor as a new regimen for aiding in the collection of blood stem cells from adults and adolescents with sickle cell disease (SCD). The proof-of-concept trial, a collaboration between Magenta Therapeutics and Bluebird Bio, may establish this…

Treatment with Oxbryta (voxelotor) may be a feasible alternative to blood transfusions for sickle cell disease (SCD) patients who are experiencing respiratory distress and pneumonia related to COVID-19, a case report suggests. These findings are particularly important during the ongoing pandemic in the U.S., in which rising blood…

FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering a Phase 1 clinical trial in healthy volunteers, the company announced. The trial (NCT04586985), not yet enrolling, aims to recruit 88 adults at its single Altasciences Clinical Kansas site. The…

Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high unmet…