Posts by: Joana Carvalho, PhD

Joana Carvalho, PhD

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

August 27, 2020August 27, 2020

European Commission Grants Orphan Drug Status to Imara’s IMR-687 for Sickle Cell Disease

News

The European Commission has granted orphan drug designation to IMR-687, Imara’s potential new therapy for sickle cell disease (SCD). In the European Union, this designation is given to investigative ... Read more

August 25, 2020August 25, 2020

EDIT-301, Experimental Cell Therapy for SCD, Earns Rare Pediatric Disease Designation

News

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, a potential cell therapy for the treatment of sickle cell disease (SCD). This designation is ... Read more

August 18, 2020August 18, 2020

First Sickle Cell Patient Dosed in Phase 2b Trial of Imara’s IMR-687

News

A Phase 2b clinical trial that is investigating the safety and effectiveness of IMR-687 in people with sickle cell disease (SCD) has dosed its first participant, according to the potential ... Read more

July 16, 2020July 16, 2020

Umbilical Cord Blood Transplant Treats Children With Genetic Disorders, Trial Finds

News

An umbilical cord blood transplant, coupled with a reduced intensity conditioning (RIC) regimen, can safely and effectively treat children with genetic disorders that include sickle cell disease (SCD) and thalassemia, data from ... Read more

June 25, 2020June 25, 2020

Mitapivat Safely Increases Hemoglobin Levels in SCD Patients, Phase 1 Trial Shows

News

Mitapivat (AG-348) safely increased the amount of hemoglobin and changed the levels of biomarkers of red blood damage and energy production in adults with sickle cell disease (SCD), early ... Read more

June 23, 2020September 24, 2020

First SCD Patient Treated With CTX001 Remains VOCs-free After 9 Months, Trial Results Show

News

The first patient with severe sickle cell disease (SCD) who received a single infusion of CTX001, an experimental gene-editing cell therapy, remains free of vaso-occlusive crises (VOCs) nine months ... Read more

June 4, 2020June 4, 2020

Sickle Cell Society Survey Looks to Better Understand Pandemic’s Affect

News

The Sickle Cell Society of the U.K. has created a survey to gather feedback from people with sickle cell disease (SCD) and their caregivers during the COVID-19 pandemic. Overall, the society aims ... Read more

June 2, 2020June 2, 2020

IMR-687 Safely Increases Fetal Hemoglobin Levels in SCD Patients, Phase 2a Trial Shows

News

IMR-687 safely increases the levels of fetal hemoglobin and the number of fetal hemoglobin-producing cells in people with sickle cell disease (SCD), according to early data from an ongoing ... Read more

May 14, 2020May 14, 2020

Agios to Decide Next Steps for Mitapivat as SCD Treatment by Mid-year

News

Agios Pharmaceuticals announced that, despite the current COVID-19 outbreak, it remains on track to decide by mid-year next steps for mitapivat (AG-348) as a potential treatment of sickle cell disease. Mitapivat is in an ongoing ... Read more

May 12, 2020May 12, 2020

Gene-editing Therapy CTX001 Granted RMAT Status by FDA for Severe SCD

News

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to CTX001, an investigational gene-editing cell therapy, for the treatment of severe sickle cell disease ... Read more