Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The three-year collaboration will leverage Orna’s proprietary lipid nanoparticle (LNP) delivery system. LNPs are tiny vesicles made up of fatty molecules that can be used…
News
The Bill and Melinda Gates Foundation is investing up to $50 million in Tessera Therapeutics to support the development of a new gene-editing medicine for sickle cell disease (SCD), the company said. “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for…
A newborn screening program in Catalonia improved outcomes for children with sickle cell disease (SCD), with patients being diagnosed earlier and having lower rates of major disease complications. “The data presented justify screening in countries where it has not yet been implemented, as it reduces patient morbidity and…
The European Commission has granted orphan medicinal product status to mitapivat, Agios Pharmaceuticals’ oral investigational therapy for sickle cell disease (SCD). This designation is granted to treatments for rare diseases, or those affecting fewer than five in 10,000 people in the European Union, that have the potential…
Nearly all the patients with severe sickle cell disease (SCD) who were treated with renizgamglogene autogedtemcel (reni-cel) remained free of vaso-occlusive events (VOEs) for up to two years, new data from the Phase 1/2/3 RUBYÂ clinical trial shows. The treatment also increased total hemoglobin levels and was well tolerated,…
Etavopivat, Novo Nordisk’s investigational oral therapy for sickle cell disease (SCD), may reduce the incidence of vaso-occlusive crises (VOCs) and increase hemoglobin levels. That’s according to data from the phase 2 part of the Phase 2/3 HIBISCUS trial (NCT04624659), which is assessing the treatment’s safety and…
Most people with sickle cell disease (SCD) who received the gene-editing therapy Casgevy (exagamglogene autotemcel) in clinical trials have been free from vaso-occlusive crises (VOCs) over several years of follow-up, according to new data. Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings…
The vast majority of people with sickle cell disease (SCD) who received the now-approved gene therapy Lyfgenia (lovotibeglogene autotemcel) in clinical trials have been free from vaso-occlusive events — simply called VOEs — in the years following treatment. That’s according to new data that Lyfgenia’s developer Bluebird…
Kanglin Biotechnology has raised $20 million in financing to advance the development of KL003, an experimental gene therapy for sickle cell disease (SCD) and beta thalassemia. The China-based company plans to launch a pivotal Phase 2 trial next year to test KL003 in people with beta thalassemia, a…
Two centers in California are enrolling nine people with severe sickle cell disease (SCD) for a clinical trial of a new gene editing therapy called CRISPR_SCD001. The Phase 1/2 trial (NCT04774536) is open to adolescents and adults, ages 12 through 35, who in the two years before enrollment…
Recent Posts
- New reimbursement deal in Germany expands access to Casgevy for SCD
- New York study finds regional gaps in sickle cell disease hospitalizations
- When a sickle cell pain crisis interrupts a stretch of good health, part 1
- Scientists discover how stressed red blood cells make hemoglobin
- How my sickle cell disease advocacy has evolved over time
- New study links sickle cell anemia to lower ovarian reserve in women
- $3M Breakthrough Prize honors duo whose work changed SCD treatment
- Etavopivat Phase 3 results support potential approval in sickle cell disease
- New diagnoses result in new grief and a new relationship with my body
- Study finds lower treatment use among SCD patients in sub-Saharan Africa