6 days ago · Clinical trial data on pociredir as SCD therapy expected by midyear. Fulcrum Therapeutics is planning to report, by midyear, new data from the ongoing PIONEER Phase 1b clinical trial that’s testing its oral therapy candidate pociredir (FTX-6058) in people with sickle cell disease (SCD), according to a new corporate presentation.

SCD patients free of VOEs after treatment with gene-editing therapy. 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 RUBY clinical trial shows.

Jan 4, 2022 · Sickle cell disease (SCD) is an inherited disorder caused by a mutation in the gene encoding a subunit of hemoglobin, the protein that carries oxygen in red blood cells. Mutant hemoglobin — called sickle hemoglobin or hemoglobin S — tends to clump together in red blood cells, causing them to take on an irregular “sickle-like” shape that gives the disease its name.

1 day ago · Symptoms of SCD include anemia, fatigue, swelling of the extremities, recurrent infections, and vision problems. Sickled red blood cells can also block blood flow, especially in small blood vessels, leading to painful vaso-occlusive crises and other serious complications.. Stem cell transplants are available, and have the potential to cure SCD. But such treatment carries significant risks and ...

Jan 9, 2025 · “We look forward to advancing a genetic medicine that can potentially reshape the treatment landscape for sickle cell disease worldwide,” Michael Severino, MD, Tessera’s CEO, said in a company press release.. SCD is caused by mutations in a gene that provides instructions for making hemoglobin, the protein that red blood cells use to carry oxygen throughout the body.

Dec 12, 2024 · Vertex Pharmaceuticals, which developed Casgevy alongside CRISPR Therapeutics, shared the findings at the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 7-10 in San Diego and virtually. The presentation was titled “ Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease.” Data specifically showed that, among 42 patients ...

Jan 8, 2025 · Complications vary from person to person. While numerous potential complications are associated with sickle cell disease, it’s important to note that not every patient will experience the same issues. Just because one patient suffers a stroke, for example, doesn’t mean others will face the same fate, even if they have identical genotypes.. …

Dec 29, 2022 · CTX001 is a gene-editing therapy that aims to boost the production of HbF in blood cells. Its developers CRISPR Therapeutics and Vertex announced the launch of a Phase 3 trial called VX21-CTX001-151 (NCT05329649) to test the therapy in children ages 2 to 11 with severe SCD who either don’t respond to hydroxyurea or are intolerant to it. A similar trial, called VX21-CTX001-141 (NCT05356195 ...

Sickle Cell Disease News is a digital platform intended to provide the sickle cell disease (SCD) community with the most recent news and information on SCD, as well as first-hand community perspectives from our patient and caregiver columnists.. All articles on Sickle Cell Disease News are original content produced by in-house writers and editors. . …

Nov 18, 2024 · The extensive research, funding, and other resources required for gene therapy to be approved and for a patient to complete treatment can’t be understated.It also takes extreme courage and patience to be the first at anything in medicine. I commend Cromer and his family for taking this leap. Hopefully a long line of cured patients will follow.