More than two years after its approval, only about 60 patients across the U.S., Middle East, and Europe have been treated with the gene-editing therapy. Specialists at four sickle centers told STAT ...

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

Two Pee Dee siblings are officially cured of sickle cell disease after more than a decade of living with the genetic blood ...

At the weekend in Kampala, a piece of news landed that could reshape the future of one of the world’s most neglected diseases. The United States Patent and Trademark Office (USPTO) had accepted a ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

New Patient Voice Dynamix™ research highlights that even with patients’ high awareness of options and active engagement in their care, systemic barriers and personal limitations from ongoing severe ...

Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting ...

The approved CRISPR-Cas9 gene therapy involves isolating hematopoietic stem cells (HSCs) from SCD patients and using CRISPR-Cas9 gene editing to reactivate fetal hemoglobin (HbF) genes that are ...

Scribe Therapeutics Co-Founder, President and CEO Benjamin L. Oakes, PhD A growing landscape for sickle cell disease (SCD) treatments, a growing relationship between the companies, and the potential ...

Drive adoption of QUELIMMUNE for ultra-rare pediatric AKI, expanding revenue and doubling the customer base Advance SeaStar Medical’s first-in-class SCD therapy for the adult AKI indication – a ...

Beam Therapeutics’ experimental gene-editing therapy for sickle cell disease now has its first cut of patient data showing signs of treating the rare blood disorder in a way that could differentiate ...