Doha, April 22 (QNA) - Chief Medical Officer of Sidra Medicine, Prof. Ibrahim Janahi, said offering advanced gene-editing therapy for sickle cell disease and thalassemia reflects Sidra Medicine's global standing in science and research.

In a statement to Qatar News Agency (QNA) Wednesday, he said that Sidra Medicine's delivery of this type of complex treatment undoubtedly underscores the global confidence it enjoys in providing cutting-edge, specialized, and revolutionary therapies.

Sidra Medicine had announced yesterday the provision of the first advanced gene-editing treatment for sickle cell disease and thalassemia for the first time in Qatar, following the center's accreditation by Vertex Pharmaceuticals as one of the few hospitals worldwide authorized to administer Casgevy, a groundbreaking one-time gene therapy based on CRISPR/Cas9 gene-editing technology.

Prof. Janahi noted that the treatment is now available at Sidra Medicine for patients aged 12 and above suffering from transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD).

He explained that Sidra Medicine is the only hospital in Qatar authorized to provide this type of treatment, alongside a limited number of centers globally and regionally where thalassemia and sickle cell disease are prevalent.

Prof. Janahi pointed out that the new treatment is among the most expensive therapies, with a treatment period ranging from six to eight months and sometimes extending to a full year, particularly as this type of therapy falls under gene therapy aimed at correcting the genetic mutation in patients with sickle cell disease and thalassemia.

For his part, Chief Research Officer at Sidra Medicine Prof. Khaled Fakhro said that Casgevy represents a global breakthrough and that its availability at Sidra underscores the hospital's pioneering role in precision medicine driven by research and development.

He added to Qatar News Agency (QNA) that making this treatment available in the State of Qatar aligns with Sidra Medicine's vision to be among the world's leaders in precision medicine based on the concept of treating causes rather than symptoms, through reliance on genomics as a fundamental science in understanding disease sequencing and identifying causative mutations.

He pointed out that Sidra Medicine places strong emphasis on treating rare diseases and offering hope to patients who have long suffered from such complex conditions.

Casgevy is a one-time gene therapy that addresses the root cause of both severe sickle cell disease and transfusion-dependent beta thalassemia.

The treatment can lead to sustained reductions in pain crises among patients with severe sickle cell disease and reduce reliance on blood transfusions for patients with beta thalassemia.

The treatment process is complex, and access to this type of therapy remains limited, currently restricted to a small number of specialized centers worldwide, including Sidra Medicine.

Sidra Medicine has entered into a partnership with Vertex, which is working on studying the use of gene therapies targeting the root causes of serious hemoglobin disorders, including severe sickle cell disease and transfusion-dependent beta thalassemia.

Notably, the Advanced Cell Therapy Center at Sidra Medicine houses a Good Manufacturing Practice facility responsible for processing hematopoietic stem cells and cryopreserving them.

The center also supports gene therapy readiness, including treatments such as Casgevy for transfusion-dependent thalassemia and severe sickle cell disease.

In a related context, QNA reviewed, during a tour of Sidra Medicine's facilities today, the extensive capabilities and advanced technologies of the Advanced Cell Therapy Center laboratory and its specific role in gene therapy, in addition to its role in the Casgevy treatment protocol.

The tour also covered Qatar's cord blood storage facility at Sidra Medicine, the country's only in-state bank, offering GMP-accredited services with global access, supporting treatment of over 80 diseases. (QNA)