gene therapy

The hospital’s team modified the patient’s hematopoietic stem cells to correct the underlying genetic defect, then reinfused the corrected cells back into the body.

The acquisition centers on anitocabtagene autoleucel, known as anito-cel, an investigational CAR-T cell therapy targeting multiple myeloma.

The acquisition gives Lilly access to Orna’s innovative platform, which combines circular RNA (circRNA) with lipid nanoparticle-based therapies.

Once the transaction successfully closes, the combined company plans to operate under the PrimeGen US name or through a successor entity.

Eli Lilly will obtain exclusive global rights to advance these programmed recombinases through preclinical studies, clinical development, and eventual commercialization.

This cutting-edge approach represents a major advancement in how doctors can treat genetic diseases by directly modifying the faulty genes responsible for these conditions.

Fakeeh University Hospital became the first facility in the region to administer Duvyzat (givinostat), an innovative therapy used to manage Duchenne muscular dystrophy (DMD).

Waskyra is specifically indicated for pediatric patients aged six months and older, as well as adults diagnosed with WAS who have a mutation in the WAS gene.

The therapy delivers the full-length human frataxin gene using a dual-administration approach that combines direct intradentate nucleus (IDN) injection with intravenous (IV) infusion.