These mice exhibit a severe bleeding tendency and a leukocyte adhesion defect reminiscent to the phenotype in LAD-III patients. Kindlin-3 belongs to a family of three cytoplasmic proteins ...

Conditions include chronic granulomatous disease and leukocyte adhesion deficiency syndrome ... and then delivered to the patient via an IV or subcutaneous injection weekly, biweekly, or monthly.

Patients in the EU with Fanconi anaemia ... from the FDA on Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency-I (LAD-I), its lead product, which is due by 30th June ...

The company is developing its gene therapy candidate, Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder.

The company’s solutions include ex vivo lentiviral vector treatments for Fanconi anemia, leukocyte adhesion deficiency-I ... Rocket also finished enrolling patients in its Phase 1 trial for ...