siRNA

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SiSaf’s small interfering RNA (siRNA) therapeutic for skeletal disorder Autosomal Dominant Osteopetrosis Type 2 (ADO2). In addition, due to the serious manifestations of this rare disease in children, SIS-101-ADO has been granted Rare Pediatric Disease Designation for the treatment of Autosomal Dominant […]

Aldena Therapeutics, a biotech company that is pioneering siRNA-based therapies for dermatological indications, has updated its immuno-dermatology pipeline, appointed a Scientific Advisory Board (SAB), and announced $30 million financing by Medicxi.  Pipeline  Under the leadership of Thibaud Portal, CEO of Aldena, the company’s goal is to develop advanced, long-lasting treatments for dermatological diseases based on […]

Biopharmaceutical company VBI Vaccines has revealed interim data from its Phase II study evaluating the combination of VBI-2601, VBI’s HBV immunotherapeutic candidate, and BRII-835, an HBV-targeting siRNA candidate, in chronically infected hepatitis B (HBV) patients.   The data demonstrate that the combination therapy was generally well-tolerated, restored strong anti-HBsAg antibody responses, and led to improved HBsAg-specific […]

The overall potential of siRNA-based therapeutics has attracted Chinese pharma companies to tap opportunities and bring promising innovative therapies across the globe, according to reporting by GlobalData. Against this backdrop, the Chinese pharma companies have increased their focus towards the siRNA pipeline through strategic alliances.  China pharma companies have executed four deals with siRNA players […]

Michael Smyth is Medical Director at Galapagos UK, responsible for launching innovative therapies in the UK to help change the course of inflammatory disease for patients. Having studied medicine at Liverpool University, one of his personal missions is lifelong learning which is reflected in his role to strive for new treatment options that lead to better […]