YolTech doses first subject in Phase I trial for YOLT-201 therapy
China-based YolTech Therapeutics has dosed the first patient as part of its Phase I clinical trial for gene editing therapy…
China-based YolTech Therapeutics has dosed the first patient as part of its Phase I clinical trial for gene editing therapy…
Spur Therapeutics has announced that its gene therapy candidate for Gaucher disease will soon be moving to a Phase III…
CSL Behring has announced the treatment of two patients with HEMGENIX (etranacogene dezaparvovec) gene therapy for haemophilia B, an inherited…
US-based therapeutics company Unravel Biosciences has partnered with the Pitt Hopkins Research Foundation to carry out a proof-of-concept trial of…
A man who set up a rare disease biotech in a bid to cure his son’s disease will soon be…
Shanghai-based Refreshgene Therapeutics has announced that ten of the 11 patients dosed in a trial of its otoferlin (OTOF) gene…
Atsena Therapeutics has announced positive preliminary data for its gene therapy candidate ATSN-201 for the treatment of X-linked retinoschisis (XLRS).…
Prime Medicine has been granted approval by the US Food and Drug Association (FDA) to initiate trials of the first-ever…
The US National Institutes of Health (NIH) has reported that etidronate, an existing drug typically used to treat bone diseases,…
Vertex Pharmaceuticals has confirmed the progression of inaxaplin (VX-147) into the Phase III portion of the international Phase II/III pivotal…