Sickle cell disease comprises a group of inherited disorders caused by mutations of...
Global Blood Therapeutics leads in world sickle cell disease trials
Sickle cell disease comprises a group of inherited disorders caused by mutations of haemoglobin subunit beta.
Protalix and Chiesi close enrolment in Phase III Fabry disease trial
Protalix BioTherapeutics and Chiesi Farmaceutici (Chiesi Group) have concluded patient enrolment in the Phase III BALANCE clinical trial of pegunigalsidase alfa (PRX 102) to treat Fabry disease.
FDA puts clinical hold on Abeona’s Phase III study of EB-101
The US Food and Drug Administration (FDA) has sent a clinical hold letter to Abeona Therapeutics rejecting a planned Phase III VIITAL clinical trial of the company’s EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Novartis pushes back regulatory filing of Rett syndrome gene therapy
Novartis completed its acquisition of AveXis in May 2018 for $8.7bn and successfully brought AveXis’ most advanced asset, Zolgensma past FDA approval on May 2019 for spinal muscular atrophy.
Spruce’s rare disease drug yields positive data in Phase IIa trial
Spruce Biosciences has reported positive data from a Phase IIa clinical trial of tildacerfont to treat patients with congenital adrenal hyperplasia (CAH).
Rocket Pharmaceuticals begins gene therapy in LAD-I study
Rocket Pharmaceuticals has started treatment with its investigational gene therapy, RP-L201, in a Phase I/II clinical trial to treat severe Leukocyte Adhesion Deficiency-I (LAD-I).
ReveraGen reports positive data for vamorolone in DMD
Santhera Pharmaceuticals has announced ReveraGen BioPharma’s report of positive results from the Phase IIa extension study of vamorolone to treat Duchenne muscular dystrophy (DMD) patients.
Fulcrum launches Phase IIb trial of losmapimod to treat FSHD
Fulcrum Therapeutics has started a Phase IIb clinical trial of losmapimod in patients suffering from facioscapulohumeral muscular dystrophy (FSHD).
ProQR to study new drug candidate for retinitis pigmentosa
ProQR Therapeutics has announced plans to conduct a Phase I/II clinical trial to evaluate QR-1123 for the treatment of patients with autosomal dominant retinitis pigmentosa (adRP).
Arrowhead initiates Phase II/III trial of RNAi therapeutic ARO-AAT
Arrowhead Pharmaceuticals has initiated a potentially pivotal Phase II/III clinical trial of ARO-AAT, its second generation subcutaneously administered RNA interference (RNAi) therapeutic.