Genetic Disorders

University of Cambridge trialling deferiprone for brain disorder

The University of Cambridge in the UK has begun a clinical trial to investigate the potential of deferiprone for treating…

Sionna raises $182m to double down in cystic fibrosis drug development

Sionna Therapeutics has continued raising capital, closing a $182m Series C financing round to help advance additional cystic fibrosis candidates…

iECURE’s gene therapy trial for OTC deficiency receives MHRA approval

iECURE has received the Medicines & Healthcare Products Regulatory Agency (MHRA) approval for its clinical trial authorisation application (CTA) to…

Pasithea begins recruiting patients for neurofibromatosis therapy trial

Pasithea Therapeutics has started enrolling patients in a Phase I trial investigating its candidate for the treatment of neurofibromatosis type…

ReCode doses first subject in Phase I primary ciliary dyskinesia trial

ReCode Therapeutics has dosed the first subject in a Phase I clinical trial of its inhaled messenger ribonucleic acid (mRNA)-based…

BioLineRx doses first subject with motixafortide in sickle cell disease trial

BioLineRx has dosed the first subject in the Phase I clinical trial of its CXCR4 inhibitor, motixafortide, for CD34+ hematopoietic…

Immusoft doses first subject in Phase I mucopolysaccharidosis type I trial

Immusoft has dosed the first subject with its investigational engineered B cell therapy, ISP-001, in a human Phase I clinical…

Australian TGA grants clearance for iECURE’s trial of ECUR-506

The Australian Therapeutic Goods Administration (TGA) has granted clearance for iECURE’s clinical trial notification (CTN) to commence a Phase I/II clinical…

Palvella and Ligand partner for Phase III rare lymphatic tumour trial

Palvella Therapeutics has partnered with Ligand Pharmaceuticals for the Phase III clinical trial investigating topical Qtorin rapamycin for the treatment…

Aro Biotherapeutics enrols first subject in Phase I Pompe disease trial

Aro Biotherapeutics has enrolled the first subject in its Phase I clinical trial of ABX1100 to treat Pompe disease.  ​​Launched…

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