Fabry disease is a rare X-linked lysosomal storage disorder where a deficiency in α-galactosidase A (GLA) results in the pathological accumulation of globotriaosylceramide (Gb3 or GL-3) and other glycosphingolipids in vascular endothelial cells, nerve cells, cardiomyocytes and renal cells.
Andera Partners closed its Biodiscovery 6 fund at €456 million (US$442 million). The venture capital firm will maintain its custom of allocating about two-thirds of the fund to biotechnology and one-third to medical devices and medical technology. The fund just edges past its target of €450 million. It is considerably larger than its immediate predecessor, Biodiscovery 5, which raised €345 million.
The FDA has placed Logicbio Therapeutics Inc.’s phase I/II clinical trial of LB-001, an investigational AAV genome-editing therapy for treating pediatric patients with methylmalonic acidemia (MMA), on a clinical hold. So far, four patients have been dosed in the study and two have had serious adverse events related to the candidate, the company’s lead asset.
HONG KONG – Canbridge Pharmaceuticals Inc. signed a collaboration and licensing agreement that could be worth $591 million, gaining global rights to develop, manufacture and commercialize gene therapy candidates from Logicbio Therapeutics Inc. for the treatment of Fabry and Pompe diseases. The candidates are based on Logicbio’s adeno-associated virus (AAV) sL65, the first produced from its Saavy capsid development platform.
HONG KONG – Canbridge Pharmaceuticals Inc. signed a collaboration and licensing agreement that could be worth $581 million, gaining global rights to develop, manufacture and commercialize gene therapy candidates from Logicbio Therapeutics Inc. for the treatment of Fabry and Pompe diseases. The candidates are based on Logicbio’s adeno-associated virus (AAV) sL65, the first produced from its Saavy capsid development platform.
Pediatric gene editing specialist Logicbio Therapeutics Inc. has revealed an FDA clinical hold on a planned phase I/II trial of its lead candidate, LB-001, an investigational therapy for rare inherited metabolic disorder methylmalonic acidemia (MMA).
