Biotech Companies continue to show resurgence even in these challenging times, with more and more companies persevering with bringing their clinical candidates to fruition. While Vaccine development and therapeutics to combat the COVID-19 pandemic are at the forefront of development activities in the biotech sector,...
Abeona Therapeutics (NASDAQ: ABEO), a clinical-stage biopharmaceutical Company pioneering cell and gene-based therapies for rare genetic disease, announced positive interim data from two ongoing phase I/II clinical trials evaluating ABO-102 and ABO-101, indicated for the treatment of MPS IIIA and MPS IIIB. The study being...
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...
Neuronal Ceroid Lipofuscinosis (NCL), or more commonly known as Batten disease, is a group of rare lysosomal storage disorders attributed to the dysfunction in the autosomal recessive gene, which causes mutations that hinder efficient cell functioning. They disrupt the cell’s natural waste disposal mechanism leading...
