Tagged: cell therapy

How we can help The BBCR team provides knowledge in cell, biologics and gene therapies BBCR addresses sponsors’ questions in the ever-changing regulatory environment We offer clinical strategy for orphan diseases and precision medicine RWE can be used to build orphan diseases evidence to support regulatory decisions BBCR serves small and medium sized Biotech Companies, […]

Duchenne Muscular Dystrophy is the most common fatal genetic disorder to affect children around the world. The first patient, a six year old boy, received, in November 2021, an infusion of DT-DEC01, a novel cell therapy containing Dystrophin Expressing Chimeric Cells (DEC),  in Phase I pilot clinical study for the treatment of Duchenne muscular dystrophy (DMD). […]

Progress and limitation of therapeutic approaches targeting DMD By: Dr. Maria Niu Duchenne muscular dystrophy (DMD) is an X—a chromosome-linked recessive disorder caused by mutations in the gene coding muscle cytoskeletal protein dystrophin. Because of the X-linked inheritance, nearly 1 in 3,500 males are affected worldwide, while females occasionally develop symptoms. There is no curative […]

Emerging cell and gene therapy may offer sustained long-term correction for LSD patients Dr. Maria Niu Lysosomal storage diseases (LSDs) are rare inherited metabolic diseases and characterized by the accumulation of substrates in excess in various organs’ cells due to lysosomes’ defective functioning. The combined incidence of LSDs is between 1 in 5000 to 1 […]