Pharmaceutical Technology on MSN
Potentially “groundbreaking” FDA gene therapy pathway leaves key issues unresolved
A new FDA pathway could speed bespoke gene therapies, but key questions over scope and commercial viability remain.
Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...
Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...
Despite high awareness, biosimilars remain on the periphery—particularly in the first-line setting. While many physicians are open to their use in principle, uptake remains modest, constrained in part ...
Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...
Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
Ocugen, Inc. (NASDAQ: OCGN) will present its latest advances in gene therapy for blindness diseases at NobleCon21, the annual ...
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. Roche described the restructuring in its ...
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