admin
The Basics of Hemophilia A
Hemophilia A is a disorder that makes a person predisposed to bleeding. This can cause issues ranging from prolonged bleeding from a minor wound to …
Gene therapy shows promise against bleeding events in hemophilia
Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers …
ASC Therapeutics Appoints Gene and Cell Therapy Manufacturing Veteran Gary Potter to lead Global Operations
Experienced in manufacturing of next-generation hemophilia gene therapies at uniQure Experienced in transformational cell culture manufacturing Consolidates global leadership team of gene and cell therapy industry veterans September 27, 2021 08:00 PM Eastern Daylight Time MILPITAS, Calif.–(BUSINESS WIRE)–ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and … Read more
We must unlock the full potential of cell and gene therapies
In July, I was delighted to chair a Policy Connect roundtable on cell and gene therapies, to consider how they can contribute to better patient care, …
ASC Therapeutics and Professor Steven W. Pipe Publish
Expert Review on Novel Gene Therapies of Hemophilia A
Second-generation gene therapies can increase protein synthesis and secretion Minimizing cellular stress can increase durability of liver-targeted gene therapies A peer-reviewed, open-access, comprehensive seminal review MILPITAS, Calif.–(BUSINESS WIRE)–ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic, metabolic and other rare diseases … Read more
Hemophilia A gene therapy: current and next-generation approaches
Next-generation gene therapies incorporating chimeric DNA sequences in the transgene can increase clotting factor synthesis and secretion, and advance the efficacy, safety, and durability of gene replacement therapy for hemophilia A as well as other blood clotting disorders.
Clinical applications of gene and cell therapies: case studies for the relevance of precision medicine
With the rapid growth of gene and cell therapies, biotechnology and pharmaceutical companies face a call to action: we must establish proper selection and monitoring protocols to provide patients with the safest and most effective therapeutic options for genetic diseases. This article presents two clinical programs for gene and allogeneic cell therapies and provides a primer for the relevance of precision medicine applications.