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In this article, Dr Alaa Hamed, Global Head of Medical Affairs for Rare Disease at Sanofi, shares why the company’s enzyme replacement therapy Xenpozyme® (olipudase alfa) has promise for the rare genetic disease acid sphingomyelinase deficiency (ASMD).
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended several new medicines, including positive opinions for treatments for rare disorders.
Expanding on his earlier podcast discussion with EPR, Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen, discusses the company’s promising modifier gene therapy candidates for ophthalmic disorders.
Acquisition of Forge Biologics by Ajinomoto Co., Inc. is set to extend global capabilities in AAV and plasmid gene therapy manufacturing for Ajinomoto, Co. Inc.
The Committee for Medicinal Products for Human Use (CHMP) recommended two new medicines for approval in its May meeting, including a neurosteroid for epileptic seizures.
Lentiviral gene therapy reduces infections and bleeding episodes in children with a severe immune disorder Wiskott–Aldrich syndrome (WAS), finds new study.
John Liddell, Chief Technologist at the Centre for Process Innovation (CPI), writes about the challenges associated with gene therapy products, which constitute a major portion of the overall cell and gene therapy market.
ProQR and Lilly announced a collaboration to combat genetic disorders in the liver and nervous system using Axiomer® RNA editing platform.
The global medical marijuana market will show impressive growth, a new report has said, but this could be hindered due to some doctors being reluctant to prescribe these products.
