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New mass spectrometry Chapter could “revolutionise” biopharma quality assurance
Launch of the USP general method chapter signals a breakthrough in pharmaceutical drug impurity control, according to biopharma experts.
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Genetic disorders
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Enzyme replacement therapy: improving outcomes in rare disease
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).
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CHMP meeting highlights – June 2024
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.
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Modifier gene therapy – clinical development and manufacturing considerations
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.
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New acquisition to support global gene therapy manufacturing
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.
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CHMP meeting highlights – May 2023
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.
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Gene therapy shows promise in Wiskott–Aldrich syndrome
Lentiviral gene therapy reduces infections and bleeding episodes in children with a severe immune disorder Wiskott–Aldrich syndrome (WAS), finds new study.
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Challenges in downstream purification of advanced therapies
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.
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ProQR and Lilly collaborate to develop RNA therapeutic
ProQR and Lilly announced a collaboration to combat genetic disorders in the liver and nervous system using Axiomer® RNA editing platform.
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Report predicts the medical marijuana market will reach $3.2bn by 2025
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.
