Will 2020 see the debut of promising gene therapy for hemophilia A? It’s up to the FDA.

| January 8, 2020

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The clotting disorder hemophilia A may become the third gene therapy that the US Food and Drug Administration approves, joining treatments for a form of retinal blindness in 2017, and spinal muscular atrophy in 2019. Biomarin Pharmaceutical Inc. has submitted a biologics license application to FDA and documentation of clinical trial results to the European Medicines Agency, with reviews slated to begin early this year at both organizations.

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AbbVie

At AbbVie, we believe the world needs new approaches to addressing today’s health issues — from life-threatening illness to chronic conditions. We combine advanced science, expertise and passion to solve serious health issues and have a remarkable impact on people’s lives. With ~29,000 employees and medicines in over 175 countries, AbbVie is uniting the best of pharma and the boldness of biotech, to innovate end-to-end approaches that make a real difference in people’s lives.

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MEDICAL

Better Purification and Recovery in Bioprocessing

Article | August 2, 2021

In the downstream portion of any bioprocess, one must pick through the dross before one can seize the gold the biotherapeutic that the bioprocess was always meant to generate. Unfortunately, the dross is both voluminous and various. And the biotherapeutic gold, unlike real gold, is corruptible. That is, it can suffer structural damage and activity loss. When discarding the dross and collecting the gold, bioprocessors must be efficient and gentle. They must, to the extent possible, eliminate contaminants and organic debris while ensuring that biotherapeutics avoid aggregation-inducing stresses and retain their integrity during purification and recovery. Anything less compromises purity and reduces yield. To purify and recover biotherapeutics efficiently and gently, bioprocessors must avail themselves of the most appropriate tools and techniques. Here, we talk with several experts about which tools and techniques can help bioprocessors overcome persistent challenges. Some of these experts also touch on new approaches that can help bioprocessors address emerging challenges.

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DIAGNOSTICS

Making Predictions by Digitizing Bioprocessing

Article | April 20, 2021

With advances in data analytics and machine learning, the move from descriptive and diagnostic analytics to predictive and prescriptive analytics and controls—allowing us to better forecast and understand what will happen and thus optimize process outcomes—is not only feasible but inevitable, according to Bonnie Shum, principal engineer, pharma technical innovation, technology & manufacturing sciences and technology at Genentech. “Well-trained artificial intelligence systems can help drive better decision making and how data is analyzed from drug discovery to process development and to manufacturing processes,” she says. Those advances, though, only really matter when they improve the lives of patients. That’s exactly what Shum expects. “The convergence of digital transformation and operational/processing changes will be critical for the facilities of the future and meeting the needs of our patients,” she continues. “Digital solutions may one day provide fully automated bioprocessing, eliminating manual intervention and enabling us to anticipate potential process deviations to prevent process failures, leading to real-time release and thus faster access for patients.” To turn Bioprocessing 4.0 into a production line for precision healthcare, real-time release and quickly manufacturing personalized medicines will be critical. Adding digitization and advanced analytics wherever possible will drive those improvements. In fact, many of these improvements, especially moving from descriptive to predictive bioprocessing, depend on more digitization.

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Spotlight

AbbVie

At AbbVie, we believe the world needs new approaches to addressing today’s health issues — from life-threatening illness to chronic conditions. We combine advanced science, expertise and passion to solve serious health issues and have a remarkable impact on people’s lives. With ~29,000 employees and medicines in over 175 countries, AbbVie is uniting the best of pharma and the boldness of biotech, to innovate end-to-end approaches that make a real difference in people’s lives.

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