Remove Hot-Topics Cell-Gene-Therapies
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HLTH 2022 – Day 2

pharmaphorum

The day will continue with other hot topics for pharma, including health equity, cell and gene therapy, oncology, and pharmacy model disruption. Then we’ll hear from Aptar Digital Health’s Sai Shankar and Pierre Laurent in a session all about patients as partners in healthcare.

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AZ, Scorpion strike deal on elusive transcription factor targets in cancer

pharmaphorum

AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases.

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Protein misfolding specialist Gain nets $40m from IPO

pharmaphorum

The company has developed a toolkit called Site-Directed Enzyme Enhancement Therapy or SEE-Tx, which uses computational techniques to find small-molecule drugs that are able to bind to sites on misfolded proteins, restore their shape and function and in some cases prevent them clumping together.

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Introducing CoP Leader Profiles: Rachel Owen

ISPE

Rachel is excited about hot topics and potential task teams being discussed within IPNA-EU CoP, including cell and gene therapy. Cell and gene therapy is moving us toward more and more personalized medicine, which isn’t a new concept, but it is becoming more of a reality.

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The Europe Hackathon Inspires Creative Solutions to Real-World Challenges

ISPE

This year the problem statement was set by our hackathon sponsor, Roche, and it focused on the hot topic of ATMPs (Advanced Therapy Medicinal Products - also known as Cell & Gene Therapy in the USA).

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CRISPR therapies targeting the next breakthrough in oncology

pharmaphorum

The potential of CRISPR technology has been a hot topic in the industry ever since it was first developed, but as trials progress further into the clinic, what therapeutic areas could be set to benefit? coli, and their research in turn formed the foundation for the CRISPR-based therapies being explored to create new treatment options.

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OTAT Becomes the Office of Therapeutic Products (OTP) and Kicks off Town Hall Series – They appear to be tired of writing the same “Complete Response” Letters

The FDA Law Blog

Lewis, Senior Regulatory Device & Biologics Expert — On September 29, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to gene therapy chemistry, manufacturing, and controls. Depending on the change (e.g.,