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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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FDA-approved drug sensitises brain cancer cells to radiotherapy

Drug Discovery World

This action sensitised GBM cells to radiotherapy both in vitro and in vivo (in mouse models). “Our research has revealed cladribine as a radiosensitiser for GBM treatment by drug repurposing, which can offer multiple advantages,” says Prof Youn. “Our GBM is a WHO grade IV brain tumour with dismal prognosis.

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Overcoming diverging regulatory expectations to bring CGTs to market

Drug Discovery World

While CGT is still an emerging market, its growth has accelerated over the last five years, resulting in a wave of products approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). And the robust CGT pipeline suggests there could be an influx of new approvals on the horizon.

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Biologics – The Next Step in Revolutionary Medication

Roots Analysis

Given the ongoing scientific advancements and the rise of FDA-approved biologics, the pharmaceutical industry seems to be approaching the era of biologics. In fact, biologics are anticipated to play a significant role in shaping the pharmaceutical industry and will eventually serve as an integral part of traditional medicines.

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What the Glycome Can Tell Us About Persistent HIV Infection

XTalks

Related: FDA Approves Rukobia for HIV Patients with Limited Treatment Options. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

RNA 52
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Life Science Trends to Look Out for in 2024

XTalks

In 2023, Dr. Varshney notes that the US Food and Drug Administration (FDA) issued a request for industry comment on the integration of AI in drug and biologics development. This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease.

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Can liquid biopsies transform precision medicine?

Drug Discovery World

Brett Swansiger, Chief Commercial Officer at ANGLE discusses the importance of liquid biopsies in precision medicine trials for cancer. As such there is a need to develop fast and accurate methods to assess drug safety and efficacy, facilitating early success or failure.