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CAR-T therapy could “alter the treatment paradigm” of multiple sclerosis

Drug Discovery World

CAR T-cell therapy involves modifying a patient’s T cells to recognise and remove B cells in the patient’s body. KYV-101, specifically targets CD19, a protein expressed on the surface of B cells, which is involved in various types of autoimmune diseases.

Therapies 312
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FDA approves first gene therapy for Duchenne muscular dystrophy

Drug Discovery World

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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How does circVec technology compare to conventional mRNA expression? 

Drug Discovery World

As a result, the data provides an important proof-of-concept for Circio’s circular RNA platform, which we expect will translate into improved gene therapies for patients in the future,” said Dr Thomas Hansen, CTO at Circio. “In design, which increases protein expression by 2-4 -fold vs circVec 2.1

RNA 147
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Enhancing gene therapy with Circio

Drug Target Review

Secondly, circRNAs can be engineered for more efficient protein expression by carefully selecting and optimising the IRES element, a sequence motif derived from viruses and used to initiate cap-independent translation from circRNA. There are three major reasons for why Circio has selected AATD as its lead gene therapy program.

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Novel CAR T-cell vein-to-vein process could reduce time and cost

Drug Discovery World

A paper in BioRxiv has described a new process that could improve the chimeric antigen receptor (CAR) T-cell patient experience and reduce costs through a minimisation of the CAR T-cell therapy vein-to-vein process. Leukapheresis – a specific type of apheresis – is a conventional starting material for CAR T-cell therapy.

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Curcumin activates the JNK signaling pathway to promote ferroptosis in colon cancer cells

Chemical Biology and Drug Design

Curcumin modulates the gene and protein expression levels of ferroptosis mediators via JNK signaling. Ferroptosis, a new form of regulated cell death, plays a vital role in the pathogenesis and therapy of cancers.

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Companies partner to identify biomarkers for renal cell carcinoma

Drug Discovery World

Immune-checkpoint inhibitor (ICI) therapy has transformed treatment outcomes for RCC. OncoHost and Dana-Farber Cancer Institute have launched a new study to identify biomarkers for renal cell carcinoma (RCC). However, ICIs can also trigger immune-related adverse events and may not benefit all patients.