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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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The future outlook for mRNA therapies

Drug Discovery World

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43 mRNA-based approaches can produce proteins / peptides by using protein synthesis which is processed in a transfected cell 1.

Therapies 130
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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. “As

Disease 137
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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.