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

Drug Discovery World

. “This approval is a critically necessary step that paves the way to enrol patients with treatment-refractory progressive MS for whom there are no currently available treatment options in the KYSA-7 trial. CAR T-cell therapy involves modifying a patient’s T cells to recognise and remove B cells in the patient’s body.

Therapies 312
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Dr Gonzalez-Aseguinolaza recognised for gene therapy research

Drug Discovery World

Dr Gonzalez-Aseguinolaza said: “This is a tremendous honour, and I am very pleased that the RFS Society recognises my dedication and work in the field of gene therapy. She then moved into rare diseases and has been working on rare hepatic diseases for the last two decades, with a focus on the development of advanced therapies.

Therapies 130
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First engineered cell therapy for a solid tumour gets US approval

Drug Discovery World

Adaptimmune Therapeutics’ Tecelra (afamitresgene autoleucel) has become the first engineered cell therapy for a solid tumour cancer approved in the US. It is also the first new therapy option in more than a decade for synovial sarcoma, which is a rare, soft tissue cancer that most commonly impacts young adults.

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The curative potential of CAR-T cell therapy

Drug Discovery World

As we gear up for Phase I clinical trials in the first half of 2024, we’re aiming to evaluate the safety of our innovative CD5 knockout strategy enabled by our novel cell therapy and manufacturing platform. Beyond VIPER-101, we’re developing subsequent programmes for autoimmune disease and solid tumours.

Therapies 147
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Gene therapy restores vision in first-of-its-kind trial

Drug Discovery World

A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. The trial also tested the safety profile of the treatment. The gene therapy itself caused mild inflammation that was treated with steroids.

Therapies 147
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Researchers design genetic therapy to prevent brain deterioration

Drug Discovery World

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases.

Therapies 147
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Funding to advance gene therapy for cerebrotendinous xanthomatosis treatment 

Drug Discovery World

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. At present there is no cure for CTX, only treatment to slow disease progression. Vivet Therapeutics has received €4.9 CrisTauX will enable accelerated clinical development of VTX-806 upon completion of the programme.

Therapies 130