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This week in drug discovery (13-16 May) 

Drug Discovery World

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields. News round-up for 13-16 May by DDW Senior Digital Content Editor Diana Spencer.

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Conversations from ESMO Targeted Anticancer Therapies Congress 2024 

Drug Discovery World

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. In vivo, METTL1 inhibitors induce tumour growth inhibition in both immune-deficient and immune-competent mouse strains. We were there to present the latest findings from our research.

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AACR: A focal point of the cancer research community

Drug Discovery World

To support this goal, Heidelberg Pharma presented safety and first efficacy data from the Phase I/IIa clinical trial with lead ATAC product candidate HDP-101 as well as preclinical data on other drug candidates from the company’s proprietary antibody-drug conjugate (ADC) technology platforms.

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Spotlighting Lupus Awareness Month: CAR-T Technology Creates New Avenues for Treatment of a Devastating Disease

WCG Clinical

The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.

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Multispecific antibody shows promise in B cell lymphoma

Drug Discovery World

A multispecific antibody candidate for B cell lymphoma preferentially engaged CD47 in presence of co-engagement of CD20 and CD19, in preclinical investigations. One dose of Nutcracker Therapeutics’ NTX-472 in vivo rapidly depleted B cells with no detectable binding to red blood cells.

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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Emerging as a new treatment option in rare and orphan disease areas, oligonucleotide therapeutics have matured into a drug class with a broad indication spectrum. Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake.

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The challenges and trends of cell & gene therapies 

Drug Discovery World

There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. Among these include Zolgensma, Novartis’ gene therapy drug for spinal muscular atrophy (SMA), which made headlines in the UK for its £1.79 billion, compared to $19.9 million price point per dose. Cancer is king .