April 2023

Jodas Expoim > News > 2023 > April

Current Scenario of Clinical Trials of Cell, Gene and RNA Therapies

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Current Scenario of Clinical Trials of Cell, Gene and RNA Therapies

Constant emergence of new gene therapies as well as refinement of the existing ones changes the global landscape of the cell and gene therapies clinical trials, where the US, China, and Europe are leading in respect of the number of trials conducted. As per Global Data, China showed 15% faster growth in cell and gene therapy clinical trials making the Asia-Pacific region contributes for one-third of the trial activities. As a result, the Asia Pacific region is witnessing 50% faster growth than the rest of the world (ROW). Asia Pacific region leads globally in terms of CAR-T cell gene therapy clinical trials for the time period 2015-2022 since China alone conducted ~60% of all CAR-T trials. Till April 2022, there are 19 approved gene therapies, 17 RNA-approved therapies while 56 non-genetically modified approved cell therapies (Figure 1). Details of the approved location of the clinical trials of gene therapies and RNA therapies drug product are provided in Table No.1 and Table No. 2 respectively, which presents a bird’s-eye view of the landscape of the clinical trials of the approved gene and RNA therapies.

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Insectome: A treasure trove of pharmaceutical compounds

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Quite often we hear people talking about microbiome disturbance leading to unhealthy aging and going back to our ancestral habits including paleo diet has the potential to cure many diseases. This folk wisdom is supported by some recent scientific publications. However, we have majorly neglected the fact that paleo diet-eating and cave-dwelling ancestors of ours had several insects on their bodies, and inside caves, they constantly fought with insects. Thus we argue, if modern habits are responsible for the current epidemic of metabolic/cardiovascular/neurological and other degenerative diseases, may be insects also had some role to play in the healthier aging of our ancestors as compared to us. In this blog post, we would like to review the benefits of insect bitings/stings published in the literature. Thus we will examine, if an apparent parasitic interaction between humans and insects is a mutualistic relationship in disguise. When an insect bites/stings us, it releases a barrage of biologically active compounds, including those with potential to act as anticoagulant/vasodilator. Can these chemicals be exploited to cure Cardio-Vascular-Diseases/dissolve internal blood clots? More importantly, there are other chemicals which have virucidal, anti-cancer and antimicrobial properties, which in either native or modified form can be repurposed for pharmaceutical applications.

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Engineered Nanoparticles for the Delivery of Anticancer Therapeutics

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Engineered Nanoparticles for the Delivery of Anticancer Therapeutics

Therapeutic agents in cancer treatment are aimed at rapidly dividing cells, limiting their multiplication, and promoting apoptosis. The lack of selectivity of these conventional methods resulted in needless damage to normal cells leading to severe adverse effects. Nanotechnology in medicine gratifies the constraint in conventional treatment by delivering conventional drugs to the targeted tissue or organ and plays an important role in targeting the delivery, thereby avoiding systemic toxicity and increasing the bioavailability and therapeutic index of the drug. The advantage of using nanoparticles as drug carriers are in their binding competence and reversing multidrug resistance. Using active and passive targeting strategies, nanoparticles enhance intracellular drug concentrations. The present review focuses the on the basic pathophysiology of cancer and the various types of nanoparticulate drug delivery systems that have been explored so far, taking advantage of the tumor vasculature and other molecular mechanisms which differentiates cancer cells from normal ones, for the delivery of anticancer therapeutics for effective management of cancer. The article also aims to focus on the various surface-engineered nanoparticles for the targeted delivery of cancer.

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Base Editing: A Promising Endeavour in Gene Therapy

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Moving ahead with our blog series we are bringing up Base Editing a new feather in the cap of gene editing therapy. Base Editing Therapy is a technology that introduces single-nucleotide variants (SNVs) precisely and efficiently at targeted genomic sequences without causing double-stranded breaks in the DNA enabling it as an efficient technique of genome editing. Nearly half of known pathogenic genetic variants are due to SNVs and base editing therapy holds enormous potential for the treatment of these genetic disorders by either temporary RNA or permanent DNA base alterations. Correction of single point mutations will be a major point of interest in the upcoming times for the scientific community for precision medicine.

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