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March 2025

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October 2021 Issue, Drug Discovery News

October 2021

Sean McClain

EHEC grown on an agar plate.

Scientists combined engineered <em>E. coli</em> with artificial intelligence to produce effective, high-quality protein and antibody-based drugs at a large scale.

E. coli may hold the key to manufacturing hard-to-make drugs

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Alessio Fasano leads the international GEMMA study with the hope of understanding how genetics, the environment, the microbiome, and the metabolome affect autism.

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Sarkis Mazmanian studies the influence of the gut microbiome on the brain.

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two individuals in lab coats are working in a lab 

Genome Environment Microbiome and Metabolome in Autism (GEMMA) 

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Scientists leverage the gut microbiome-brain connection to develop new treatments for autism spectrum disorders.

Targeting gut bacteria to treat autism

Veterinarian with syringe ready to give vaccine shot to pigs at cattle farm

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A group of people are standing together in a classroom. An individual wearing an orange shirt is holding a certificate.

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Scientists continue to ring alarm bells about the risks associated with the continued misuse of antimicrobials and advocate for innovative treatments, improved surveillance, and greater public health education. 

Antimicrobial resistance: The silent pandemic

Microbiology

Flies are good models of the nervous system, as demonstrated in larva. Red higlights  neural stem cell nuclei and green labels cells that derive from neuroblasts.

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ACOX1-driven neurodegeneration is one of the diseases that Bellen’s team has modeled in flies to find drug treatments. In a fly without ACOX1, the immune system attacks the fly’s own tissue. 

Researchers use fruit flies to figure out how genetic mutations disrupt biology and how drugs might reverse these effects.

Genetic studies of rare undiagnosed diseases take flight with model organisms

A person with brown hair is wearing glasses and a blazer. They are smiling at the camera.

Courtney Young

Disabled boy in wheelchair holding mother's hand on path

An engineer turned biology researcher co-founded a bio-pharma start-up with the goal of using CRISPR to reverse the mutation causing the degenerative muscle disorder Duchenne muscular dystrophy.

CRISPR-based muscular dystrophy therapy

A drawing of a person with a healthy arm muscle is shown on the left. An arrow points to a drawing of a person with a dystrophied muscle in a circle on the right.

Dystrophied muscle

CRISPR editing for treating Duchenne muscular dystrophy

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Researchers are using CRISPR gene editing to restore function of the mutant protein that causes Duchenne muscular dystrophy, a genetic disease that causes muscle degeneration.

Infographic: CRISPR editing for treating Duchenne muscular dystrophy

Rare Diseases

Cellarity uses machine learning algorithms to digitize all of the gene expression changes occurring in single cells as the cells transition from healthy to diseased, allowing them to identify the cellular behaviors that drive disease.

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A person wearing glasses in a blue shirt is smiling

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Big Data futuristic background

Machine learning paired with single-cell analysis helps scientists identify pathways that cause disease and potential therapeutics.

Decoding cell behavior and disease with AI and single-cell transcriptomics

Shape Therapeutic’s guide RNAs (gRNA) direct a cell’s own RNA editing enzymes, ADARs, to swap adenosines implicated in disease to guanosines.

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A DNA molecule is shown in blue with one edited piece of a strand in red.

Genetic engineering

A person with red hair is wearing a white shirt and blue blazer and is smiling at the camera

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By redirecting cells&rsquo; endogenous RNA editing machinery, scientists can correct disease-causing mutations, bringing a new kind of gene therapy to the clinic.

Harnessing cells&#39; own RNA editing tools for new therapeutics&nbsp;

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Oisin Biotechnologies’ gene therapy involves two components: a proteolipid delivery vehicle that relies on a viral peptide to bind any cell, and a DNA logic gate that uses p16 promoters to control expression of pro-apoptotic caspase-9

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Some of the recent and ongoing clinical trials targeting cell death pathways for indications range from oncology to neurodegeneration, autoimmunity to ophthalmology, and beyond.

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The scalable time-lapse analysis of cell death kinetics (STACK) screening platform monitors the overall ratio of living (red) and dead (green) cells and provides insights on the real-time kinetics of cell death in response to test compounds. 

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Researchers redefine cell death in disease and drug discovery.

Special report: Live and let die

Cell Biology

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Black swan

How can scientists bridge the communication gap between themselves and the public? 

The black swan

Editors Insight

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Thermo Fisher Scientific

Next-generation natural refrigerant cooling system for centrifuges, offering a lower Global Warming Potential and reduced energy consumption.

Thermo Scientific&trade; Centrifuges with GreenCool Technology

Video

Clear sample tubes are shown in a clear tote with red lids in a sample prep robot with a blue and silver industrial lab background.

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Discover how better sample preparation can unlock improved assay accuracy and analytical results.

The crucial role of sample preparation in biotherapy manufacturing

Webinars

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Embrient

Malaria continues to drive urgent research worldwide, with new therapies and tools emerging to combat the parasite&rsquo;s complex lifecycle and global burden.

October 2021 Volume 17, Issue 10