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By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.
My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0
Because these proteins have such a profound effect on cells, there is typically a second hurdle of regulation that needs to be overcome to make the proteins. eFFECTOR Therapeutics has developed a novel class of cancer drugs known as Selective Translation Regulator Inhibitors (STRIs), which directly target the eIF4F complex.
In recent years, genetic editing technologies and other gene therapy approaches have given scientists the ability to alter the genes inside living cells. This limits our ability to design gene therapies that only effect certain cell types in the human body." Tags: Genome regulation Machine Learning Pardis Sabeti
The researchers found that copy number is influenced by the nucleus and pinpointed 92 locations in the nuclear genome that regulate copy number, many of which have been linked to rare genetic disorders related to mitochondrial DNA maintenance. This is contrary to previous studies, which did not account for these factors.
A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. The authors suggest natural tracr-L regulates autoimmunity. And removing tracr-L altogether greatly increased CRISPR-Cas9 expression.
Regarding therapy resistant melanomas, why is the alteration of the epigenetic landscape considered significant, and what has been lacking in terms of validated targetable epigenetic mechanisms? What is remarkable here is that we show that the addition of corin to BRAFi-resistant tumour cells resensitises them to BRAFi therapies.
Zhang’s arrest was for “the suspected offense of smuggling goods prohibited by the import and export regulations under the laws of the PRC,” according to a GenScript statement. Human genetic goods are tightly regulated by the Chinese government. Cilta-cel snagged Breakthrough Therapy Designation from the U.S.
But what happens if you restore these cone cells, using gene therapy? ” Wellcome Collection , London I wrapped up my series on “30 Days of Great Biology Papers.” ” This was a series of tweets in which I told brief stories behind seminal papers, mostly in molecularbiology and biophysics.
But what happens if you restore these cone cells, using gene therapy? ” Wellcome Collection , London I wrapped up my series on “30 Days of Great Biology Papers.” ” This was a series of tweets in which I told brief stories behind seminal papers, mostly in molecularbiology and biophysics.
SRP-001 targets the central nervous system (CNS) by producing N-arachidonoylaminophenol (AM404) in the midbrain’s periaqueductal grey (PAG) region, crucial for pain sensation and regulation. Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies?
Biomarkers are an essential advancement that will help determine risk for developing Alzheimer’s disease, diagnose the disease before symptoms have progressed significantly, predict how quickly patients may experience cognitive decline and evaluate how well new therapies are working. Progress in molecularbiology and translational science.
The only subject in school that held my interest was biology. As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. I wanted to use molecularbiology to create drugs. I did a postdoc with Paul Berg at Stanford, studying transcriptional regulation. Arrakis is the capstone of my career.
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chPD1 will be used in the Company’s proprietary chimeric antigen receptor therapy (CAR-T) platform using gamma-delta T-cells (GD-T).
Barber’s work will give Kiromic a significant acceleration in the clinical development of its therapy platform and an even more significant advantage over its competitors.
In biology , scientific research on cell metabolism focuses on different topics such as understanding the fundamental metabolic processes, metabolic changes and their role in diseases (including cancer cell and tumor metabolism), the development of therapies, the identification of unknown metabolic processes and metabolites, etc.
Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. Nevertheless, phage therapy did have a stint in the U.S. I’ll post an update soon. coli in mice.
Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. Nevertheless, phage therapy did have a stint in the U.S. I’ll post an update soon. coli in mice.
Even after a half-century of molecularbiology research, scientists didn’t know until recently how gas vesicles physically trap gas while occluding water. For more than a century, scientists thought of gas vesicles as little more than a natural curiosity and, later, as a way for microbes to regulate buoyancy.
The History of the ICH Q5A(R1) Guideline The International Council on Harmonization (ICH) Q5A(R1) Guideline, Viral Safety Evaluation of Biotechnology Products Derived from Cell Lines of Human or Animal Origin , was first adopted by regulators in 1999. cell line type ) and the final product type. The Specific Precautions section (6.2.7)
Their capacity to provide an unlimited supply of cells means that iPSCs could revolutionise cell therapies. Together, they shared valuable insights into the progress of iPSC-based therapies, the key obstacles ahead and the exciting opportunities for future breakthroughs.
As a result, patients receiving ESC-derived therapies must also take immunosuppressive drugs potentially for life. The process can yield a renewable stockpile of cells for everything from CAR-T cancer therapies to tissue and organ transplants. This can trigger an immune response that causes the body to reject these therapeutic cells.
Link Gene Expression & Regulation →Genetic regulatory mechanisms in the synthesis of proteins , by Jacob F. & Journal of MolecularBiology (1961). Gene Expression and Regulation. Nature Reviews Molecular Cell Biology (2008). Link Molecular Cloning Techniques , by AddGene. & Xie X.S.
Link Gene Expression & Regulation →Genetic regulatory mechanisms in the synthesis of proteins , by Jacob F. & Journal of MolecularBiology (1961). Gene Expression and Regulation. Nature Reviews Molecular Cell Biology (2008). Link Molecular Cloning Techniques , by AddGene. & Xie X.S.
I’ve chosen these two because I think they are the linchpin by which we’ll be able to build broadly useful AI models for cell and molecularbiology. But looking ahead to the second bottleneck, what does it mean to say that biology is “complex”? It involves pragmatism and cultural sensitivity.
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