Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Drug Target Review

APRIL 17, 2024

HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immune responses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immune responses and facilitating antibody production.

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Drug Target Review

JUNE 6, 2023

We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. Generally, IO has been focused on harnessing the anti-tumour activity of certain cancer-fighting T-cells , a key cell type involved in the adaptive immune defense system.

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Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

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The Pharma Data

JANUARY 18, 2021

Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy. Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. gene therapy company (hereafter Neurophth), and AAVnerGene Inc. ,

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The Pharma Data

DECEMBER 23, 2020

” Dr. Stanley Plotkin , Professor Emeritus at The Wistar Institute, said, “INOVIO’s DNA vaccine appeared to be quite safe with few significant reactions but yet induced both antibody and T cell responses to SARS-CoV-2.” About INOVIO’s DNA Medicines Platform. mg and 2.0

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DrugBank

APRIL 24, 2025

Epigenetics , the study of changes in gene activity that occur without altering the DNA sequence, has revolutionized our understanding of gene expression regulation. These therapies aim to reverse abnormal epigenetic patterns, restore normal gene function, and offer hope for previously uncurable diseases.

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DrugBank

MAY 15, 2024

In the early 20th century, the discovery process was largely random , focused on cytotoxic agents that kill cells by disrupting key cellular functions like DNA replication. 5 This period also saw the rise of biologics in the 1980s, which have played a crucial role in modern therapies, particularly for diseases like cancer.

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Conversations in Drug Development Trends

JUNE 21, 2024

Antibody-Drug Conjugates: Advancing Precision Therapy ADCs represent a compelling combination of targeted antibody therapy and potent chemotherapy, aiming to enhance treatment efficacy while minimizing systemic toxicity. Currently, there are 12 approved ADC drugs, and several hundred more are in the development pipeline.

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PerkinElmer

MAY 6, 2022

Coined immunotherapy, this umbrella term describes several types of treatment, including immune checkpoint inhibitors, immune system modulators, and adoptive cell therapies. Each triggers the immune system in a different way to fight cancer.

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Drug Target Review

APRIL 3, 2024

Everything started in school with an experiment on isolating DNA from bananas. Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences.

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The Pharma Data

MAY 15, 2023

The team at XinThera has developed research assets with the potential to target the DNA damage repair pathway in treating cancer and direct the body’s immune response in inflammatory diseases, both of which may improve outcomes for people living with these diseases,” said Flavius Martin, M.D.,

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The Pharma Data

NOVEMBER 18, 2020

.” Ming-Tain Lai, PhD, Chief Scientific Officer at OBI Pharma stated, “In the trial, OBI-833 demonstrated a favorable safety profile and generated detectable anti-Globo H IgM/IgG responses. ” Presentation number: 397P / Poster: ID 680. Additional information can be found at www.obipharma.com.

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Dark Matter Blog

JULY 16, 2021

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. Along with work on IFN-β protein, which became the multiple sclerosis drug Avonex ® , my team worked extensively on adenovirus-mediated IFN-β gene therapy.

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LifeSciVC

MARCH 1, 2023

Learning from Past Failures to Drive Toward a More Durable Immune Response Following the initial clinical successes of blocking inhibitory receptors, like CTLA4 and PD1, many in the immuno-oncology field explored the potential to further enhance anti-tumor immunity by simultaneously enhancing co-stimulatory pathways.

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The Pharma Data

DECEMBER 27, 2020

NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today provided a year-end review and an outline of its plans for 2021. CARLSBAD, Calif.–( –( BUSINESS WIRE )– Lineage Cell Therapeutics, Inc.

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The Pharma Data

OCTOBER 14, 2020

(Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic.

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Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?

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Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

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Codon

FEBRUARY 13, 2024

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.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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Broad Institute

OCTOBER 11, 2023

His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.

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Drug Target Review

JANUARY 15, 2024

Unlike almost every other cell type (except B cells), T cells do not have the exact same chromosomal DNA sequences as other cells in the body. To be therapeutically useful, antigenic peptides must be presented in a way that allows immune responses to destroy cancer cells without causing unacceptable damage to healthy tissue.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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PLOS: DNA Science

OCTOBER 3, 2024

The idea to redirect an immune response against a pathogen to fight cancer goes back to 1891, when Manhattan bone surgeon William Coley became intrigued by a man’s neck tumor that melted away after he contracted a nasty Streptococcus skin infection. For many patients, BCG stops the cancer.

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Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 9 In addition, host immune responses further add to the complexity of developing cell-specific AAV capsids for clinical applications. Molecular Therapy 20 , 1831-1832 (2012).

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The Pharma Data

APRIL 8, 2021

Leadership in DNA damage response demonstrated in multiple presentations for AZD5305, a next-generation PARP1 selective inhibitor. Data for AZD5305 will demonstrate how the next wave of DNA damage response medicines can build on the success of PARP inhibitors, potentially allowing patients to stay on treatment longer.

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Drug Target Review

MAY 14, 2024

Since cancer cells are under chronic stimulation of ER stress, the polypeptides cannot activate innate immune sensors in cancer cells even upon the polypeptide treatment. Which natural agonists impede the activation of intracellular DNA sensors in APCs, and what challenges do they pose?

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Codon

JULY 27, 2023

Their cone cells, which are responsible for color vision, don’t work like normal. But what happens if you restore these cone cells, using gene therapy? They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. LEGO robot to make sucrose gradients.

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Codon

JULY 27, 2023

Their cone cells, which are responsible for color vision, don’t work like normal. But what happens if you restore these cone cells, using gene therapy? They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. LEGO robot to make sucrose gradients.

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PLOS: DNA Science

JANUARY 4, 2024

The end-of-year FDA approval of the first CRISPR-based therapy , for sickle cell disease, came a mere dozen years after Jennifer Doudna and Emmanuelle Charpentier introduced the technology. The microbes deploy them to dismantle the genetic material of infecting viruses, a little like an immune response.

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DrugBank

FEBRUARY 26, 2025

This stagnation has created a perfect storm, threatening the effectiveness of current therapies and limiting the pipeline of new drugs. Targeting Novel Bacterial Pathways Traditional antibiotics often target essential bacterial processes such as cell wall synthesis or DNA replication.

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PLOS: DNA Science

SEPTEMBER 5, 2024

This removes the cancer’s blocking of the immune response, so that T cells can fight the cancer. Keytruda’s classification based on mechanism, rather than its structure, is immune checkpoint inhibitor. Analyzing DNA patterns using AI may replace time-consuming process DNA and genomic sequencing tests, Yu said.

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Codon

APRIL 23, 2023

Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain. These ribonucleoproteins also caused a lower immune response compared to the viruses, and are much easier to make in the lab. Nature Communications. Nature Communications. Twitter thread.

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Codon

APRIL 23, 2023

Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain. These ribonucleoproteins also caused a lower immune response compared to the viruses, and are much easier to make in the lab. Nature Communications. Nature Communications. Twitter thread.

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Codon

APRIL 30, 2023

Yeast die for two reasons: Either their nucleolus (where the DNA is kept) degrades and dies, or their mitochondria whimpers out and they stop making energy. The vaccine printer can make lots of different types of vaccines, including protein, DNA, and mRNA ones, but I’m sure this is all quite expensive right now. From Zhang et al.

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