Drug Target Review

SEPTEMBER 28, 2023

After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. These are synthetic, not the live virus, so do not pose a risk of infection.

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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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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Broad Institute

DECEMBER 13, 2023

They transferred 101 serial sections, spanning the volume of a single mouse brain, onto arrays of beads covered in unique DNA barcodes, which bound to the mRNA transcripts in the brain tissue. In their study, the researchers focused on a variant of an engineered recombinant adeno-associated virus (rAAV) known as PHP.eB.

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PLOS: DNA Science

FEBRUARY 8, 2024

CRISPR may make sense for replacing a single mutant gene, like the one behind sickle cell disease, for which a CRISPR-based gene therapy was recently approved. DNA Science recently covered perfumes resurrected from extinct plants, but using older and less precise recombinant DNA technology. appeared first on DNA Science.

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addgene Blog

MAY 30, 2023

Adeno-associated virus (AAV) is a single stranded, Parvoviridae DNA virus, packaged in a non-enveloped icosahedral capsid, that can be used to express genes of interest in cell and animal models. This post was contributed by guest blogger Didem Goz Ayturk with edits and updates from Addgenies Karen Guerin and Susanna Stroik.

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NIH Director's Blog: Drug Discovery

MARCH 8, 2016

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain. PRISM consists of two key components.

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DrugBank

MARCH 12, 2025

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. This approach can be personalized for each patient by sequencing the tumor's DNA and RNA to identify unique mutations.

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PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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Codon

JUNE 11, 2024

And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.

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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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Broad Institute

MAY 14, 2025

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells. Like Cas9, IscB enzymes cut DNA at sites specified by an RNA guide.

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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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Drug Target Review

APRIL 17, 2024

His lab applies approaches from computational biology and machine learning to unravel the molecular determinants of response to immune checkpoint blockade therapy, understand the co-evolution of tumour cells and the human immune system during cancer evolution, and learn the complex rules that govern T cell epitope recognition and immune escape.He

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The Pharma Data

JANUARY 27, 2021

. – Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. “We

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The Pharma Data

OCTOBER 21, 2020

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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Codon

JULY 10, 2025

The event hosts will provide snacks and drinks, and the winning teams will get signed copies of our DNA books and free hats. Register here Issue 07 also features: Leech Therapy. The tools for delivering gene therapies into cells have improved radically since the use of retroviruses in the early 1990s. Please RSVP to join.

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The Pharma Data

NOVEMBER 5, 2020

5 of its ongoing Phase 2 extension study, Study 211, examining vebicorvir (VBR, or ABI-H0731) in patients with hepatitis B virus infection. The company’s other two Phase 2 trials, Study 201 and 202, showed that the addition of VBR to NrtI therapy achieved a quicker, deeper level of viral suppression than with NrtI alone.

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The Pharma Data

OCTOBER 19, 2020

. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,

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KIF1A

FEBRUARY 4, 2024

In addition to sequencing patients, the researchers looked for antibodies to the HTLV-1 virus in the patients’ spinal fluid. We each have two DNA copies of the KIF1A gene; each copy is called an allele and makes its own RNA, which is then translated into protein. What is an allele specific ASO?

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The Pharma Data

OCTOBER 14, 2020

The research is part of an ongoing collaboration between Columbia University and Tonix that focuses on T cell and antibody responses to SARS-CoV-2 (CoV-2), the virus that causes COVID-19. solution), which is in Phase 2 development for the treatment of life-threatening cocaine intoxication and has FDA Breakthrough Therapy designation.

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Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. Certain CRISPR components can add short DNA sequences from the genomes of defeated viruses into the bacterium’s own genome, creating a type of protective “memory.” pyogenes , making it more specific.

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Broad Institute

MAY 27, 2025

Gene editing disrupts Huntingtons mutation in mice By Allessandra DiCorato May 27, 2025 Breadcrumb Home Gene editing disrupts Huntingtons mutation in mice Making single-letter edits in stretches of repeated DNA stopped or reversed the genetic change that causes Huntingtons disease and Friedreichs ataxia. That gave Matuszek and Arbab an idea.

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Broad Institute

JANUARY 14, 2025

The treatment, which uses base editing to make a single-letter change in DNA, reduced levels of the disease-causing prion protein in the brain by as much as 60 percent. Theres still a long way to go to make this a therapy, Minikel said. But the researchers needed to deliver the base editors to the brain.

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Elrig

FEBRUARY 23, 2023

It brings together leading scientists, researchers, and entrepreneurs with bold ideas, and one shared ambition: to develop the next generation of therapies to cure more patients around the world. This year, R&I runs from 29–30 th March at the Hinxton Hall Conference Centre in Cambridgeshire, UK. So, that’s the focus of my work.”

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New Drug Approvals

APRIL 18, 2023

Approximately 76% of HIV-patients with measurable viral load are infected with a strain of virus that is resistant to one or more classes of antiretroviral agents, thus reducing treatment options. AMD-070 targets the CXCR4 receptor on HIV and prevents the virus from entering and infecting healthy cells. They are for reference only.

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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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Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? DNA vectors in mouse models?

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Drug Target Review

JANUARY 15, 2024

Each T cell expresses a TCR specific for an HLA‑presented peptide signal, and when that signal is detected a T cell expressing the relevant TCR can launch a complex array of immune effector responses that can suppress or destroy tumours or virus-infected cells.

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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. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012).

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PLOS: DNA Science

APRIL 20, 2023

DNA is a sleek double helix, with “rungs” consisting of a purine base paired with a smaller pyrimidine base: adenine (A) with thymine (T) and guanine (G) with cytosine (C). . ” DNA encodes amino acid sequences comprising proteins, which impart traits. Watson and F.

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PLOS: DNA Science

JULY 3, 2025

When I saw a news release describing a test in development to sequence pathogen genomes in diarrhea, my geneticist side was, at first, impressed – why not track DNA to identify a specific virus, bacterium, parasite, or fungus? A research team from the University of Liverpool published their findings in Genome Medicine.

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Drug Target Review

SEPTEMBER 4, 2023

Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. We discovered it is highly effective and environmentally friendly.

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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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