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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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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DrugBank

JUNE 13, 2024

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots. This approach targets the fundamental instructions within a cell's DNA, either by correcting faulty genes or introducing entirely new ones to combat disease.

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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

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

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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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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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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Drug Target Review

DECEMBER 28, 2023

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. This allows the cancer to evolve and develop resistance to the therapy.

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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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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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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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Codon

SEPTEMBER 29, 2024

mRNA was the intermediate stage between DNA and protein, a dynamic entity that shifted depending on the second-to-second needs of the cell, able to point out if a cell was cancerous or stressed, what kind of cell it was, and so on. Of particular interest was how genetic therapies were delivered.

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Codon

MARCH 20, 2023

Signal Transduction and Targeted Therapy. Digital nanoreactors to control absolute stoichiometry and spatiotemporal behavior of DNA receptors within lipid bilayers. CRISPR-induced DNA reorganization for multiplexed nucleic acid detection. Molecular Therapy. Gene Therapy. Szymczak P. Nature Communications.

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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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PLOS: DNA Science

SEPTEMBER 7, 2023

As genetics morphed into genomics, artificial intelligence stepped in, layering the combinatorial information of comparative genomics onto DNA sequences. Train an algorithm on the DNA sequences of a known disease-causing gene, then search for identical or highly similar sequences in cells from other individuals to assist diagnosis.

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Broad Institute

MARCH 22, 2024

Staying power The mRNA in today’s COVID vaccines is so effective because very little is needed – once injected into the body, it stimulates the production of proteins that resemble parts of the COVID virus. They named those modified molecules “mRNA-oligo conjugates” or mocRNAs.

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Codon

JULY 27, 2023

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. approved a gene therapy for hemophilia A for the first time. They’re talking about a patient who has a rare genetic disease.

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Codon

JULY 27, 2023

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. approved a gene therapy for hemophilia A for the first time. They’re talking about a patient who has a rare genetic disease.

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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.

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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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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

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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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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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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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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Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Thousands of transcription factors — proteins that bind DNA and control gene expression — were studied in human cells. Molecular Therapy. Gene Therapy.

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The Pharma Data

NOVEMBER 23, 2021

Approval is for Cytomegalovirus, a Type of Herpes Virus. Livtencity works by preventing the activity of human cytomegalovirus enzyme pUL97, thus blocking virus replication. CMV is a type of herpes virus that commonly causes infection in patients after a stem cell or organ transplant. Today, the U.S.

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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. Unlike gene therapy as originally-envisioned since the 1950s, which adds a functioning copy of a gene, CRISPR can add, remove, or replace a DNA sequence.

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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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Codon

MAY 18, 2023

shelves, it’s easy to find DNA in the air , and some weird insect cybernetics. 2/ Your DNA is Everywhere. A study published Monday in Nature Ecology & Evolution shows that tiny amounts of human DNA, shed into the soil or air, can be easily detected using little more than a $1,000 Nanopore MinION. Read more in WIRED.

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Codon

MAY 18, 2023

shelves, it’s easy to find DNA in the air , and some weird insect cybernetics. 2/ Your DNA is Everywhere. A study published Monday in Nature Ecology & Evolution shows that tiny amounts of human DNA, shed into the soil or air, can be easily detected using little more than a $1,000 Nanopore MinION. Read more in WIRED.

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Codon

APRIL 2, 2023

But now, by studying DNA extracted from microbes in the blood of almost 10,000 healthy people, this paper shows that there is no such thing. Read Transcription factors bind to DNA and control gene expression. Read Switchable hydrophobic pockets in DNA protocells enhance chemical conversion. Nature Microbiology. Meeussen J.V.W.

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Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. The SOP should list these symptoms and any available prophylaxis for post-exposure treatment.

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Codon

MAY 22, 2023

And experiments at Tune Therapeutics showed that CRISPR epigenome editing — which doesn’t cut DNA at all, but merely silences genes by adding chemical groups to them — can curb “bad cholesterol” by more than 50% in monkeys. These tools do not cut or nick DNA, and so they may be safer than other options.

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The Pharma Data

SEPTEMBER 28, 2021

By testing only what’s medically relevant and necessary, we enable targeted therapy, avoid misuse of antibiotics and reduce unnecessary intervention.”. TaqMan® DNA probes that hybridise with the target strand are labeled with a fluorescein allowing real-time measurements during the PCR process.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Gene Delivery Systems Genetic material, in the form of DNA or RNA, does not easily enter cells without the aid of a delivery system.

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