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. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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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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The Pharma Data

MARCH 18, 2022

Columbia Engineering researchers report that they have developed a “cloaking” system that temporarily hides therapeutic bacteria from immune systems, enabling them to more effectively deliver drugs to tumors and kill cancer cells in mice. Sheng Professor of Biomedical Engineering. Using Bacteria For Therapy.

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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. The limitations of natural AAV serotypes in meeting all clinical needs has spurred growing interest in AI-driven AAV engineering approaches.

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Broad Institute

JULY 10, 2024

The new method, published today in Nature Biomedical Engineering , precisely and durably corrects the mutation in human lung cells, restoring cell function to levels similar to that of Trikafta. Nature Biomedical Engineering. Paper cited Sousa AA and Hemez C et al. Online July 10, 2024. DOI:10.1038/s41551-024-01233-3.

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Broad Institute

NOVEMBER 13, 2023

Liquid biopsies enable clinicians to find and analyze tumor DNA in a patient’s blood sample to detect cancer early, monitor cancer recurrence, assess the patient’s response to treatment, and measure other clinically important features in real time, without invasive procedures.

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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

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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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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Codon

MAY 18, 2023

shelves, it’s easy to find DNA in the air , and some weird insect cybernetics. And the article doesn’t mention how Pairwise engineered the mustard to be “less pungent.” 2/ Your DNA is Everywhere. ” DNA from the air gave the longest reads. ” DNA from the air gave the longest reads.

DNA 52

DNA Engineering Therapies Virus 52

Codon

MAY 18, 2023

shelves, it’s easy to find DNA in the air , and some weird insect cybernetics. And the article doesn’t mention how Pairwise engineered the mustard to be “less pungent.” 2/ Your DNA is Everywhere. ” DNA from the air gave the longest reads. ” DNA from the air gave the longest reads.

DNA 52

DNA Engineering Therapies Virus 52

Drug Target Review

APRIL 26, 2023

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. To achieve this, scientists utilise various technologies that function similarly to scissors, precisely cutting the DNA at a particular location.

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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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PLOS: DNA Science

OCTOBER 3, 2024

This criticism, along with the development of radiation therapy and chemotherapy, caused Coley’s Toxins to gradually disappear from use. ’” Coaxing BCG to Tame TB and then Cancer The novel vaccine strategy builds on the accumulation of natural mutations when DNA copies itself as cells divide.

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Drug Target Review

APRIL 16, 2024

Can you explain how Radioligand Therapy (RLT) is applicable in the treatment of both chemotherapy-resistant tumours and in naïve patients who have not undergone prior cancer treatment? By delivering radiation directly to the tumour cells, RLT can induce DNA damage, leading to cell death and tumour regression.

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Creative Biolabs

MAY 25, 2022

These cytotoxic drugs include DNA base analogs, antimetabolic drugs, tubulin inhibitors, etc. Cancer has become the second largest health threat in the world, with about 10 million people dying of cancer in 2020. For decades, cytotoxic-based chemotherapy has been the main treatment for all kinds of cancers.

Therapies 52

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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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Broad Institute

JANUARY 8, 2024

A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. Prime editing, described in 2019 by Liu’s group, can make longer and more diverse types of DNA changes than other types of editing.

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The Pharma Data

JANUARY 25, 2021

a pre-clinical stage biotechnology company pioneering a new class of fully personalized neo-antigen T cell therapies to treat cancer, today announced that the company’s co-founder, Board member and Chairman of its Scientific Advisory Board, Ton Schumacher, Ph.D., has been awarded the 2021 Jeantet-Collen Prize for Translational Medicine.

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The Pharma Data

SEPTEMBER 11, 2021

By leveraging our unique ability to manufacture DNA at scale, we can construct proprietary antibody libraries precisely designed to match sequences that occur in the human body. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by “writing” DNA on a silicon chip.

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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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The Open Targets Blog

AUGUST 15, 2023

Research in mice has shown that RBM3 expression is protective against misfolded protein damage, suggesting the protein as an interesting target in the development of therapies for neurodegenerative conditions such as Alzheimer’s disease. RBM3 is quite special though, because in this case the poison exon is regulated by temperature.

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The Pharma Data

DECEMBER 14, 2020

engine along with algorithms tuned to predict BBB permeability played an important role in helping determine which CNS cancers and which genomically-defined subtypes of CNS cancer should be prioritized for development. . At the stage of GBM relapse and recurrence, no effective therapy strategies currently exist.

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Codon

MAY 11, 2023

Scroll to the end for a beautiful chart about the Anthropocene & some details on insect engineering companies. link ) A more representative human genome sequence, based on DNA from 47 ethnically diverse people, was published yesterday. link ) Emily Whitehead was the first child to receive a CAR-T cell therapy.

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Codon

MAY 11, 2023

Scroll to the end for a beautiful chart about the Anthropocene & some details on insect engineering companies. link ) A more representative human genome sequence, based on DNA from 47 ethnically diverse people, was published yesterday. link ) Emily Whitehead was the first child to receive a CAR-T cell therapy.

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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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LifeSciVC

DECEMBER 6, 2022

These hair clip-like molecules clamp onto DNA, thereby turning many genes on and off. Darnell’s 2002 paper Transcription Factors as Targets for Cancer Therapy , in which he wrote: “A limited list of transcription factors are overactive in most human cancer cells, which makes them targets for the development of anticancer drugs.

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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. iPSC-derived lymphocytes, eg, T cells and natural killer (NK) cells, engineered to express targeting molecules such as chimeric antigen receptors (CARs) have shown clinical promise to treat haematological malignancies.

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Drug Target Review

JULY 7, 2023

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

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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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Therapies RNA DNA Engineering 52

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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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. Because I was put into the highest tier, I was automatically mapped to the engineering track.

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The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov. USE IN SPECIFIC POPULATIONS.

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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. In previous decades, isolation, sequencing and characterisation of DNA encoding a specific TCR was laborious and technically challenging. Engineering soluble T-cell receptors for therapy.

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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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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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