STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham. Continue to STAT+ to read the full story…

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Gene Editing Gene Genetic Disease Gene Therapy 126

Drug Discovery World

SEPTEMBER 20, 2022

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate.

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Gene Therapy Gene Genome Genomics 52

Drug Discovery World

APRIL 17, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

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Worldwide Clinical Trials

JANUARY 24, 2024

Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their genetic diseases. Genetic counselors are critical in this pathway, especially as more is learned about the human genome and relevant incidental findings.

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Genetics Trials Clinical Trials Clinical Trials 184

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drug Discovery World

NOVEMBER 1, 2022

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development In-Vivo Genome Genomics 130

Drug Discovery World

JULY 10, 2023

If there’s one thing I would take from the Summer 2023 issue of Drug Discovery World , it would be that the application of technologies and processes throughout the pharmaceutical industry are changing how scientists pursue the development of therapies. This was done to assess the accuracy of the team’s machine-learning algorithms.

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Pharmaceutical Technology

FEBRUARY 3, 2023

The field of genomic medicine has reached a true turning point. With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

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Drug Discovery World

MAY 6, 2024

A more established Amsterdam-based enterprise is gene therapy company uniQure, developer of etranacogene dezaparvovec, the first gene therapy for haemophilia B, which was approved for use in the US in November 2022 and in the EU in February 2023. The government and organisations here are so positive and enthusiastic.

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Drugs Life Science Gene Editing Gene 59

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

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Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. billion in funding. This is reflected by our recent share price performance outperforming the index in the past year.”

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Drugs Life Science Gene Therapy Genome 76

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

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Gene Expression Gene Genetic Disease RNA 59

Scienmag

OCTOBER 7, 2020

Credit: UC Berkeley Biochemist Jennifer Doudna, a professor at UC Berkeley and faculty scientist at the Department of Energy’s Lawrence Berkeley National Laboratory (Berkeley Lab), is co-winner of the 2020 Nobel Prize in Chemistry for “the development of a method for genome editing.”

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Genome Genomics Scientist Development 89

pharmaphorum

JANUARY 24, 2023

Though instances of this kind of therapy are rare, it is a growing area. The cost of testing per human genome in 2006 was approximately $14 million , and in less than two decades, an average consumer-purchased genetic test costs $100. Scientists are able to study the aggregated, de-identified genetics of these individuals.

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Drug Discovery World

MAY 11, 2023

Although various approaches such as gene therapy exist that offer hope of slowing or blocking the progression of photoreceptor cell loss, these techniques cannot restore lost cells. When these cells degenerate due to disease, they are not replaced and the patient suffers vision loss that can progress to total blindness.

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Gene Therapy Gene Scientist Research 52

Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach. References “Approved Cellular and Gene Therapy Products.”

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Gene Therapy Gene Manufacturing Development 52

pharmaphorum

MAY 18, 2022

Genomics has unlocked entirely new insights to what exactly may cause certain neurological conditions. Also, there is particular emphasis on exploring the potential of gene therapy to develop targeted treatments for these and further neurodegenerative conditions. Conclusion.

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Gene Therapy Gene Genetics Development 80

The Pharma Data

MARCH 16, 2022

The research scientist at the Max Planck Institute for Medical Research received the award in recognition of his ground-breaking work in establishing various approaches to protein labeling in living cells that have enabled far-reaching advances in chemical and cell biology. Dr. Connor W.

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Protein Scientist Gene Development 52

Delveinsight

DECEMBER 15, 2020

The combined entity will put forward two rapidly converging, patient-centric models of care delivery with combined strengths in immunology, biologics, genomics and oligonucleotides to drive future medicine innovation. Welcome the new entrant in Gene therapy market: InnoSkel.

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XTalks

MARCH 8, 2022

From leading COVID-19 vaccine developments to being at the helm of the latest gene therapies and inventing ground-breaking DNA technologies like CRISPR, women have been at the forefront of some of the most leading-edge scientific discoveries in recent years, both as innovators and leaders.

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Life Science Gene Therapy DNA Vaccination 105

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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The Pharma Data

SEPTEMBER 21, 2020

. A biotech company spun out of the University of Edinburgh will utilise research to develop new gene therapies to target different types of cancer. The funding will be used to develop a pipeline of ‘highly selective’ gene therapies targeting cancer stem cells in a variety of solid tumour types.

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Gene Therapy Gene Development Genome 52

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Gene-based therapeutics and vaccines hold immense potential for more efficient and personalised treatment and prevention of complex diseases.

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Production Gene Protein Vaccination 52

Drug Discovery World

AUGUST 10, 2022

Later, scientists took advantage of antibodies’ natural tendency to seek out specific antigens and created antibody therapies that guide T cells towards the cells they are supposed to destroy. To overcome the challenge of identifying the ideal target, scientists are using several creative methods.

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Manufacturing Gene Genome Genomics 98

Scienmag

MARCH 11, 2021

The CRISPR-based gene editor, C-to-G Base Editor (CGBE), opens up treatment avenues for up to 40 per cent of genetic disorders caused by single-nucleotide mutations Credit: Agency for Science, Technology and Research (A*STAR), Genome Institute of Singapore (GIS) A team of researchers from the Agency for Science, Technology and Research’s (A*STAR) (..)

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Gene Scientist Genome Genomics 74

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

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Drug Discovery World

SEPTEMBER 30, 2022

Elsewhere researchers have discovered new ways of identifying cancer cells and a manufacturing system could advance the development and production of cell and gene therapies. Up to 8 recipients to be awarded grants for genomic research. Cell analysis system could streamline gene therapy manufacturing .

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Gene Therapy Drugs Genome Genomics 52

Drug Discovery World

FEBRUARY 21, 2023

For example, CRISPR Classroom, a biotechnology education company that discusses cutting edge advances in cell and gene therapy on TikTok boasts a following of over 60,000 people of which >79% are older than 25. Furthermore, marketing teams can tailor the organisation’s following to feature specific demographics.

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Marketing Branding Gene Therapy DNA 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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Drug Discovery World

FEBRUARY 29, 2024

They are called: Sitting down with… PharmEnable , Is single-cell gene expression the next trend in next generation sequencing? In the second article, we hear from Dr Dina Finan, PhD, Product Manager at 10x Genomics and Dr Nick Downey, PhD, NGS Collaborations Lead at Integrated DNA Technologies.

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Gene Expression DNA Genome Genomics 52

Drug Discovery World

OCTOBER 12, 2023

New approaches and thought processes When it comes to diversity in drug discovery, this includes our scientists’ and pharma workforce’s approach to innovation which will help advance the industry. Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity.

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Drugs Life Science Genome Genomics 59

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Additionally, the CRISPR-Cas system can be used to create TCR T cells that can specifically recognise these neoantigens, expanding its applications beyond traditional CAR T therapy.

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Drug Discovery World

APRIL 30, 2024

mRNA exists in every cell within the human body and this, along with its role in the development of proteins, are the reasons why mRNA therapies have such potential in treating many diseases. Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells.

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Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

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