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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Drug Discovery World

FEBRUARY 27, 2023

DDW’s Diana Spencer speaks to Raphael Townshend PhD, Founder and CEO of Atomic AI, about the company’s successes so far, plans for the future and the unique approach of combining RNA structural biology and AI models. DS: How is Atomic AI “unlocking the next generation of RNA drug discovery”? What is unique about your engine?

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Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Limitations of AAV gene therapy . We’re hoping to use what we’ve learned so far about LNPs to develop an improved gene editor delivery system.”

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Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy.

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Drug Discovery World

FEBRUARY 29, 2024

Study reveals therapeutic potential of mRNA in rare diseases Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Read more.

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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. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

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Scienmag

MARCH 4, 2021

Findings explain how plants use RNA to defend against fungal invaders Credit: Nicole Ward Gauthier/University of Kentucky New research reveals an essential step in scientists’ quest to create targeted, more eco-friendly fungicides that protect food crops.

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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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The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. This space will provide dedicated and configurable lab and office space, access to Lilly scientists, and opportunities for collaboration. in the rapidly expanding Seaport district of Boston.

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Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. billion in funding.

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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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pharmaphorum

NOVEMBER 24, 2021

Here’s a snapshot of our world class speaker line-up: Andreas Kuhn, Senior VP – RNA Biochemistry & Manufacturing, BioNTech . Dariush Mohammadyani, Senior Scientist, Johnson & Johnson . Elaine Peters, Director, Analytical – Individualized Cell & Gene Therapies, Genentech.

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pharmaphorum

JUNE 28, 2022

Chairs for the conference are industry expert Ekkehard Leberer, Senior Consultant, Elbiocon, and Jim Weterings, Principal Scientist, AstraZeneca. The 2022 Conference theme is on the latest advances in oligonucleotide discovery and delivery. Interested parties can register for the conference and workshop at: [link].

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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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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA).

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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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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

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Another approach to address the limited availability of CAR T therapies consists of using CRISPR technology to generate CAR T-cell therapeutics derived from inducible pluripotent stem cells (iPSCs) 5.

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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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Gene Editing Gene Engineer 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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DNA Gene Gene Silencing Genome 52

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? and Preparing for new-era drug modalities with technology.

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Gene Expression DNA Genome Genomics 52

Drug Discovery World

OCTOBER 17, 2023

RA: With spatial biology being cross-disciplinary – in that it uses techniques in both gene expression analysis and immunofluorescence – is expertise in the field limited? The first step to reach this goal will be to generate protein and RNA data from the same sample. Let’s take CAR T cells as an example.

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Drug Discovery World

NOVEMBER 13, 2023

The presentations are as follows: Fabian Schmich, PhD, Senior Data Scientist, pRED Informatics, Roche Diagnostics Deutschland, on: ‘scifAI: An explainable machine learning framework applied to functional characterisation of therapeutic antibodies’.

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Protein Antibody Engineer Scientist 59

XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. Onureg (azacitidine), a leukemia therapy, also saw large growth of 70 percent to $124 million in 2022, and the multiple sclerosis drug Zeposia (ozanimod) generated $250 million in 2022, an 87 percent increase. billion in 2022.

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Drug Discovery World

FEBRUARY 22, 2024

Neuroscience advances At the time of writing The Guardian newspaper led with the headline:” Scientists hope weight-loss drugs could treat addiction and dementia”, reporting that “medications with semaglutide such as Ozempic and Wegovy are being studied to see if they can help other conditions.”

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XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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Pharmaceutical Technology

FEBRUARY 20, 2023

DDL was founded in 1994, also originally started by a group of scientists operating in a university, also specialising in infectious diseases, but with more of a focus on HPV and Hepatitis. Viroclinics Biosciences – what it was called before the DDL purchase – was also well known for its specialist logistics infrastructure.

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