Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

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Gene Therapy Gene Antibody FDA Approval 59

Drug Discovery World

OCTOBER 19, 2022

Yapan Bio has expanded its capabilities with a new process development facility at Genome Valley, Hyderabad, India, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. .

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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. David Williams, uses a virus to introduce RNA into the extracted bone marrow that turns off BCL11A. SATURDAY, Dec.

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Pharmaceutical Technology

FEBRUARY 23, 2023

The gene editing platform of Life Edit provides an extensive and diverse library of base editors and RNA-guided nucleases (RGNs), which are smaller in size than conventional nucleases. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

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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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pharmaphorum

APRIL 28, 2022

Harness Safe & Efficacious Lipid-Based Delivery for Novel Therapeutics Beyond RNA & Hepatic Tissue. The Next Generation Lipid-Based Nanoparticles Delivery Summit is your definitive industry-led meeting to design and engineer 2nd generation of lipid nanoparticles for emerging payloads beyond RNA into clinic.

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RNA Drug Delivery Engineer Gene Therapy 52

XTalks

NOVEMBER 30, 2023

Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV. percent in the placebo group.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Roots Analysis

FEBRUARY 23, 2022

Further, NGS library preparation kits offer several benefits, such as low requirement of starting material, generation of longer RNA / DNA sequences and production of superior quality nucleotide libraries, thereby, catering to existing unmet needs of the research domain. Next Generation Sequencing (NGS) Library Preparation Kits.

Genetics 52

Genetics DNA RNA Reagent 52

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

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Marketing Gene Sequencing DNA Genome 52

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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Gene Expression Gene Genetic Disease RNA 59

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.”. pyogenes dCas9.

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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.

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Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. DNA vectors compared to standard linear mRNA-based expression. “We

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RNA In-Vivo Gene Therapy Genetic Disease 52

XTalks

JANUARY 8, 2024

Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. Getting a drug to market used to be a relatively simple and straightforward affair. That’s just not the case anymore. Her advice revolves around two main strategies.

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Clinical Trials Clinical Trials Trials Gene Therapy 105

pharmaphorum

AUGUST 5, 2021

Vividion’s in-house R&D efforts are still at the preclinical stage, but include drug candidates in oncology and immune diseases targeting transcription factors – which are involved in the process of transcribing DNA into RNA and thereafter into proteins. billion ($5.3

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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 without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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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

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. It has a vibrant community of life-science companies, from global pharma to smaller start-ups.

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Drugs Life Science Gene Therapy Genome 75

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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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.

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Life Science Vaccination Vaccine Gene 111

pharmaphorum

OCTOBER 12, 2022

The payload, Del Bourgo tell us, is the therapeutic DNA or RNA sequence that will cure or fix damaged cells. Inactive viruses often act as the viral vector, as they’re able to deliver genetic therapies efficiently. and France to accelerate the development of genomic therapies. So far, we’ve seen promising results.”.

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Genome Genomics Medicine Development 99

Roots Analysis

AUGUST 31, 2023

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors.

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Gene Gene Therapy Gene Expression Regulation 40

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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Roots Analysis

MARCH 16, 2023

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein.

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Vaccination Vaccine DNA Protein 52

Drug Discovery World

MARCH 14, 2023

The success of messenger RNA (mRNA) vaccines in combatting the global SARS-CoV-2 crisis brings a new era of vaccine development. IVT is a complex reaction, requiring a mixture of RNA polymerase and supplementary components (e.g., Also keep in mind that optimisation is not linear and can be process- and product-dependent.

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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. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g.,

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Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al. In one example, Choi et al.

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Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

AUGUST 10, 2022

How ddPCR technology detects and quantifies DNA ddPCR technology is a highly sensitive and accurate tool used to detect and quantify known genetic sequences. Unlike qPCR, which requires users to generate a standard curve to estimate the concentration of DNA or RNA in a sample, ddPCR technology quantifies nucleic acids directly.

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Manufacturing Gene Genome Genomics 98

Drug Discovery World

JANUARY 23, 2023

Such stocks have already been created using conventional CRISPR-Cas9 approaches; however, cells edited with CRISPR-Cas9 have an increased risk of random insertions and deletions (indels) which are introduced during a cell’s intrinsic DNA repair process following a DNA double strand break (DSB) from Cas9. Dis Model Mech. Nat Commun.

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Gene Editing Gene Engineer DNA 52

Drug Discovery World

FEBRUARY 29, 2024

In the first article, Reece Armstrong speaks to Natalia Mateu, Co-Founder, Jelena Aleksic, Co-Founder and Peter Curran, Chemoinformatics Scientist, about utilising 3D chemistry and AI to get small molecule therapies from the clinic to the bedside.

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Gene Expression DNA Genome Genomics 52

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression.

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Trials Antibody Gene Therapy Clinical Trials 52

Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. One of the most exciting recent developments is the use of gene editing tools to create cell and gene therapies.

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Drug Discovery World

AUGUST 8, 2023

Before that, in October 2022, Yapan Bio expanded its capabilities with a new process development facility at Genome Valley, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. million) at the city’s life sciences hub 14.

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Research Drugs Genome Genomics 52

Drug Discovery World

OCTOBER 17, 2023

This is an invaluable approach for precision medicine because it promises a detailed view of desired and undesired effects of treatments with cell and gene therapy agents or antibodies. 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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Gene Expression Drugs Protein Gene 52

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

NOVEMBER 13, 2023

Steven Quistad, PhD, Senior Applications Scientist, DNA Script, on: ‘Rapidly assemble genes in your laboratory using automated enzymatic oligo synthesis’. Vaccines In his session, there will be two presentations following chairperson’s remarks from Viktor Lemgart, PhD, Research Fellow, Tidal Therapeutics, a Sanofi Company.

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Protein Antibody Engineer Scientist 59

Pharmaceutical Technology

APRIL 24, 2023

Since then, the field of nanomedicine has steadily progressed to reach high points such as the successful use of nanotechnology to deliver messenger RNA (mRNA)-based Covid-19 vaccines. Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Langer, now a David H.

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