Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

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Gene Editing Gene In-Vivo Gene Therapy 147

Drug Discovery World

DECEMBER 13, 2022

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.

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RNA Genetics Gene In-Vitro 52

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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Medical Xpress

FEBRUARY 9, 2023

When cells copy DNA to produce RNA transcripts, they include only some chunks of genetic material known as exons and throw out the rest. The resulting product is a fully-mature RNA molecule, which can be used as a template to build a protein.

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RNA DNA Gene Genetics 75

Scienmag

APRIL 27, 2022

CHAPEL HILL, NC – Inside embryonic cells, specific proteins control the rate at which genetic information is transcribed from DNA to messenger RNA – a crucial regulatory step before proteins are created. Then, organs develop and hopefully function properly.

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Scientist Gene RNA Protein 83

Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Clinical holds are becoming more common, especially in gene therapy programs.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

Drug Discovery World

SEPTEMBER 27, 2023

While genetics has made significant strides in healthcare, the field of epigenetics unveils a new dimension in precision medicine, offering insights into the dynamic interplay between genes and experiences that shape our health and disease.

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DNA Genetics Gene Medicine 115

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

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The Pharma Data

MARCH 7, 2022

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

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Protein Gene Genome Genomics 52

Scienmag

MAY 5, 2021

Transcription is a vital process in bacterial cell, where genetic information in DNA is transcribed to RNA for the translation of proteins that perform cellular function. Credit: The Hong Kong University of Science and Technology The emergence and spread of new forms of resistance remains a concern that urgently demand new antibiotics.

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Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. When it gives the green light, H3K4me3 allows RNA polymerase II to move along DNA, transcribing it into RNA as it moves.

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Gene Expression Gene DNA RNA 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

Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. We were excited to unveil data showing that two distinct chemical series exhibit METTL1 inhibition in vitro at low nanomolar concentrations with minimal interference with other RNA and protein methyltransferases.

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Scienmag

JUNE 29, 2022

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy, characterized by progressive muscle wasting and weakness and caused by abnormally repetitive DNA segments that are transcribed into toxic molecules of RNA. Rett […].

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Scienmag

MARCH 17, 2021

An international team of scientists from the University of Turku, Finland and PennState University, USA have solved a long-standing mystery of how living organisms distinguish RNA and DNA building blocks during gene expression paving the way for the design of new antiviral drugs. All cellular […].

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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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Gene Editing Gene Gene Therapy Genetics 98

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. Although immune features remained relatively stable, RNA-based features related to DNA damage repair pathways changed significantly.

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Pharmaceutical Technology

FEBRUARY 14, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. These systems include human and mouse cell lines, and even in vivo in live animals.

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

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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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Scienmag

MAY 27, 2021

Capturing how RNA polymerase enzymes kick off transcription On scales too small for our eyes to see, the business of life happens through the making of proteins, which impart to our cells both structure and function.

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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

BioTech 365

APRIL 13, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Scaling up genome editing in tiny worms.Understanding the effects of specific mutations in gene regulatory regions—the sections of DNA and RNA that turn genes on and off—is … Continue reading →

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Drug Discovery World

JANUARY 20, 2023

Precision medicine uses information about an individual’s genes, proteins, and environment to prevent, diagnose, and treat diseases, including cancer. Differences in DNA, RNA, and protein can be identified in individual tissue samples, and even in individual cells.

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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.

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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

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. This well-read article featured in the DDW Winter 22/23 issue’s Cell & Gene Therapy Guide.

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Gene Therapy Gene Antibody FDA Approval 59

pharmaphorum

JANUARY 5, 2023

The company develops mRNA therapeutics and vaccines , and the move will enable it to utilise the synthetic biology and enzyme tech of 2018-founded Japanese company OriCiro, which develops cell-free DNA synthesis and amplification technologies, and thereby expand Moderna’s portfolio.

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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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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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Gene Therapy Gene Gene Editing Genetics 52

Scienmag

APRIL 14, 2021

In the cell nucleus histones play a crucial role packaging DNA into chromatin. Histones are however very sticky to both DNA and RNA, so to ensure they are transported to the cell nucleus after synthesis and bind to the right portion of DNA to organize the chromatin, they are guarded by complexes of histone chaperones. […].

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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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Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely.

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Genetics Gene Expression DNA Antibody 130

XTalks

JUNE 20, 2022

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

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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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XTalks

APRIL 6, 2022

Cancerous cells and tumors release small fragments of DNA into the bloodstream called circulating tumour DNA (ctDNA). The researchers at the Cancer Research UK Cambridge Institute published this data from the LUng cancer CIrculating tumour DNA (LUCID) study in the journal Annals of Oncology.

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DNA In-Vitro Doctors Doctor 105

pharmaphorum

MARCH 11, 2021

Join the Epigenetic Therapeutic Targets Summit – the only virtual summit dedicated to the clinical translation and optimization of therapeutic candidates targeting epigenetic mechanisms of gene expression.

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