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

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. We can see this versatility in the various treatments that are currently being researched.

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Roots Analysis

AUGUST 31, 2023

Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate.

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Gene Gene Therapy Gene Expression Regulation 40

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

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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DNA Genome Genomics RNA 98

Drug Discovery World

AUGUST 10, 2022

To overcome this evasion tactic, the CAR protein was developed to recognise other markers on cancer cells. CAR is a fusion protein composed of an extracellular antibody fragment that recognises antigens and an intracellular domain that directs the T cell to the tumour. Identifying the right CAR T cell target.

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Manufacturing Gene Genome Genomics 98

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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DNA Gene Gene Silencing Genome 98

Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

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Protein Antibody Engineer Scientist 59

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

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

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. At PhoreMost, we have been particularly excited about the emergence of targeted protein degradation (TPD) as a new therapeutic modality.

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Research Drugs Protein RNA 52

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

OCTOBER 17, 2023

Today, spatial omics technologies enable imaging-based identification of cells in their native tissue context while simultaneously detecting hundreds of RNAs or tens of protein markers in the same cells. The first step to reach this goal will be to generate protein and RNA data from the same sample.

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Gene Expression Drugs Protein Gene 52

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. The drug is an autologous IL13Ralpha2-CAR-T cell product.

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Trials Antibody Gene Therapy Clinical Trials 52

XTalks

AUGUST 2, 2023

Johnson & Johnson’s Top 5 Best-Selling Drugs of 2022: 1) Stelara (ustekinumab) Stelara is an immunosuppressant biologic therapy that blocks the IL-12 and IL-23 proteins that play a role in plaque psoriasis and Crohn’s disease. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A.

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Sales Manufacturing FDA Approval Life Science 98

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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