Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Therapies that don’t precisely target diseased cells can have unintended effects in other healthy cells.” .

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Gene Expression Gene Packaging Packaging 52

Drug Discovery World

APRIL 17, 2024

JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. What are the challenges of drug discovery in this area?

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Gene Expression Gene Genetic Disease RNA 59

XTalks

DECEMBER 4, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies.

Gene Therapy 69

Gene Therapy Gene Clinical Trials Clinical Trials 69

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

NOVEMBER 27, 2023

This follows the news that the MHRA was the first regulatory authority to authorise Vertex’s gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Kaftrio is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface.

Protein 52

Protein Gene Medicine Gene Therapy 52

Cloudbyz

MARCH 6, 2024

Understanding Biologics: Biologics are a class of therapeutic agents derived from living organisms, such as cells, tissues, or proteins. Personalized Medicine: Biologics offer the potential for personalized treatment approaches by taking into account individual variations in genetics, immune responses, and disease characteristics.

Gene 72

Gene Immune Response Manufacturing Gene Therapy 72

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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Gene Therapy Drugs Gene Manufacturing 52

XTalks

NOVEMBER 30, 2023

Its mechanism of action involves blocking the protein shell (capsid) of the HIV-1 virus, disrupting multiple crucial stages of the viral lifecycle. This candidate, derived from a person’s own genetically modified cells, aims to restore the Gag-specific CD4+ T cell response in those receiving antiretroviral therapy.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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.”

Drugs 75

Drugs Life Science Gene Therapy Genome 75

pharmaphorum

SEPTEMBER 12, 2022

Alongside drugs and therapeutics, data collection and technology enhancements have redefined the traditional healthcare experience, especially within oncology, and genomic profiling has become a significant factor in allowing for personalised care. In other words, every person’s cancer has its own genetic construct. Tailored oncology.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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Gene Therapy Gene Gene Editing DNA 94

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. A genetic treatment for epilepsy Most treatments for epilepsy aim to treat seizures, which is the main symptom of this condition, says Ferraro.

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Research Gene Gene Therapy Genome 264

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. But this kind of change can’t happen in a bubble.

Gene Therapy 52

Gene Therapy Gene Manufacturing Development 52

Pharmaceutical Technology

JUNE 4, 2023

Innovation S-curve for the pharmaceutical industry Transcription factors for AAV is a key innovation area in pharmaceutical Adeno-associated virus (AAV) vectors are widely used for gene therapy. AAV genomes are highly compact, with overlapping coding regions, alternate splicing schemes, and multiple transcription initiation codons.

Gene 262

Gene Gene Therapy Genetics DNA 262

Delveinsight

JULY 23, 2020

The cause behind the FCS is believed to be the mutations in genome makeup of an afflicted person that disrupts the normal functioning of the enzyme required to break down the large molecules of triglycerides. 2017, Stroes et al. 2017, Stroes et al. Therapeutic Options and Unmet Needs.

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Marketing Gene Therapy Genome Genomics 61

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.

Vaccination 52

Vaccination Vaccine DNA Protein 52

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. As such, teams are now researching a variety of medical applications for mRNA-based drugs.

RNA 52

RNA Protein Vaccination Vaccine 52

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

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

OCTOBER 12, 2023

Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity. When it comes to the technologies driving drug discovery, she thinks that the ability to specifically edit regions of the genome using CRISPR/Cas9 is particularly revolutionary.

Drugs 59

Drugs Life Science Genome Genomics 59

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. Host cell proteins (HCPs) – proteins derived from the host cells used for viral production—are one class of process-related impurities.

Production 52

Production Gene Protein Vaccination 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 through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

Drug Discovery World

FEBRUARY 8, 2023

Svea Cheeseman , Refeyn, explains why better efficiency is needed to advance the production of viral vectors for use in gene therapies. Adeno-associated viruses (AAVs) are a promising, widely used vector for delivering gene therapies. A crucial part of the AAV workflow is the purification of viral vectors.

Gene Therapy 52

Gene Therapy Gene Antibody Production 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. Cas12 protein creates staggered double-strand breaks (DSBs) and promotes the HDR mechanism instead of utilizing both non-homologous ends joining (NHEJ) and HDR.

DNA 98

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

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

MAY 30, 2023

At PhoreMost, we have been particularly excited about the emergence of targeted protein degradation (TPD) as a new therapeutic modality. The concept of harnessing the ubiquitin-proteosome system to ‘destroy’ or degrade disease causing proteins has been established for some time.

Research 52

Research Drugs Protein RNA 52

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Most antibody drugs and vaccines have been developed to target parts of the spike protein.

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Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

APRIL 27, 2023

When combined with rapid advancement in delivery technologies, mRNA has the potential to lead to breakthroughs in the development of tailored treatment approaches in a wide range of indications from cancer to rare genetic disorders while enabling rapid responses to emerging threats. billion by 2028.

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