Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Scienmag

APRIL 15, 2021

High resolution electron microscopy imaging shows how damage-sensing proteins recognize DNA breaks and then bridge them together Credit: He Lab From cancer treatment to sunlight, radiation and toxins can severely damage DNA in both harmful and healthy cells.

DNA 46

DNA Protein Gene Therapy Gene 46

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform.

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin. Eventually, they will need assistance with breathing.

Gene Therapy 94

Gene Therapy Gene FDA Approval DNA 94

Delveinsight

DECEMBER 3, 2020

Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences.

Gene Therapy 52

Gene Therapy Gene Antibody Research 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?

Gene Expression 59

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

pharmaphorum

JANUARY 29, 2021

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. First of all it will be used for ultra-rare monogenic disease.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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.”.

DNA 98

DNA Gene Gene Silencing Genome 98

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.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

Roots Analysis

JANUARY 14, 2024

Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. Insect cell lines have been frequently used as recombinant protein expression systems. cerevisiae) and Pichia pastoris.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Cloudbyz

MARCH 6, 2024

Understanding Biologics: Biologics are a class of therapeutic agents derived from living organisms, such as cells, tissues, or proteins. Unlike traditional small molecule drugs, which are chemically synthesized, biologics are produced through complex biotechnological processes, often involving recombinant DNA technology.

Gene 72

Gene Immune Response Manufacturing Gene Therapy 72

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

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.

Life Science 111

Life Science Vaccination Vaccine Gene 111

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.

Gene Therapy 94

Gene Therapy Gene Gene Editing DNA 94

Drug Discovery World

MAY 7, 2024

This means we are able to hijack the cell machinery to be even more efficient than before to produce more of what we want (mAb) and less of what we don’t want (impurities) through the different genetic editing tools for cell engineering. RA: How can anion exchange (AEX) chromatographic approaches improve production processes for antibodies?

Antibody 59

Antibody Production Manufacturing Engineer 59

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

RNA 52

RNA In-Vivo Gene Therapy Genetic Disease 52

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

Drugs 75

Drugs Life Science Gene Therapy Genome 75

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. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

Gene 262

Gene Gene Therapy Genetics DNA 262

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.

Gene 40

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.

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

STAT News

NOVEMBER 4, 2022

His goal was to find a treatment for his brother’s form of Duchenne muscular dystrophy, which is caused by a mutation that impeded his ability to make a crucial muscle protein called dystrophin. It was the first time that a gene editing therapy was custom-made to address a single individual’s unique genetic mutation.

Gene Editing 105

Gene Editing Gene Gene Therapy Clinical Trials 105

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.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

Drug Discovery World

AUGUST 2, 2022

CureHeart is using gene therapy technologies to edit or silence the faulty genes that cause inherited heart muscle diseases. . Also known as genetic cardiomyopathies, they often go undiagnosed and are a major cause of heart transplants due to how current treatments do not prevent the condition from progressing. .

Gene Editing 52

Gene Editing Gene Research Genetics 52

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

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

The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. PGRN is a complex and highly conserved protein. The rapid progression of FTD results in an average survival of eight years after onset of symptoms.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

NOVEMBER 9, 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. Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’.

Engineer 52

Engineer Antibody Protein In-Vivo 52

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. Data were provided by the Cell and Gene Therapy Catapult (London, UK).

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

DNA 98

DNA Genome Genomics RNA 98

The Pharma Data

MARCH 20, 2023

As the number of patients treated with gene therapy around the world continues to grow, our goal is that more patients, and even new SMA patient populations, will be able to experience the transformative impact of this treatment,” said Sitra Tauscher-Wisniewski, MD, Vice President Clinical Development & Analytics, Novartis Gene Therapies.

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

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

The Pharma Data

DECEMBER 27, 2020

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. TSHA-101 is an investigational gene therapy designed to deliver the HEXA and HEXB genes driven by a single promoter within the same AAV9 construct, dubbed a bicistronic vector.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 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. 2016/17 saw the emergence of this novel CRISPR-Cas9-based approach.

Gene Editing 52

Gene Editing Gene Engineer DNA 52