pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. Through base editing, drugs replace single nucleotide in the DNA strand with another, without making double-strand breaks (DSBs) in the gene.

Gene Editing 98

Gene Editing Trials Drugs In-Vivo 98

pharmaphorum

FEBRUARY 19, 2021

Once someone is infected with HIV it stays with them for life thanks to the retrovirus’s ability to inject its DNA code into the host. We have proven the technology and candidate programs in vitro and in vivo in both small animal models and primate models.

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

BioTech 365

SEPTEMBER 2, 2020

(Nasdaq: SRNE, “Sorrento”) and SmartPharm Therapeutics, Inc. (“SmartPharm”) announced today that Sorrento has completed the acquisition of SmartPharm, a gene-encoded protein therapeutics company developing non-viral DNA and … Continue reading →

DNA 40

DNA In-Vivo Protein Gene 40

Scienmag

NOVEMBER 16, 2020

Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. doi:10.1186/s13073-020-00788-5… SINGAPORE, 3 Nov 2020 – Long RNA molecules carrying DNA codes that […]. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).

In-Vivo 53

In-Vivo RNA DNA Genome 53

BioTech 365

MARCH 9, 2021

an important non-canonical DNA secondary structure in vivo and in vitro, G-quadruplexes (GQs) have been widely investigated in many fields including biology, … Continue reading →

Scientist 40

Scientist In-Vivo In-Vitro DNA 40

Roots Analysis

MARCH 1, 2023

Disruption of the integrity and function of DNA at the gene or chromosomal level can lead to heritable mutations, resulting in genetic disorders, birth abnormalities, or cancer. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.

Genotoxicity 52

Genotoxicity DNA Genetics Bacteria 52

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

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Life Science Gene Editing Development Clinical Trials 119

XTalks

MAY 22, 2024

PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision. In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology.

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

The Pharma Data

NOVEMBER 22, 2020

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

XTalks

OCTOBER 1, 2020

Leaving academia for industry is a route that academic scientists and professors did not traditionally pursue in the past due to perceived differences in skillsets, experiences and even associated stigmas. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52

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. Zinc fingers. billion in funding.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

FEBRUARY 15, 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. However, not all innovations are equal and nor do they follow a constant upward trend.

In-Vivo 162

In-Vivo Genome Genomics Gene Editing 162

Pharmaceutical Technology

FEBRUARY 15, 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. Nucleases are enzymes that hydrolytically cleave the phosphodiester backbone of DNA.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

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.

In-Vivo 130

In-Vivo DNA Antibody Gene 130

Pharmaceutical Technology

MARCH 9, 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. However, not all innovations are equal and nor do they follow a constant upward trend.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

XTalks

DECEMBER 13, 2023

The CRISPR system is dubbed to be molecular “scissors” that can cut out parts of genes that are abnormal, deactivating or replacing them with new strands of normal DNA. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

AUGUST 12, 2020

Despite the effectiveness of anti-retroviral therapy (ART) in significantly improving health, quality of life and reducing mortality among HIV-positive individuals, new research shows that persistent infections may be due to the ability of the virus to take refuge in distinct subsets of immune T cells.

RNA 52

RNA Protein DNA In-Vivo 52

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. These genetic switches assist transcription factors in binding to the promoter region.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Roots Analysis

AUGUST 8, 2023

Furthermore, a wide variety of drugs and macromolecules, including DNA , proteins, and imaging agents, can be encapsulated in liposomal vesicles due to their unique ability to entrap both lipophilic and hydrophilic substances. Bangham, in 1961 at the Babraham Institute, University of Cambridge.

Drug Delivery Systems 52

Drug Delivery Systems Drug Delivery Drugs In-Vivo 52

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry.

Engineer 52

Engineer Containment In-Vivo Production 52

The Pharma Data

OCTOBER 14, 2020

VBL has recently demonstrated ex-vivo activity of anti-MOSPD2 antibodies in patients with relapsing-remitting and progressive multiple sclerosis (MS), as well as in animal models of rheumatoid arthritis (RA), nonalcoholic steatohepatitis (NASH) and inflammatory bowel disease (IBD). 1. -- -->. -- [if lte IE 8]--> .

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing In-Vivo 52

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

The Pharma Data

NOVEMBER 29, 2020

Compared to placebo, participants vaccinated with three HB-101 doses had: a 48 percent reduction in CMV viremia (presence of CMV DNA in the blood); a 42 percent reduction in the use of antiviral therapy; and. CMV can cause severe complications in kidney transplant recipients including organ rejection and death.

Vaccine 40

Vaccine Vaccination Clinical Trials Clinical Trials 40

Roots Analysis

JANUARY 14, 2024

Different types of expression system for the production of biologics have been mentioned in the following sections: Insect Expression Systems: Baculovirus is a double stranded DNA (dsDNA) lytic virus that can be effectively amplified in insect cells belonging to the Lepidoptera family.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

The Pharma Data

JUNE 7, 2023

Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Source link: [link]

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

pharmaphorum

NOVEMBER 24, 2021

Nykode’s Vaccibody technology generates DNA plasmids directed against neoantigens, which are only found on tumours. They are an attractive target for cancer immunotherapies as they may be recognised as foreign and attacked by the immune system, while leaving healthy tissue unscathed.

Vaccination 52

Vaccination Vaccine In-Vivo DNA 52

WCG Clinical

MAY 28, 2024

The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

Roots Analysis

AUGUST 23, 2024

Unveiling The Potential of mRNA Technology mRNA is a single-stranded molecule that relays the genetic instructions needed to make proteins from DNA in the cell nucleus to ribosomes. In recent years, mRNA technology has rocked the world of medicine, making it possible to prevent and treat a vast array of diseases and disorders.

Protein 40

Protein Vaccination Vaccine Immune Response 40

Roots Analysis

JANUARY 27, 2025

This can be done through various techniques such as PCR, DNA sequencing or gene synthesis. Vector Construction: Vector construction involves designing and assembling a DNA molecule (vector) that carries the gene encoding the protein of interest along with regulatory elements necessary for protein expression.

Protein 40

Protein Reagent Marketing Gene 40