pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

Scienmag

JANUARY 27, 2021

— Researchers used single-molecule imaging to compare the genome-editing tools CRISPR-Cas9 and TALEN. Their experiments revealed that TALEN is up to five times more efficient than CRISPR-Cas9 in parts of the genome, called heterochromatin, that are densely packed. Credit: Composite photo by L. Brian Stauffer CHAMPAIGN, Ill.

Genome 52

Genome Genomics DNA Research 52

Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. These therapies involve modifying a patient’s cells or genes to treat or prevent diseases, with the promise of long-lasting effects.

Gene Therapy 52

Gene Therapy Gene Medicine In-Vitro 52

XTalks

MAY 22, 2024

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

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

XTalks

JANUARY 21, 2025

The Shield test provides a non-invasive approach to colorectal cancer screening by analyzing blood for key DNA changes linked to cancer. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals. The diagnostic tests for more than 500 genes to profile tumors.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

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.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

XTalks

DECEMBER 20, 2023

Beam Therapeutics : A company focused on base editing, a more precise form of CRISPR editing that allows for single DNA base changes without making double-stranded breaks in the DNA. In 2024, we are likely to see more advancements and potentially new CRISPR therapies emerging from these and even more companies in the field.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

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

Roots Analysis

FEBRUARY 23, 2022

Over the past few years, several companies have started offering a diverse range of genome sequencing products and services using various second and third generation sequencing technologies. Currently, close to 60 industry players are actively offering NGS kits for preparing DNA / RNA libraries. Players Engaged in the Domain.

Genetics 52

Genetics DNA RNA Reagent 52

Cloudbyz

JUNE 26, 2023

Patient Risk Stratification: Generative AI techniques can analyze patient data, including electronic health records (EHRs) and genomic information, to identify individuals at higher risk for certain diseases or adverse events. This can lead to shorter trial durations, reduced costs, and more accurate evaluation of treatment efficacy.

Clinical Research 97

Clinical Research Research Clinical Trials Clinical Trials 97

Pharmaceutical Technology

FEBRUARY 3, 2023

The field of genomic medicine has reached a true turning point. With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

Genome 244

Genome Genomics Medicine Gene Therapy 244

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 98

Gene Therapy Gene Gene Editing DNA 98

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.”. The genomic medicine journey.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

pharmaphorum

OCTOBER 12, 2022

David Del Bourgo (CEO and co-founder, Whitelab Genomics) has always been passionate about introducing disruptive, innovative technologies to markets. We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says.

Genome 105

Genome Genomics Medicine Development 105

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

Pharmaceutical Technology

MARCH 29, 2023

Pharmaron is receiving a £151 million investment in capital to support the growth of operations in Liverpool, quadrupling production capacity for gene therapy and vaccine components. The government is giving Touchlight £14 million for commercial scale manufacturing of its “doggybone DNA” vector at its Hampton, London base.

Life Science 276

Life Science Manufacturing Gene Therapy Medicine 276

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.

DNA 98

DNA Gene Gene Silencing Genome 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

Cloudbyz

MARCH 6, 2024

Unlike traditional small molecule drugs, which are chemically synthesized, biologics are produced through complex biotechnological processes, often involving recombinant DNA technology. This allows for the creation of highly specific and targeted therapies that can modulate biological pathways with remarkable precision.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

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

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

MARCH 8, 2022

From leading COVID-19 vaccine developments to being at the helm of the latest gene therapies and inventing ground-breaking DNA technologies like CRISPR, women have been at the forefront of some of the most leading-edge scientific discoveries in recent years, both as innovators and leaders.

Life Science 105

Life Science Gene Therapy DNA Vaccination 105

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., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

Pharmaceutical Technology

FEBRUARY 20, 2023

This may lead to therapies and dosing regimes being less suitable for minority populations – and according to Hans Bunschoten, chief strategy officer at clinical research company Cerba Research, part of Cerba Healthcare, this is a major problem. The technology platforms are almost the same.

Research 130

Research Clinical Trials Clinical Trials Trials 130

Advarra

OCTOBER 10, 2024

These resistance genes are commonly found on small circles of DNA called plasmids. Phages deliver their genetic material into the bacteria so, when the bacteria flourishes and reproduces, the bacteria also copies the viral genome and hands it down to bacterial progeny.

Bacteria 52

Bacteria Genetics Genetic Engineering Gene 52

FDA Law Blog

DECEMBER 15, 2024

Valentine We recently published the first part of our review of FDAs draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. If you make a tissue product you may produce one lot per patient where a gene therapy may only need a handful of lots through Phase 3.

Gene Therapy 52

Gene Therapy Gene Production Development 52

Roots Analysis

JANUARY 27, 2025

Gene Identification: The process starts with identifying the gene that codes for the desired protein i.e. gene of interest. This can be done through various techniques such as PCR, DNA sequencing or gene synthesis.

Protein 40

Protein Reagent Marketing Gene 40

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. Infectious disease expert, epidemiologist and director of the Yale Institute for Global Health at Yale University, Saad B.

Life Science 111

Life Science Vaccination Vaccine Gene 111