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 RNA 40

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

DNA 56

DNA Gene Gene Editing Scientist 56

Drug Discovery World

SEPTEMBER 25, 2023

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers.

Genome 52

Genome Genomics Gene Editing Medicine 52

Drug Discovery World

JANUARY 17, 2023

Limitations of AAV gene therapy . They will lead research into using LNPs to deliver a gene editing tool that could delete bad genes in the photoreceptor cells and replace them with correctly functioning genes. . Sahay and Ryals have received a $3.2

Gene Editing 98

Gene Editing Gene Gene Therapy Genetics 98

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Selim Corbacioglu from the University of Regensburg, relies on CRISPR-Cas9 gene editing to switch off BCL11A.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 52

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

Gene Therapy 59

Gene Therapy Gene Antibody FDA Approval 59

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

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

Research 118

Research Gene Editing Gene Genetics 118

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

Gene Editing 98

Gene Editing DNA Gene Gene Sequencing 98

Scienmag

JULY 2, 2021

The ongoing effort to explore […].

Gene Silencing 75

Gene Silencing Gene Editing Gene DNA 75

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

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

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 81

Gene Therapy Gene Gene Editing DNA 81

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 83

Gene Immune Response Manufacturing Gene Therapy 83

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 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

Drug Discovery World

JULY 5, 2024

The last few days have seen some interesting developments related to gene editing, including the discovery of a new mechanism for genetic programming and evidence in favour of redosing CRISPR-based therapies, as well as significant investment and new indications for gene therapies.

Gene Therapy 59

Gene Therapy Gene Editing In-Vivo Gene 59

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The treatment used a new version of CRISPR to turn on a backup copy of the dystrophin gene that mysteriously lies dormant in our muscles.

Gene Editing 105

Gene Editing Gene Gene Therapy Clinical Trials 105

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

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. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 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. . It’s estimated that one in 250 people worldwide are affected by genetic cardiomyopathies, with a 50:50 risk they will pass their faulty genes on to each of their children. .

Gene Editing 52

Gene Editing Gene Research Genetics 52

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

Gene Editing 52

Gene Editing Gene Engineer DNA 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.

DNA 52

DNA Gene Gene Silencing Genome 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. Additionally, the CRISPR-Cas system can be used to create TCR T cells that can specifically recognise these neoantigens, expanding its applications beyond traditional CAR T therapy.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

MAY 30, 2023

The intersection of cell, protein, RNA and DNA analysis at the single cell level has advanced significantly over the last five years and is particularly informative to our understanding of cancer biology and drug responsiveness. One of the most exciting recent developments is the use of gene editing tools to create cell and gene therapies.

Research 52

Research Drugs Protein RNA 52

XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. Onureg (azacitidine), a leukemia therapy, also saw large growth of 70 percent to $124 million in 2022, and the multiple sclerosis drug Zeposia (ozanimod) generated $250 million in 2022, an 87 percent increase.

Sales 98

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. CRISPR Gene Editing Inventors Win Nobel Prize.

Life Science 111

Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

MAY 26, 2023

Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.

DNA 130

DNA Manufacturing Gene Therapy Gene 130

Pharmaceutical Technology

FEBRUARY 20, 2023

Gene therapy, while in its infancy, could be a gamechanger for cancer treatment and certain rare diseases and advancements in gene-editing technology relies on knowledge of viral factors. In addition, safety oncology and immunotherapy, which is a focus area for Cerba Research has close ties to virology.

Research 130

Research Clinical Trials Clinical Trials Trials 130