Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

Gene Editing 235

Gene Editing DNA Gene Gene Therapy 235

Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Limitations of AAV gene therapy . Sahay and Ryals have received a $3.2

Gene Editing 98

Gene Editing Gene Gene Therapy Genetics 98

Drug Discovery World

JUNE 22, 2023

“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.

Gene Therapy 52

Gene Therapy Gene Editing Drug Delivery Gene 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 57

Gene Therapy Gene Antibody FDA Approval 57

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. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 52

XTalks

NOVEMBER 30, 2023

Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV. percent in the placebo group.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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

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

DNA 98

DNA Genome Genomics RNA 98

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. greater safety, transient and reversible effect on gene expression). References.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Delveinsight

SEPTEMBER 14, 2021

ADARx bags USD 75 Million to advance its RNA tech pipeline. ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. SR One Capital Management and OrbiMed Advisors co-led Series B. Simeon George, M.D.,

RNA 67

RNA Clinical Trials Clinical Trials Drugs 67

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

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

Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. billion in funding. Cambridge is a focal point for many pharmaceutical companies working in this therapeutic area.

Drugs 73

Drugs Life Science Gene Therapy Genome 73

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenetic Editing with CRISPR.

DNA 98

DNA Gene Gene Silencing Genome 98

Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. 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

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

FEBRUARY 22, 2024

Indeed, we have shown that the causative mutations of some leukodystrophies affect the subunits of an important cellular enzyme, RNA polymerase III, preventing its normal assembly – it turned out that Riluzole can counteract this assembly defect,” says Maxime Pinard, the researcher responsible for the project in Coulombe’s lab. ‘’For

Drugs 57

Drugs Gene Clinical Trials Clinical Trials 57

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. billion in 2022.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

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