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., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

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. David Williams, uses a virus to introduce RNA into the extracted bone marrow that turns off BCL11A. SATURDAY, Dec.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 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 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 Genomics 98

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

pharmaphorum

APRIL 28, 2022

Harness Safe & Efficacious Lipid-Based Delivery for Novel Therapeutics Beyond RNA & Hepatic Tissue. The Next Generation Lipid-Based Nanoparticles Delivery Summit is your definitive industry-led meeting to design and engineer 2nd generation of lipid nanoparticles for emerging payloads beyond RNA into clinic.

RNA 52

RNA Drug Delivery Engineer Gene Therapy 52

Roots Analysis

FEBRUARY 23, 2022

Further, NGS library preparation kits offer several benefits, such as low requirement of starting material, generation of longer RNA / DNA sequences and production of superior quality nucleotide libraries, thereby, catering to existing unmet needs of the research domain. Next Generation Sequencing (NGS) Library Preparation Kits.

Genetics 52

Genetics DNA RNA Reagent 52

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

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 Vaccine 105

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.

Vaccine 52

Vaccine Vaccination DNA Protein 52

XTalks

JANUARY 8, 2024

Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. Getting a drug to market used to be a relatively simple and straightforward affair. That’s just not the case anymore. Her advice revolves around two main strategies.

Clinical Trials 105

Clinical Trials Clinical Trials Trials Gene Therapy 105

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.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines.

Genetics 52

Genetics Medicine RNA Scientist 52

pharmaphorum

OCTOBER 12, 2022

The payload, Del Bourgo tell us, is the therapeutic DNA or RNA sequence that will cure or fix damaged cells. Inactive viruses often act as the viral vector, as they’re able to deliver genetic therapies efficiently. and France to accelerate the development of genomic therapies. So far, we’ve seen promising results.”.

Genomics 105

Genomics Genome Medicine Development 105

Roots Analysis

MAY 21, 2024

Several LNP-based therapies are currently being evaluated in the clinical trials; majority of these are conducted for treating breast cancer, ovarian cancer and lung cancer. In addition, these can be modified with ligands in order to improve the specificity and selectivity of the gene therapy and reduce off-target effects.

Drug Delivery 40

Drug Delivery Drugs Gene Therapy Bioavailability 40

pharmaphorum

AUGUST 5, 2021

Vividion’s in-house R&D efforts are still at the preclinical stage, but include drug candidates in oncology and immune diseases targeting transcription factors – which are involved in the process of transcribing DNA into RNA and thereafter into proteins. billion ($5.3

DNA 52

DNA Gene Therapy Protein Gene 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 Genomics 52

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Gene Delivery Systems Genetic material, in the form of DNA or RNA, does not easily enter cells without the aid of a delivery system.

Engineer 52

Engineer Genetics Clinical Research Research 52

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression.

Trials 52

Trials Antibody Gene Therapy Gene 52

WCG Clinical

MAY 28, 2024

In contrast to B cell malignancies, a hallmark of lupus is not uncontrolled cellular proliferation, but rather uncontrolled production of antibodies that bind to “self” targets — in particular to nucleic acids (DNA and RNA). WCG has many ways to support cell and gene therapy clinical trials for lupus.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

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

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.

Life Science 111

Life Science Vaccine Vaccination Gene 111

Pharmaceutical Technology

APRIL 24, 2023

Since then, the field of nanomedicine has steadily progressed to reach high points such as the successful use of nanotechnology to deliver messenger RNA (mRNA)-based Covid-19 vaccines. Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Langer, now a David H.

Drug Delivery 147

Drug Delivery Vaccine Vaccination Research 147

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. The most obvious is that both firms are highly scientific and so that presents a lot of opportunity to exchange knowledge and research.

Research 130

Research Clinical Trials Clinical Trials Trials 130