pharmaphorum

APRIL 28, 2022

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. The industry is now poised to exploit the full therapeutic potential of these vehicles for emerging payloads and extrahepatic delivery.

RNA 52

RNA Drug Delivery Engineer Gene Therapy 52

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. In addition, Vir Biotechnology, Inc.

FDA Approval 110

FDA Approval Gene Therapy Gene Clinical Trials 110

Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient.

Gene Therapy 130

Gene Therapy Gene Marketing Manufacturing 130

Scienmag

JULY 14, 2021

Researchers have found a way to enhance radiation therapy using novel iodine nanoparticles Credit: Mindy Takamiya/Kyoto University iCeMS Cancer cell death is triggered within three days when X-rays are shone onto tumor tissue containing iodine-carrying nanoparticles. The findings, by scientists at Kyoto […].

DNA 97

DNA Containment Scientist Research 97

NY Times

FEBRUARY 22, 2021

Two patients in a gene therapy study developed cancer years after treatment. It is not clear whether the therapy was responsible.

Gene Therapy 54

Gene Therapy Trials Gene Research 54

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

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 103

Life Science Gene Therapy DNA Vaccination 103

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. Since 2017, the US Food and Drug Administration (FDA) has approved six CAR T-cell therapies.

Research 109

Research Gene Editing Gene Genetics 109

Scienmag

JULY 2, 2021

The ongoing effort to explore […].

Gene Silencing 75

Gene Silencing Gene Editing Gene DNA 75

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.”.

Genome 105

Genome Genomics Medicine Development 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

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

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.

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Gene Gene Therapy Genetics DNA 262

Pharmaceutical Technology

JANUARY 10, 2023

The multi-year collaboration will focus on discovering precision genetic medicines by developing a new platform for complete gene insertion by delivering a single vector of gene editing and DNA cargo. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review? What Does an IBC Review?

Genetic Engineering 98

Genetic Engineering Gene Therapy Genetics Engineer 98

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. From a technical standpoint, such diseases should be easier to treat with current gene therapy technology.

Gene Therapy 52

Gene Therapy Development Gene Drugs 52

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

STAT News

NOVEMBER 4, 2022

Many scientists hope that the approach proves to be a safer alternative to gene editing therapies that make permanent changes to DNA itself. One aspect of the therapy that was not entirely new was the use of engineered viruses to deliver instructions for the gene editing therapy into Terry’s cells.

Gene Editing 105

Gene Editing Gene Gene Therapy Scientist 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

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 96

DNA Gene Gene Silencing Genome 96

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.

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 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

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

DECEMBER 27, 2020

Taysha Gene Therapies announced that Queen’s University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its Phase I/II trial of TSHA-101 for infantile GM2 gangliosidosis. RESTORE-1 is a Phase II trial of an intracerebral AAV-based gene therapy for Parkinson’s disease.

Trials 52

Trials Antibody Gene Therapy Gene 52

Advarra

OCTOBER 10, 2024

These resistance genes are commonly found on small circles of DNA called plasmids. Figure 3 illustrates some of these examples: Bacteria can secrete enzymes that inactivate antibiotics (e.g., Bacteria can exchange plasmids through conjugation, a process where bacteria connect with a sex pilus and plasmids are shared.

Bacteria 52

Bacteria Genetics Genetic Engineering Gene 52

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 108

Life Science Vaccination Vaccine Gene 108

Pharmaceutical Technology

APRIL 24, 2023

Langer’s engineering lab was instrumental in the development of these lipid nanoparticles, playing into his role in co-founding Moderna. Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Lipid nanoparticle drug delivery was in the spotlight during the Covid-19 pandemic.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147