Pharmaceutical Technology

JANUARY 12, 2023

The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

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. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

Drugs 76

Drugs Life Science Gene Therapy Genome 76

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

APRIL 27, 2023

The first quarter of 2023 has seen deal flow continue for UK life science and biotech companies at all stages of development, new data from the UK BioIndustry Association and Clarivate shows. million to advance its gene therapy platform to treat cancer. million across 20 deals, with an average deal size of £13 ($16.1)

Life Science 52

Life Science Gene Therapy Bacteria RNA 52

Drug Discovery World

OCTOBER 4, 2022

Taking place at the ExCel in London from 4-5 October, ELRIG Drug Discovery 2022 is set to provide visitors with access to the latest trends, technologies and expertise driving the life sciences sector. . Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7 billion, compared to $19.9

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

XTalks

OCTOBER 26, 2021

Related: Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA. AHP is a life-threatening genetic condition caused by an inherited deficiency in the metabolic enzyme hydroxymethylbilane synthase (HMBS), also known as porphobilinogen deaminase (PBGD).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

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

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA).

RNA 52

RNA Protein Vaccination Vaccine 52

XTalks

MAY 29, 2024

Novartis also purchased siRNA-focused DTx Pharma for $500 million upfront, which includes a preclinical therapy for an inherited neuromuscular disorder. RNA, radioligand and cell and gene therapy are three technology platforms that Novartis is focused on besides biologics and traditional small molecules.

Pharma Companies 52

Pharma Companies Sales FDA Approval Vaccination 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

XTalks

DECEMBER 14, 2022

We wanted to work toward what gene therapy was supposed to do at its original inception, which is to replace genes, not just correct individual mutations.”. The PASTE gene editing technique was recently published in Nature Biotechnology. How does the Gene Editing Tool PASTE Work?

Gene Editing 98

Gene Editing DNA Gene Gene Sequencing 98

pharmaphorum

AUGUST 22, 2022

” For example, Torrance recently met with an expert from the Broad Institute who has been looking into gene therapies and says gene therapy can completely change how drugs are built. We identify what trends we want to look into, and we’ll come together and think about themes driving change. About the author .

Gene Therapy 102

Gene Therapy Antibody Gene Medicine 102

Drug Discovery World

AUGUST 8, 2023

It was commissioned in 1999 and is considered India’s first structured hub for life sciences R&D. million) at the city’s life sciences hub 14. million) at the city’s life sciences hub 14. The growth and potential in Genome Valley are expressed in the continuous investment in the hub.

Research 52

Research Drugs Genome Genomics 52

Drug Discovery World

AUGUST 10, 2022

Unlike qPCR, which requires users to generate a standard curve to estimate the concentration of DNA or RNA in a sample, ddPCR technology quantifies nucleic acids directly. They’ve also induced lymphomas in humans when delivered directly in the form of gene therapy 17. Gene Therapy. Marwan obtained his M.S.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

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. The system shows potential as a therapy against multiple melanomas.

DNA 98

DNA Genome Genomics RNA 98

XTalks

JUNE 4, 2024

Today, there’s been a shift towards more complex, large molecules or biologics like monoclonal antibodies, antibody-drug conjugates (ADCs) and advanced therapies like RNA-based treatments and cell and gene therapies. Our mission at Fortrea is to deal with and manage the complexity in the space,” says Dr.

Clinical Development 110

Clinical Development Development Clinical Trials Clinical Trials 110

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. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. Epigenetic Editing with CRISPR.

DNA 98

DNA Gene Gene Silencing Genome 98

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

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. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. However, a drawback is that the dose and neuronal specificity of gene therapy is still poorly understood even in translational models.

Trials 83

Trials Drug Delivery Drugs Gene 83

XTalks

DECEMBER 24, 2020

The pandemic propelled the life science and healthcare sectors onto center stage, and they rose to the occasion against the most unprecedented health challenge in recent times. While COVID-19 has undoubtedly been the biggest story in the life science industry in 2020, it was a busy and positive year in many other areas.

Life Science 111

Life Science Vaccination Vaccine Gene 111

Drug Discovery World

NOVEMBER 13, 2023

The track will be opened by chairperson remarks from Daniel Chen, MD, PhD, Founder, Engenuity Life Sciences, which will be followed by the track’s keynote presentation by Bent Jakobsen, PhD, FMedSci, CEO & Founder, Accession Therapeutics. It is called, ‘An unexpected future for immune oncology therapies?’

Protein 59

Protein Antibody Engineer Scientist 59