Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

pharmaphorum

MARCH 9, 2022

billion takeover of Bioverativ in 2018 – is extended in the body by fusing it with von Willebrand factor (vWF), a protein that binds to factor VIII in the blood and makes it more stable, protecting it from degradation. The half life of the new drug – one of the main draws for Sanofi’s $11.6

Protein 98

Protein Gene Therapy Sales Regulation 98

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

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

AUGUST 5, 2021

billion upfront to claim ownership of Vividion ‘s drug discovery platform, which uses chemoproteomics to identify new binding sites on proteins and find small-molecule compounds that interact with them, with another $500 million on the table if pipeline candidates meet targets. ” Bayer is paying $1.5 billion ($5.3

DNA 52

DNA Gene Therapy Protein Gene 52

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

Marketing 52

Marketing Gene Drugs Protein 52

pharmaphorum

JULY 11, 2022

Sanofi R&D in its core haemophilia category is advancing on three fronts – engineered proteins, RNA interference drugs and gene therapies – and highlighted results in two of those at the International Society on Thrombosis and Haemostasis (ISTH) congress over the weekend.

Engineer 52

Engineer Gene Therapy Sales Drugs 52

The Pharma Data

MARCH 5, 2021

This will be adapted to the needs of messenger RNA vaccine production for CureVac’s CVnCoV, the manufacturing of which is highly complex. At the Kundl site, Novartis is a pioneer and has decades of experience in pharmaceutical production of proteins and in more recent years of nucleic acids.

Manufacturing 52

Manufacturing Vaccination Vaccine Production 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. What About Small Proteins and Antibodies Reaching Extracellular CNS Targets? Next Steps for Targeted CNS Delivery.

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Trials Drug Delivery Drugs Gene 83

Pharmaceutical Technology

APRIL 20, 2023

Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Initially reported in 2011, CircRNA is a naturally occurring class of RNA. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 240

Gene Therapy RNA Gene Development 240

Pharmaceutical Technology

APRIL 27, 2023

Orbital Therapeutics has raised $270m in a Series A round led by ARCH Venture Partners to advance a portfolio of programmable RNA therapeutics. Orbital will use the new funding to increase the application of RNA-based medicines for use in the fields of new vaccines, immunomodulation and protein replacement.

RNA 130

RNA Medicine Gene Therapy Gene 130

XTalks

APRIL 1, 2025

Management typically involves on-demand treatment or preventive (prophylactic) therapy using FVIII or FIX replacement products, or agents that mimic the function of these factors. Qfitlia differs from traditional factor replacement therapies. Among these are three gene therapies.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Pharmaceutical Technology

FEBRUARY 1, 2023

A major breakthrough from the National Institutes of Health (NIH), in mapping the structure of the RSV protein significantly changed the landscape, allowing companies to leap into RSV drug research with better efficacy results. However, subsequent RSV drugs, like Medimmune’s motavizumab, failed in clinical trials.

Vaccination 162

Vaccination Vaccine Antibody Development 162

XTalks

NOVEMBER 30, 2023

Its mechanism of action involves blocking the protein shell (capsid) of the HIV-1 virus, disrupting multiple crucial stages of the viral lifecycle. 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.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Roots Analysis

AUGUST 31, 2023

Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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Clinical Development Gene Therapy Development Gene 52

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

Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Beyond our expertise in small molecules, we have built a solid reputation with biopharmaceutical companies for the characterisation and analysis of monoclonal antibodies, therapeutic proteins and biosimilars.

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Development Production Manufacturing Vaccination 130

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

Pharmaceutical Technology

APRIL 27, 2023

GlobalData pharmaceutical analysts project that RNA-based gene therapies for oncology will grow from zero in 2022 to $4.6 Makowiecki says the same benefits apply to the development and manufacture of RNA-LNPs as in other biopharmaceutical manufacturing, while accommodating for their unique characteristics.

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Manufacturing RNA Contamination Vaccination 130

The Pharma Data

JANUARY 10, 2021

FBX-101 is a first-in-human AAV gene therapy. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1.

Gene Therapy 52

Gene Therapy Trials Gene Clinical Trials 52

Delveinsight

SEPTEMBER 23, 2021

Boehringer stated that the Abexxa’s generation aims at cancer-specific proteins situated throughout the mobile versus proteins at the mobile membrane broadening the selection of possible cancer antigen objectives. 858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Jeffrey Stafford, Ph.D.,

RNA 61

RNA Gene Therapy Gene Protein 61

Worldwide Clinical Trials

MAY 30, 2024

Tofersen (Intrathecal injection) This is an antisense oligonucleotide (ASO) that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient.

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Gene Clinical Trials Clinical Trials Gene Therapy 204

pharmaphorum

NOVEMBER 24, 2021

Here’s a snapshot of our world class speaker line-up: Andreas Kuhn, Senior VP – RNA Biochemistry & Manufacturing, BioNTech . Elaine Peters, Director, Analytical – Individualized Cell & Gene Therapies, Genentech. Danilo Casimiro, CSO, Sanofi Pasteur. Patrick Finn, Senior Director – Rare Diseases, Moderna.

Vaccination 52

Vaccination Vaccine Gene Therapy RNA 52

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. The drug is an autologous IL13Ralpha2-CAR-T cell product.

Trials 52

Trials Antibody Gene Therapy Gene 52

XTalks

AUGUST 2, 2023

Johnson & Johnson’s Top 5 Best-Selling Drugs of 2022: 1) Stelara (ustekinumab) Stelara is an immunosuppressant biologic therapy that blocks the IL-12 and IL-23 proteins that play a role in plaque psoriasis and Crohn’s disease. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

Roots Analysis

MAY 21, 2024

LNPs comprise of a lipid bilayer that surrounds a hydrophobic core, which can be loaded with therapeutic agents ( such as drugs, genetic material and proteins ). 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.

Drug Delivery 40

Drug Delivery Drugs Gene Therapy Bioavailability 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.

Life Science 111

Life Science Vaccination Vaccine Gene 111

XTalks

JANUARY 6, 2025

From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111