Pharmaceutical Technology

JULY 29, 2022

And we are very prepared and have data that will answer those questions, what would be the advantage of shifting to an Omicron-containing vaccine, whether it's monovalent or bivalent.” Although the UK has fewer facilities, around 70% of those offer dose manufacturing with containment.

Manufacturing 130

Manufacturing Vaccination Vaccine Packaging 130

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

APRIL 13, 2021

synuclein, a protein involved in the formation of amyloid deposits in the brain characteristic of Parkinson’s disease and other neurodegenerative disorders, is also implicated in melanoma. The scientists found that the amyloid-forming protein is expressed at higher levels in melanoma cells than in normal healthy skin. In addition to ?-synuclein,

Research 98

Research Protein In-Vitro Gene Therapy 98

Drug Discovery World

OCTOBER 27, 2023

With cues from a diverse population of tissue-specific cell types, extracellular matrix proteins, and biomechanical forces, cells grown in organ-chips closely emulate their in vivo counterparts and replicate tissue-level functions. How do organ-chips support gene therapy delivery? What are the limitations of the technology?

Research 73

Research Gene Therapy In-Vivo In-Vitro 73

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

Gene Therapy 98

Gene Therapy Gene Medicine Trials 98

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

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.

Development 130

Development Manufacturing Production Vaccination 130

Drug Discovery World

JULY 26, 2022

RA: Could this type of treatment be used alongside other oncology therapies such a cell & gene therapies? BM: Oncolytic viruses can be used in combination with other cancer treatments, including cell & gene therapies. The virus is allowed to replicate within the cells, which are then frozen.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 52

The Pharma Data

NOVEMBER 22, 2020

They use mRNA that codes for the spike protein in the SARS-CoV-2 virus, which is embedded in a nanolipid particle—basically a minuscule fat molecule. When injected into the body, cells churn out the spike protein, which causes the body’s immune system to recognize the virus. It contains the genetic materials of the spike protein.

Vaccination 52

Vaccination Vaccine Protein Genetics 52

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

Drug Discovery World

JANUARY 9, 2024

2024 is anticipated to be a transformational year for the pharma industry, with new modalities like antibody drug conjugates and AI/machine learning providing therapies for patients with limited treatment options.

Drugs 69

Drugs Antibody Gene Editing Medicine 69

Roots Analysis

APRIL 15, 2024

These complex entities, such as antibodies, antibody drug conjugates, cell therapies, gene therapies, and therapeutic proteins, are highly specific molecules that are designed to precisely target biomarkers associated with a particular disease pathway.

Manufacturing 40

Manufacturing Production Contract Manufacturing Contamination 40

Advarra

MARCH 10, 2023

Red blood cells contain hemoglobin, a protein that carries oxygen. These are reasons for biotech organizations to renew a focus on improving diversity in clinical research while bringing more sickle cell gene therapies to market.

Clinical Trials 98

Clinical Trials Clinical Trials Trials Doctors 98

The Pharma Data

MARCH 5, 2021

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. This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Disclaimer. Source link:[link].

Manufacturing 52

Manufacturing Vaccination Vaccine Production 52

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

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

The Pharma Data

OCTOBER 21, 2020

Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, today announced the pricing of its public offering of 4,687,500 shares of its common stock at a public offering price of $40.00 BOSTON, Oct. 22, 2020 (GLOBE NEWSWIRE) — Replimune Group, Inc.

Gene Therapy 40

Gene Therapy Immune Response Sales Gene 40

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Host cell proteins (HCPs) – proteins derived from the host cells used for viral production—are one class of process-related impurities.

Production 52

Production Gene Protein Vaccination 52

Drug Discovery World

AUGUST 10, 2022

To overcome this evasion tactic, the CAR protein was developed to recognise other markers on cancer cells. CAR is a fusion protein composed of an extracellular antibody fragment that recognises antigens and an intracellular domain that directs the T cell to the tumour. Identifying the right CAR T cell target.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. About Passage Bio.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

JUNE 26, 2021

4 Ranibizumab is a vascular endothelial growth factor (VEGF) inhibitor designed to bind to and inhibit VEGF-A, a protein that is believed to play a critical role in the formation of new blood vessels and the leakiness of the vessels. 13 PDS contains a customised formulation of ranibizumab not approved by regulatory authorities.

Drug Delivery 52

Drug Delivery Clinical Trials Clinical Trials Medicine 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

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Drug Discovery World

OCTOBER 12, 2022

Professor Michael Butler, head of the National Institute for Bioprocessing Research and Training (NIBRT) Cell Technology Group (CTG), explains that the characterisation and analysis of glycans is important for the biopharma sector as it can help in the development of therapeutic agents such as monoclonal antibodies and other recombinant proteins.

Protein 52

Protein Gene Therapy Antibody Production 52

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Roche’s Chief Medical Officer and Head of Global Product Development.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Drug Discovery World

FEBRUARY 8, 2023

Svea Cheeseman , Refeyn, explains why better efficiency is needed to advance the production of viral vectors for use in gene therapies. Adeno-associated viruses (AAVs) are a promising, widely used vector for delivering gene therapies. Data were provided by the Cell and Gene Therapy Catapult (London, UK).

Gene Therapy 52

Gene Therapy Gene Antibody Production 52

The Pharma Data

AUGUST 25, 2020

In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. In certain countries outside the U.S., Cautionary Statement Regarding Forward-Looking Statements.

Trials 52

Trials FDA Approval Development Clinical Trials 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. It’s a great tool for controlling gene expression.”. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

The Pharma Data

OCTOBER 14, 2020

About VBL’s VB-600 Platform VBL is conducting two parallel drug development programs that are exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases and cancer.

Antibody 52

Antibody Gene Therapy Containment Clinical Trials 52

The Pharma Data

JANUARY 3, 2021

The vaccine contains a full-length, pre-fusion spike protein and the company’s Matrix-M adjuvant. AT132 is an AAV8 vector containing a functional copy of the MTM1 gene. Eprenetapopt is a small molecule that reactivates the mutant and inactivated p53 protein. Vyrologix is a CCR5 antagonist. and Mexico.

Trials 52

Trials Protein Containment Vaccination 52

XTalks

MAY 15, 2023

Protalix BioTherapeutics focuses on the production, development and commercialization of recombinant therapeutic proteins. Elfabrio is available as a clear and colorless solution in single-dose vials, containing 20 mg/10 mL (2 mg/mL) of pegunigalsidase alfa-iwxj. The administration of Elfabrio is performed by a healthcare professional.

FDA Approval 98

FDA Approval Gene Genetics Clinical Trials 98

Drug Discovery World

JUNE 14, 2023

One example we are following with anticipation are the two FDA approvals for the first gene therapies for sickle cell disease. We read every day about biotech’s and their work to develop new treatment options. umbilical cord blood, leukopaks, HSCs, MSCs, PBMCs, NK, and T cells).

Manufacturing 52

Manufacturing Production Gene Therapy Development 52

Pfizer

SEPTEMBER 28, 2022

Monovalent refers to any authorized and approved COVID-19 vaccine that contains or encodes the spike protein of only the Original SARS-CoV-2 virus . The information contained in this release is as of September 28, 2022. . • receipt of the most recent booster dose with any authorized or approved monovalent COVID-19 vaccine. .

Vaccination 101

Vaccination Vaccine Medicine Clinical Trials 101

XTalks

AUGUST 2, 2023

It contains the antiviral medications nirmatrelvir and ritonavir. 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. billion in 2022. billion in 2021.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

Pfizer

OCTOBER 19, 2022

Monovalent refers to any authorized and approved COVID-19 vaccine that contains or encodes the spike protein of only the Original SARS-CoV-2 virus. The information contained in this release is as of October 19, 2022. The European Commission will review the CHMP recommendation and is expected to make a final decision soon.

Vaccination 40

Vaccination Vaccine Clinical Trials Clinical Trials 40

Drug Discovery World

DECEMBER 15, 2022

Potential solutions: For CRISPR systems, the likelihood of off-target editing has been shown to be influenced by a variety of factors, ranging from the design of the gRNA 1 and Cas9 protein 2 , to the targeted cell type 3 , to name a few examples. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52