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

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

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

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 40

Antibody Gene Therapy Containment Clinical Trials 40

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

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

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

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

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

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

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

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

Gene Therapy 97

Gene Therapy Gene Manufacturing Clinical Trials 97

The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris).

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 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 Gene 52

Pharmaceutical Technology

APRIL 24, 2023

Biologics are costly to produce and sell, and the more recent modalities, such as cell and gene therapies, can be difficult to manufacture. There are fewer sites for cell and gene therapies and vaccines – 90 companies with 127 facilities, as per GlobalData figures.

Contract Manufacturing 130

Contract Manufacturing Manufacturing Protein Sales 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. It’s a great tool for controlling gene expression.”. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

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

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.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

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

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.”. Epigenome Editing with CRISPR. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

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 Production Manufacturing Vaccination 130

The Pharma Data

NOVEMBER 30, 2020

The AstraZeneca-Oxford vaccine leverages a viral vector using a weakened version of a common cold (adenovirus) virus that contains the genetic materials of SARS-CoV-2 spike protein. The adenovirus is what is typically used in gene therapies.

Vaccination 52

Vaccination Vaccine Regulation Trials 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

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

The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. This indication is approved under accelerated approval based on tumor response rate and duration of response.

Trials 52

Trials Hormones Clinical Trials Clinical Trials 52

The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. This indication is approved under accelerated approval based on tumor response rate and duration of response.

Trials 52

Trials Hormones Clinical Trials Clinical Trials 52

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

The Pharma Data

SEPTEMBER 15, 2020

20vPnC showed a safety and tolerability profile that was similar to Prevnar 13 ® Pneumococcal 13-valent Conjugate Vaccine [Diphtheria CRM197 Protein]. Based on the acceptable safety profile and the favorable immune response data, including the 4th dose response data, Pfizer received Breakthrough Therapy Designation.

Gene Therapy 52

Gene Therapy Vaccination Vaccine Trials 52

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

The Pharma Data

APRIL 27, 2021

A new study to select the most appropriate antigen dosage for Phase 3 evaluation of an adjuvanted recombinant protein COVID-19 vaccine candidate (SP0253) was initiated and already completed enrollment. This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended.

Sales 52

Sales Vaccination Vaccine Medicine 52

FDA Law Blog

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.

Gene Therapy 59

Gene Therapy Gene Production In-Vivo 59

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

The Pharma Data

DECEMBER 20, 2020

It is an optimized, non-chemically modified mRNA that codes for the prefusion stabilized full-length spike protein of the SARS-CoV-2 virus. The therapy is remestemcel-L, which contains culture-expanded mesenchymal stromal cells derived from the bone marrow of an unrelated donor. FLT180a is an AAV gene therapy.

Trials 52

Trials Gene Therapy Vaccination Vaccine 52