pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin. Eventually, they will need assistance with breathing.

Gene Therapy 98

Gene Therapy Gene FDA Approval DNA 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

Delveinsight

AUGUST 6, 2020

The Indian company informed that the DNA vaccine was well tolerated in the first stage of the adaptive phase 1/2 trial. ZyCoV-D is designed to introduce DNA that encodes for a viral protein into human cells. By ordering human cells to make the protein, ZyCoV-D could cause an immune response against the coronavirus.

Allergies 53

Allergies Vaccination Vaccine Gene Therapy 53

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

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

pharmaphorum

APRIL 7, 2021

The collaboration will focus on combining Artios’ DNA damage response (DDR) drug candidates with Novartis’ radioligand therapies, which consist of tumour-targeting molecules carrying radioactive isotopes that can deliver a cell-killing dose of radiation to malignant cells. It’s also providing research funding.

DNA 52

DNA Licensing Licensing Gene Therapy 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

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

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

pharmaphorum

JANUARY 29, 2021

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Macrae explains. “So Most of them are rare mutations.”.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Delveinsight

DECEMBER 3, 2020

Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

Pharmaceutical Technology

JUNE 8, 2023

GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. The core objective of the collaboration is to make the therapies affordable and accessible to patients.

Gene Therapy 130

Gene Therapy Gene Development Antibody 130

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 98

DNA Gene Gene Silencing Genome 98

Cloudbyz

MARCH 6, 2024

Understanding Biologics: Biologics are a class of therapeutic agents derived from living organisms, such as cells, tissues, or proteins. Unlike traditional small molecule drugs, which are chemically synthesized, biologics are produced through complex biotechnological processes, often involving recombinant DNA technology.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

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

DECEMBER 4, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies.

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Gene Therapy Gene Clinical Trials Clinical Trials 69

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. This candidate, derived from a person’s own genetically modified cells, aims to restore the Gag-specific CD4+ T cell response in those receiving antiretroviral therapy.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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

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

STAT News

NOVEMBER 4, 2022

His goal was to find a treatment for his brother’s form of Duchenne muscular dystrophy, which is caused by a mutation that impeded his ability to make a crucial muscle protein called dystrophin. Many scientists hope that the approach proves to be a safer alternative to gene editing therapies that make permanent changes to DNA itself.

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Gene Editing Gene Gene Therapy Scientist 105

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., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

The Pharma Data

MARCH 22, 2021

Radioligand therapy combines a targeting compound that binds to markers expressed by tumors and a radioactive isotope, causing DNA damage that inhibits tumor growth and replication. We would not be able to realize our commitment to reimagining medicine without the partnership of patients and their families.”.

Hormones 52

Hormones Gene Therapy Medicine Trials 52

Roots Analysis

JANUARY 14, 2024

Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. Insect cell lines have been frequently used as recombinant protein expression systems. cerevisiae) and Pichia pastoris.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

The Pharma Data

DECEMBER 27, 2020

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. TSHA-101 is an investigational gene therapy designed to deliver the HEXA and HEXB genes driven by a single promoter within the same AAV9 construct, dubbed a bicistronic vector.

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

Roots Analysis

JANUARY 27, 2025

Protein therapeutics have completely revolutionized the healthcare segment, greatly influencing the study and treatment of human diseases by conferring precise targeting ability to therapeutic modalities. Considering their vast potential, close to 50% of the top selling drugs in 2023 were protein therapeutics.

Protein 40

Protein Reagent Marketing Gene 40

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. FLT180a is an AAV gene therapy. Sio Gene Therapies reported positive six-month follow-up data from the low-dose cohort of its dose escalation Phase I/II trial of AXO-AAV-GM1.

Trials 52

Trials Gene Therapy Vaccination Vaccine 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 111

Life Science Vaccination Vaccine Gene 111

pharmaphorum

JULY 26, 2022

At a basic level, the virus’s protein ‘coat’ and the enzymes it uses to gain control of the host cell’s DNA mutate frequently, making it difficult to target with therapeutics. The team found that HIV Pol is formed by two proteins bound together, which was unexpected as other similar viral proteins are single-protein assemblies.

Gene Therapy 140

Gene Therapy Protein Vaccination Vaccine 140