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). million price tag of Elevidys, a one-time gene therapy. Options for managing the symptoms of DMD have been limited.

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Drug Discovery World

DECEMBER 5, 2022

Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. . In the study, researchers used ‘fossilised’ virus DNA sequences contained within animal genomes to reconstruct the long-term evolutionary history of parvoviruses.

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Drug Discovery World

JANUARY 27, 2023

Exclusive CGT Guide This issue contains an exclusive Cell & Gene Therapy Guide covering the latest innovations and challenges within the sector.

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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).

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Drug Discovery World

OCTOBER 19, 2022

A new blood donor centre in Glasgow, Scotland has been opened to support cell and gene therapy research. . The donor center will be used primarily for producing LEUKOMAX leukopaks, which contain concentrated white blood cells and are used for cell and gene therapy research. .

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Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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The Pharma Data

OCTOBER 26, 2020

DALLAS–( BUSINESS WIRE )– Taysha Gene Therapies Inc. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency.”.

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Pharmaceutical Technology

DECEMBER 12, 2022

It leverages the new D-Domain binder of Arcellx and contains autologous T cells genetically modified for targeting multiple myeloma. The latest development comes after Kite Pharma and Daiichi Sankyo modified their collaboration agreement signed in 2017 for the former’s CAR T-cell therapy, Yescarta (axicabtagene ciloleucel).

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Pharmaceutical Technology

AUGUST 1, 2022

The mRNA-1273.222 vaccine containing the BA.4/5 Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Last month, the company announced the advancement of two bivalent candidates based on various population health security approaches in different countries.

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Pharmaceutical Technology

MAY 12, 2023

The in vivo study, conducted in mice, involved a phosphatidylserine-containing nano-formulation of mRNA supplied by BioNTech, distinct from traditional lipid nanocrystals (LNCs). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Matinas announced the news in a May 10 statement.

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Pharmaceutical Technology

AUGUST 15, 2022

The updated, Omicron-containing bivalent vaccine that acts on two coronavirus variants is indicated as a booster dose for active immunisation for the prevention of Covid-19 in people of this age group. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 1 Omicron variant of concern.

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The Pharma Data

JANUARY 18, 2021

Taysha’s commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community.”. About Taysha Gene Therapies. Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. Forward-Looking Statements.

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Scienmag

MAY 28, 2021

Genetics toolkit targets less researched Culex mosquitoes, which transmit West Nile virus and avian malaria Credit: Gantz Lab, UC San Diego Since the onset of the CRISPR genetic editing revolution, scientists have been working to leverage the technology in the development of gene drives that target pathogen-spreading mosquitoes such as Anopheles and (..)

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Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Drug Discovery World

OCTOBER 19, 2022

Yapan Bio has expanded its capabilities with a new process development facility at Genome Valley, Hyderabad, India, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. .

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Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

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BioSpace

JUNE 9, 2022

Following a marathon session of the FDA's Cell, Tissue and Gene Therapies Advisory Committee, bluebird bio passed its first critical hurdle in approval for the lentiviral vector gene therapy, eli-cel.

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Drug Discovery World

JANUARY 11, 2024

Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy. Until recently, gene therapy employed modified viruses to bring the therapeutic gene to the disease cells, but these viral systems can cause severe adverse effects.

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Pharmaceutical Technology

OCTOBER 18, 2022

Gavi CEO Dr Seth Berkley said: “This agreement with Moderna represents a critical step for equitable access, helping COVAX adjust its portfolio to current demand and ensuring lower-income countries have access to variant-containing vaccines to use where appropriate.”. 5 Omicron-targeting bivalent booster vaccine for Covid-19.

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Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient.

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Pfizer

SEPTEMBER 25, 2022

Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment. Hospira Issues A Voluntary Nationwide Recall For One Lot of Propofol Injectable Emulsion (containing benzyl alcohol), Due To The Potential Presence of Visible Particulates. Leadership. 09.22.2022.

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Drug Discovery World

NOVEMBER 14, 2022

Find out more: Atrogi gets go-ahead to begin Phase I study of type 2 diabetes therapy . AAV vectors for diabetes gene therapy. The end goal is to develop AAV vectors that target specific pancreatic cell types and contain payloads that express therapeutic genes under control of cell-specific regulatory elements.

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XTalks

JANUARY 2, 2024

Filsuvez topical gel is a sterile botanical drug product designed for topical use, containing birch triterpenes within an oil base. For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector.

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Pharma Packaging Solutions

SEPTEMBER 22, 2023

There are a few common types of biologics, including extracted (taken directly from a living system), semi-synthesized (produced with recombinant DNA technology), vaccines, and gene therapies. Covid-19, flu, coronavirus liquid vaccine vial bottle and syringe set in plastic package container preparing for injection on white background.

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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.

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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.

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Drug Discovery World

OCTOBER 27, 2023

How do organ-chips support gene therapy delivery? Organ-chips are also playing a role in helping scientists overcome the challenge of developing safe and efficient delivery vehicles for gene therapies, like lipid nanoparticles (LNP). What are the limitations of the technology?

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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

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Drug Discovery World

NOVEMBER 8, 2022

The bioanalytical laboratory will contain 48 workbenches in an open space, with services that include mass spectrometry and ligand binding assays, flow cytometry, biomarkers, as well as cell culture. This will help with our recruiting efforts, as we launch our first clinical gene therapy trial in humans next year.”.

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

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Scienmag

JULY 14, 2021

Researchers have found a way to enhance radiation therapy using novel iodine nanoparticles Credit: Mindy Takamiya/Kyoto University iCeMS Cancer cell death is triggered within three days when X-rays are shone onto tumor tissue containing iodine-carrying nanoparticles. The findings, by scientists at Kyoto […].

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The Pharma Data

JANUARY 26, 2021

The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Scopus BioPharma Inc.

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Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies.

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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

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XTalks

NOVEMBER 10, 2023

Treatments for cTTP includes prophylactic plasma-based therapy for individuals with chronic disease to reduce the risk of clotting/bleeding by restoring the low amounts or absence of the ADAMTS13 enzyme. For prophylactic ERT, Adyznma is given to help reduce the risk of disease symptoms.

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pharmaphorum

JULY 9, 2021

Myocarditis is generally the result of a viral infection, so could be unrelated to the mRNA vaccines, particularly as neither of them contain any live virus. Tens of thousands of cases of myocarditis and pericarditis are thought to be diagnosed in Europe every year.

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