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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XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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BioSpace

MAY 11, 2021

Nearly 50 infants with "bubble baby" disease developed a working immune system after they received a gene therapy that contained the AIDS virus, according to a new study.

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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). It is indicated for patients who do not have a pre-existing medical reason preventing treatment with the therapy.

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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.

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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

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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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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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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Pharmaceutical Technology

JANUARY 31, 2023

The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for Moderna ’s investigational mRNA vaccine candidate, mRNA-1345, for respiratory syncytial virus (RSV) in adult patients. It contains a single mRNA sequence encoding for a stabilised prefusion F glycoprotein.

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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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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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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.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 3, 2021

Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the company’s lead product candidate, PBGM01, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of infantile GM1 gangliosidosis (GM1). to 1 in 100,000 live births. “I About Passage Bio.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

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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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

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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Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

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XTalks

JANUARY 21, 2025

The liquid portion of the blood (serum or plasma) containing antibodies is separated and tested using the Liason Biotrin parvovirus B19 IgG plus test on a Liason XL analyzer. The test collection kit includes a container for stool collection and a separate sampler for hemoglobin testing, requiring only a single stool sample to collect both.

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Pharmaceutical Technology

AUGUST 31, 2022

ILC Dover, specialists in the innovative design of biopharmaceutical manufacturing solutions, continuously monitors the market in order to provide in-demand equipment such as flexible containment solutions that reduce bottlenecks by providing closed, Grade-A, aseptic environments for fill and finish activities.

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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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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.

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Gene Therapy Gene Trials Clinical Trials 52

The Pharma Data

JANUARY 18, 2021

SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant. About Taysha Gene Therapies. Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.

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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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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.

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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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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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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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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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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.

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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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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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Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Our QC testing capabilities range from solids, semi-solids and liquids for oral, inhaled and injectable use to packaging materials, including rubber closures, glass and plastic containers. HQ ADDRESS: Römerpark 2.

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Camargo

DECEMBER 13, 2021

An application submitted under 351(a), also known as a “stand-alone” application, must contain all safety and effectiveness information for a biological product and cannot depend on any other biological product. Section 351(a) is the traditional pathway for approving biologics under the PHS Act. Product Type-Specific Analysis.

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Roots Analysis

JANUARY 27, 2025

The American Society of Gene and Cell Therapy (ASGCT) defines cell therapy as a therapeutic modality that involves the administration of either normal or modified cells to patients for the treatment of various diseases. Such procedures require sterile and higher-grade clean rooms, along with regular checks on containment levels.

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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. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease.

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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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