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

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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Development Gene Therapy Clinical Trials Clinical Trials 264

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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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

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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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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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

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.

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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 PBGM01.

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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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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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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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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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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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FDA Approval Gene Therapy Gene Trials 111

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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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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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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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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Roots Analysis

JANUARY 27, 2025

Some of the key challenges faced by cell therapy manufacturers have been briefly summarized below: It is difficult to scale-up the manufacturing procedures of cell therapy products. Such procedures require sterile and higher-grade clean rooms, along with regular checks on containment levels.

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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.

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Camargo

DECEMBER 10, 2021

In October 2017, the European Commission (EC) and the European Medicines Agency (EMA) collaborated with the Member States’ authorities to launch a plan to reduce discrepancies across the EU in applying certain legislation to advanced therapy medicinal products (ATMPs) containing or consisting of genetically modified organisms (GMOs).

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XTalks

APRIL 13, 2021

synuclein amyloid to a test tube containing the amyloid-forming region of Pmel (known as the repeat, or RPT, domain) and found that the ?-synuclein For example, gene therapy approaches are being investigated to downregulate ?-synuclein -synuclein and Pmel proteins were present in the melanosomes of human melanoma cells.

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The Pharma Data

NOVEMBER 2, 2020

In the second half of 2019, Sensorion launched two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. www.sensorion-pharma.com. www.sensorion-pharma.com. Disclaimer.

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XTalks

JUNE 5, 2024

Tune into the episode to learn about CERo’s next-gen T-cell technology and its lead therapeutic candidate, a CER T cell product that contains a phagocyte receptor component. Hear about how it is being employed for AML, a blood cancer that remains among the most challenging cancers to treat.

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The Pharma Data

JANUARY 18, 2021

In the second half of 2019, Sensorion initiated two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. www.sensorion-pharma.com. www.sensorion-pharma.com. Disclaimer.

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The Pharma Data

NOVEMBER 19, 2020

In the second half of 2019, Sensorion launched two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. www.sensorion-pharma.com. www.sensorion-pharma.com. Disclaimer.

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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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FDA Approval Gene Therapy Gene Clinical Trials 59

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.

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Roots Analysis

MARCH 16, 2023

Protein Replacement Therapies: Gene therapy can be defined as transplantation of normal genes, in order to replace missing or defective genes, with an aim to correct genetic disorders or promote inactive beneficial mechanisms or pathways.

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The Pharma Data

DECEMBER 17, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (Paris:LYS) (FR0013233475 – LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, announces a change in the governance and control of KGA, a company co-owned by Karen Aiach and which currently owns approximately 6% of Lysogene’s capital.

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FDA Law Blog

JANUARY 12, 2025

Moreover, an application (NDA or BLA) based upon a surrogate or intermediate clinical endpoint seeking accelerated approval must still meet the substantial evidence of effectiveness standard and contain sufficient information to demonstrate that the drug is safe for use under the conditions of the proposed labeling.

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The Pharma Data

JANUARY 6, 2021

In the second half of 2019, Sensorion initiated two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. [link]. [link]. Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN.

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XTalks

MAY 15, 2023

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. The recommended treatment regimen consists of a 1 mg/kg intravenous infusion every two weeks.

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The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

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XTalks

JUNE 29, 2022

XTALKS WEBINAR: Live Cell Metabolic Analysis — Paving the Way for Metabolic Research and Cell & Gene Therapy. Register for this free webinar to learn how live cell metabolic analysis paves the way not only for metabolic research, but also the manufacturing of significant cell and gene therapy (CGT) products.

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