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

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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The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. Dr. John Greinwald is a tenured professor of Otolaryngology and Pediatrics with over 20 years of experience with a focus on the genetic causes and treatment of deafness. To register for the call, please click here.

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Drug Discovery World

MAY 29, 2024

Marc Hummersone, Senior Director of Research and Development (R&D) at Astrea Bioseparations, shares insight on the challenges and opportunities in cell and gene therapy (CGT) with DDW’s Megan Thomas. That takes a lot of effort, and some very clever people to get that amount of genetic information into a single plasmid DNA.

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Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy.

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The Pharma Data

JANUARY 18, 2021

This disease is a debilitating form of genetic epilepsy in children that significantly impacts movement, motor control, cognition and quality of life, and there remains a need to alter the course of this disease early in life.”. “As About Taysha Gene Therapies. Assistant Professor in Pediatric Neurology at UT Southwestern.

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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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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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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

OCTOBER 27, 2023

Animal models, Ewart explains, are limited by their genetic and physiological differences from humans. How do organ-chips support gene therapy delivery? Organ-on-a-chip technology provides an alternative to other preclinical research models, and can overcome some of the issues of inaccuracy.

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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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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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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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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

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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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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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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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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The Pharma Data

OCTOBER 27, 2020

NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. There are currently no disease-modifying therapies for Krabbe disease, and we believe incidence may be 2.6 PHILADELPHIA, Oct. About PBKR03.

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The Pharma Data

NOVEMBER 22, 2020

It contains the genetic materials of the spike protein. It deploys a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees. After vaccination, the cells produce the spike protein, stimulating the immune system to attack the SARS-CoV-2 virus.

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Roots Analysis

SEPTEMBER 20, 2023

Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

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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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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.

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The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LEXINGTON, Mass., About LogicBio Therapeutics.

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Roots Analysis

APRIL 15, 2024

Over the years, these large molecules have demonstrated promising therapeutic outcomes in the treatment of myriad of chronic and challenging disorders, including autoimmune disorders, infectious diseases, genetic disorders, and oncological disorders.

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The Pharma Data

MARCH 5, 2021

Furthermore, Novartis Gene Therapies entered into a manufacturing agreement with Massachusetts Eye and Ear and Massachusetts General Hospital to produce its novel genetic COVID-19 vaccine candidate called AAVCOVID8. More information about the Novartis response to COVID-19 is available at www.novartis.com/COVID-19. Disclaimer.

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The Pharma Data

JANUARY 3, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. GM1 is a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available. PHILADELPHIA, Jan. About PBGM01.

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Roots Analysis

AUGUST 31, 2023

Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals.

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XTalks

DECEMBER 20, 2023

Novo Nordisk’s GLP-1 weight loss-specific drug Wegovy (semaglutide) has been facing similar shortages, as it contains the same active ingredient as Ozempic. The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy.

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pharmaphorum

JANUARY 27, 2023

In the morning, I can be talking about gene therapy for hearing loss, for example, [and] in the afternoon, I can be talking about the treatment of a very specific tumour. The ABPI report also contained recommendations regarding this. So, it gives me much more breadth of exposure to be able to make that difference.”

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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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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. The gene therapy utilizes an AAV1 viral vector to deliver a modified DNA encoding the GRN gene to a patient’s cells.

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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.

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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.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Drug Discovery World

AUGUST 10, 2022

Cells from solid tumours are genetically heterogeneous both between and within tumours, making it challenging to identify a single, selective antigen target for treatment. Furthermore, even if the transgene enters the cell in the correct numbers, the location where the gene integrates into the genome is somewhat random.

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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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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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