Drug Discovery World

JANUARY 11, 2024

Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification. 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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Research Gene Therapy RNA Gene 59

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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Development Gene Therapy Drugs Gene 52

Cloudbyz

SEPTEMBER 16, 2023

Addressing data privacy and data protection concerns when implementing ChatGPT in clinical trial operations management is crucial to maintain compliance with regulations, safeguard sensitive patient information, and build trust among stakeholders. This may involve conducting regular audits and assessments to maintain compliance.

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Clinical Trials Clinical Trials Trials Compliance 59

pharmaphorum

JULY 9, 2021

The EU medicines regulator has said that there is some evidence to suggest a possible link between mRNA-based vaccines for COVID-19 and rare cases of heart inflammation. . 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.

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Vaccination Vaccine Gene Therapy Regulation 105

Camargo

DECEMBER 13, 2021

The regulations regarding BLAs for therapeutic biological products are included in 21 CFR parts 600 , 601 , and 610. 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.

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Development Production Immune Response Licensing 161

XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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FDA Approval Gene Therapy Gene Clinical Trials 59

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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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

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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Containment Gene Therapy Gene Regulation 40

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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Containment Gene Therapy Gene Development 52

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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Containment Gene Therapy Gene Development 40

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.

Genetics 52

Genetics Containment Genetic Disease Gene Therapy 52

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.

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Development Production Manufacturing Vaccination 130

Advarra

FEBRUARY 24, 2023

How do you strategically plan your therapy and research objectives to improve the chances you will satisfy what the regulators are looking for and ultimately get your therapy approved for marketing? Sponsors need to carefully consider how they will prove safety and efficacy in a manner sufficient to satisfy the regulators.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Regulation 52

The Pharma Data

JANUARY 7, 2021

As CEO of Freeline, a liver-directed gene therapy company, she scaled the company from preclinical stage to a fully integrated biotechnology organization, which included a broad, internally developed pipeline, two programs in clinical development and a commercial-scale, high-quality CMC and manufacturing platform.

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Gene Therapy Life Science Production Clinical Development 52

The Pharma Data

NOVEMBER 22, 2020

As the Pfizer and BioNTech COVID-19 vaccine goes to regulators and the Moderna vaccine approaches the end of its Phase III trials, AstraZeneca and the University of Oxford announced high-level results from an interim analysis of their COVID-19 vaccine, AZD1222. It contains the genetic materials of the spike protein. Source link.

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Vaccination Vaccine Protein Genetics 52

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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Gene Gene Therapy Gene Expression Regulation 40

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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Gene Therapy Gene Gene Expression Regulation 208

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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Manufacturing Vaccination Vaccine Production 52

The Pharma Data

JANUARY 4, 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.

Development 52

Development Containment Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 21, 2020

Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, today announced the pricing of its public offering of 4,687,500 shares of its common stock at a public offering price of $40.00 BOSTON, Oct. 22, 2020 (GLOBE NEWSWIRE) — Replimune Group, Inc.

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Gene Therapy Immune Response Sales Gene 40

FDA Law Blog

SEPTEMBER 20, 2021

We have seen first-hand the difficult task faced by CBER review staff to keep up with the flood of innovative cell and gene therapy products, as well as the taxing influx of COVID-19 pandemic related applications requiring review resources in both Centers.

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Development Gene Therapy Production Drugs 52

Drug Discovery World

MARCH 29, 2023

However, stakeholders should be aware that the leaked review contains no such analysis. What of the increased possibilities for ‘replacement pharmacy compounding’ for weekly hospital supplies, and for routine manufacturing of cell and gene therapies without a marketing authorisation?

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Clinical Trials Clinical Trials Production Medicine 52

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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Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

AUGUST 10, 2022

For example, it is challenging to regulate transgene copy numbers in each T cell, posing safety risks. If the gene does not integrate into the genome, the cell will not become a CAR T cell. But if the cell accepts too many copies of the gene, the cell can cause cytokine release syndrome (CRS) or a ‘cytokine storm’.

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Manufacturing Gene Genome Genomics 98

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Gene-based therapeutics and vaccines hold immense potential for more efficient and personalised treatment and prevention of complex diseases.

Production 52

Production Gene Protein Vaccination 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling gene expression.”.

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DNA Gene Gene Silencing Genome 98

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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Antibody Gene Therapy Containment Clinical Trials 52

Drug Discovery World

DECEMBER 15, 2022

Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo. In one example, Choi et al. In all cases, viral delivery vectors have limited payload capacity which can limit various use cases.

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Gene Editing Gene In-Vivo Genome 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling gene expression.”.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

DECEMBER 8, 2020

They are based on targets with strong human validation, represent diverse modalities and leverage new platform capabilities in cell therapy, gene therapy and data sciences. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

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Containment Engineer FDA Approval Vaccination 52

XTalks

AUGUST 2, 2023

It contains the antiviral medications nirmatrelvir and ritonavir. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. In September 2022, Roche acquired Good Therapeutics for an upfront payment of $250 million, and has access to their PD-1-regulated IL-2 program.

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Sales Manufacturing FDA Approval Life Science 98

Pharma Marketing Network

JANUARY 20, 2021

Assuming these findings are replicated in clinical trials, this vaccine, with its more-appealing route of administration and convenient (refrigerator) storage requirements could make an important contribution to containing the disease.

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Vaccination Vaccine In-Vivo Genetics 52

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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Clinical Development Gene Therapy Development Gene 52

The Pharma Data

NOVEMBER 30, 2020

The rolling review process began on October 6, which allowed European regulators to evaluate the data as it was submitted. The UK regulators have begun analyzing the Pfizer vaccine data and is expected to begin evaluating the AstraZeneca product soon. The adenovirus is what is typically used in gene therapies.

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Vaccination Vaccine Regulation Trials 52

The Pharma Data

MAY 15, 2021

I was recently reminded of my journey out of the HCT nightmare as I watched a documentary about gene therapy. Gene therapy is a huge deal – some gene therapies are already achieving jaw-dropping results for people with previously untreatable illnesses. It’s fascinating stuff. So long, HCT.

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Bacteria Gene Gene Therapy Gene Expression 52