Drug Discovery World

OCTOBER 3, 2023

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

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Pharmaceutical Technology

DECEMBER 19, 2023

In this issue: Fixing drug nomenclature to avoid confusing names, gene therapies for sickle cell disease, childproof packaging for drugs, and UK pharma manufacturing gears up for a shake up in 2024

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Drug Discovery World

JULY 5, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

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Gene Therapy Gene Bioinformatics Life Science 52

Drug Discovery World

JUNE 29, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Gene Therapy 52

Gene Therapy Gene Bioinformatics Life Science 52

Drug Discovery World

JUNE 22, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Gene Therapy 52

Gene Therapy Gene Bioinformatics Life Science 52

FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Gene Therapy Gene FDA Approval Production 119

Pharma Manufacturing

AUGUST 15, 2022

Optimal packaging and labeling solutions help ensure the delivery of safe, effective viral vector products

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Cloudbyz

MARCH 30, 2023

Cell and gene therapies are rapidly becoming an essential component of modern medicine, offering hope to patients with previously untreatable diseases. However, the development and delivery of cell and gene therapies present significant operational challenges that must be navigated carefully to ensure successful clinical trials.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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Gene Therapy Gene Development Production 52

Drug Discovery World

APRIL 17, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.

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Pharmaceutical Technology

AUGUST 16, 2022

Large (market cap $10–100bn) and mega-cap (market cap >$100bn) sponsors also require contract manufacturers and packagers with specialist injectable capabilities in the case of cell and gene therapies, as shown in the report titled Contract Injectable Packaging Trends in the Bio/Pharma Industry (August 2022).

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Gene Therapy Packaging Packaging Contract Packaging 162

Drug Discovery World

APRIL 24, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.

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Genome Genomics DNA Gene Therapy 52

Pharma Packaging Solutions

JUNE 30, 2023

Gene Therapy: Gene therapy is a biologic treatment that involves manipulating a virus to carry a certain, desirable piece of genetic material which can then target a patient’s cells. As biopharma technology progresses, becoming both more prevalent and available, the need for high-quality biopharma packaging increases.

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Recombinant DNA Technology Packaging Packaging Gene Therapy 52

Pharmaceutical Technology

NOVEMBER 22, 2022

Despite China producing a significant proportion of the world’s API supply (mostly small molecule), it manufactures relatively few biosimilar and innovator drugs and no cell and gene therapies for the western markets of Europe and the US despite investments and an increasing number of startups to improve innovative manufacture.

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Manufacturing Gene Therapy Gene Contract Manufacturing 162

The Pharma Data

MAY 26, 2023

The expansion forms part of Catalent’s ongoing global strategy to increase its ability to handle, store and manage advanced therapies for clinical supply, and follows investments at its facilities in Philadelphia, Singapore, and Shanghai, China, in specialized, ultra-low temperature storage capabilities.

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Clinical Supply Gene Therapy Gene Development 52

Drug Discovery World

JUNE 23, 2023

Advanced Therapy Medicinal Products (ATMPs) have been making the news this week. FDA approves first gene therapy for Duchenne muscular dystrophy Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US.

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Gene Therapy Drugs Gene FDA Approval 52

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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Recombinant DNA Technology Packaging Packaging Pharma Packaging 52

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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Gene Therapy Gene Manufacturing Production 52

Pharmaceutical Technology

APRIL 11, 2023

The Phase III ENDEAVOUR study, which is part of the SRP-9001’s BLA package, used a primary endpoint centered on measuring micro-dystrophin expression, a surrogate marker that has been used in the regulatory approvals for exon-skipping therapies.

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Gene Therapy Gene Packaging Packaging 130

Drug Discovery World

NOVEMBER 23, 2022

One critical bottleneck in Cell and Gene Therapy (CGT) manufacturing is the production of high-quality plasmid DNA (pDNA), a vital building block for mRNA-based vaccines, viral vector-based cell or gene therapies that require pDNA as a starting material.

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DNA Manufacturing Gene Therapy Gene 52

Drug Discovery World

MARCH 19, 2024

AT-108 is a first-in-class, off-the-shelf gene therapy that directly reprogrammes tumour cells into antigen-presenting dendritic cells, ultimately leading to a personalised anti-tumour immune response. We believe this breakthrough strategy will give rise to the next generation of cell therapies.

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In-Vivo Gene Therapy Immune Response Packaging 52

Drug Discovery World

NOVEMBER 1, 2022

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Therapies that don’t precisely target diseased cells can have unintended effects in other healthy cells.” . Splicing-linked expression design.

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Gene Expression Gene Packaging Packaging 52

Pharmaceutical Technology

AUGUST 17, 2022

These molecules synthesised newly are packaged densely into custom lipid nanoparticles (LNPs), which Orna has made to act on the body’s crucial tissues. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

JUNE 14, 2023

Navigating the complexities of pharma and biotech packaging services can be difficult. With the growing number of complex therapies that require specialized packaging and handling requirements, selecting the right contract packaging organization (CPO) involves evaluating what services and additional benefits they can bring to your business.

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Contract Packaging Packaging Packaging Development 130

Drug Discovery World

JANUARY 17, 2023

Limitations of AAV gene therapy . The research aims to develop solutions for the limitations associated with adeno-associated virus (AAV), the current primary means of delivery for gene editing. . AAV has limited packaging capacity compared to LNPs and it can prompt an immune system response,” Sahay said. “It

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Pharmaceutical Technology

JULY 29, 2022

Injectables and their packaging requirements have never been more important to marketed treatments, given the volume of vaccines being packaged that are required to tackle the Covid-19 pandemic, as well as the rising presence of biologics in pipelines and their marketed approvals.

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Manufacturing Vaccination Vaccine Packaging 130

BioSpace

NOVEMBER 23, 2022

BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its BLA for Roctavian an AAV gene therapy for adults with severe hemophilia A.

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FDA Law Blog

NOVEMBER 2, 2023

While on the surface this may not seem like major advance for the field, this could be a game changer for the gene therapy space. These meetings have a question-and-answer format to provide clarity to sponsors about the particular topics at hand.

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pharmaphorum

AUGUST 20, 2020

US biotech BioMarin has hit back with a filing for a rare disease drug, after suffering what was described by analysts as a “major setback” when the FDA rejected its gene therapy for haemophilia A. It also has three other gene therapies in its pipeline for phenylketonuria, hereditary angioedema, and hypertrophic cardiomyopathy.

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Camargo

JULY 30, 2021

But now it’s a warning in their package insert. That has to do a lot with the work in gene therapy and rare disease and the ultra-rare space because there are 7,000 rare diseases that have been identified. But I believe that the future of pediatrics is going to be in gene therapy, cell therapy.

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Pharmaceutical Technology

MAY 26, 2023

The acquisition targets had a wide variety of manufacturing services, including API and dose manufacturing, packaging, and analytical services. These acquisitions focused on advanced cell and gene therapy capabilities.

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Camargo

JANUARY 12, 2022

As we noted in a separate blog post , the forthcoming reauthorization of the FDA’s Prescription Drug User Fee Act (PDUFA VII) includes several provisions that are expected to help advance cell and gene therapy products, as well as therapies designed to treat rare diseases. Receipt of Background Package. Response Time.

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Roots Analysis

FEBRUARY 1, 2024

Further, the cold chain storage process involves the cold chain storage, cold chain transportation, and monitoring temperature controlled packaging products. Therefore, the pharmaceutical, biotech, food and chemical industries usually rely on cold chain packaging for the transportation of their products.

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Camargo

AUGUST 11, 2021

Meeting Package Due. A letter of intent, analogous to an FDA meeting request, and briefing package must be submitted by the sponsor either three weeks (when seeking scientific advice without a meeting) or approximately seven weeks (when requesting a pre-submission meeting) prior to the intended start of the scientific advice procedure.

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pharmaphorum

AUGUST 19, 2020

Our community of supply chain experts are looking forward to connecting with you in the digital world, including: Christopher Baldwin , Supply Chain Director, Cell & Gene Therapy¸ GSK. Reggie Foster , Senior Director Labelling, Logistics & Packaging, Kite: a Gilead Company.

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Gene Therapy Packaging Packaging Branding 52

Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach. link] “COVID-19 and cell and gene therapy: How to keep innovation on track.”

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The Pharma Data

JUNE 7, 2023

Originally launched in 2019 for early-phase protein therapy development, OneBio Suite offers customers an integrated service to accelerate programs from development to manufacturing, including fill/finish and packaging, and support for clinical supply and commercial launch.

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