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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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.

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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.

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Gene Therapy Gene Bioinformatics Life Science 52

Pharmaceutical Technology

AUGUST 16, 2022

Even giant pharma companies with large internal production capabilities are leaning on outsourcing to meet market demand for huge numbers of injectables, including high-value biologics and Covid-19 vaccines, according to a GlobalData analysis. Why do large and mega-cap sponsors outsource?

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Drug Discovery World

MAY 7, 2024

Where the bottlenecks of achieving higher cell density and higher titers were once thought to be issues of the past, hurdles have now shifted to achieving speed and scale in mAb production without sacrificing quality. DC: Antibodies have and remain a large product class within the biopharmaceutical market.

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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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Camargo

OCTOBER 15, 2020

Often, to gain clinical trial design insights, a sponsor reviews the physician package inserts from approved New Drug Applications (NDAs) and Biologics License Applications (BLAs) with similar indications or in the same therapeutic area. Natural history data becomes critical for product development when there is no concurrent control.

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Pharma Packaging Solutions

JUNE 30, 2023

Biopharmaceuticals, also called biologics, are drug products that are manufactured using biological sources. There are a few major classes of biopharma products, including the following: Extracted: Biologics extracted directly from living systems (of either animals or humans) are the oldest form of biopharmaceuticals.

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Pharma Packaging Solutions

SEPTEMBER 22, 2023

In many ways, biopharmaceutical products have revolutionized the medical industry. Biologics are drug products that are manufactured using materials from living sources. 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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Roots Analysis

APRIL 15, 2024

These complex entities, such as antibodies, antibody drug conjugates, cell therapies, gene therapies, and therapeutic proteins, are highly specific molecules that are designed to precisely target biomarkers associated with a particular disease pathway.

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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. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

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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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Gene Therapy Gene Manufacturing Production 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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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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The Pharma Data

MAY 26, 2023

State-of-the-art cryogenic freezers alongside material transfer equipment have been installed that are designed to retain the integrity of investigational advanced therapy products by minimizing their time-out-of-environment. Catalent helps accelerate over 1,000 partner programs and launch over 150 new products every year.

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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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STAT News

APRIL 6, 2023

Meanwhile, we hope you have a meaningful and productive journey today. They may involve infusions of a gene packaged inside the shell of a virus, which can lead to potential immune complications and can require close monitoring of patients. You can digest the results if you scroll down the screen.

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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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Camargo

AUGUST 11, 2021

As regulatory requirements become increasingly harmonized across the globe, the development and marketing of pharmaceutical products worldwide are also becoming more streamlined. The CHMP evaluates the product’s quality, safety, and efficacy and provides an opinion to the European Commission (EC).

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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. Merck will also make royalty payments on any approved products developed out of the partnership. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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The Pharma Data

AUGUST 7, 2020

The governing body claims this proposal could have positive effects on mitigating burdensome requirements for gene therapy clinical trials in the future. Most importantly, we are continuing to see patients benefit from the profound therapeutic effects of both approved products and those currently in clinical development.

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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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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. The expansion will be operational in 2024.

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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. Despite challenging business conditions in 2021 and 2022, large CMOs were still involved in acquisitions to enhance their capabilities or scale of production. Catalent Inc.

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Pharmaceutical Technology

DECEMBER 9, 2022

M&A – Commended PRODUCT LAUNCHES – Commended RESEARCH AND DEVELOPMENT – Commende d. The company is ranked in the M&A, PRODUCT LAUNCHES and RESEARCH AND DEVELOPMENT categories in the 2022 Pharmaceutical Technology Excellence Rankings. Pharmaceutical Technology Excellence Rankings – The Verdict.

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The Pharma Data

JULY 11, 2021

Eli Lilly and Company (NYSE: LLY) announced new Phase 2 data showing that gene expression changes induced by mirikizumab in patients with ulcerative colitis (UC) over a 12-week induction treatment were maintained for up to one year. For methodology, see the “About the Studies” section below.

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FDA Law Blog

NOVEMBER 2, 2023

It is not especially novel even within CBER, as previous publications had encouraged the use of standards in product development. FDA needs assurances regarding the safety, purity, and potency of regenerative medicine products to approve them.

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Drug Discovery World

FEBRUARY 27, 2024

BS: Genetic tuning, also called epigenetic editing, actively manipulates the epigenetic machinery to influence gene transcription. Orchestrating the naturally occurring conductors of gene expression can shift the functional state of cells and alter gene expression. MT: How will genetic tuning improve CAR T therapies?

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Roots Analysis

FEBRUARY 1, 2024

In terms of science, cold chain industry requires the understanding of chemical and biological processes associated with the product. This technology usually relies on physical methods to ensure desirable temperature conditions for perishable products throughout the supply chain.

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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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pharmaphorum

MARCH 10, 2022

Biologics product development has increased steadily over the past decade, with the volume of business forecast to double by 2030. This trend will continue in the near future with a steady increase in market demand over the coming years, alongside further development in cell and gene therapies,” says Magnelli. The rise of biologics.

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Roots Analysis

OCTOBER 5, 2023

It is worth highlighting that the manufacturing of polymers for biopharma application needs to be carried out in a controlled environment with strict adherence to regulatory guidelines in order to prevent deterioration of the product quality. This allows for precise customization and faster production of medical devices.

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pharmaphorum

MARCH 3, 2022

The UK biopharma industry has welcomed a new tranche of funding from the government aimed at bolstering manufacturing of medicinal products and developing the national health data infrastructure.

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Drug Discovery World

JANUARY 10, 2023

For over 50 years, Bethyl Laboratories (now a Fortis Life Sciences brand) has been dedicated to improving lives by supporting scientific discovery through qualified antibody products and custom services. This pillar uses gene knockout or knockdown to reduce the levels of target protein available for detection. Our areas of expertise.

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Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

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pharmaphorum

NOVEMBER 16, 2020

During his tenure the EMA also implemented new rules to encourage the development of advanced therapy medicinal products (ATMPs) like gene and cell therapies, and the introduction of the PRIME fast-track review for promising medicines. Emer Cooke.

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Drug Discovery World

MARCH 29, 2023

The review builds on several data sets, including large IQVIA/MIDAS data sets of products authorised between 2002 and 2020. They explain that a number of studies have demonstrated how incentives influence company development and innovation decisions, such as through the industry-standard risk-adjusted Net Present Value (rNPV) model.

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