pharmaphorum

MARCH 15, 2021

Novartis has said its gene therapy pricing model, involving a steep one-off payment, could be a template for the hundreds of similar treatments that could follow its Zolgensma for Spinal Muscular Atrophy (SMA). . There are around 350 gene therapies in various studies.”. Zolgensma is model for gene therapy pricing appeared first on.

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Gene Therapy Gene Pharma Companies Clinical Trials 96

XTalks

OCTOBER 4, 2023

Heather McDonald: My previous role at Bayer was a global-level role with a specific focus on cell and gene therapies. In addition, AstraZeneca just announced a major acquisition of Pfizer’s cell and gene therapy portfolio. How is the shift toward value-based healthcare affecting drug pricing and reimbursement?

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Marketing Development Drugs Gene Therapy 105

Camargo

MARCH 9, 2021

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. The FDA revealed that pharma companies are not forthcoming when they publicly disclose the reasons behind a refuse-to-file (RTF) letter from the Agency.

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XTalks

DECEMBER 27, 2022

Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, so it cannot multiply in human cells. However, more than 50 percent of patients who had an initial treatment with BCG will go through disease recurrence and progression within one year, with many of these patients developing disease that is unresponsive to BCG.

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Gene Therapy FDA Approval Gene Clinical Trials 52

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 52

Drug Discovery World

JANUARY 25, 2024

Japan-based pharma company Kyowa Kirin has successfully completed the acquisition of gene therapy developer Orchard Therapeutics. Libmeldy is known as OTL-200 in the US, where it is currently an investigational drug under Priority Review by the Food and Drug Administration (FDA).

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Gene Therapy Gene Pharma Companies Drugs 52

Delveinsight

SEPTEMBER 3, 2021

Thus, missed or late diagnosis is a serious concern; however, it can be due to the heterogeneity of the condition, late age of onset of symptoms, slow and gradual development of symptoms, and so on. Additionally, a lack of curative and approved therapies in the CMT disease market is a serious unmet need.

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Pharma Companies Marketing Clinical Trials Clinical Trials 52

pharmaphorum

NOVEMBER 30, 2020

Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for gene therapies. This is particularly pertinent in rare diseases, as a number of gene therapy products use viral delivery methods to deliver the transgene to target organs.

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Gene Therapy Gene Antibody Clinical Trials 105

pharmaphorum

JUNE 1, 2022

Astellas has become the latest big pharma company to sink some money into immuno-oncology start-up GO Therapeutics, forging an alliance to develop antibodies targeting two glycoprotein targets. The Japanese drugmaker is paying $20.5 The Japanese drugmaker is paying $20.5

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Gene Therapy Clinical Development Antibody Gene 98

XTalks

MARCH 28, 2024

Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein. Bettica said the company’s focus is to now “make Duvyzat available as a treatment for DMD management in the US as quickly as possible.” The drug generated domestic sales of $200.4

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Drug Discovery World

MAY 6, 2024

In this article, DDW’s Diana Spencer highlights several prominent hotspots that are taking the lead on advancing European drug discovery and development. The oldest, still active pharmaceutical company worldwide, Merck, started life in 1668 in Darmstadt, Germany. The CDD will sponsor and manage KWF-supported trials.

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Drugs Life Science Gene Editing Gene 59

pharmaphorum

MAY 21, 2021

Read below for a preview: What are some of the challenges of developing innovative therapies for ultra-rare patient populations? This is especially relevant for the new era of gene therapies that are disease modifying, offering hope for some of the most intractable and devastating diseases, many of which affect children.

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Gene Therapy Gene Clinical Trials Clinical Trials 119

pharmaphorum

DECEMBER 14, 2022

Last month we saw some movement in the pharma world, but also in some of the orbiting worlds of agencies, consultancies, and pharma investors. In addition, two team members are taking on new roles at the company, with Glen Webster named EVP of global operations and Jennifer White named global head of growth. New CEO joins UBC.

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Life Science Gene Therapy Gene Editing Gene 98

pharmaphorum

SEPTEMBER 29, 2020

As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. The industry is asking itself how to stay innovative, how to develop and bring to market higher quality therapies to patients – and how to do this faster and more efficiently.

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Research Gene Editing Gene Development 132

XTalks

FEBRUARY 16, 2022

In this episode, Ayesha discussed the NHS’s recommendation of Libmeldy, a gene therapy for the treatment of the rare disease MLD, which is officially the world’s most expensive drug with a list price of almost $4 million USD. These include breaches and leakages of items like email addresses, passwords and even credit/banking information.

Life Science 52

Life Science Gene Therapy Drugs Pharma Companies 52

Drug Discovery World

OCTOBER 16, 2023

Cambridge boasts the world-renowned university alongside a biotech and pharma cluster situated throughout the city and its surrounding science parks, all of which helps attract top scientific talent.” At ELRIG’s Scottish Research and Drug Development Forum meeting, DDW’s Diana Spencer provided a snapshot of drug discovery in Scotland.

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Drugs Life Science Gene Therapy Genome 75

pharmaphorum

JANUARY 24, 2023

Drug development has long been an issue for the pharma industry, due to the expense and the high failure rate of potential treatments. Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates.

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Genetics Drugs Genome Project Biobanking 98

pharmaphorum

SEPTEMBER 3, 2020

King’s Cross is also home to Google’s DeepMind artificial intelligence company, which has already been involved in life sciences projects such as using the technology to predict acute kidney injury. She added: “Using technologies like AI is a critical part of helping us to discover and develop medicines for serious diseases.”.

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Drug Discovery World

AUGUST 4, 2023

With so many exciting novel treatments emerging from the labs, biotechs and pharma companies need to understand the potential unknowns as well as the known considerations if they are to optimise their impact in the real world. This means having GMP-trained operators and high-quality process development.

Gene Therapy 52

Gene Therapy Gene Manufacturing Marketing 52

Drug Discovery World

APRIL 3, 2023

I quickly recognised the commercial value of aptamers as being able to address the gap in the market where antibodies fail to perform, and together we established the business to begin aptamer development. DS: The company now has clients across big pharma. To what do you attribute your success?

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Pharmaceutical Technology

JULY 6, 2022

This period of hype saw companies going public earlier, which could explain why we may see some companies being merged or bought out, says Jason Foster, CEO of Ori Biotech, a private cell and gene therapy manufacturing company. The bear market has also impacted venture capital investment into private pharma companies.

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Marketing Gene Therapy Manufacturing Pharma Companies 324

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7

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Gene Therapy Drugs Gene Manufacturing 52

pharmaphorum

APRIL 21, 2022

One of the leading companies in the bid to bring a new class of alpha-synuclein targeting drugs to market for Parkinson’s disease – AbbVie – is ducking out of the challenge. ” She added that BioArctic will now “investigate options to continue the development of this asset.”

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Antibody Protein Gene Therapy Pharma Companies 97

XTalks

OCTOBER 22, 2020

Pharmaceutical development of novel drugs is a complex and tedious process. To achieve transparency and actionability of data, pharmaceutical companies are evolving their manufacturing processes by digitalizing the data from all of their equipment. Nevertheless, they have succeeded to continue the process development to the next stage.

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The Pharma Data

JANUARY 24, 2021

.–( BUSINESS WIRE )– Faze Medicines , a biotechnology company pioneering therapeutics based on the groundbreaking new science of biomolecular condensates, today announced that it has appointed Philip Vickers, Ph.D., a company developing first-in-class therapeutic antibodies targeting the tumor microenvironment.

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pharmaphorum

OCTOBER 12, 2022

Overall, the aim is to improve the efficiency of drug discovery by identifying genetic variants that contribute to human disease in omics data – a broad category that includes DNA profiles, gene transcripts, and protein expression.

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Genome Genomics DNA Drugs 64

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. Here are four life sciences trends to look out for in 2023.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

Pharmaceutical Technology

JANUARY 31, 2023

The US omnibus bill passed in late December 2022 will give the agency greater powers to regulate accelerated approval drugs, while the Inflation Reduction Act’s lowering of drug prices will create an uncertain environment for drug developers, he said. That leaves three years for pharma companies to prepare. It’s much needed.

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Drug Discovery World

DECEMBER 27, 2023

Developing. Ng is working on critical challenges in clinical trials, specifically in planning and recruitment, and has been building innovative solutions designed to benefit patients and researchers – with a focus on efficiency, transparency and diversity in clinical development. DDW Highlights Innovative. Listen here. Listen here.

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Clinical Trials Clinical Trials Drugs Development 69

XTalks

DECEMBER 20, 2023

These advancements and trends addressed important industry challenges and offered opportunities to foster innovations and solutions in areas including drug development, healthcare and medical devices. Top 30 Pharma Companies in 2023: Statistics and Trends Xtalks assembled a comprehensive list of the top pharma companies in 2023 by revenue.

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pharmaphorum

DECEMBER 29, 2022

A new study by AspenTech in collaboration with FT Longitude confirms, for example, how digital transformation is on the rise in pharma to meet the demands of such new modalities as cell and gene therapies, and biologics. Crisis readiness and digital workflows. Removing manual pen-on-paper processes should be an obvious first step.

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Manufacturing Medicine Pharma Companies Engineer 111

pharmaphorum

NOVEMBER 23, 2021

The DISC is the biggest investment AZ has ever made, and has been hailed by the pharma company’s chief executive Pascal Soriot as the centrepiece of what he believes could one day become a biotech hub to rival Boston and San Francisco in the US.

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Gene Editing Life Science Pharma Companies Clinical Trials 105

pharmaphorum

APRIL 28, 2022

Although they have, by definition, small addressable markets, the treatments cost just as many resources to develop as therapies for more common diseases – sometimes more, because of the added difficulty of finding sufficiently large patient populations for trials and finding disease experts to run them. Alexion’s new development hub.

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Pharma Companies Branding Development Marketing 59

Pharmaceutical Technology

JUNE 14, 2023

Biologics form 51% of the current global drugs pipeline (highest stage of development as pipeline) according to GlobalData’s Drugs database (as of February 7, 2023). It is common for requirements to evolve and specifications to develop as the project unfolds.

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Contract Packaging Packaging Packaging Development 130

Drug Discovery World

FEBRUARY 10, 2023

Scandinavia represents one of the most exciting but perhaps underrepresented markets for the discovery and development of pharmaceuticals. Sweden’s place in the market Scandinavia is home to some of the strongest research and development hubs, with highly respected academic institutes that are helping drive the sector forward.

Life Science 52

Life Science Drugs Gene Therapy Manufacturing 52

pharmaphorum

JANUARY 6, 2021

The Jefferies team also noted that the French biotech is also partnered with AbbVie to develop an oral successor to its inflammatory diseases blockbuster Humira (adalimumab). Proof-of-concept phase 1b data is due from the drug codenamed ABBV-157 is due this quarter, which will help AbbVie decide on whether to continue development.

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Gene Silencing Regulation Pharma Companies Doctors 52

Pharmaceutical Technology

JULY 6, 2022

This period of hype saw companies going public earlier, which could explain why we may see some companies being merged or bought out, says Jason Foster, CEO of Ori Biotech, a private cell and gene therapy manufacturing company. The bear market has also impacted venture capital investment into private pharma companies.

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Marketing Gene Therapy Manufacturing Pharma Companies 162