pharmaphorum

DECEMBER 22, 2021

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. The one-shot therapy aims to treat GA by delivering the gene for complement factor I (CFI) to the eye using an adeno-associated virus (AAV) vector.

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Bio Pharma Dive

JULY 22, 2022

The pharma company plans to spend $70 million on the South San Francisco facility, which will house West Coast employees of Astellas Gene Therapies.

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pharmaphorum

MAY 25, 2021

The UK has been a global leader in introducing cell and gene therapies that can potentially transform the lives of patients with debilitating diseases – but there are still many barriers to wider adoption. This includes increasing the prominence of cell and gene therapy in access policy discussions.

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pharmaphorum

NOVEMBER 23, 2022

uniQure and CSL have become the first pharma companies to get regulatory approval for a gene therapy for haemophilia B anywhere in the world, after the FDA cleared their Hemgenix therapy for adults with the bleeding disorder in the US. The post uniQure, CSL claim first okay for haemophilia B gene therapy appeared first on.

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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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pharmaphorum

OCTOBER 13, 2020

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. He said: “These big pharma companies have every type of technology available to them.

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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. The pharma company will expand its manufacturing capacity for commercial scale vector production for oncology treatment. How Does the Gene Therapy Adstiladrin Work?

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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.

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Pharma Times

JUNE 10, 2021

German pharma company announces progress in its 'two-pronged' cell and gene therapy approach to Parkinson's disease

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Delveinsight

SEPTEMBER 3, 2021

The post Charcot-Marie-Tooth Disease Market Size Expects Growth Owing to Increasing Prevalence, Influx of Pharma Companies and Novel Therapies appeared first on DelveInsight Business Research.

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XTalks

JANUARY 4, 2023

With a handful of significant gene therapy approvals in 2022, the cell and gene therapy space continues to rapidly expand, and hence we can expect more ground-breaking gene therapy approvals in the coming year, particularly for rare diseases. Read the full article here: 4 Life Sciences Trends for 2023.

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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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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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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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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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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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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

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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.

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BioTech 365

OCTOBER 15, 2020

ISTANBUL–(BUSINESS WIRE)– Company name: Er-Kim Ilac Presentative: Cem Zorlular, CEO AnGes, Inc.,

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Drug Discovery World

MAY 6, 2024

A more established Amsterdam-based enterprise is gene therapy company uniQure, developer of etranacogene dezaparvovec, the first gene therapy for haemophilia B, which was approved for use in the US in November 2022 and in the EU in February 2023. More than 400 companies now make up Lithuania’s life sciences sector 4.

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pharmaphorum

MAY 21, 2021

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. How can we ensure pharma companies continue to innovate if there are these hurdles?

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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. Other challenges involve talent gaps, particularly now that cell and gene therapy manufacturing is maturing.

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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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Pharmaceutical Technology

MAY 26, 2023

Pharma company merger and acquisition (M&A) activity in the first half of 2023 has remained relatively low, consistent with the second half of last year, but investors predict deals will increase later in 2023. Experts at PWC, Goldman Sachs, and Morgan Stanley agree that pharma-related M&A is expected to pick up in 2023.

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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.

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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.

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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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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. There are a growing number of biotech and pharma companies that have established or are establishing such end-to-end cell therapy capabilities, which can also play into how collaborations are structured in the field.

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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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pharmaphorum

SEPTEMBER 22, 2022

Companies can only do so much to market digital therapeutics until certain information infrastructure is in place, says Kristin Milburn, managing director of Healthware Labs. Gene therapies for prevalent diseases in Europe. Moving forward the evolution of digital maturity in pharma.

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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. Then, in January, Sanofi stumped up $75 million upfront for a preclinical-stage bispecific antibody from South Korea’s ABL Bio in a $1 billion deal.

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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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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.

Gene Editing 104

Gene Editing Life Science Pharma Companies Clinical Trials 104

Drug Discovery World

APRIL 3, 2023

As the industry’s awareness of alternatives to traditional antibodies continues to grow, there is increasing demand for these alternatives, which we are seeing particularly within the therapeutic sector, to enable targeted therapeutics and enable the emerging gene therapy market. DS: The company now has clients across big pharma.

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pharmaphorum

NOVEMBER 30, 2022

The biggest gathering of pharma companies in Europe right now on the commercial side, the Veeva Systems Summit permits the sharing of best practices and of new ideas. Facts that they gleaned included: Across Europe, only 52% of HCPs will accept a visit from a pharma company (i.e.

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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., Preclinical and clinical programs at Northern Biologics were acquired by big pharma companies in 2020.

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pharmaphorum

JANUARY 24, 2023

Pharma has used this to begin creating targeted treatments, such as gene therapies , which are able to hone in on gene mutations that cause health issues and eradicate or replace the faulty gene.

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