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.”. The post Novartis says £1.8m

Gene Therapy 96

Gene Therapy Gene Pharma Companies Clinical Trials 96

pharmaphorum

JANUARY 29, 2021

Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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.

Gene Therapy 143

Gene Therapy Pharma Companies Gene Research 143

Pharma Times

JUNE 10, 2021

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

Gene Therapy 121

Gene Therapy Pharma Companies Gene 121

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genome 264

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.

Gene Therapy 59

Gene Therapy Gene FDA Approval Trials 59

BioPharma Reporter

OCTOBER 31, 2023

The London and New York-based gene therapy player MeiraGTx Holdings has received a $30 million investment from Sanofi in addition to potential strategic deals with the big pharma company down the road.

Gene Therapy 52

Gene Therapy Gene Pharma Companies Regulation 52

pharmaphorum

NOVEMBER 12, 2020

UCB has made a new foray into the gene therapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its gene therapy drive with Handl acquisition appeared first on.

Gene Therapy 52

Gene Therapy Gene Manufacturing Contract Manufacturing 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

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?

Gene Therapy 162

Gene Therapy Packaging Packaging Contract Packaging 162

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. In serious cases, higher levels of pre-existing NAbs can even cause a dangerous immune reaction when such therapies are administered.

Gene Therapy 105

Gene Therapy Gene Antibody Clinical Trials 105

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?

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

Fierce Pharma

MARCH 21, 2024

Three large pharma companies plan to expand their manufacturing footprints. million gene therapy set a new world pricing record. Three large pharma companies plan to expand their manufacturing footprints. million gene therapy set a new world pricing record. Kyowa Kirin’s $4.25 Kyowa Kirin’s $4.25

Gene Therapy 72

Gene Therapy Pharma Companies Gene Manufacturing 72

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.

Marketing 324

Marketing Gene Therapy Manufacturing Pharma Companies 324

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?

Gene Therapy 119

Gene Therapy Gene Clinical Trials Clinical Trials 119

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

Gene Therapy 98

Gene Therapy Clinical Development Antibody Gene 98

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.

Life Science 52

Life Science Gene Therapy Gene Pharma Companies 52

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.

Life Science 98

Life Science Gene Therapy Gene Editing Gene 98

Pharmaceutical Technology

JUNE 14, 2023

Future waves of approvals for ATMPs (advanced therapy medicinal products, e.g., cell and gene therapies) will heighten demand on the contract packaging industry, further increasing the need for advanced cold chain solutions and specialized packaging designed to withstand cryogenic temperatures.

Contract Packaging 130

Contract Packaging Packaging Packaging Development 130

World of DTC Marketing

JANUARY 31, 2021

The venture capitalists funding them are looking for a big payday when a pharma company acquires them. I have been recruited by these companies who promise huge payouts when they are acquired if their drug shows promise in clinical trials. In pharma R&D returns have declined to 1.8 percent—a slight decrease of 0.1

In-Vivo 187

In-Vivo Generic Drugs Drugs Gene Therapy 187

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

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

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. Many novel therapies have been successfully developed through such partnerships. Case Study: From Technology Agreement to co-development and co-commercialisation partnership.

Research 132

Research Gene Editing Development Gene 132

XTalks

MAY 29, 2024

The top ten pharma companies in 2023 by revenue accounted for about 35 percent of the market value, with total earnings of $559.5 The top big pharma players have remained relatively consistent in their market positions over the past several years. Here is a look at the top ten pharma companies in 2024 based on 2023 revenue.

Pharma Companies 52

Pharma Companies Sales FDA Approval Vaccination 52

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?

Marketing 105

Marketing Development Drugs Gene Therapy 105

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. Ori raised over $100 million (EUR97.52

Marketing 162

Marketing Gene Therapy Manufacturing Pharma Companies 162

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.

Life Science 130

Life Science Manufacturing Contract Manufacturing Gene Therapy 130

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.

Antibody 97

Antibody Protein Gene Therapy Pharma Companies 97

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.

Life Science 52

Life Science Gene Therapy Drugs Pharma Companies 52

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.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

DECEMBER 20, 2023

“In 2024, pharma companies and emerging biotechs will improve candidate safety profiles and reduce off-target effects by exploiting predictive intelligence unlocked by AI,” she predicts. CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

pharmaphorum

AUGUST 4, 2021

This changed with the 2017 release of Biogen’s Spinraza, the first FDA-approved therapy for SMA. The drug – the first of a new generation of gene therapies – has been demonstrated to significantly impact disease progression. About the author.

Gene Therapy 145

Gene Therapy FDA Approval Pharma Companies Development 145

Pharmaceutical Technology

JANUARY 31, 2023

While insulin price caps will begin this year, larger effects will be delayed until 2026 – when the US federal government will be allowed to negotiate Medicare prices and demand rebates from drug companies that price gouge. That leaves three years for pharma companies to prepare. It’s much needed. It’s a hot area.”

Drugs 130

Drugs Gene Therapy Regulation Insulin 130

XTalks

OCTOBER 22, 2020

In this sense, pharmaceutical companies have developed different strategies to produce a wide variety of advanced therapy medicinal products (ATMP) and biological products, such as monoclonal antibodies, recombinant proteins, gene therapy viral vectors, CAR-T cells and oncolytic viruses, in order to deliver new medical needs in our society.

Manufacturing 98

Manufacturing Development Production Gene Therapy 98

pharmaphorum

MARCH 22, 2022

The efforts underway to strengthen the manufacturing process across the industry have become more important than ever, as the therapies the industry is developing increase in complexity. However, with a number of deals and investments being made in the space, the demand is there to develop more efficient methods of training and manufacturing.

Manufacturing 137

Manufacturing Production Contamination Gene Therapy 137

pharmaphorum

JUNE 11, 2021

It’s a big disappointment for the specialist in drugs and gene therapies for ocular diseases, although the decision to embark on a COVID-19 programme has been viewed as a somewhat maverick move for the biotech.

Vaccination 83

Vaccination Vaccine Clinical Trials Clinical Trials 83

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.

Manufacturing 111

Manufacturing Medicine Pharma Companies Engineer 111

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. Frontiers Health 2022.

Gene Therapy 52

Gene Therapy Marketing Gene Sales 52