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 88

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).

Gene Therapy 52

Gene Therapy Gene Pharma Companies Drugs 52

Delveinsight

SEPTEMBER 3, 2021

Key pharmaceutical companies such as Pharnext, MedDay Pharmaceuticals, among others are proactively working in the domain; however, the CMT disease pipeline constitutes only a few therapeutic agents such as MD1003 and PXT3003 in advanced stages of clinical trials.

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Pharma Companies Marketing Clinical Trials Clinical Trials 52

Drug Discovery World

MAY 31, 2024

Cell and gene therapy delivery is another area where current digital tracking solutions are needed to replace the paper-based documentation often used in clinical trials. Especially, as they require removing cells from a patient or donor, modifying them, and then reintroducing to a patient.

Biobanking 52

Biobanking Gene Therapy Pharma Companies 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.

Gene Therapy 104

Gene Therapy Gene Antibody Clinical Trials 104

XTalks

MAY 11, 2023

Clinical trials are essential for the development of new treatments for rare diseases, but they can be complex and challenging to execute. Patient input is a crucial component of rare disease clinical trials, as it can provide valuable insights into the patient experience and inform decisions about study design and implementation.

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Clinical Trials Clinical Trials Trials Development 97

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

MAY 21, 2021

Once all of that is addressed and a treatment is discovered, it goes through years and sometimes decades of clinical trials and final regulatory review. How can we ensure pharma companies continue to innovate if there are these hurdles? The challenge is for the patient to be able to access the treatment.

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Gene Therapy Gene Clinical Trials Clinical Trials 117

Drug Discovery World

MAY 6, 2024

billion on R&D during 2021, with Germany and Switzerland being the biggest contributors, and around one fourth of clinical trials worldwide were performed in Europe 1. The city is host to many innovative biotech companies, including relative newcomers Orexa, a company that recently launched its first Phase II clinical trial.

Drugs 59

Drugs Life Science Gene Editing Gene 59

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

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

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

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. AZ/Takeda are meanwhile running phase 2 trials of their MEDI 1342 antibody candidate. Interest in the target has also prompted a flurry of licensing deals.

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Antibody Protein Gene Therapy Pharma Companies 97

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. Aligning with the focus of the Cancer Moonshot Research Initiative , PACT aimed to “retrospectively analyse patient data” from past clinical trials with the goal of “predicting future patient outcomes.”.

Research 132

Research Gene Editing Gene Development 132

XTalks

DECEMBER 20, 2023

‘No Time to Wait’ Ad Campaign from BMS-Pfizer Features Basketball Legend Kareem Abdul-Jabbar for AFib Awareness As pharma TV advertising remained popular in 2023, so did partnerships between pharma companies and celebrities for disease and drug campaigns.

Life Science 59

Life Science Clinical Trials Clinical Trials FDA Approval 59

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

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

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

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.”

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Drugs Regulation Gene Therapy Insulin 130

pharmaphorum

JANUARY 6, 2021

Analysts are continuing to back Inventiva’s lanifibranor as a potential “best-in-class” drug for the fatty liver disease NASH, as the company prepares for the launch of a phase 3 trial in spring. The trial will take a while however, with results not due until the second half of 2023, with sales forecast to peak at around $2.6

Gene Silencing 52

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.

Marketing 162

Marketing Gene Therapy Manufacturing Pharma Companies 162

pharmaphorum

NOVEMBER 22, 2022

Francis, who is currently CEO of gene therapy specialist Purespring Therapeutics and cardiovascular drug developer company Forcefield Therapeutics, is due to take the top job at Teva on 1 January, and will serve as the pharma company’s president. Richard Francis.

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Sales Gene Therapy Pharma Companies Gene 57

Drug Discovery World

DECEMBER 27, 2023

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. Listen here. Listen here. Listen here.

Clinical Trials 69

Clinical Trials Clinical Trials Drugs Development 69

pharmaphorum

DECEMBER 23, 2020

The American Society of Clinical Oncology (ASCO) annual conference was one of the highlights of the year in cancer research and the fact it was held virtually did not distract from some stunning clinical lung cancer trial results presented by AstraZeneca. In the 500 patient trial, patients treated with Trodelvy lived for a median of 12.1

Marketing 87

Marketing Gene Silencing Gene Drugs 87

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. billion in funding.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

pharmaphorum

JUNE 11, 2021

Ocugen added that it is now “in discussions with the FDA to understand the additional information required to support a BLA submission,” but will likely have to carry out an additional clinical trial. It was given the go-ahead in January, well before Bharat reported phase 3 data suggesting a protective efficacy of 78%.

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Vaccination Vaccine Clinical Trials Clinical Trials 77

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.

Life Science 98

Life Science Pharma Companies Sales Marketing 98

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. How big pharma tackles rare diseases.

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Pharma Companies Branding Development Marketing 57

The Pharma Data

OCTOBER 20, 2020

In hopes to advance the best and brightest, MD Anderson, in partnership with The Focus Fund GP, has launched the Cancer Focus Fund to propel compelling investigational cancer therapies from preclinical development through Phase II clinical trials. Targeted therapies, genetic sequencing, gene editing, immunotherapies.

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Clinical Trials Clinical Trials Gene Editing Trials 52

Delveinsight

JULY 26, 2020

However, pharma companies such as TauRx Therapeutics , Axon Neuroscience SE , Prevail Therapeutics , Avid Radiopharmaceuticals , and Alector are actively working to buildup the Frontotemporal dementia pipeline. The Frontotemporal dementia market is expected to increase with a CAGR of 21.41% for 7MM during the study period.

Marketing 54

Marketing Pharma Companies Manufacturing Clinical Trials 54

Drug Discovery World

SEPTEMBER 28, 2022

Its application in pharma, most commonly in drug discovery, has been steadily growing with global partnerships between tech vendors and pharma companies becoming more commonplace. According to Hampleton Patners’ latest Healthtech M&A market report2, digital health companies raised a total of $57.2

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

Delveinsight

DECEMBER 15, 2020

Gannex Pharma has received Fast Track designation approval from FDA for its drug candidate ASC42 for non-alcoholic steatohepatitis (NASH). The FTA designation will help the pharma company to advance its research and development in the NASH landscape and help its commercialization in the market.

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Vaccination Vaccine Gene Therapy Gene 54

Drug Discovery World

MARCH 29, 2023

Moreover, an extra six months of RDP for drugs with full comparator clinical trials (rather than showing only that a drug is no worse than the standard of care). Will this be a viable solution? Clinical trials The draft outlines the requirement for clinical trials in children where a drug is approved in adults for a different disease.

Clinical Trials 52

Clinical Trials Clinical Trials Production Medicine 52

pharmaphorum

APRIL 26, 2022

Research into the area is being made possible through advancements in technology, such as being able to sequence genes at scale, allowing researchers to gain more detailed genetic data on the microbiome. In the trial, Seres announced that the product candidate had met its primary endpoint, showing a statistically significant 30.2%

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Containment Development Production Immune Response 113

Delveinsight

FEBRUARY 14, 2021

Developing a new drug requires years of research, time, and money; and in cases where winds are against the market forces and profit margins appear shallow, conducting and bearing the cost for such trials can prove to be a pretty penny. Conducting clinical trials for rare diseases? Aftermath of clinical trials.

Marketing 52

Marketing Clinical Trials Clinical Trials Trials 52

XTalks

APRIL 16, 2024

The drug is a calcitonin gene-related peptide (CGRP) receptor inhibitor that blocks CGRP receptors in the brain to reduce migraine symptoms of headache, nausea, vomiting, light sensitivity and sound sensitivity. Backlash against celebrities for partnering with pharma companies isn’t new. million in 2023.

Drugs 52

Drugs Pharma Companies Doctors Doctor 52

pharmaphorum

NOVEMBER 16, 2020

Under Rasi, the EMA implemented a suite of reforms including a drive towards transparency – for example making clinical study reports (CSRs) publicly available – and the implementation of sweeping EU directives on pharmacovigilance, clinical trials and falsified medicines.

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