Pharmaceutical Technology

JULY 14, 2022

It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Go-to-market strategies.

Drugs 147

Drugs Medicine Marketing Development 147

Rethinking Clinical Trials

AUGUST 18, 2022

People are “underrepresented” because they have been historically excluded and marginalized. There are barriers at the health care professional and researcher level. There are barriers at the health care professional and researcher level. How far along are we in the reframing of looking at the true underlying factors?

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Clinical Trials Clinical Trials Trials Insulin 130

Drug Discovery World

MARCH 2, 2023

But the new findings answer a fundamental and longstanding question – how epigenetic proteins regulate the processes of transcription and gene expression, through which our genes are read and translated into proteins. But, until now, scientists lacked an understanding of what the chemical tag does, despite many years of research.

Gene Expression 52

Gene Expression Gene DNA RNA 52

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. Currently, all approved CAR-T therapies are autologous, where T cells taken from patients are modified and then re-infused. There are issues of geography, but also issues of economics.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

XTalks

OCTOBER 26, 2021

The treatment came with an initial price tag of $39,000 per vial, which amounts to a total of $575,00 each year. AHP is a life-threatening genetic condition caused by an inherited deficiency in the metabolic enzyme hydroxymethylbilane synthase (HMBS), also known as porphobilinogen deaminase (PBGD).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

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Drugs Gene Therapy Genetic Disease Gene 98

Drug Discovery World

JANUARY 30, 2024

Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

XTalks

DECEMBER 13, 2023

Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. The CRISPR system is dubbed to be molecular “scissors” that can cut out parts of genes that are abnormal, deactivating or replacing them with new strands of normal DNA. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

JUNE 8, 2021

SMA is a rare genetic condition that leads to a loss of motor neurons that results in progressive muscle weakness and wasting, paralysis and, when left untreated in its most severe form, breathing difficulties leading to permanent ventilation or death for most patients by the age of two. With a price tag of over $2.5

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

The Pharma Data

MARCH 23, 2022

To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase. Early in infection, it appears that host cellular proteins are in defense mode against Ebola virus polymerase and the viral genetic material. Catching polymerase in the act.

Protein 52

Protein Genome Genomics Research 52

Drug Discovery World

JANUARY 10, 2023

We are vertically-integrated from immunisation through manufacturing and validation. All of our antibodies are highly validated using six pillars of validation, meaning that our antibodies do what we say they’ll do. Bethyl antibodies are highly regarded for passing strict validation testing before arriving in customer hands.

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Antibody Protein Reagent Scientist 52

pharmaphorum

DECEMBER 23, 2022

But along with strong efficacy come some considerable risks and costs: cytokine release syndrome (CRS), where a patient’s immune system becomes dangerously over-stimulated, and neurotoxicity are potential side effects that need to be managed. These are particularly important in the progression of certain blood cancers.

Development 52

Development Drugs Clinical Development Antibody 52

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

DNA 52

DNA In-Vivo Genetics Medicine 52

pharmaphorum

AUGUST 18, 2022

Zynteglo is a customised, one-time treatment created using a patient’s own bone marrow stem cells hat are genetically modified to produce functional beta-globin, which is mutated in the disease. million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

The Pharma Data

OCTOBER 21, 2020

The disease is a subtype of ALL in which the cancer cells have a genetic abnormality called the Philadelphia chromosome (Ph). Traditionally, Ph-positive ALL had a poor prognosis, but in the past two decades that has changed with the introduction of drugs that target the genetic anomaly. The patients were followed for up to two years.

Drugs 52

Drugs Genetics Trials Research 52

The Pharma Data

OCTOBER 21, 2020

The disease is a subtype of ALL in which the cancer cells have a genetic abnormality called the Philadelphia chromosome (Ph). Traditionally, Ph-positive ALL had a poor prognosis, but in the past two decades that has changed with the introduction of drugs that target the genetic anomaly. The patients were followed for up to two years.

Drugs 52

Drugs Genetics Trials Research 52

pharmaphorum

FEBRUARY 22, 2021

If a causal link is shown, it could have “significant ramifications” on the use of lentiviral vectors, a type of self-inactivating virus that underpins both approved cell therapies like Novartis’ CAR-T Kymriah as well as a host of experimental therapies that involve genetic manipulation of cells. million in Europe.

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Gene Therapy Gene Gene Sequencing Gene Expression 52

Roots Analysis

JANUARY 28, 2024

Velocity: Big Data needs to be handled promptly as businesses generate data at an unprecedented speed, driven by the growing adoption of Internet of Things (IoT), and technologies, such as RFID tags, sensors, and smart meters are used to deal with the high velocity of data in real-time.

Big Data 40

Big Data Medicine Marketing Clinical Trials 40

pharmaphorum

JUNE 2, 2021

The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people with the rare genetic disorder who might be eligible to receive it. Zolgensma was backed for type 1 SMA in draft guidance issued last month, even though the gene therapy’s £1.79

Gene Therapy 52

Gene Therapy Gene Drugs Medicine 52

XTalks

AUGUST 17, 2020

There are several different types of immunotherapies, including chemical inhibitors, cell-based therapies (i.e. Immune checkpoint inhibitors : these are drugs that block immune checkpoints, allowing immune cells to elicit enhanced responses to cancer cells. protein-based biologics) : are those which bind to specific components (i.e.

Protein 98

Protein Engineer Immune Response In-Vivo 98

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. Tools like PRO measures (PROMs) are being used more frequently to assess the impact of treatments from the patient’s perspective.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

Delveinsight

DECEMBER 3, 2020

There are presently no medical treatments, which can regenerate retinal cells that have atrophied, though gene therapies and stem cell therapies are the leading R&D hopes in this arena. The researchers concentrated on the “Yamanaka factors,” which are four transcription factors—Oct4, Sox2, Klf4 and c-Myc.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

pharmaphorum

DECEMBER 7, 2021

They used computational techniques to sift through genetic and other biologic data as well as insurance claims information from a database of more than 7 million patients, and found that sildenafil had the greatest potential as an Alzheimer’s drug.

Drugs 52

Drugs Clinical Trials Clinical Trials Scientist 52

Drug Discovery World

NOVEMBER 9, 2023

Thereafter, there will be two presentations, which are: Jan Terje Andersen, PhD, Professor, Department of Pharmacology, University of Oslo; Research Group Leader, Department of Immunology, Oslo University Hospital, on: ‘Biology-based engineering of versatile antibody and albumin technologies’.

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Engineer Antibody Protein In-Vivo 52