Pharmaceutical Technology

MAY 1, 2023

CTH-004 is developed by genetically altering patient T cells for inserting a chimeric antigen receptor (CAR) to target a marker (TAG-72) on ovarian cancer cells and delete genes which are involved in T cell function suppression. Shunxi will also hold an option to negotiate rights to other CAR-T products, which include the licenced IP.

Gene Therapy 130

Gene Therapy Gene Development Clinical Trials 130

Pharmaceutical Technology

MAY 2, 2023

Chimeric antigen receptor T-cell (CAR-T) therapies are the only genetically modified cell therapies to have received regulatory approval, and they are currently utilised in relapsed/refractory settings. Cost-related unmet needs also scored highly.

Genetic Engineering 130

Genetic Engineering Antibody Clinical Trials Clinical Trials 130

Drug Discovery World

MARCH 2, 2023

New understanding of epigenetics It has been known for more than 20 years that the enzymes placing H3K4me3, a chemical tag added to DNA, are crucial for normal cell development, as well as being linked to leukaemia, breast, bowel and pancreatic cancers. The team found that H3K4me3 acts like a traffic light at a busy intersection.

Gene Expression 52

Gene Expression Gene DNA RNA 52

The Pharma Data

FEBRUARY 22, 2022

As part of its on-going work to track variants, WHO’s Technical Advisory Group on SARS-CoV-2 Virus Evolution ( TAG-VE ) met yesterday to discuss the latest evidence on the Omicron variant of concern, including its sublineages BA.1 1 in its genetic sequence, including some amino acid differences in the spike protein and other proteins.

Protein 52

Protein Vaccination Vaccine Genetics 52

Scienmag

SEPTEMBER 9, 2020

Credit: © 2020 KAUST Purifying specific protein molecules from complex mixtures will become easier with a simpler way to detect a molecular “tag” commonly used as a handle to grab the proteins.

Protein 60

Protein Genetics 60

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. These countries will have a smaller patient pool due to population size and will likely carry a price tag that is significantly below that of higher GDP countries.”.

Drugs 147

Drugs Medicine Marketing Development 147

Rethinking Clinical Trials

MAY 10, 2023

Participants shared that genetic information is a type of information they want to get back. Tags #pctGR, @Collaboratory1 The post Grand Rounds May 5, 2023: All of Us Research Program: Improving Health Through Diverse Technology, Huge Cohorts, and Precision Medicine (Joshua C. Returning value is a key part of the program.

Medicine 130

Medicine Research Clinical Trials Clinical Trials 130

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.

Drugs 91

Drugs Gene Therapy Genetic Disease Gene 91

Rethinking Clinical Trials

AUGUST 18, 2022

If we accept and acknowledge that race is a social construct, not a category that is tied to biological or genetic differences, then what does that mean when we see clear differences and disparities in health outcomes for these populations? Discussion Themes. -If Learn more: Improving Representation in Clinical Trials and Research.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Insulin 130

The Pharma Data

MARCH 23, 2022

Early in infection, it appears that host cellular proteins are in defense mode against Ebola virus polymerase and the viral genetic material. This special enzyme, once fused to Ebola virus polymerase, enables the polymerase to add a molecular tag to any other protein it interacts with.

Protein 52

Protein Genome Genomics Research 52

XTalks

JUNE 28, 2023

DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. million price tag of Elevidys, a one-time gene therapy. It primarily affects boys, affecting approximately one in 3,300 to 3,600 male births worldwide. In a media call, Sarepta CEO Douglas Ingram revealed the $3.2

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.” Vertex-CRISPR’s Casgevy has a US list price of $2.2 million and bluebird’s Lyfgenia is 40 percent higher at $3.1

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

Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Other Challenges: Limited insurance coverage: Many insurance companies are hesitant to cover CGTs due to their high price tags and limited long-term data.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

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

XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). With a list price of $3.5

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetics 52

pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. million price tag. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

Gene Therapy 59

Gene Therapy Gene Genetic Disease Manufacturing 59

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

DECEMBER 23, 2022

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Add to this the considerable cost of these medications – the first approved CAR-T, Novartis’ Kymriah (tisagenlecleucel), had a U.S.

Development 52

Development Drugs Clinical Development Antibody 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.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

Drug Discovery World

JANUARY 10, 2023

Pillar 5: Protein OE/Epitope Tags. Pillar 6: Genetic Strategies. This pillar takes advantage of the unique biology associated with some protein targets. This pillar uses gene knockout or knockdown to reduce the levels of target protein available for detection.

Antibody 52

Antibody Protein Reagent Scientist 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. But I think we’ve never had more tools than we have now in synthetic biology, genetic engineering, enhanced potency design switches and CRISPR. But there is that significant challenge.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

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

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

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. It is loosely arranged into categories using meta tags.

Big Data 40

Big Data Medicine Marketing Clinical Trials 40

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. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

The Pharma Data

MARCH 8, 2022

Omicron is comprised of several genetically related sublineages, including BA.1, This statement highlights the global epidemiological situation, challenges of streamlining vaccine composition and provides the current position of the Label-CO-VAC. 3, each of which is being covered by WHO and mates. At a global position, BA.1

Vaccination 52

Vaccination Vaccine Manufacturing Development 52

Delveinsight

DECEMBER 3, 2020

It has been speculated that modifications to the epigenome drive cell ageing, especially a process known as DNA methylation, by which methyl groups are tagged onto DNA. Ovid showed a phase 2 data on OV101 in Angelman, a genetic nervous system disorder, in 2018. Merck chooses TriNKET cancer immunotherapy program.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

XTalks

AUGUST 17, 2020

A growing area in the immunotherapy space is the development of biological modulators, which are genetically engineered proteins that target specific components of the immune system. The beauty with Neo-STAT is that you can make the entire [HLA-based] scaffold along with the IL-2 elements for cancer immunotherapy without a peptide.

Protein 98

Protein Engineer Immune Response In-Vivo 98

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

Marine Houdou, Genetic Engineering Specialist, Polyplus, on: ‘From modular DNA assembly to recombinant protein production at Polyplus’. Lena Tholen, Director, Cell line and Bioprocess Development, FyoniBio, on: ‘Fyonibio’s versatile cell line expression platforms for the development of complex molecules’.

Engineer 52

Engineer Antibody Protein In-Vivo 52