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? There is plenty of data that shows that. The social circumstances affect health outcomes.

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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

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 98

Drugs Gene Therapy Genetic Disease Gene 98

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

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.

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Gene Therapy Gene Genetic Disease Manufacturing 60

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.” Data on the long-term effects of the therapies is limited, as trials evaluating the therapies were only two years long.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

DECEMBER 23, 2022

Here, Richard Staines reports on phase I/II data unveiled at the American Society of Hematology conference that supports further clinical development of inobrodib in multiple myeloma and other blood cancers. Oral therapy data presented at ASH. Oral therapy data presented at ASH. CellCentric, a U.K-based

Development 52

Development Drugs Clinical Development Antibody 52

XTalks

JUNE 8, 2021

In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. With a price tag of over $2.5 million, Zolgensma is currently the world’s most expensive drug. It was first approved in the US in 2019 to treat children below the age of two with SMA.

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Gene Therapy Gene Protein Trials 110

Drug Discovery World

JANUARY 10, 2023

Scientists purchase antibodies with the expectation that they will recognise their intended targets and produce reliable data, but the presence of improperly validated antibodies on the market can make this process both time-consuming and expensive. Pillar 5: Protein OE/Epitope Tags. Pillar 6: Genetic Strategies.

Antibody 52

Antibody Protein Reagent Scientist 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

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.

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Gene Therapy Gene Engineer Production 147

pharmaphorum

AUGUST 18, 2022

An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June. 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, which compares to the $1.7

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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

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 study commenced in September 2019 (NCT03978689) and interim data is expected later this year.

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

Research 59

Research Drugs Gene Therapy Clinical Trials 59

XTalks

FEBRUARY 11, 2021

This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics. Rosalind Franklin.

Genetics 119

Genetics DNA Genome Genomics 119

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

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

Sven Malik, Mr, Senior Application Specialist, Applications, Bruker Daltonics SPR, on: ‘Bruker’s SPR Analyser 4 – State of the art SPR data analysis’. Marine Houdou, Genetic Engineering Specialist, Polyplus, on: ‘From modular DNA assembly to recombinant protein production at Polyplus’.

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Engineer Antibody Protein In-Vivo 52