pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

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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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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.

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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.

Immunity 52

Immunity Immunization Vaccines 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.

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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.

Drug Development 52

Drug Development Immunity Immunization Hospitals 52

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.”.

Pharmaceutical Companies 59

Pharmaceutical Companies Pharmaceutical Companies Hospitals 59

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.

Immunity 52

Immunity Immunization 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

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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

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Documentation Communication 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.

Immunity 59

Immunity Immunization 59

PharmaShots

MARCH 9, 2023

She developed various mobile technologies and pioneered early electronic labels including NFC tags, which helped Thin Film Electronics win the Wall Street Innovation Award and the Global Technology Award. Other Affiliations: She holds New York State Department of Health certification in Molecular Genetics and Molecular Oncology.

Clinical Pharmacology 40

Clinical Pharmacology Immunity Immunization Pharmaceutical Companies 40

European Pharmaceutical Review

MAY 11, 2023

Rare, genetic diseases like SMA are prime targets for gene therapy, but by their very nature, early signs can be difficult to recognise, delaying diagnosis and treatment. 1 This is assuming a $1M price tag for all gene therapies. What are the challenges in treating conditions such as spinal muscular atrophy?

Drug Development 70

Drug Development 70

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.

Compounding 52

Compounding Diabetes 52

European Pharmaceutical Review

NOVEMBER 10, 2022

BT-474 cell line was gene-edited to incorporate a green fluorescent protein (GFP) tag into late endosomes/lysosomes (green). Her expertise lies in developing cellular models by applying genetic engineering techniques, including viral vectors and CRISPR/Cas. Physical characterisation. During her PhD at St.

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Labelling Compounding 96

European Pharmaceutical Review

DECEMBER 27, 2023

This is in addition to its considerable price tag. He established the first public cord blood bank in Israel and performed the first cord blood transplantations from related and unrelated donors in genetic and malignant haematological diseases.

Adverse Reactions 83

Adverse Reactions Immunity Immunization Medical Schools 83