Drug Discovery World

NOVEMBER 20, 2023

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

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Drug Discovery World

NOVEMBER 20, 2023

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

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Drug Discovery World

NOVEMBER 20, 2023

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

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BioSpace

OCTOBER 14, 2020

Cyclerion is a clinical-stage biopharmaceutical company developing treatments for serious and orphan diseases affecting the central nervous system (CNS).

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pharmaphorum

JUNE 30, 2021

bluebird bio’s sickle cell disease (CD) gene therapy LentiGlobin is the latest recipient of an ‘innovation passport’ introduced in the UK earlier this year to speed up NHS access to promising new medicines. That was relaxed earlier this year and treatment is resuming.

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Drug Discovery World

JULY 15, 2022

News round-up by DDW’s Reece Armstrong for 11 – 15 July. First treatment for sickle cell side-effect in 20 years approved. The first new treatment in 20 years that prevents a common, painful side-effect in patients with sickle cell disorder (SCD) has been issued interim acceptance by the Scottish Medicines Consortium (SMC).

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pharmaphorum

FEBRUARY 17, 2022

Global Blood Therapeutics’ run of positive regulatory news for its oral sickle cell disease treatment Oxbryta has continued with an approval for the drug in Europe for patients aged over 12. The post GBT claims EU approval for sickle cell drug Oxbryta appeared first on.

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Drug Discovery World

NOVEMBER 27, 2023

This follows the news that the MHRA was the first regulatory authority to authorise Vertex’s gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Kaftrio is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface.

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STAT News

OCTOBER 6, 2022

… Multimillion-dollar prices for a rival’s treatments with the potential to cure rare diseases are “in the right ballpark,” according to Stuart Arbuckle, chief operating officer at Vertex Pharmaceuticals, which is preparing to market its own treatment for sickle-cell disease , Bloomberg News tells us. Even

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pharmaphorum

APRIL 23, 2021

CRISPR Therapeutics is to receive a hefty $900m payment from Vertex after the companies amended a collaboration to develop, manufacture and market a gene editing therapy for sickle cell disease and beta thalassemia.

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pharmaphorum

NOVEMBER 18, 2022

Next up in the news pipeline will be the presentation of first clinical data with EDIT-301 in sickle cell disease, although that relies on the use of CRISPR to modify cells ex vivo.

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pharmaphorum

FEBRUARY 22, 2021

Last week, Bluebird said it was pausing its Zynteglo (betibeglogene autotemcel) programme in Europe after a phase 1/2 trial in patients with sickle cell disease (SCD) – another red blood cell disorder – turned up two cases of blood cancers, one of acute myeloid leukaemia (AML) and another of myelodysplastic syndrome (MDS).

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XTalks

DECEMBER 24, 2020

The spike protein is critical for binding to ACE2 receptors on host cells — this interaction mediates the entry of the virus into cells. This is an internal protein in the virus, made in large quantities, so is not that important for humoral immunity, but it is a good target for cell-mediated immunity.”. Drug Approvals.

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Pharma Times

MAY 2, 2024

The inherited disorder is caused by genetic mutations that affects haemoglobin production

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The Pharma Data

NOVEMBER 25, 2020

Some other stroke risk factors, like diabetes and sickle cell anemia, are also more common among Black adults. Yet those “classic” risk factors do not tell the whole story, Dr. Hooman Kamel, the lead researcher on the new study, said in a journal news release.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

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Pharma Times

JANUARY 24, 2024

Patients living with sickle cell disease and thalassaemia will be eligible for the test

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Drug Discovery World

NOVEMBER 17, 2023

News round-up for 13-17 November by DDW Digital Content Editor Diana Spencer. The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing.

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pharmaphorum

NOVEMBER 7, 2022

Blood cells grown in a laboratory have been given to people for the first time in a clinical trial being carried out by researchers in the UK, in the hope that plentiful supplies of rare blood groups can be manufactured to order.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy Casgevy, a cell-based gene therapy, is approved for the treatment of SCD in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs).

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XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. she said. “We

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pharmaphorum

AUGUST 8, 2022

Pfizer’s reported interest in acquiring sickle cell disease specialist Global Blood Therapeutics (GBT) has been confirmed, with the $68.50-per-share The post Pfizer vaults into sickle cell market as GBT deal confirmed appeared first on. per-share deal valuing GBT at $5.4

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pharmaphorum

NOVEMBER 15, 2021

Patients with sickle cell disease are facing racism in the NHS that is putting their lives a risk, claims a new report. . “This is the most comprehensive report on sickle cell care that our All-Party Group has ever published,” he said.

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pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). “Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option,” he added.

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pharmaphorum

JANUARY 29, 2023

The EMA’s human medicines committee has started a review of Novartis’ sickle cell disease (SCD) therapy Adakveo, shortly after the company reported that it failed to show efficacy in a phase 3 trial. That study showed that Adakveo lowered the median annual rate of VOCs to 1.63 compared to 2.98

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pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. HbF produces normal, healthy red blood cells, rather than the misshapen cells produced by faulty haemoglobin in the two disorders.

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pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease.

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Scienmag

FEBRUARY 15, 2022

Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored by bluebird bio.

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Scienmag

FEBRUARY 16, 2022

(Boston)—Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed growth or puberty.

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pharmaphorum

SEPTEMBER 2, 2022

Novo Nordisk has doubled down on position in the sickle cell disease (SCD) category with a $1.1 The post Novo Nordisk steps up sickle cell push with $1.1bn Forma buy appeared first on. billion agreement to acquire Forma Therapeutics, and its therapeutic candidate etavopivat, in phase 2/3 testing.

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pharmaphorum

AUGUST 8, 2022

Pfizer is on the brink of announcing a deal to buy Global Blood Therapeutics (GBT) and its oral therapy Oxbryta for sickle cell disease for around $5 billion, according to press reports. The post Pfizer bid for sickle cell drug developer GBT said to be imminent appeared first on.

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Drug Discovery World

JULY 13, 2022

The first new treatment in 20 years that prevents a common, painful side-effect in patients with sickle cell disorder (SCD) has been issued interim acceptance by the Scottish Medicines Consortium (SMC). . For too long, the sickle cell community has gone without new innovations to help manage this challenging condition.

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pharmaphorum

OCTOBER 5, 2021

People living with sickle cell disease (SCD) in England and Wales will be able to get access to treatment with Novartis’ Adakveo, thanks to a special arrangement between the drugmaker and NICE. The post Joy as sickle cell patients get access to Novartis’ Adakveo after NICE ruling appeared first on.

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pharmaphorum

AUGUST 24, 2021

The latest deal allows Vertex to tap into Arbor’s technology platform to develop cell therapies for diseases like type 1 diabetes and blood disorders sickle cell disease (SCD) and beta thalassaemia.

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pharmaphorum

APRIL 16, 2021

In some cases, they reduce the number of times patients must visit health facilities: Adakveo reduces the number of vaso-occlusive crises that sickle cell anaemia patient suffer – crises which typically land patients in an emergency room.

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The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. Peer went on to state that he believes that when cancer cells are treated in this manner, they never become active again. This is not the only good news out of the CRISPR realm as of late.

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