Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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Pharmaceutical Technology

MAY 1, 2023

Australian biotechnology company Cartherics has granted licence for CTH-004 , its autologous CAR-T cell product, to Chinese company Shunxi. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 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

IO agents include the classes of immune checkpoint modulators, cell therapies, bispecific antibodies, oncolytic viruses, therapeutic vaccines, and cytokines. All currently approved CAR-Ts are autologous, with the patient’s T-cells being genetically engineered to target antigens expressed by the cancerous cells.

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Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Scienmag

JULY 1, 2022

One way cells can control the activities of their genes is by adding small chemical modifications to the DNA that determine which genes are turned on or off. Methyl groups are one of these chemical modifications or tags.

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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. The mutation causes red blood cells to develop a crescent or “sickle” shape.

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Scienmag

APRIL 23, 2021

Credit: RIKEN Researchers led by Katsunori Tanaka and Kenward Vong at the RIKEN Cluster for Pioneering Research (CPR) in Japan have demonstrated that tumor growth can be reduced by therapy that tags cancer cells with different therapeutic molecules.

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Scienmag

APRIL 29, 2021

Credit: UC San Diego Health Sciences Researchers at University of California San Diego School of Medicine have discovered one way in which SARS-CoV-2, the coronavirus that causes COVID-19, hijacks human cell machinery to blunt the immune response, allowing it to establish infection, replicate and cause disease.

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Immune Response Genome Genomics Medicine 52

Scienmag

NOVEMBER 17, 2020

Credit: © Andreas Reicher / CeMM Until now, scientists typically studied the changes of proteins and their roles in the cell by using a fluorescent tag to label and follow one protein at a time. This approach limited the number of proteins that could be studied and precluded unbiased discovery approaches.

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Drug Discovery World

JANUARY 9, 2023

Harvard investigators have developed a new cell therapy approach to eliminate established tumours and induce long-term immunity. . Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” .

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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. AZ: Cell and gene therapies often come with a high price.

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Drug Discovery World

MARCH 2, 2023

Scientists at The Institute of Cancer Research, London have revealed how a key epigenetic signal called H3K4me3 determines when and how DNA should be read and translated into proteins within our cells. But, until now, scientists lacked an understanding of what the chemical tag does, despite many years of research.

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Pharmaceutical Technology

DECEMBER 5, 2022

CB1 receptors are primarily expressed in the brain and central nervous system, whereas CB2 receptors are expressed in T-cells in the immune system. Collectively, cannabinoid receptors (CB1 and CB2) are currently the most popular targets in preclinical stage of development, with 391 drugs tagged in total.

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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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BioTech 365

AUGUST 21, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Researchers create bioluminescent tag to detect DNA break repair.A

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Drug Discovery World

OCTOBER 31, 2023

In the combined Phase I and II clinical trial, five children under one year of age with the condition also known as mucopolysaccharidosis type II (MPS II) will be treated with autologous hematopoietic stem cell (HSC) gene therapy.

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Drug Discovery World

NOVEMBER 28, 2023

Mission Therapeutics develops therapeutics that promote mitophagy, which enables the removal of dysfunctional mitochondria, thereby improving cell health and function. Importance of healthy mitochondrial function Dysfunctional mitochondria are usually tagged for removal via mitophagy with a protein ‘flag’ called ubiquitin.

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In-Vivo Protein Drugs Research 52

Pharmaceutical Technology

NOVEMBER 9, 2022

million price tag. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The post Can gene therapies for haemophilia defend their high price tags? After its approval, bluebird had set Zynteglo’s price at roughly EUR1.6 million ($1.8 million at the time).

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Rethinking Clinical Trials

FEBRUARY 20, 2024

Due to the lack of evidence, indications for red blood cell transfusion in patients with MI are controversial. Tags #pctGR, @Collaboratory1 The post Grand Rounds February 16, 2024: Clinical Implications of the MINT Trial: p=0.07 (Jeffrey Carson, MD, MACP) appeared first on Rethinking Clinical Trials.

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Trials Clinical Trials Clinical Trials Medicine 130

Drug Discovery World

OCTOBER 31, 2022

Click’ chemistry used to tag proteins made by cancer cells. A new method to study the proteins released by cells, which could lead to new biomarkers for diseases including cancer, has been developed by scientists at the Francis Crick Institute and Imperial College London. .

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XTalks

FEBRUARY 27, 2024

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.

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Pharmaceutical Technology

FEBRUARY 10, 2023

This is not the first treatment to come with a high price tag. Using this categorization, programmed cell death protein 1 (PD-1)/programmed death ligand 1 (PD-L1) inhibitors saw the greatest percentage of RSAs granted. million, according to GlobalData’s Price Intelligence (POLI), making it the most expensive drug in the world.

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pharmaphorum

JUNE 12, 2022

Cases of cancer associated with insertional mutagenesis, caused when the viral vectors used to deliver the gene therapy to host cells, have been seen with bluebird’s gene therapies but have been more apparent in trials involving its CALD and sickle cell disease candidates than beti-cel.

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World of DTC Marketing

SEPTEMBER 7, 2022

In pharma, growth depends on new products with hefty price tags when over 80% of voters want lower costs for their prescription drugs. More recently developed agents, such as CAR T-cell therapy, may cost almost USD 500,000 annually. Wall Street wants growth, as do investors, but that may be impossible for pharmaceutical companies.

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The Pharma Data

MARCH 7, 2022

Researchers from the University of Washington School of Medicine in Seattle describe this finding in the journal Cell Reports. “The beauty of this approach is we can safely upregulate specific genes to affect cell activity without permanently changing the genome and cause unintended mistakes,” Levy said.

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Drug Discovery World

JUNE 14, 2023

We are a cell therapy company that uses a very specific receptor to engineer T cells and administer these engineered T cells back to solid tumour patients. We consider ourselves a next-generation engineered cell therapy company. AB : I think it is accomplishing on cell therapy in solid tumours as a whole.

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The Pharma Data

JANUARY 31, 2021

Both vaccines induced high levels of neutralizing antibodies comparable to or exceeding levels in human convalescent sera as well as a strong Th1-biased cell-mediated immunity. Utilizing Clover’s proprietary Trimer-Tag© technology, S-Trimer is a trimeric SARS-CoV-2 spike (S)-protein subunit vaccine candidate.

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XTalks

JANUARY 28, 2022

Kimmtrak is part of a novel class of bispecific T cell immunotherapies being developed by Immunocore. Kimmtrak has also become the first bispecific T cell engager to be FDA-approved for the treatment of a solid tumor. Kimmtrak has a price tag of $18,760 per vial, which amounts to a weekly dose.

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The Pharma Data

MARCH 23, 2022

Their investigation reveals that an experimental drug that targets GSPT1 for degradation can also halt Ebola virus infection in human cells. who co-led the Cell Reports study with Scripps Research Professor Juan Carlos De La Torre, Ph.D., The team found that polymerase succeeds by biding its time in host cells.

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The Pharma Data

MARCH 16, 2022

The research scientist at the Max Planck Institute for Medical Research received the award in recognition of his ground-breaking work in establishing various approaches to protein labeling in living cells that have enabled far-reaching advances in chemical and cell biology. Protein labeling (e.g.

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pharmaphorum

AUGUST 25, 2022

The double-headed antibody binds to CD3 on T cells and redirects them to BCMA-expressing myeloma cells, with the aim of stimulating an immune attack on the tumour, which remains incurable despite a slew of new therapies reaching the market in recent years.

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Rethinking Clinical Trials

MARCH 1, 2023

S1800A was a Phase II randomized study of ramucirumab plus pembrolizumab versus standard of care for NSCLC patients previously treated with immunotherapy performed within the Lung-MAP platform. The aim of S2302 is to validate the improvement in overall survival in S1800A.

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pharmaphorum

JANUARY 5, 2022

Teliso-V (telisotuzumab vedotin) is pitching to become the first targeted cancer treatment for people with non-small cell lung cancer (NSCLC) whose tumours overexpress c-Met. The post FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag appeared first on.

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pharmaphorum

APRIL 5, 2022

Immunocore now has approval on both side of the Atlantic for Kimmtrak – the first cancer therapeutic based on T cell receptor (TCR) technology – after getting a green light from the European Commission. It’s the first treatment for mUM in the EU, as well as the first BiTE drug to treat a solid tumour.

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pharmaphorum

JANUARY 11, 2021

While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA. The gene therapy firm will be focused on its most important therapies in beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US And Europe.

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The Pharma Data

DECEMBER 3, 2020

Strong Th1 cell-mediated immune responses were also observed for the vaccine candidates with either adjuvant. Utilizing Clover’s proprietary Trimer-Tag © technology, S-Trimer is a trimeric SARS-CoV-2 spike (S)-protein subunit vaccine candidate. About Trimer-Tag © Technology. S-Trimer is intended to be adjuvanted.

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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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