Pharmaceutical Technology

NOVEMBER 9, 2022

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

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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

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester. The modified HSCs are then infused back into the patient to engraft in the bone marrow.

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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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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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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. Biogen’s failure should be a warning to other pharma companies. ” The U.S.

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Drug Discovery World

NOVEMBER 28, 2023

An experimental drug has shown potential as a disease-modifying therapy for Parkinson’s disease, according to a new study published in Nature Communications. Mission Therapeutics develops therapeutics that promote mitophagy, which enables the removal of dysfunctional mitochondria, thereby improving cell health and function.

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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

JUNE 29, 2022

This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans.

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Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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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

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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Pharmaceutical Technology

FEBRUARY 10, 2023

On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 This is not the first treatment to come with a high price tag.

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pharmaphorum

JUNE 12, 2022

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8

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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. There are a lot of successes that have been achieved with CAR T therapies.

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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). It is indicated for patients who do not have a pre-existing medical reason preventing treatment with the therapy.

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Gene Therapy Gene Protein FDA Approval 98

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. Preliminary results from that study showed a 53.8%

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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. million price tag. The post bluebird bio to split into oncology and gene therapy specialists appeared first on.

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Drug Discovery World

MAY 22, 2023

Ryan Leahy , a science communication expert in cell and gene therapy, at Phacilitate, says there is a lot to celebrate but also much to assess, question and challenge in this sector. But are these price tags a barrier to access? Advanced Therapies Week highlights the dynamic landscape of CGTs. million per dose.

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Drug Discovery World

OCTOBER 31, 2022

The last month has seen huge strides forward in our understanding of cancers, particularly in how they develop resistance to therapies and how we can ‘outsmart’ them using gene editing or different therapeutic pathways, but also how we can better target drugs to individuals and accurately predict treatment outcomes.

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pharmaphorum

DECEMBER 23, 2022

There’s been huge progress in treatments for blood cancer in recent years – but drawbacks of expensive CAR-T cell and injected antibody therapies have led drug developers to look at novel oral therapies as patient-friendly alternatives. Oral therapy data presented at ASH. CellCentric, a U.K-based

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pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. .

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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. The post Immunocore gets EU nod for TCR cancer therapy Kimmtrak appeared first on. ” Image by Pexels from Pixabay .

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pharmaphorum

DECEMBER 23, 2021

The designation – which is awarded to therapies that could represent a significant improvement over standard of care in either efficacy, safety, or both – has been awarded on the strength of phase 1 results in patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer (NSCLC).

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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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pharmaphorum

APRIL 14, 2022

US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. gene therapy is cost-effective appeared first on. The post ICER says bluebird bio’s $2.1m

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pharmaphorum

APRIL 27, 2022

A breakthrough designation recognises that a drug could offer a substantial improvement over available therapies for serious or life-threatening diseases. The post Enhertu gets breakthrough tag in HER2-low breast cancer appeared first on. billion upfront to license rights to the drug in a deal that could be worth up to $6.9

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pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. million for chelation therapy to remove excess iron from the body. million, which compares to the $1.7

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Gene Therapy FDA Approval Gene Clinical Trials 52

Delveinsight

FEBRUARY 9, 2021

Breyanzi, BMS’ Blood Cancer CAR-T Therapy Receives FDA Nod. Breyanzi targets CD19, a protein that is highly expressed on the surface of B cells, which remain there following the transformation of these cells from normal to malignant. Ukoniq’s approval was backed by Phase 2 UNITY-NHL Trial (NCT02793583).

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pharmaphorum

APRIL 20, 2021

Novartis has secured backing from NICE for its relapsing multiple sclerosis (RMS) therapy Kesimpta in the UK, just two weeks after the drug was approved by the national drugs regulator. . The post NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta appeared first on. Novartis took ownership of the drug in 2015.

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Rethinking Clinical Trials

MARCH 1, 2023

For the standard of care arm, treatment is determined by the treating investigator and participant, with the recommendation that the choice of drug is based on NCCN guidelines and dosing administration based on the participant’s previous therapy and disease. The aim of S2302 is to validate the improvement in overall survival in S1800A.

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pharmaphorum

JULY 25, 2022

The EMA’s human medicines committee has recommended approval of Johnson & Johnson’s Tecvayli as a fourth-line therapy for multiple myeloma , joining a growing group of BCMA-targeted therapies for the blood cancer.

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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. About Dynavax.

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XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. With a price tag of over $2.5 With a price tag of over $2.5 Baby Arthur: First UK Patient.

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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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XTalks

AUGUST 17, 2020

Immunotherapy continues to be a burgeoning field, with immune-based therapies being among the leading treatments being developed in oncology and autoimmunity. Cell-Based Immunotherapy vs. Immune Biologics. cells, receptors) to treat disease. cells, receptors) to treat disease. Immune system modulators (i.e.

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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., who co-led the Cell Reports study with Scripps Research Professor Juan Carlos De La Torre, Ph.D.,

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