pharmaphorum

MARCH 20, 2024

Orchard Therapeutics has revealed the US price of Lenmeldy, its gene therapy for rare disease MLD, placing a $4.25m price tag on the one-shot treatment

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Gene Therapy Gene Medicine 111

Fierce Pharma

JULY 14, 2023

The potentially curative promise of gene therapies often carries a steep price tag. | The potentially curative promise of gene therapies often carries a steep price tag.

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Gene Therapy Gene Medicine 79

BioSpace

NOVEMBER 7, 2023

Cell and gene therapy represents the most promising breakthrough in cancer treatment for decades, but it comes with a hefty price tag. Here’s how to drive costs down.

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Gene Therapy Gene 76

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). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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Gene Therapy Gene FDA Approval Licensing 147

Pharmaceutical Technology

MAY 1, 2023

Cartherics will retain all the development and commercialisation rights for the therapy outside Greater China. 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.

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Gene Therapy Gene Development Clinical Trials 130

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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Gene Therapy FDA Approval Gene In-Vivo 98

Pharmaceutical Technology

APRIL 5, 2023

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023.

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Gene Therapy Gene Engineer Production 147

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.

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Gene Therapy Gene Trials Clinical Trials 52

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

XTalks

AUGUST 31, 2022

Ayesha shared news about another significant FDA approval for a gene therapy to treat transfusion-dependent beta thalassemia. Bluebird bio was awarded the approval for its gene therapy Zynteglo (beti-cel), which is a one-time treatment for the rare blood disorder. Bluebird’s $2.8M

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Gene Therapy FDA Approval Life Science Gene 52

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

JANUARY 9, 2023

Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

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Gene Therapy Genetics Gene Clinical Trials 119

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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Gene Therapy Gene Marketing Trials 59

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 Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy?

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Gene Therapy Gene Protein Trials 110

Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The therapy was also recently approved by the FDA for patients in the US. .

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Gene Therapy Gene Clinical Trials Clinical Trials 52

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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Drugs FDA Approval Marketing Development 246

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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Gene Therapy Manufacturing Gene Clinical Trials 71

STAT News

OCTOBER 6, 2022

million tag as cost effective for a Bluebird Bio drug called Zynteglo for the blood disorder beta thalassemia, Arbuckle said in an interview. Bluebird also recently said it would charge $3 million for Skysona, the gene therapy it just launched for a rare brain-wasting disease. Continue to STAT+ to read the full story…

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Gene Therapy Gene Insulin Drugs 98

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. The entire gene Tx field is watching.

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Gene Therapy FDA Approval Gene Clinical Trials 52

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.

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Gene Therapy Gene Marketing Clinical Trials 64

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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Scientist Gene Editing Gene Engineer 52

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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Gene Therapy Gene Drugs Contamination 52

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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Drugs Research Gene Editing Protein 52

pharmaphorum

JANUARY 5, 2022

The c-Met protein thought to be both a driver of cancer itself, as well as a resistance mechanism that tumours can develop to protect them from some widely-used NSCLC therapies, including EGFR inhibitors. The post FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag appeared first on.

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Antibody Drugs Protein Gene 111

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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Gene Therapy Gene Genetic Disease Manufacturing 66

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. . million in Europe. million in Europe.

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Gene Therapy Gene Gene Sequencing Gene Expression 52

pharmaphorum

NOVEMBER 19, 2021

The agreement has been hailed by patient associations, as it adds to current treatment options for the progressive muscle wasting disease – Biogen’s injectable Spinraza (nusinersen) and one-shot gene therapy with Novartis’ Zolgensma (onasemnogene abeparvovec) – which are also backed by NICE.

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Drugs Gene Therapy Genetic Disease Gene 98

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Gene Silencing Gene Clinical Trials Clinical Trials 98

The Pharma Data

MARCH 7, 2022

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

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Protein Gene Genome Genomics 52

pharmaphorum

OCTOBER 21, 2021

Analysts were predicting sales of Biogen’s recently approved Alzheimer’s therapy Aduhelm may start to gather a little momentum in the third quarter, but the drug seems to be going into reverse. Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug.

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Sales Doctors Doctor Gene Therapy 122

The Pharma Data

MARCH 16, 2022

using SNAP-tag) involves labeling cell proteins with a fluorescent dye, thereby enabling researchers to observe how proteins interact in the living cell and where in the cell they are located. With Spirochrome, he commercialized fluorescent probes for cell biology, while with Covalys, he marketed SNAP-tag and other protein labeling methods.

Protein 52

Protein Scientist Gene Development 52

pharmaphorum

JUNE 2, 2021

The orally-administered drug is the third approved treatment for SMA after Biogen’s injectable Spinraza (nusinersen) and Novartis’ one-shot gene therapy Zolgensma (onasemnogene abeparvovec), which was administered to the first NHS patient this week. — Spinal Muscular Atrophy UK (@SMA_UK_) June 2, 2021.

Gene Therapy 52

Gene Therapy Gene Drugs Medicine 52

Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

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Gene Therapy Gene Sales Protein 246

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. If they eliminated the T cells, the therapy stopped working.

DNA 52

DNA In-Vivo Genetics Medicine 52

pharmaphorum

NOVEMBER 16, 2022

The advancements made in drug development over the last decade have seen the arrival of a number of treatments that offer one-time cures and more effective therapies for conditions in smaller patient populations. However, alongside this progress, Ben Hargreaves finds that there is also a conundrum over how to price these therapies.

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Gene Therapy Gene Marketing Development 131

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. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Research Drugs Gene Therapy Clinical Trials 59

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

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Gene Therapy FDA Approval Gene Clinical Trials 95