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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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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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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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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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

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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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 58

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

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.

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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. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely.

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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? million per dose.

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

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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

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

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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 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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XTalks

DECEMBER 21, 2022

The past year has been a strong one for life sciences industries, from pivotal gene therapy approvals to continued innovations in the biotech and medical device spaces. Gene Therapy Approvals. Bluebird bio is a notable mention as it secured FDA approvals for two of its gene therapies, Zynteglo and Skysona.

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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

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. Researchers from the University of Washington School of Medicine in Seattle describe this finding in the journal Cell Reports.

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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. Preliminary results from that study showed a 53.8%

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XTalks

SEPTEMBER 27, 2022

With a list price of $3 million and a recent accelerated US Food and Drug Administration (FDA) approval, bluebird bio’s gene therapy Skysona (elivaldogene autotemcel, eli-cel), has officially become the world’s most expensive drug. Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia.

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FDA Approval Gene Therapy Drugs Gene 52

Delveinsight

DECEMBER 3, 2020

Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences.

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Gene Therapy Gene Antibody Research 52

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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Protein Scientist Gene Development 52

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. Beam employs three approaches to deliver its genetic medicines to cells. Electroporation is used for ex vivo delivery of therapies to blood and immune cells.

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DNA In-Vivo Genetics 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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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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pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. BioMarin has said Roctavian will be available at a ‘net’ price of around €1.5 million spread over five years.

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Gene Therapy Gene Antibody Marketing 110

pharmaphorum

FEBRUARY 16, 2021

It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. There is a curative treatment for people who rely on blood transfusions to survive and maintain their levels of red blood cells.

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Gene Therapy Gene Medicine Development 76

Drug Discovery World

NOVEMBER 9, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Kielczewska will present: ‘Discovery of antibody-based therapeutics to challenging targets: Platform considerations’.

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Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

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Protein Antibody Engineer Scientist 59

Roots Analysis

AUGUST 28, 2022

Recent advances in multi-omics approaches, including genomics, transcriptomics, proteomics, metabolomics, cytometry and imaging, in combination with bioinformatics and biostatistics, have been translated into several popular bioanalytical platforms, such as next-generation sequencing (NGS), single-cell analysis, flow cytometry and mass spectrometry.

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The Pharma Data

MAY 5, 2021

Affinia Therapeutics raises $110 million for gene therapy treatments ( STAT ). Athenex shares soar with buyout of natural killer T cell player ( Endpoints ). Startup sets out to tackle the gene therapy manufacturing crisis ( Endpoints ). Posted 05 May 2021 | By Kari Oakes .

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