Clinical Research Informer

Zynteglo halt re-ignites viral vector safety concerns; analysts

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. . The post Zynteglo halt re-ignites viral vector safety concerns; analysts appeared first on.

Gene Therapy

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What challenges does the advanced therapies sector face?

Drug Discovery World

JANUARY 30, 2024

Natalia Elizalde, Chief Business Development Officer, VIVEbiotech, says that Dr Naldini’s award represents the significant benefits and demonstrated safety of the therapies developed using lentiviral vectors that are being used for more and more clinical trials and more prevalent diseases.

Gene Therapy

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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

APRIL 13, 2023

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. Bluebird already uses a lentiviral vector in its approved gene therapy for beta-thalassemia called Zynteglo. Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

Gene Therapy

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First gene therapy trial for Hunter syndrome opens in Manchester, UK

Drug Discovery World

OCTOBER 31, 2023

The gene therapy works by collecting HSCs from the patient and inserting a working copy of the gene into the HSCs using a lentiviral gene therapy vector. Next generation stem cell gene therapy Children with Hunter syndrome have a missing gene, meaning they cannot produce an important enzyme called iduronate-2-sulfatase or IDS.

Gene Therapy

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Second unanimous FDA adcomm vote boosts bluebird bio

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.

Gene Therapy

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Leading innovators in peptide nano-particle conjugates for the pharmaceutical industry

Pharmaceutical Technology

MAY 15, 2023

Adeno-associated virus vectors, alcohol dehydrogenase compositions, and antibody serum stabilisers are some of the accelerating innovation areas, where adoption has been steadily increasing. Among maturing innovation areas are anti-influenza antibody compositions and anti-interleukin-1, which are now well established in the industry.

Antibody

Antibody DNA Development Vaccination

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

XTalks

JUNE 28, 2023

million price tag of Elevidys, a one-time gene therapy. It has taken Sarepta six years to develop Elevidys with several roadblocks in its journey, including two clinical holds due to safety concerns. In a media call, Sarepta CEO Douglas Ingram revealed the $3.2

Gene Therapy

Gene Therapy Gene Protein FDA Approval

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

While Lyfgenia delivers a functional copy of hemoglobin A to replace the abnormal one in hematopoietic (blood) stem cells via a lentiviral vector, Casgevy edits the HBB gene using CRISPR technology to increase the production of fetal hemoglobin in blood stem cells. Both Casgevy and Lyfgenia work to produce normal versions of hemoglobin.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

XTalks

JUNE 8, 2021

Zolgensma is an adeno-associated virus vector (AAV)-based gene therapy that delivers a functional copy of the SMN1 gene into target motor neurons to replace the mutant copy. With a price tag of over $2.5 million, Zolgensma is currently the world’s most expensive drug. Why is Zolgensma So Expensive?

Gene Therapy

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The Covid cash cow – a look at the Covid vaccine sales figures

pharmaphorum

NOVEMBER 19, 2021

Barely a week later, Moderna’s mRNA rival jab entered the fray, followed quickly by the AstraZeneca/Oxford University viral vector vaccine, and then this year, Janssen’s single-shot vaccine got its finger in the pie. Indeed, the price tags for Pfizer’s and Moderna’s jabs are substantial. Pfizer/BioNTech’s vaccine dominates the market.

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Roctavian okay sets up another gene therapy test for Europe

pharmaphorum

AUGUST 26, 2022

Roctavian delivers a working copy of the Factor VIII gene into cells via an adeno-associated virus (AAV) vector, and offers a one-shot, potentially curative therapy for haemophilia A. million spread over five years.

Gene Therapy

Gene Therapy Gene Antibody Marketing

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

XTalks

SEPTEMBER 27, 2022

The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. The price tag reflects “the clinical benefit [it] provides as an urgently needed treatment option to slow the progression of neurologic dysfunction in children impacted by progressive, irreversible and fatal rare disease,” Obenshain added.

FDA Approval

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Hemgenix Approved as First Gene Therapy for Hemophilia B

XTalks

NOVEMBER 24, 2022

The adeno-associated virus vector-based gene therapy delivers a functional copy of Factor IX, a protein involved in blood clotting that is missing or not made in enough amounts in patients who have the disease due to mutations in the gene that codes for it.

Gene Therapy

Gene Therapy Gene FDA Approval Genetics

What to expect from PEGS Europe 2023: Day 2

Drug Discovery World

NOVEMBER 9, 2023

Samuel Lai, PhD, Professor, Pharmacoengineering & Molecular Pharmaceutics, University of North Carolina at Chapel Hill, on: ‘Viral vectors for highly specific immunoengineering of b and t cells in situ’. The session ends with two presentations and two luncheon presentations.

Engineer

Engineer Antibody Protein In-Vivo

What to expect from PEGS Europe 2023: Day 3

Drug Discovery World

NOVEMBER 13, 2023

Wouter Verdurmen, PhD, Assistant Professor, Medical Biosciences, Radboud University Nijmegen, on: ‘Adenovial vector-mediated local IgA production: A novel anti-cancer immunotherapy’. Fabian Mohr, PhD, Vice President Research & Development, IBA Lifesciences, on: ‘Developing a robust affinity tag platform using engineered streptavidin’.

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Protein Antibody Engineer Scientist

Clinical Research Informer

Zynteglo halt re-ignites viral vector safety concerns; analysts

What challenges does the advanced therapies sector face?

Trending Sources

Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

First gene therapy trial for Hunter syndrome opens in Manchester, UK

Second unanimous FDA adcomm vote boosts bluebird bio

Leading innovators in peptide nano-particle conjugates for the pharmaceutical industry

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

The Covid cash cow – a look at the Covid vaccine sales figures

Roctavian okay sets up another gene therapy test for Europe

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

Hemgenix Approved as First Gene Therapy for Hemophilia B

What to expect from PEGS Europe 2023: Day 2

What to expect from PEGS Europe 2023: Day 3

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