Bio Pharma Dive

SEPTEMBER 13, 2022

Drug regulators have not ordered a trial hold, however. The development comes three weeks after Roctavian was approved in Europe and ahead of a planned regulatory submission in the U.S.

Gene Therapy 200

Gene Therapy Trials Gene Regulation 200

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

Gene Therapy 104

Gene Therapy Gene Trials Protein 104

Bio Pharma Dive

APRIL 28, 2022

The regulator has asked Pfizer to closely monitor patients in a hospital setting for a week as part of a deal to end a study suspension that's lasted more than four months.

Gene Therapy 308

Gene Therapy Gene Regulation Trials 308

Camargo

DECEMBER 20, 2021

Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies. The increase is designed to strengthen CBER’s capacity and capabilities for regulating cell and gene therapies. 2024: 48 staffers. 2025: 29 staffers.

Gene Therapy 184

Gene Therapy Gene Development Production 184

Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity.

Gene Therapy 108

Gene Therapy Gene Trials FDA Approval 108

pharmaphorum

SEPTEMBER 14, 2021

There has been a fourth patient fatality in Astellas’ clinical trial of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), which has been halted twice due to safety concerns. The post Astellas reports fourth death in halted gene therapy trial appeared first on.

Gene Therapy 86

Gene Therapy Gene Trials Clinical Trials 86

Cloudbyz

MARCH 30, 2023

Cell and gene therapies are rapidly becoming an essential component of modern medicine, offering hope to patients with previously untreatable diseases. However, the development and delivery of cell and gene therapies present significant operational challenges that must be navigated carefully to ensure successful clinical trials.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.

Gene Therapy 64

Gene Therapy Gene In-Vitro Development 64

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.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

FDA Law Blog

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

Bio Pharma Dive

JANUARY 10, 2022

regulators, CEO Doug Ingram said Monday, though it still expects to need data from an ongoing late-stage trial to win approval. The biotech will take new Phase 2 data to U.S.

Gene Therapy 278

Gene Therapy Gene Regulation Trials 278

pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

Gene Therapy 75

Gene Therapy Gene Genetic Disease Genetics 75

Bio Pharma Dive

MARCH 10, 2021

and European regulators in hopes of resuming study of the treatment as well as sales of a related product. The biotech is discussing its findings with U.S.

Gene Therapy 271

Gene Therapy Gene Sales Regulation 271

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

Gene Therapy 155

Gene Therapy Gene Packaging Packaging 155

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. The post Roctavian okay sets up another gene therapy test for Europe appeared first on. million spread over five years.

Gene Therapy 107

Gene Therapy Gene Antibody Marketing 107

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

Gene Therapy 105

Gene Therapy Gene Genome Genomics 105

pharmaphorum

DECEMBER 22, 2021

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. AAV vectors are well-suited to gene therapies for DMD because they are effective at targeting muscle tissue.

Gene Therapy 57

Gene Therapy Gene Trials Clinical Trials 57

pharmaphorum

SEPTEMBER 1, 2021

Astellas has halted dosing in a study of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM) for the second time after another serious adverse event (SAE) linked to possible liver damage. The post More trouble for Astellas as AT132 gene therapy trial is halted again appeared first on.

Gene Therapy 78

Gene Therapy Gene Trials Regulation 78

BioPharma Reporter

APRIL 29, 2021

Adverum Biotechnologies has reported a setback in its trial exploring gene therapy for the treatment of diabetic macular edema (DME).

Gene Therapy 98

Gene Therapy Gene Trials Regulation 98

FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Gene Therapy 119

Gene Therapy Gene FDA Approval Production 119

pharmaphorum

DECEMBER 22, 2021

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. It is vying to become the first therapy for the disease, which affects around eight million people globally, according to Novartis.

Gene Therapy 86

Gene Therapy Gene Licensing Licensing 86

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 52

Pharmaceutical Technology

DECEMBER 18, 2023

In this issue: Diabetes researchers oppose the regulation of islet transplantation as a biologic in the US, why haemophilia gene therapies are so expensive, how AI will help with trial recruitment.

Gene Therapy 130

Gene Therapy Regulation Gene Trials 130

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

pharmaphorum

JANUARY 12, 2022

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. point increase on the NSAA scale with the gene therapy, while the control group saw a 0.7-point

Gene Therapy 86

Gene Therapy Gene Regulation Trials 86

Drug Discovery World

NOVEMBER 24, 2022

A new report by Charles River Associates for the European Federation of Pharmaceutical Industries and Associations (EFPIA has revealed that Europe is falling behind the US and Asia in its pharmaceutical R&D investment, particularly when it comes to Advanced Therapies Medicinal Products (ATMPs) like tissue, gene and cell therapies. .

Gene Therapy 98

Gene Therapy Gene Clinical Trials Clinical Trials 98

pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle.

Gene Therapy 52

Gene Therapy Gene Gene Expression Trials 52

BioPharma Reporter

JANUARY 18, 2024

The Cell and Gene Therapy Catapult (CGT Catapult), an independent organization specialising in the advancement of cell and gene therapies, has revealed that the UK has remained an attractive destination for commercial trials, with this type of clinical trial accounting for 81% of trials in 2023 and 80% in 2022.

Clinical Trials 105

Clinical Trials Clinical Trials Gene Therapy Trials 105

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

Gene Therapy 52

Gene Therapy Gene Trials Gene Expression 52

BioPharma Reporter

MAY 10, 2022

Regenxbio says it is to delay dosing of patients in its DMD gene therapy clinical trial following a quality issue at an unnamed third-party contract manufacturer.

Contract Manufacturing 52

Contract Manufacturing Gene Therapy Manufacturing Gene 52

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

Gene Therapy 52

Gene Therapy Gene Development Production 52

Drug Discovery World

SEPTEMBER 16, 2022

Here, DDW Editor Reece Armstrong explores the global hubs of innovation for cell and gene therapies. Since the approval of Kymirah in 2017, the cell and gene therapy sector has emerged as an exciting and innovative market, representing some of the most promising drugs the pharmaceutical industry has ever seen. .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

BioPharma Reporter

JUNE 7, 2021

The FDA has lifted the clinical holds on bluebird bioâs trials of gene therapies in sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), an inherited blood disorder.

Gene Therapy 59

Gene Therapy Gene Trials Regulation 59

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”

Gene Therapy 66

Gene Therapy Gene Genetic Disease DNA 66