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.

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Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

DECEMBER 18, 2023

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. The researchers plan to conduct a first-in-human clinical trial in the next five years.

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Gene Therapy Gene Regulation Engineer 105

Pharmaceutical Technology

MAY 24, 2023

The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies. The regulator granted RMAT designation based on the data obtained from the ongoing Phase I RP-L301 clinical trial.

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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.

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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.

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Gene Therapy Clinical Trials Clinical Trials Gene 52

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?

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Gene Therapy Gene Trials Clinical Trials 98

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.

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Gene Therapy Gene Trials Clinical Trials 52

Cloudbyz

OCTOBER 19, 2023

Clinical trials are the backbone of drug development, and managing these trials efficiently is paramount. In recent years, the need for a unified clinical trial management platform has become increasingly evident. Cost Efficiency: Centralized data management reduces administrative overhead, minimizing trial costs.

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Clinical Trials Clinical Trials Trials Compliance 86

Cloudbyz

JUNE 11, 2023

This transformation has permeated the domain of clinical trials, which is now embracing the concept of decentralized clinical trials (DCTs). This paper delves deep into the universe of DCTs, their distinctiveness from conventional trials, their inherent benefits, existing hurdles, and measures to bolster their adoption.

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Clinical Trials Clinical Trials Trials Compliance 71

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.

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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.

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Gene Therapy Gene Trials Clinical Trials 86

Cloudbyz

JUNE 10, 2023

21 CFR Part 11 is a crucial component of regulatory compliance in clinical trials and, more broadly, any industry subject to the regulations of the U.S. This blog post will serve as a comprehensive guide to understanding and implementing 21 CFR Part 11 compliance in your clinical trials. Let’s explore.

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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.

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Cloudbyz

SEPTEMBER 16, 2023

Addressing data privacy and data protection concerns when implementing ChatGPT in clinical trial operations management is crucial to maintain compliance with regulations, safeguard sensitive patient information, and build trust among stakeholders. This may involve conducting regular audits and assessments to maintain compliance.

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Clinical Trials Clinical Trials Trials Compliance 59

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.

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Gene Therapy Gene Trials Clinical Trials 57

Cloudbyz

SEPTEMBER 25, 2023

Overview – What They Are A Clinical Trial Management System (CTMS) is a software system that manages and organizes tasks and processes related to clinical trials. A unified CTMS is aimed at keeping track of every relevant part of your trials and involves integrating a long list of functionalities into your CTMS solution.

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Clinical Trials Clinical Trials Trials Compliance 80

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). Additionally, the FDA said a “clinical benefit of Elevidys, including improved motor function, has not been established.”

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Gene Therapy Gene Protein FDA Approval 98

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.

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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.

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Contract Manufacturing Gene Therapy Manufacturing Gene 52

Drug Discovery World

NOVEMBER 24, 2022

Despite the importance of the pharmaceutical industry to national economies and population health, the report paints a picture of falling competitiveness, with the global share of R&D investment, clinical trials and manufacturing output all decreasing. . Overall clinical trial activity in the region is also on the decline.

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Gene Therapy Gene Clinical Trials Clinical Trials 98

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.

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XTalks

FEBRUARY 10, 2023

According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. Among 20 recent trials, 17 experienced delays in meeting key milestones.

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Clinical Trials Clinical Trials Trials Gene Therapy 52

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.

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Gene Therapy Gene Production Clinical Trials 52

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.

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Gene Therapy Gene Genome Genomics 105

Cloudbyz

AUGUST 11, 2023

Setting the initial stages right in a clinical trial is akin to laying the foundation for a building. A stumble here can have a cascading effect on the entire trial. Have they handled similar trials before? Let’s delve deeper into each of the best practices for an effective site start-up.

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Clinical Trials Clinical Trials Trials Regulation 52

XTalks

MAY 20, 2021

Today is Clinical Trials Day 2021 — a day for recognizing the importance of clinical research in protecting and improving global health. Amid the alarmist headlines posted by other media outlets, Xtalks has remained balanced in its reporting of COVID-19 clinical trial results and safety data.

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Clinical Trials Clinical Trials Trials Vaccination 105

Cloudbyz

JUNE 13, 2023

Introduction: Clinical trials are essential for evaluating the safety and efficacy of new medical treatments, therapies, and interventions. Developing an accurate and comprehensive budget is crucial for successful trial planning and execution. Site Selection: Careful site selection is critical for optimizing trial costs.

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Drug Discovery World

SEPTEMBER 12, 2022

Chaired by Karin Blumer, Director of Global Patient Engagement at Novartis, the first event in this track, called ‘Patient Engagement: Driving Patient Advocacy to Inform Patient Centric Development’, looked into the multi-layered importance of involving patients in the drug development process, particularly in this case for gene therapy.

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Clinical Trials Clinical Trials Trials Gene Therapy 52

Drug Discovery World

MARCH 21, 2023

New EU legislation designed to improve public health is delaying clinical trials and blocking access to new treatments for cancer and rare diseases, says the European Federation of Pharmaceutical Industries and Associations (EFPIA). The In Vitro Diagnostic Regulation (IVDR) came into effect in May 2022.

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In-Vitro Regulation Clinical Trials Clinical Trials 52

pharmaphorum

JUNE 11, 2021

Abel Archundia discusses how COVID-19 has highlighted the importance of securing clinical trials against cyber threats. However, data-intensive, decentralised clinical trials also present a major risk to healthcare participants through cybercrime and data privacy vulnerability.

Clinical Trials 101

Clinical Trials Clinical Trials Trials Compliance 101

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.

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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. .

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Pharmaceutical Technology

JUNE 9, 2023

RP-A601 is an adeno-associated virus-based gene therapy currently being evaluated as a one-time, potentially curative treatment to improve survival and quality of life for PKP2-ACM patients. The existing standard of care for the disease includes medical therapy, ablation procedures and implantable cardioverter defibrillators (ICDs).

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Gene Therapy Gene FDA Approval Trials 147

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. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

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Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? According to Foster, the industry needs to work together to address the problems from the earliest phases of pre-clinical development. DDW’s Megan Thomas finds out.

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Gene Therapy Gene Manufacturing Marketing 52

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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Gene Therapy Gene Manufacturing Production 52

Advarra

FEBRUARY 24, 2023

In most cases, the results you are really looking for and trying to prove in a trial can be complex. How do you strategically plan your therapy and research objectives to improve the chances you will satisfy what the regulators are looking for and ultimately get your therapy approved for marketing?

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