XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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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 8, 2023

Over the past few years, there has been a significant expansion in the cell and gene therapy landscape, with an increasing number of therapies entering clinical trials and receiving regulatory approvals. “I think if we all go into these relationships with this mindset, we’ll achieve great things together.”

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AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

Researchers see a greater need for new generation therapeutics to propel precision medicine. The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.

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XTalks

JANUARY 16, 2025

Meanwhile, Medtronics BrainSense Adaptive DBS, newly approved in Europe , could help advance neuromodulation therapy for Parkinsons with real-time brain stimulation adjustments as it becomes available in 2025.

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Pharmaceutical Technology

JANUARY 17, 2023

The Joint Cancer Immunotherapy Clinical Research Unit of the Hospital Universitario 12 de Octubre and the Spanish National Cancer Research Center (CNIO) created the STAb-T therapy led by Dr Luis Álvarez-Vallina and the Josep Carreras Leukaemia Research Institute team Dr Pablo Menéndez and Dr Diego Sánchez-Martínez.

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Scienmag

JANUARY 28, 2021

— In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition. ROCHESTER, Minn.

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Medical Xpress

NOVEMBER 21, 2022

In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).

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XTalks

APRIL 8, 2025

As Thomas says, The accessibility of clinical trials and clinical research is what drives a significant amount of that cross-country movement. Global clinical trials for rare diseases have various accessibility needs and considerations ( Figure 1 ). This gap calls for well-planned logistical solutions.

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Cloudbyz

JUNE 26, 2023

Generative artificial intelligence (AI) techniques are increasingly being applied in clinical research to enhance various aspects of medical data analysis, diagnosis, treatment planning, and drug discovery. These notes have been used to advance research in NLP and clinical decision support systems.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023. Why is Casgevy a Breakthrough Gene Therapy for Patients with Sickle Cell Disease?

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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. We also discuss how a centralized biosafety review process can benefit this type of research.

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Worldwide Clinical Trials

JANUARY 24, 2024

However, clinical research around Fragile X is considerably robust, making the possibility of clinical trial participation a potential motivation for parents to choose to undergo genetic testing for their children. As of July 2023, the American Society of Cell + Gene Therapy stated there were 3,905 therapies in development.

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Pharmaceutical Technology

JUNE 16, 2023

Benefiting from Abu Dhabi’s improved genomics capabilities, the deal seeks to expand research into genomic medicine and genetic diseases to provide patients in the UAE and beyond with improved access to new tools and treatments.

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Worldwide Clinical Trials

MAY 20, 2022

The past few years we’ve had exciting opportunities to work on more cell and gene therapy programs for our sponsors. The industry is just at the tip of the iceberg for exploring the potential of these therapies in revolutionizing how we treat disease, and as the approvals start to trickle in, the pipeline only grows.

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BioSpace

AUGUST 8, 2021

Voyager’s primary focus will be on developing gene therapies for Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), and pre-clinical research in spinal muscular atrophy and diseases linked to GBA1 mutations.

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Clinical Trial Gurus

AUGUST 26, 2021

Clinical research nurses have the background and the experience to virtually demand a great amount of interest from prospective employers. In this video I interview Michele Blue, a research nurse from an academic medical center (AMC). We discuss her career, job prospects, and a lot of other topics related to clinical research.

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XTalks

APRIL 15, 2025

XTALKS CLINICAL EDGE: Issue 4 Rare Disease Trials with Takeda’s VP, US Hematology and Transplant Franchise Head Anthea Cherednichenko Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials.

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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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BioPharma Reporter

FEBRUARY 18, 2021

Massachusetts-based clinical research organization (CRO), Charles River Laboratories, is to acquire Cognate BioServices, a cell and gene therapy contract development and manufacturing organization (CDMO) for around US$875m in cash.

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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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Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. These therapies involve modifying a patient’s cells or genes to treat or prevent diseases, with the promise of long-lasting effects.

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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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The Pharma Data

OCTOBER 18, 2020

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. Ricci told BioSpace the company has been “investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area.”

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Advarra

MARCH 20, 2023

The Food and Drug Administration (FDA) recently released new guidance regarding cellular and gene therapy products, one of which may significantly impact early-phase clinical trials of such products. This is to ensure a sufficient level of scientific and clinical understanding is developed prior to Phase I trials.

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Pharmaceutical Technology

MAY 3, 2023

Both C-CAR039 and the anti-CD20 CAR-T therapy C-CAR066 have been studied in the treatment of non-Hodgkin’s lymphoma and have received investigational new drug (IND) application clearance from the FDA. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Velocity Clinical Research

OCTOBER 4, 2023

Velocity Clinical Research, the leading multi-specialty clinical sites business, announced it is opening greenfield clinical research sites in Bristol, Leicester, and Romford, demonstrating the company’s commitment to the U.K. The sites launch at a time when clinical trial activity in the U.K.

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Roots Analysis

AUGUST 29, 2023

Over the years, cell and gene therapies (CGT) and other advanced therapy medicinal products (ATMPs) have managed to capture the interest of both drug developers and healthcare investors. Their popularity can be attributed to various factors, such as minimal side effects, clinical efficacy and their personalized nature.

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BioTech 365

DECEMBER 2, 2020

4D Molecular Therapeutics Strengthens Leadership Team with Key Appointments in Clinical Research and Development 4D Molecular Therapeutics Strengthens Leadership Team with Key Appointments in Clinical Research and Development EMERYVILLE, Calif.–(BUSINESS

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Cloudbyz

AUGUST 9, 2023

The clinical research landscape is rapidly evolving. As it becomes more complex with growing volumes of data, evolving regulations, and the pressure for faster drug development, traditional methods of clinical research management are no longer sufficient. This is where the platform approach comes into play.

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Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review?

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XTalks

JANUARY 17, 2024

Changes in what clinical performance data is used to inform the trial design and site selection, new approaches to patient recruitment and the rise of advanced treatments such as cell and gene therapy are rewriting the script. Vidal is a certified medical oncologist with extensive experience in oncology drug development.

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Worldwide Clinical Trials

OCTOBER 24, 2023

Seeking relief at the genetic cause of disease Engaged discussion with the audience followed my joint presentation on this topic with Matthew Fuller, PhD, PMP, Executive Director of Gene Therapy Research at Ultragenyx. Roughly 80% of rare diseases are due to a known genetic driver.

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XTalks

JANUARY 8, 2024

Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. It’s important to make sure that with this increase in clinical trial complexity, we don’t make our trials overly burdensome to sites or patients,” says Markham.

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XTalks

DECEMBER 16, 2024

Maria Hopfgarten Head of Global Medical Writing PPD Clinical Research Business of Thermo Fisher Scientific Artificial intelligence (AI) is revolutionizing industries worldwide, and medical writing is no exception. Addressing Risks in AI-Assisted Medical Writing The integration of AI into medical writing isnt without risks.

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ACRP blog

FEBRUARY 25, 2025

[ Editors Note: In recognition of Rare Disease Day being observed on February 28, ACRP is pleased to present this, the second of two blogs contributed by subject matter experts offering insights on how rare diseases are being focused on by the clinical research enterprise.

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Velocity Clinical Research

MAY 23, 2024

Standard Velocity site features: Private exam rooms Comfortable patient reception areas Facilities for extended-stay PK studies Ample parking Secure monitoring rooms and workstations equipped with phones and high-speed internet Individual and secure workstations for research staff Regulatory document processing area EDC capabilities Secure record storage (..)

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