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

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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Medicine Research Gene Therapy Genetics 189

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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Trials Clinical Trials Clinical Trials Gene Therapy 104

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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Gene Therapy Gene In-Vitro Clinical Research 74

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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Gene Therapy Gene Genome Genomics 73

Velocity Clinical Research

OCTOBER 3, 2024

As Nick Spittal states in this Advarra press release, membership in the Gene Therapy Ready (GTR) site network “allows Velocity to start studies over a month faster and provides a meaningful credential and important validation that increases sponsors’ confidence in our specialized capabilities to conduct complex clinical research safely.”

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Gene Therapy Gene Genetic Engineering Clinical Research 52

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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Gene Therapy Gene Research Genetics 52

Outsourcing Pharma

OCTOBER 31, 2022

Thermo Fisherâs clinical research subsidiary, PPD, invests $59 million in sample management and testing capacity for vaccines and cell and gene therapy products with its expansion of laboratory operations in Kentucky.

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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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Life Science Genetic Disease Genome Genomics 130

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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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

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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Gene Therapy Gene Clinical Research Manufacturing 52

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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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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Genetic Engineering Gene Therapy Genetics Engineer 98

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. The post Risk Assessment for use of Engineered Genetic Materials in Clinical Research appeared first on Advarra.

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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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Clinical Trials Clinical Trials Trials Gene Therapy 105

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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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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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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XTalks

FEBRUARY 24, 2023

“Many rare disease communities are seeing gene therapies as potential game changers, since most rare diseases are genetically driven,” Dr. Raymond says. Hence, one conversation that needs to take place is between the sponsors developing gene therapies and the payers who in effect “gate” patient access to these novel treatments.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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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.

Life Science 52

Life Science Gene Therapy Gene Clinical Trials 52

Cloudbyz

MARCH 22, 2024

In the realm of clinical research, the efficient management of data is paramount. However, traditional methods of data entry, particularly from diverse and unstructured documents, have long posed challenges for researchers, leading to inefficiencies, errors, and compliance risks. Scalability: Cloudbyz’s ClinExtract.AI

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