Changing cell & gene therapy landscape and implications on clinical trials
Bio Pharma Dive
APRIL 3, 2023
Cell & gene therapy clinical research may help treat diseases and change how we approach healthcare.
Ophthalmologists eye gene therapies for vision-threatening conditions as NGS facilitates precision diagnosis
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 9, 2024
The convergence of gene therapies and clinical research is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.
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Eisai and National Cancer Center partner for tazemetostat clinical research
Pharmaceutical Technology
APRIL 3, 2023
Eisai and the National Cancer Center have signed an agreement to partner on investigator-initiated clinical research for the anti-cancer agent, tazemetostat (Tazverik Tablets 200 mg), based on the Patient-Proposed Healthcare Services system.
Scientists Just Identified Hundreds of Genes That Could Cause Cancer
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 22, 2024
(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.
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This Rare Gene Variant Seems to Protect You From Alzheimer’s Disease
AuroBlog - Aurous Healthcare Clinical Trials blog
JUNE 25, 2024
Family members with just a single copy of her version of a particular gene – what’s known as the Christchurch variant of apolipoprotein E (APOE) – seem to also be protected against genetically determined early-onset dementia, researchers […]
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The State of Pediatric Clinical Research: An Interview with Paidion CEO Barry Mangum, PharmD, FCP
Camargo
JULY 30, 2021
Barry Mangum talks the importance of pediatric research, its evolution over the years, and the obstacles and opportunities that the industry faces as it moves forward. How long have you been in pediatric clinical research, and how did you enter the field? So I’ve been working in that space about 43 years.
Vivet wins $5.3m grant to develop gene therapy for rare metabolic disorder
Pharmaceutical Technology
FEBRUARY 1, 2024
The French government grant will cover preclinical and clinical research for Vivet’s gene therapy for cerebrotendinous xanthomatosis.
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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment
Worldwide Clinical Trials
NOVEMBER 27, 2023
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.
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July 31, 2024: Gene Editing Therapies and Population Health, in This Week’s PCT Grand Rounds
Rethinking Clinical Trials
JULY 31, 2024
Adrian Hernandez In this Friday’s PCT Grand Rounds, Adrian Hernandez of Duke University will present “Precision Health to Population Health: Opportunities and Challenges for Gene Editing Therapies.” ” The Grand Rounds session will be held on Friday, August 2, 2024, at 1:00 pm eastern. Join the online meeting.
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Type 2 Diabetes: Challenges & Emergent Strategies for Clinical Research
Worldwide Clinical Trials
JULY 29, 2024
Partnering with a CRO that emphasizes the complexity of the patient’s voice and harnesses the nuances of clinical trial design is crucial for executing successful clinical research. DEXA (Dual-Energy X-ray Absorptiometry) is the optimal method for accurately measuring body composition in clinical research.
Avance Clinical Expands Gene Technology Clinical Trial Services to Meet $17.4 billion Market Demand
Pharma Mirror
MAY 7, 2021
The leading Australian CRO for biotechs and Frost & Sullivan Asia-Pacific CRO Market Leadership Award winner, Avance Clinical, has expanded its gene technology clinical trial services to meet the increasing global demand predicted to reach $17.4 billion by 2023.
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Emmes launches cell and gene therapy center
BioPharma Reporter
DECEMBER 8, 2022
Emmes has announced the creation of a dedicated center for cell and gene therapy research: which will focus on supporting clinical trials for the Clinical Research Organizationâs (CRO) clients.
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3 Key Takeaways from Global Genes Summit 2023
Worldwide Clinical Trials
OCTOBER 24, 2023
Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”
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Experiment Reveals Human Genes Can Be Controlled With Electricity
AuroBlog - Aurous Healthcare Clinical Trials blog
AUGUST 7, 2023
New research from ETH Zürich in Switzerland could see future wearable devices (with perhaps a few implants and a touch of genetic engineering) boost our health directly. Fitness trackers help you stay healthy by keeping count of your steps and monitoring your heart rate, driving you on to hit those cardio goals.
The future of the gene therapy market: driving innovations in biotherapeutics
Pharmaceutical Technology
OCTOBER 26, 2022
The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient. The future of cell and gene therapies.
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Aging And Schizophrenia Share Striking Similarities in 2 Types of Brain Cells
AuroBlog - Aurous Healthcare Clinical Trials blog
MARCH 17, 2024
US researchers examined gene expression in more than a million brain cells collected post-mortem from 191 donors. Those with schizophrenia and older adults were found to have a comparitively reduced expression of specific genes in both […]
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BlueRock Advances Bemdaneprocel Cell Therapy to Phase III Trial for Parkinson’s Disease
XTalks
JANUARY 16, 2025
This year has started strong for Parkinsons disease research with progress in several clinical trials and medical devices. BIALs Phase II ACTIVATE trial of BIA 28-6156, which targets GBA1 gene mutations, is ongoing, with results expected in 2026 to determine its potential to modify disease progression.
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Scientists Accidentally Made a Mouse Grow Legs in Place of Genitals
AuroBlog - Aurous Healthcare Clinical Trials blog
APRIL 8, 2024
Turning off a gene early in mouse development led researchers to end up with an accidental six-legged embryonic mammal. This strange result took the spinal cord research of developmental biologists Anastasiia Lozovska and Moisés Mallo and their colleagues at Portugal’s Gulbenkian Science Institute in a new direction.
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Experimental Alzheimer’s Vaccine Shown to Delay Symptoms in Mice
AuroBlog - Aurous Healthcare Clinical Trials blog
AUGUST 3, 2023
Mice engineered with genes that put them at greater risk of an Alzheimer’s-like disease had fewer amyloid plaques following the vaccine. An experimental vaccine that aims to slow down or prevent the progression of Alzheimer’s disease has been trialed in mice with promising early results.
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We’ve Discovered A Subtle Genetic Imbalance That May Drive Aging
AuroBlog - Aurous Healthcare Clinical Trials blog
DECEMBER 21, 2022
Researchers from Northwestern University have revealed animals like mice, rats, killifish, and even humans show a gradual imbalance of long and short genes in virtually every cell in their […].
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Unborn Babies Use a Special Gene to Steal Nutrients From Mum
AuroBlog - Aurous Healthcare Clinical Trials blog
JULY 19, 2023
Genetic inheritance is a physical link that tethers our birth parents; one-half of two sets of chromosomes meshing together to form our own unique set of molecular instructions for life.
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Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio
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. As such, rare disease patients and their families often face little hope for effective treatments.
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Generative AI in Clinical Research: Revolutionizing Medical Data Analysis, Treatment Planning, and Innovation
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.
Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia
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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Novel CSF biomarkers signal new avenues for Parkinson’s therapeutics
Pharmaceutical Technology
OCTOBER 18, 2022
CSF biomarkers have been a popular area of interest in neurodegenerative disease research with previous studies related to Alzheimer’s and Multiple Sclerosis exploring the potential of their use. In PD clinical research, many biomarkers, such as neurofilament, can be measured within the CSF using a lumbar puncture.
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Researchers see new generation therapeutics to propel precision medicine
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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Enhancing Access and Retention in Global, Cross-Border Rare Disease Clinical Trials
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.
Biology opens up new frontiers in medicine to cure genetic diseases: Kiran Mazumdar Shaw
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 17, 2024
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.
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Cell & Gene Therapy Product Development: How Can You Build a Successful Partnership with Your CRO’s Project Manager?
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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Hospital Universitario and Josep Carreras develop cell therapy for leukaemia
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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First hybrid gene therapy shows early promise in treating long QT syndrome
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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Leveraging Genetic Testing for Enrolling Rare Disease Trials
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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Clinical Research Nurse Finds Opportunities via LinkedIn: We Discuss Gene Therapy, Career Growth!
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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WCG: Consider IBC review requirements early on in HGT clinical trial planning
BioPharma Reporter
FEBRUARY 7, 2022
Planning for Institutional Biosafety Committee (IBC) oversight is a critical and often neglected initiation step for human gene transfer (HGT) clinical research, says an expert.
Abu Dhabi DoH signs new deal to accelerate development in life sciences
Pharmaceutical Technology
JUNE 16, 2023
Along with iCGD and MGB, DOH will facilitate the clinical research and treatment associated with prioritised global genetic diseases in the fields of rare diseases, oncology, cardiovascular and metabolic diseases and neurology.
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Voyager Pipeline Transformation Takes Shape, Focused On Maximizing Novel AAV Capsids
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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Velocity Opens Three UK Greenfield Clinical Research Sites
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.
How Centralized IBC Review Can Benefit Gene Therapy Research
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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Novel suppression-replacement preclinical gene therapy study shows promise in long and short QT syndromes
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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?The Current State of Advanced Therapeutics
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. For a full look at the cell and gene therapy landscape, check out our webinar titled: “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapy.”). (For Check it out!
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Charles River acquires Cognate BioServices to expand cell and gene therapy programs
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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Why a Platform Approach is Vital for Clinical Research Management Transformation
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.
Tamibarotene Fails in Phase III Trial for a Subset of Higher-Risk MDS
XTalks
NOVEMBER 15, 2024
The trial was evaluating the drug in combination with the chemotherapy azacitidine for the treatment of newly diagnosed patients with higher-risk myelodysplastic syndrome (HR-MDS) who harbor overexpression of the RARA (retinoic acid receptor alpha) gene.
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Advanced Biomed IPO Raises $6.56M Towards Liquid Biopsy Testing
XTalks
MARCH 17, 2025
Looking ahead, the company plans to expand into North American and European markets by preparing localized production and regulatory filings, and it aims to initiate clinical research for its A+LCGuard lung cancer screening kit by mid-2025 underscoring its commitment to rapid, affordable and reliable diagnostics.
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