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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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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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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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. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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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. The IBC’s membership roster is therefore unique to the individual research location it oversees.

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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. For instance, in oncology, patients with specific genetic mutations (e.g.,

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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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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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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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Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. Synthetic mRNA-based therapeutics used in clinical research today also fall into this self-limiting class of recombinant therapeutics.

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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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Gene Therapy Development Gene Drugs 52

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.

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Genome Genomics Clinical Trials Clinical Trials 105

pharmaphorum

MAY 18, 2022

To support the wide range of patients this complex disease impacts, who suffer from a diverse range of symptoms, it is critical that research into this area continues. “At Charl believes we are witnessing the ‘decade of the brain’ with significant leaps in scientific understanding of disease pathways and genetic correlations.

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XTalks

MARCH 25, 2021

Ayesha obtained an MSc in Cancer & Genetics from McMaster University and a PhD in Medical Biophysics from the University of Toronto. She has presented her research at international conferences and published several peer-reviewed papers.

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XTalks

FEBRUARY 27, 2024

Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy. Both therapies were awarded their approvals on the same day in December 2023.

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XTalks

SEPTEMBER 12, 2023

Biomedical Research and Technologies: Get access to the most recent biomedical research news from both industry and academia, including preclinical studies and technological breakthroughs in genomics, single cell analysis, proteomics, metabolomics and much more.

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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. While mRNA usage has played several roles in clinical research , oncology researchers in particular are eager to explore the possibilities of mRNA-based cancer vaccines.

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pharmaphorum

JANUARY 27, 2023

In the morning, I can be talking about gene therapy for hearing loss, for example, [and] in the afternoon, I can be talking about the treatment of a very specific tumour. million people in the UK that have a rare disease,” she continued, “and that’s where a lot of the innovation is, in more of these rare genetic diseases.

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The Pharma Data

DECEMBER 20, 2020

eTrueNorth is actively recruiting COVID-19-positive patients about potential participation in the NIH’s ACTIV-2 clinical research trial. FLT180a is an AAV gene therapy. Sio Gene Therapies reported positive six-month follow-up data from the low-dose cohort of its dose escalation Phase I/II trial of AXO-AAV-GM1.

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Trials Gene Therapy Vaccine Vaccination 52

XTalks

MAY 17, 2024

Philipson’s transition to the biotech sector began with a position at UK-based biotech Trizell, where his efforts culminated in a gene therapy FDA submission for bladder cancer. Reflecting on the evolution of rare disease treatments, Dr. Philipson emphasizes the critical role of understanding disease biology and genetics.

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Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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Advarra

JUNE 20, 2023

Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community. Often, medical innovations are first explored in oncology research (e.g., cell and gene therapies), with other therapeutic areas then pushing it further.

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The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Prior to SpringWorks, Smith was the executive vice president of Gene Therapy at Bamboo Therapeutics.

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Development Gene Therapy Engineer Life Science 52

XTalks

JUNE 4, 2024

Borie explains how Kyverna Therapeutics’ research and development efforts are grounded in leveraging the immune system to target the pathogenesis of autoimmune diseases such as lupus nephritis and myasthenia gravis.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Antibody 52

XTalks

AUGUST 30, 2022

Clinical research experts from the CRO Medpace shared insights about the operational and regulatory considerations for neuroscience trials with direct CNS administration. There are loose correlations between the pathology going on in the brain and clinical symptoms in neurology and psychiatry,” says Dr. Vornov. “We

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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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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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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.

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Worldwide Clinical Trials

MAY 30, 2024

This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications. For a more in-depth discussion on challenges and solutions to ALS research, read our blog.

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pharmaphorum

SEPTEMBER 1, 2020

Likewise, the 1964 Declaration of Helsinki put greater ethical structures on clinical research, clearly cementing the difference between production of scientific prescription medicines and other chemicals. This knowledge of genetics and the underlying cause of many diseases, including cancer, has resulted in powerful new drugs.

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XTalks

DECEMBER 20, 2023

The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy. Check out this article to learn about the biotech companies that went public this year.

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Pharmaceutical Technology

FEBRUARY 20, 2023

This level of homogeneity within the clinical trial population may prevent the identification of variable therapy responses within population subgroups. The most obvious is that both firms are highly scientific and so that presents a lot of opportunity to exchange knowledge and research.

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Pharmaceutical Technology

FEBRUARY 20, 2023

The formal signing ceremony of the MoU today at Cerba Research’s Ghent office was led by Mario Papillon, CEO of Cerba Research, and Xu Yi, CEO of Teddy Lab, on behalf of their respective organisations. About Teddy Clinical Research Laboratory : As a joint venture company established in 2016 by Dian Diagnostics (300244.SZ)

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Pharmaceutical Technology

MAY 9, 2023

The shared expertise and resources enables Cerba Research to broaden its services within cell and gene therapy, infectious disease and oncology and access new markets in North America, Asia and Africa, the sites of some of the most exciting growth in clinical trials.

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XTalks

FEBRUARY 26, 2025

Regenerons CHORD study evaluating investigational gene therapy DB-OTO for otoferlin-related hearing loss a rare condition caused by variants in the OTOF gene has revealed that 10 of 11 children with at least one post-treatment hearing assessment experienced notable improvements, including dramatic gains in speech perception.

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Worldwide Clinical Trials

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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