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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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.

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Pharmaceutical Technology

MAY 3, 2023

The regulator has also granted orphan drug designation for the therapy to treat follicular lymphoma (FL). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The transaction is anticipated to be completed in the second quarter of 2023.

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Drug Discovery World

FEBRUARY 12, 2024

In the upcoming year, we will see the expansion of new, more complex markets and updated requirements from regulators. Ensuring sufficient and timely patient enrollment in a rapidly growing number of cell and gene therapy clinical trials is becoming a bottleneck to advancing potentially life-saving therapies.

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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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ACRP blog

APRIL 5, 2023

A special feature in the forthcoming April issue of ACRP’s Clinical Researcher journal puts a spotlight on how the life sciences sector has been undergoing an immense transformation, driven by the adoption of artificial intelligence (AI), machine learning, and natural language processing, and by the exploitation of “big data” sources in new and powerful (..)

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Drug Discovery World

SEPTEMBER 23, 2022

An interesting week for clinical research and drug approvals, with rare diseases being a key focus for both the FDA and European Commission. FDA approves gene therapy for rare neurological disorder. Breast cancer cells ‘regulate their own metastases’. News round-up for 19-23 September by DDW Editor Reece Armstrong.

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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. Food and Drug Administration (FDA).

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Pharmaceutical Technology

JULY 18, 2022

Novartis is developing icenticaftor (QBW251), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. CFTR modulators are widely used in the treatment of cystic fibrosis (CF), which is characterised by homozygous mutations in the CFTR genes.

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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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XTalks

DECEMBER 20, 2022

Therefore, it is important to be up to speed with US Food and Drug Administration (FDA) guidance documents for example and to have early discussions and engagement with regulators. This includes guidance documents on eligibility criteria , patient-reported outcome (PRO) measures and clinical trial design strategies among many others.

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Cloudbyz

JUNE 11, 2023

They often come with in-built compliance mechanisms to ensure adherence to regulations and good clinical practice guidelines. What are the different types of decentralized clinical trials? Why are clinical trials decentralized? Clinical trials are increasingly being decentralized due to a variety of reasons.

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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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Drug Discovery World

APRIL 14, 2023

Carl H June, MD, FAACR – Lifetime Achievement in Cancer Research June, a Fellow of the AACR Academy, is the Richard W Vague Professor in Immunotherapy, Director of the Center for Cellular Immunotherapies, and Director of the Parker Institute for Cancer Immunotherapy in the Perelman School of Medicine at the University of Pennsylvania.

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XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Inherited mutations in the SLC3A1 and SLC7A9 genes cause cystine to be abnormally transported within the kidney leading to cystinuria.

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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. As the efforts to develop a vaccine against SARS-CoV-2 intensified, clinical research groups found themselves designing trials to evaluate them under the confines of tighter timelines.

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Roots Analysis

MARCH 24, 2022

Genomics is the study of genetic material within a cell, and transcriptomics is the study of gene activity in different cells. The post Metabolomics: An Emerging yet Powerful Tool in Clinical Research and Development appeared first on Blog. Likewise, metabolomics refers to the study of byproducts of metabolism. Web: [link].

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Drug Discovery World

MAY 17, 2024

Deepika Khedekar , Associate Centralized Clinical Trial Lead at IQVIA Inc, looks at the challenges of utilising artificial intelligence (AI) in oncology clinical research. The researchers delved into the genetic makeup of tumours, recognising that genetic mutations heavily influence how tumours respond to drugs.

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The Pharma Data

JANUARY 7, 2021

As CEO of Freeline, a liver-directed gene therapy company, she scaled the company from preclinical stage to a fully integrated biotechnology organization, which included a broad, internally developed pipeline, two programs in clinical development and a commercial-scale, high-quality CMC and manufacturing platform.

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Gene Therapy Life Science Production Clinical Development 52

WCG Clinical

SEPTEMBER 26, 2023

We’re here today to talk about clinical research with psychedelic drugs. FDA published its first draft guidance on this topic in June in response to an increasing interest in clinical trials of psychedelic drugs to treat a range of diseases. And to be clear that we’re not trying to regulate.

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pharmaphorum

JULY 4, 2022

Together, the webinar panellists explored the potential of medical informatics solutions and methods in rare disease research, studies, data optimisation, and usability to overcome these challenges, all whilst maintaining compliance with data protection regulations and a patient-centric approach.

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The Pharma Data

DECEMBER 17, 2020

Martin Carter Professor in Clinical Investigation and Head of the Laboratory of Investigative Dermatology at The Rockefeller University and Weinblatt is the co-director of Clinical Rheumatology at the Brigham and Women’s Hospital. Prior to SpringWorks, Smith was the executive vice president of Gene Therapy at Bamboo 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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The Pharma Data

OCTOBER 18, 2020

This study is the first to show that by increasing NAD + , NR helped protect telomeres which are important in addressing life-threatening telomere-related diseases, paving the way for future clinical research. NR works by raising levels of NAD + , an important regulator that is known to naturally decline with age.

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XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Worldwide Clinical Trials

MAY 23, 2024

By: Sarah Bly, Regulatory Science and Innovation and Matt Cooper, Executive Director, Therapeutic Strategy Lead, Oncology The European Union (EU) presents a unique set of regulatory challenges and opportunities for clinical trials in oncology. However, they also bring unique regulatory challenges.

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XTalks

MAY 11, 2023

I know you also serve on the board of directors for Global Genes, which recently merged with RARE-X. Wendy: RARE-X is now the data and research arm of Global Genes, the flagship patient organization representing rare, ultra-rare, and “n-of-one” patients. . I’m very committed to working in rare disease for that reason.

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Clinical Trials Clinical Trials Trials Development 97

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 do the Regulations Say about IBC Review? What Does an IBC Review?

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Drug Discovery World

SEPTEMBER 28, 2022

Acts such as General Data Protection Regulation (GDPR) in the UK and the Health Insurance Portability and Accountability Act (HIPAA) in the US protect users’ health data from being used for commercial purposes. Which areas of drug discovery research stand to benefit most? . Looking ahead, the future is bright.

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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.

Gene Therapy 52

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Drug Discovery World

AUGUST 10, 2022

For example, one clinical trial examined the safety and efficacy of a CAR T cell that includes a suicide gene, which produces a toxic protein that causes the CAR T cells to die upon exposure to a drug 10. For example, it is challenging to regulate transgene copy numbers in each T cell, posing safety risks.

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XTalks

NOVEMBER 7, 2022

Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. The first option is to design a personalized small NGS gene panel based on the somatic mutations that were detected in the patient, and this can be used for monitoring disease progression.

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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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pharmaphorum

SEPTEMBER 1, 2020

.” This public-spirited industry still required greater oversight, however, and government regulations on medicines increased on both sides of the Atlantic. Thalidomide and the development of drug safety regulation and monitoring. George Merck on the cover of Time magazine.

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XTalks

DECEMBER 12, 2023

In some areas, say Alzheimer’s disease, we have growing collections of imaging, gene, cerebrospinal fluid (CSF) and blood markers to follow and look at target engagement of our drugs,” says James Vornov, MD, PhD, Vice President of the Medical Department at Medpace.

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