XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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.

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 52

Drug Discovery World

FEBRUARY 12, 2024

Leslie Orne, President and CEO, Trinity Life Sciences “AI and ML will play an increasingly important role in the clinical development and commercialisation of cell and gene therapies, which are typically targeted to small patient populations.

Genome 139

Genome Genomics Life Science Gene Therapy 139

XTalks

SEPTEMBER 12, 2023

Life science podcasts have emerged as an invaluable tool for building connections with audiences in the digital era. Furthermore, we’ll explore the unique advertising opportunities that this platform offers, positioning brands at the forefront of the life science industry. The result?

Life Science 105

Life Science Branding Clinical Trials Clinical Trials 105

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.

Clinical Trials 124

Clinical Trials Clinical Trials Trials Gene Therapy 124

Drug Discovery World

DECEMBER 20, 2022

As 2022 draws to a close, life sciences market analysts attempt to correctly predict what the next 12 months might bring to the drug discovery sector. There will be greater emphasis on diversity in clinical trials. In summary, 2023 could be a very exciting year for life sciences.

Drugs 52

Drugs Gene Editing Clinical Trials Clinical Trials 52

ACRP blog

APRIL 5, 2023

AI is increasingly important in drug discovery and development as well as clinical trials, operations, pharmacovigilance, and many other areas.” This has led to the emergence of a new ‘pharmaceutical intelligence’ that has allowed the industry to move away from traditional, slow-moving, and costly processes,” Lyons writes. “AI

Big Data 52

Big Data Clinical Trials Clinical Trials Life Science 52

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

Drugs 75

Drugs Life Science Gene Therapy Genome 75

pharmaphorum

NOVEMBER 1, 2022

The two venerable Indiana institutions – Lilly headquartered in Indianapolis and Purdue in nearby West Lafayette – have been working together for many years, collaborating on life sciences research in areas such as new delivery systems for injectable medicines and predictive models for clinical research.

Manufacturing 52

Manufacturing Life Science Drug Delivery Medicine 52

XTalks

MARCH 25, 2021

Xtalks is proud to announce the launch of the Xtalks Life Science podcast. Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Fresh Conversations About Life Science Topics. She focuses on news relating to the food industry and writes blogs on recruitment and HR in the life sciences.

Life Science 105

Life Science HR Clinical Trials Clinical Trials 105

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. Here are some of the top life science trends and topics from 2022. Gene Therapy Approvals. million for a single infusion.

Life Science 52

Life Science Gene Therapy Gene Clinical Trials 52

pharmaphorum

MARCH 3, 2022

This will make crucial data more securely and quickly available for research, while maintaining privacy, and help the NHS to deliver new life-saving treatments to patients faster, according to the Department of Health.

Manufacturing 52

Manufacturing Life Science Clinical Trials Clinical Trials 52

XTalks

MAY 9, 2024

As we explore Genmab’s strategic vision, technological advancements and the next frontier in antibody research, we gain insights into the future of medicine — a future where Genmab’s cutting-edge therapies are poised to transform patient care and meet the complex challenges of the next generation of healthcare.

Antibody 52

Antibody Clinical Trials Clinical Trials Medicine 52

The Pharma Data

JANUARY 7, 2021

Anne Prener has more than 25 years of leadership experience within life sciences companies, and currently serves as Chief Executive Officer of Imbria Inc. a gene therapy company focused on eye diseases. About Galecto Galecto (NASDAQ: GLTO) is a clinical stage biotechnology company incorporated in the U.S.

Gene Therapy 52

Gene Therapy Life Science Production Clinical Development 52

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.

Clinical Trials 105

Clinical Trials Clinical Trials Trials Vaccination 105

XTalks

SEPTEMBER 17, 2020

While clinical researchers occasionally need to identify and validate a new surrogate endpoint for a given trial, choosing endpoints that have been previously used as the basis of NDAs and BLAs can clear the path to eventual approval. Mutations in the gene encoding ?-Gal Prevalence: ABCG5 gene mutation: 1 in 2.6

Trials 98

Trials Gene Hormones Clinical Trials 98

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.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 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. Four years later at Calliditas Therapeutics, his contributions to drug development in IgA nephropathy led to the FDA approval of Tarpeyo.

Development 52

Development Drugs Clinical Trials Clinical Trials 52

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.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

pharmaphorum

JANUARY 27, 2023

She knows only too well how understanding the importance of patient centricity is crucial to the success of any modern life sciences company – but how has the landscape changed since 2019? “I It’s more the late-stage clinical trials that are not being commenced in the UK,” Dr Mullen elaborated. “So,

Clinical Trials 111

Clinical Trials Clinical Trials Trials Development 111

XTalks

DECEMBER 20, 2022

a given gene mutation) or expressed at higher levels (an abnormal protein that a drug is designed to target) in a certain racial demographic or gender. Patients from certain socioeconomic, racial and age demographics may not be too aware of how their participation in clinical research can be important.

Clinical Trials 97

Clinical Trials Clinical Trials Trials Regulation 97

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.

Development 52

Development Gene Therapy Engineer Life Science 52

XTalks

FEBRUARY 24, 2023

When life sciences industry partners with patients and their advocacy communities, the patient voice becomes integrated into the drug development process. It’s an increasingly common and evolving practice that has brought about meaningful change in the clinical trial process.

Development 98

Development Clinical Trials Clinical Trials Gene Therapy 98

XTalks

DECEMBER 14, 2023

Through Know Your Tumor, more than 600 genes are tested, including BRCA , BRAF , KRAS , NTRK , PALB and many others. Patients may also learn about inherited mutations that are also present that may indicate other treatment options.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Doctors 52

The Pharma Data

NOVEMBER 5, 2020

Velders is presently chairman of HollandBIO, the association of Dutch biotech companies, and provides years of experience, specifically in the cell and gene therapy areas. Prior to Lonza, she led External Science and Partnering across the globe for Sanofi R&D. Schollmeier is managing director of a consulting and investment firm.

Genome 40

Genome Genomics Medicine Life Science 40

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.

Clinical Trials 97

Clinical Trials Clinical Trials Trials Development 97

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.

Clinical Trials 105

Clinical Trials Clinical Trials Trials Gene Therapy 105

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.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

JULY 4, 2022

A very important question was raised by the webinar attendee of the diversity and inclusion of patients in clinical research, and Dr Hackett commented: “A lot of the work has been done in our Paediatric & Rare Disease network, as well as our Cell and Gene Therapy network, we are tackling the issue of diversity in data.

Genome 105

Genome Genomics Research Medicine 105

Cloudbyz

JUNE 10, 2023

The Title 21 Code of Federal Regulations Part 11, or 21 CFR Part 11, is a regulation set forth by the FDA, which applies to all industries under its regulation, including pharmaceuticals, medical devices, biotechnology, and other life sciences.

Compliance 88

Compliance Clinical Trials Clinical Trials Trials 88

Drug Discovery World

APRIL 29, 2024

Bill Dawson, Chair, Medicines Manufacturing Industry Partnership (MMIP): Dawson is Head of Biologics and Devices Manufacturing at GSK and has over 25 years’ experience in the vaccines, biopharma, pharmaceutical and clinical research (CRO) industries.

Drugs 52

Drugs Sales Gene Therapy Life Science 52

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.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

pharmaphorum

SEPTEMBER 12, 2022

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. Furthermore, each cancer has various gene changes – also referred to as the genetic makeup of cancer. In other words, every person’s cancer has its own genetic construct.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

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

Trials 83

Trials Drug Delivery Drugs Gene 83

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. If the gene does not integrate into the genome, the cell will not become a CAR T cell.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

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.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111

Drug Discovery World

FEBRUARY 6, 2023

Johannes Goll, Global Head of Emmes’ Biomedical Data Science and Bioinformatics department, explains how big data analytics can deliver improved therapies and bring clinical research one step close to realising the potential of personalised care.

Big Data 52

Big Data RNA Bioinformatics Vaccination 52