XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. According to the FDA, life expectancy for those with DMD has increased over the years, with some patients surviving beyond 30 years.

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FDA Approval Genetics Gene Therapy Gene 105

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. Of the more than 10,000 rare diseases, more than 80 percent are caused by genetic defects.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

Pharmaceutical Technology

FEBRUARY 2, 2023

The regulatory approval is based on the data obtained from the ASCEND-D trial, which is evaluating Jesduvroq’s safety and efficacy to treat patients. The ASCEND programme included five Phase III trials that enrolled more than 8,000 patients who received treatment for up to 4.26 dL) for the patients.

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FDA Approval Life Science Genetics Trials 264

XTalks

FEBRUARY 21, 2024

Black participation in clinical trials, and the underrepresentation of minority groups in general in trials, remains low. It is therefore important to work towards increasing Black participation in clinical trials, which begins with understanding the barriers to participation and taking steps to address them.

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Clinical Trials Clinical Trials Trials Life Science 59

XTalks

FEBRUARY 19, 2024

The team used the data and patient samples from a gB/MF59 Phase II trial in adolescent girls as a benchmark to assess the new mRNA-based vaccine. The comparison is particularly important as gB/MF59 demonstrated promise in Phase II clinical trials but did not advance to Phase III.

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Vaccination Vaccine Trials Clinical Trials 59

XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

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Trials Clinical Trials Clinical Trials Antibody 97

XTalks

APRIL 5, 2024

Amylyx Pharmaceuticals announced yesterday that it will be pulling its ALS (amyotrophic lateral sclerosis) drug Relyvrio from the market after failing in a confirmatory trial. Last month, Amylyx reported results from the global PHOENIX Phase III clinical trial that were unable to confirm the drug’s effectiveness.

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Trials Drugs Marketing FDA Approval 98

XTalks

MAY 29, 2024

Recognized on May 30 each year, World MS Day brings together global communities, healthcare professionals and life sciences industries to share stories, raise awareness and support those affected by multiple sclerosis (MS). These seek to protect the nervous system from MS damage and slow disease progression.

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Clinical Trials Clinical Trials Trials Life Science 52

XTalks

MAY 16, 2023

Clinical trials for pulmonary fibrosis are being conducted to evaluate the effectiveness of novel therapies and interventions, providing hope for improved outcomes in the future. Pulmonary fibrosis is a serious condition that can significantly impact life expectancy.

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Clinical Trials Clinical Trials Trials Research 98

XTalks

MARCH 20, 2024

When it comes to leadership in clinical trials today, what qualities are necessary to navigate the evolving landscape of clinical research effectively? Continue reading to explore effective leadership in clinical trials. Christine Enciso: My journey in clinical research spans over 28 years, mainly focused on clinical trial operations.

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Clinical Trials Clinical Trials Trials Clinical Research 52

pharmaphorum

DECEMBER 9, 2022

has announced that the pivotal phase 2 FELIX clinical trial of obecabtagene autoleucel (obe-cel) in relapsed/refractory (r/r) adult Acute Lymphoblastic Leukaemia (ALL) patients has met its primary endpoint of overall remission rate (ORR) at interim analysis. (Nasdaq: AUTL), a clinical-stage biopharma and a portfolio company of Syncona Ltd.,

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Trials Clinical Trials Clinical Trials Gene Therapy 98

XTalks

DECEMBER 14, 2023

In an insightful interview with Xtalks , Cassadie Moravek, the Senior Director of Clinical Trial Portfolio and Program Management at the Pancreatic Cancer Action Network (PanCAN) , shares her perspective on the organization’s efforts in advancing pancreatic cancer research and patient care. Read on to learn more from Moravek.

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Clinical Trials Clinical Trials Trials Doctors 52

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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In-Vivo Gene Editing Trials Gene 52

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables.

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Life Science Gene Editing Development Clinical Trials 119

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?

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Life Science Branding Clinical Trials Clinical Trials 105

pharmaphorum

JUNE 11, 2021

Abel Archundia discusses how COVID-19 has highlighted the importance of securing clinical trials against cyber threats. However, data-intensive, decentralised clinical trials also present a major risk to healthcare participants through cybercrime and data privacy vulnerability. These are the crown jewels of any R&D organisation.

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Clinical Trials Clinical Trials Trials Compliance 109

XTalks

JUNE 20, 2022

At the same time, investors’ eyes are on the company’s Phase III APOLLO-B trial for Onpattro in this heart disease indication. Alnylam is currently conducting the HELIOS-B study to evaluate Amvuttra for ATTR cardiomyopathy and expects patient survival data in 2024. billion in global sales by 2026.

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FDA Approval Protein Genetics RNA 98

XTalks

SEPTEMBER 11, 2020

Expanding minority Americans’ access to care and clinical trials isn’t just the right thing to do. Clinical Trials Don’t Reflect Demographics. Why, then, are clinical trials so stubbornly homogeneous? Clinical trials, however, don’t reflect that diversity. Nearly all diseases have a genetic component.

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Clinical Trials Clinical Trials Trials Drugs 98

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks Life Science Podcast Ep.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

FEBRUARY 25, 2022

There has been increasing awareness around the need for improving inclusion and diversity in clinical trials. Despite these statistics, in the US, Black people only constitute five percent of all clinical trial participants. The overwhelming majority of trial participants nationwide are white. Medical Mistrust.

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Clinical Trials Clinical Trials Trials Research 52

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. COVID-19 Clinical Trials.

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Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

MAY 7, 2021

Novavax published the latest results from a Phase IIb clinical trial conducted in South Africa evaluating the safety and efficacy of NVX-CoV2373, appearing in the NEJM ’s May 6, 2021 issue. Earlier results from a more complete analysis of trial data shared in March confirmed a high level efficacy of 86.3 1.351 variant.

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Protein Vaccination Vaccine Trials 97

XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Dr. Bahassi: There are several factors to consider when designing a trial that involves ctDNA detection.

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Clinical Trials Clinical Trials Trials FDA Approval 98

XTalks

FEBRUARY 23, 2024

XTALKS WEBINAR: A Collaborative Approach to Advancing Access and Equity in Rare Disease Clinical Trials Live and On-Demand: Thursday, February 29, 2024 , at 11am EST (4pm GMT / UK) Register for this free webinar to join the discussion with clinical trial experts on a collaborative approach to patient-friendly trials in rare disease.

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Clinical Trials Clinical Trials Genetics Trials 103

XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Diagnosis is confirmed by a differential complete blood count and genetic testing.

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FDA Approval Life Science Clinical Trials Clinical Trials 97

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. IGF-1 is an acceptable surrogate endpoint in trials of growth hormone receptor agonists like Somavert, and is preferred for pediatric patient populations.

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Trials Gene Hormones Clinical Trials 98

Drug Discovery World

MAY 11, 2023

Fundraising and investment are a core pillar of not just BioTrinity events but of the drug discovery industry at large, so events such as Tuesday’s keynote, Investment Trends for the Life Sciences Industry, are critical for members of the industry.

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Life Science Genetics Vaccination Vaccine 52

XTalks

SEPTEMBER 17, 2021

After asking for a placebo-controlled Phase III study for the drug in April that upset the ALS Association, the FDA dialed back on the request this week, agreeing to accept only Phase II trial data for evaluation of Amylyx’s drug application. People with ALS and their loved ones cannot wait.”.

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Trials Drugs FDA Approval Clinical Trials 98

pharmaphorum

FEBRUARY 17, 2021

One group excelling in this space is the Genetic Alliance. Their work ranges from developing new services in partnership with the National Health Service (NHS) to working with the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions, and the Department of Health on the UK Strategy for Rare Diseases.

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Life Science Branding Genetics Trials 98

Drug Discovery World

FEBRUARY 28, 2024

In glioblastoma, as with many other highly recurrent cancers, tumour cells that escape treatment develop multiple genetic adaptations that allow them to proliferate. The Gladstone team realised that these mutated cells have a unique genetic signature that could be targeted. “We

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Development Research Drugs Antibody 64

pharmaphorum

JULY 7, 2022

Eradicating cancer is an ambitious goal for oncologists and life science companies around the world. Read on to find out about reaching the gold standard of lung cancer screening, the promise of therapeutic cancer vaccines, why diversity is essential in oncology trials, and much more. Realising the promise of cancer vaccines.

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Vaccination Vaccine Life Science Trials 52

XTalks

APRIL 3, 2024

Voydeya’s FDA approval was based on positive results from the pivotal ALPHA Phase III trial. Results of the trial showed that at Week 12, danicopan plus ravulizumab or eculizumab led to a significant increase in hemoglobin levels from baseline compared to placebo plus ravulizumab or eculizumab.

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FDA Approval Sales Trials Development 102

pharmaphorum

OCTOBER 7, 2020

There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments,” said Claes Gustafsson, chair of the Nobel Committee for Chemistry. What started as a curiosity?driven,

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Gene Editing Bacteria DNA Gene 104

XTalks

MAY 26, 2023

DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

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Gene Therapy Gene FDA Approval Dermatology 97

Drug Discovery World

DECEMBER 14, 2023

The new hub will become a part of the already thriving life sciences sector in the Glasgow area, most of which is centred around the university, the Cancer Research UK Scotland Institute and the BioCity cluster, making Glasgow and its surrounds one of the most important regions for drug discovery and development in the UK.

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Drugs Life Science Research Scientist 75

XTalks

MARCH 26, 2024

For professionals in healthcare and life sciences industries, World Bipolar Day acts as a platform to spread awareness of the complexities of bipolar disorder and the comprehensive treatment approaches available for this condition, such as medication, psychotherapy and support for patients and their families.

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