PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

Therapies 97

Therapies Clinical Trials Clinical Research Trials 97

Drug Target Review

FEBRUARY 9, 2024

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Despite their potential, ADSC therapy faces several challenges in preclinical studies. Is there a certain disease or condition that you believe stem cell therapy holds the most promise for?

Therapies 105

Therapies Immune Response Clinical Trials Treatment 105

FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

Therapies 98

Therapies Trials FDA Clinical Trials 98

Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA.

Research 137

Research Immune Response Clinical Trials Therapies 137

Fierce BioTech

NOVEMBER 21, 2023

Cell and Gene Therapy (CGT) has the potential to transform the life trajectory of children with genetic conditions amendable to gene modification with advances in treatment and possible cures. Pediatric CGT trials have the potential to cure diseases and prolong lives.

Therapies 91

Therapies Research Trials Treatment 91

Antidote

JUNE 7, 2023

Before any new medical treatment, therapy, or device is approved, it must first be tested through clinical research studies. While some research studies seek participants with illnesses or conditions to be studied in the clinical trial, it is not always necessary to have a specific illness or condition to participate.

Research 98

Research Clinical Trials Clinical Research Trials 98

Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

Therapies 114

Therapies Disease Engineering Medical Schools 114

Antidote

JULY 27, 2023

Before any new therapy can be put on the market, it must be tested to ensure its safety and effectiveness, and the research for this process is done through clinical trials.

Clinical Research 98

Clinical Research Research Clinical Trials Packaging 98

Science Daily: Pharmacology News

JANUARY 24, 2024

A novel gene therapy for hearing loss was administered to six children in China in a clinical trial. The researchers report in a new study, after 26 weeks, five children demonstrated hearing recovery and dramatic improvements in speech perception and the restored ability to conduct normal conversation.

Therapies 88

Therapies Clinical Trials Trials Research 88

BioPharma Drive: Drug Pricing

SEPTEMBER 12, 2024

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

Therapies 116

Therapies Virus Trials Hospitals 116

PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. As a result, researchers now often use composite endpoints in lieu of the customary single primary endpoint.

Clinical Trials 98

Clinical Trials Trials Clinical Research Disease 98

Broad Institute

NOVEMBER 21, 2024

Researchers identify source of a brain cancer’s deadly transformation By Corie Lok November 21, 2024 Breadcrumb Home Researchers identify source of a brain cancer’s deadly transformation Slow-growing glioma tumors become more aggressive when their cells shift identities and acquire new genetic mutations.

Research 124

Research Medical Schools DNA Hospitals 124

BioPharma Drive: Drug Pricing

JUNE 22, 2023

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

Therapies 132

Therapies FDA Trials Disease 132

Antidote

OCTOBER 7, 2022

Different types of clinical studies are designed to investigate potential therapies, medications, and medical devices — and all of these must go through each phase of a clinical trial before they are approved.

Research 98

Research Clinical Trials Therapies Trials 98

PPD

JUNE 7, 2023

The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations. As therapies for the treatment of NASH in adult patients go this year to the U.S.

Trials 98

Trials Clinical Trials Clinical Research Disease 98

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.

Therapies 102

Therapies Disease RNA Engineering 102

Drug Target Review

FEBRUARY 1, 2024

This can present challenges when attempting to recruit an enriched patient population for clinical trials. Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? The lack of heterogeneity can introduce significant risk into mid- to late-stage studies.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

Antidote

AUGUST 29, 2022

In the world of medical research, experts are constantly looking for new drugs, treatments, and therapies for a variety of conditions — but the process of getting these treatments approved is only possible if they are tested through clinical trials.

Clinical Trials 98

Clinical Trials Trials Therapies Treatment 98

Conversations in Drug Development Trends

MAY 10, 2024

Over the past two decades, industry-sponsored clinical trials have targeted treatments for BPD, yielding some promising outcomes; however, broader psychiatric research often excludes BPD patients, a trend that extends to the emerging field of psychedelic studies.

Clinical Trials 95

Clinical Trials Trials Treatment Drug Development 95

Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

Therapies 105

Therapies Engineering Production Clinical Trials 105

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

Therapies 98

Therapies Disease Engineering RNA 98

Drug Target Review

OCTOBER 5, 2023

This study sought to shed light on the safety and effectiveness of aHSCT in routine healthcare settings, moving beyond the confines of clinical trials. The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020.

Therapies 116

Therapies Treatment Clinical Trials Disease 116

Antidote

AUGUST 8, 2022

Medical research is how new treatments and therapies move forward, but challenges surrounding patient recruitment can often lead to delays. Patients often have misconceptions about participating in a clinical trial — and if they are interested, they often aren’t sure where to begin.

Research 98

Research Clinical Trials Therapies Trials 98

Conversations in Drug Development Trends

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.

Trials 75

Trials Disease Clinical Trials Clinical Research 75

Antidote

MAY 22, 2023

Participating in a clinical trial can be a worthwhile opportunity for any individual.

Clinical Trials 98

Clinical Trials Trials Therapies Treatment 98

Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies 105

Therapies Engineering Molecular Biology Science 105

PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.

Clinical Trials 86

Clinical Trials Trials Clinical Research Doctors 86

ASPET

DECEMBER 22, 2023

This study provides a unique translational research opportunity to help both humans and dogs diagnosed with diseases that carry dismal prognoses in both species: histiocytic sarcoma (HS), hemangiosarcoma (HSA), and disseminated mastocytosis/mast cell tumor (MCT).

Clinical Trials 189

Clinical Trials Disease Drugs Therapies 189

PPD

OCTOBER 8, 2024

With FSO, all clinical trial tasks (e.g., are outsourced to a contract research organization (CRO) partner. In a hybrid model, one or more FSP offerings are added to a new or existing FSO arrangement to optimize clinical trial operations or address unforeseen circumstances and changing demands.

Trials 98

Trials Clinical Trials Clinical Development Clinical Research 98

Conversations in Drug Development Trends

OCTOBER 26, 2022

Bringing a psychedelic into a clinical trial setting is complex and requires a thorough operational approach to ensure the study’s success. Psychedelic trials need to be done with scientific rigor. Our team published recently in The Journal of Psychedelic Psychiatry exploring how we can bring more rigor to these trials.

Trials 97

Trials Clinical Trials Licensing Licensing 97

Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

Therapies 103

Therapies DNA Engineering Virus 103

Chemical Biology and Drug Design

MAY 11, 2023

Crocin, a naturally occurring carotenoid molecule, was found to have neuroprotective potential in the therapy of this disorder. However, more research on its efficacy as a treatment of Parkinson's disease is needed, specifically examining its mechanisms and the results obtained in clinical trials.

Disease 100

Disease Research Clinical Trials Treatment 100

Science Daily: Pharmacology News

MARCH 7, 2024

Four children have remained free of detectable HIV for more than one year after their antiretroviral therapy (ART) was paused to see if they could achieve HIV remission, according to new research.

Treatment 129

Treatment Clinical Trials Therapies Trials 129

DrugBank

AUGUST 9, 2024

Investment Trends in Pharmaceutical Research and Development Investing in the pharmaceutical sector presents a unique set of challenges and opportunities; let’s examine them in more detail.  These new therapies have demonstrated significant LDL cholesterol reductions, but their high cost remains a barrier to widespread adoption.

Pharmaceuticals 77

Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 77

Conversations in Drug Development Trends

APRIL 3, 2024

By: Sue Batchelor, Executive Director, Oncology Project Management Radiopharmaceuticals represent a cutting-edge frontier in oncology treatment, offering the promise of highly targeted therapy with the potential to revolutionize cancer care. Are you aware of the challenges you must address for a successful radiopharmaceutical trial?

Trials 81

Trials Clinical Trials Compliance Treatment 81

PPD

AUGUST 28, 2024

For pharmaceutical innovators and drug developers working to bring oncology therapies to market, patients are the “why” behind it all. For many patients, involvement in oncology clinical trials represents a last hope for an effective therapy. Researchers often employ cell therapy studies for oncology indications, and the U.S.

Trials 63

Trials Clinical Trials Therapies Drug Development 63

Antidote

FEBRUARY 22, 2024

Behind every new treatment is the incredible team of doctors, researchers, patients, and clinical trial specialists who worked on the research that made it possible.

Clinical Trials 69

Clinical Trials Trials Doctors Therapies 69