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.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field. In October, FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 – including one for a Phase 3 trial – totaling $17.2

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Clinical Development Production Clinical Trials Treatment 69

Codon

NOVEMBER 3, 2024

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. A 1994 review of 14 prospective trials and 12 case-control studies revealed that the BCG vaccine reduced the risk of TB by 50 percent.

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Vaccine Trials Therapies Treatment 102

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.

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Therapies Clinical Trials Cell Biology Trials 118

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?

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Therapies Disease Engineering Medical Schools 116

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Therapies FDA FDA Approval Treatment 119

Conversations in Drug Development Trends

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

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Treatment Clinical Trials Small Molecule Therapies 81

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Therapies FDA Clinical Trials Disease 107

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.

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Therapies DNA Engineering Virus 103

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.

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Therapies Disease RNA Engineering 102

Drug Target Review

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates.

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Therapies Treatment Clinical Trials Drugs 98

Drug Target Review

FEBRUARY 1, 2024

Part of the challenge is that patients present with very different kinds of clinical phenotypes, meaning the populations are heterogeneous. This can present challenges when attempting to recruit an enriched patient population for clinical trials. This has been a hot button issue in neuroscience for as long as I can remember.

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Therapies Drug Development Disease Clinical Development 104

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.

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Therapies Disease Engineering RNA 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.

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Clinical Trials Trials Treatment Drug Development 95

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. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

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Clinical Trials Trials Clinical Research Disease 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.

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Trials Disease Clinical Trials Clinical Research 75

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.

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Therapies Engineering Production Clinical Trials 105

PPD

MARCH 11, 2025

Breaking through research barriers Challenge #1: Small groups of patients Rare diseases impact a small number of individuals, making it difficult to recruit enough participants for clinical trials. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

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Drug Development Clinical Trials Disease Trials 52

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.

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Therapies Engineering Molecular Biology Science 105

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.

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Trials Clinical Trials Drug Development Therapies 69

PPD

MAY 18, 2023

Yet, four out of five care partners of people with neurodegenerative disorders have never been informed of clinical trial opportunities , according to a survey of more than 250 caregivers conducted by PPD, the clinical research business of Thermo Fisher Scientific. There’s little time to lose.

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Clinical Trials Trials Clinical Research Doctors 86

PPD

DECEMBER 16, 2024

Cost and complexity go hand-in-hand The rising costs and growing complexity in clinical trials are deeply linked, with patient recruitment, extended timelines and meeting regulatory demands emerging as some of the key drivers. Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024.

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Drug Development Clinical Trials Compliance Trials 52

The Pharma Data

OCTOBER 28, 2021

Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.

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Clinical Trials Trials Disease Hospitals 52

DrugBank

MARCH 12, 2025

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. Once delivered into the body, the mRNA instructs cells to produce these antigens, which are then presented to the immune system.

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Vaccine Treatment Clinical Trials Therapies 97

Drug Hunter

JUNE 25, 2023

If the molecule reaches approval, MZE001 could be the first oral therapy for Pompe disease, either alone or in combination with enzyme-replacement therapy. According to a company presentation, the 480 mg BID dose covers the ~paEC50 (~650 ng/mL) for >24 h, with a half-life of 12 hours. Get ahead now by requesting a trial.

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Targeted Protein Degradation Computational Chemistry Trials Licensing 130

Drug Target Review

DECEMBER 23, 2024

While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. It also enables a more targeted approach to ADC optimisation, minimising the need for trial-and-error experimentation.

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Research Treatment Therapies Drug Development 52

DrugBank

MARCH 19, 2025

Unlike the traditional “ one-size-fits-all ” model, precision medicine leverages individual patient data, genetic profiles, environmental exposures, and lifestyle factors to design therapies tailored to each person’s unique needs. Precision medicine's benefits extend far beyond cancer care.

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Therapies Clinical Trials Pharmacokinetics Treatment 90

Broad Institute

NOVEMBER 25, 2024

As a proof of concept, a drug designed to target a newly discovered biological node is showing efficacy in treating rare genetic diseases in the kidney, the eye, and the brain and is now making its way to clinical trials in collaboration with a pharmaceutical partner. You can learn more about how clinical trials work here.

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Clinical Trials Disease Trials Therapies 52

Chemical Biology and Drug Design

MAY 11, 2023

Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects. Crocin, a naturally occurring carotenoid molecule, was found to have neuroprotective potential in the therapy of this disorder.

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Disease Research Clinical Trials Treatment 100

Drug Target Review

DECEMBER 16, 2024

In addition to this, I also chaired national trials evaluating an anti-GD2 antibody in pediatric osteosarcoma. Being a woman and an immigrant presented challenges in my professional growth along the way. Throughout her academic career, she worked in translational and clinical research, focusing on novel therapies for pediatric cancers.

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Medical Schools Clinical Research Drug Development Trials 52

DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Therapies DNA RNA Disease 52

Broad Institute

OCTOBER 11, 2023

His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.

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The Pharma Data

JUNE 6, 2022

(NYSE: PFE) today announced new data from a planned interim analysis of the Phase 2 MagnetisMM-3 registration-enabling trial of elranatamab in people with relapsed/refractory multiple myeloma (RRMM) whose disease is refractory to at least one agent in each of three major classes of medications approved for the disease. and 176,000 globally.

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Trials Treatment Therapies Disease 52

Drug Patent Watch

DECEMBER 9, 2024

This includes: Preclinical studies Clinical trials Regulatory approval Manufacturing and marketing Throughout this process, pharmaceutical companies seek to protect their investments through patents. It’s a delicate balance between incentivizing research and ensuring equitable access to medicines.”

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Drugs Drug Development Pharmaceuticals Production 97

PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

Therapies 69

Therapies Immune Response DNA Virus 69

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

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Production Clinical Trials Trials Therapies 141

PPD

SEPTEMBER 7, 2023

Approved treatments and therapies have turned what was once a certain death sentence into a manageable condition for those with the disease. These approved therapies have been a game changer in the treatment of HIV and the prevention of its spread. million new infections in 2022.

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Clinical Trials Trials Clinical Research Drug Development 52