Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. alone, according to the Centers for Disease Control and Prevention.

Therapies 117

Therapies DNA Virus Clinical Trials 117

ASPET

APRIL 19, 2024

People with neurological injury (spinal cord injury, traumatic brain injury, stroke) or disease (multiple sclerosis, Parkinson's disease, spina bifida) and many elderly are unable to voluntarily initiate voiding. One cannot survive without regularly urinating and defecating.

Therapies 100

Therapies Hospitals Disease Drugs 100

Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

Therapies 116

Therapies Engineering Treatment Molecular Biology 116

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

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

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.

Therapies 111

Therapies FDA Clinical Trials DNA 111

Chemical Biology and Drug Design

FEBRUARY 2, 2024

Their use in combination therapy decreases the concentration of the reference drug used. Abstract Existing chemotherapy for neglected tropical diseases (NTDs) can often be toxic and ineffective, highlighting the necessity for new treatments. Terpenes are secondary metabolites with pharmacological potential.

Treatment 100

Treatment Disease Immune Response Therapies 100

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

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

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. He is presently the founding CEO/President of the start-up company Cartherics Pty Ltd.,

Therapies 118

Therapies Cell Biology Clinical Trials Trials 118

Chemical Biology and Drug Design

MAY 11, 2023

The figure depicts the neuroprotective role of crocin in Parkinson's disease. 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.

Disease 100

Disease Research Clinical Trials Treatment 100

Chemical Biology and Drug Design

APRIL 19, 2023

Abstract Alzheimer's disease (AD) is a progressive neurodegenerative disorder caused due to the damage and loss of neurons in specific brain regions. The present work identifies and explains compounds with considerable Acetylcholinesterase (AChE) inhibitory activities. It is the most common form of dementia observed in older people.

Disease 100

Disease Therapies Research Drugs 100

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

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

ASPET

JANUARY 23, 2024

Patients with sickle cell disease (SCD) display priapism, a prolonged penile erection in the absence of sexual arousal. Thus, there is an urgent need for new drug targets and preventive pharmacological therapies for this condition.

Disease 100

Disease Therapies Treatment Research 100

Drug Target Review

JULY 7, 2023

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

Disease 98

Disease Immune Response Therapies Treatment 98

Chemical Biology and Drug Design

NOVEMBER 23, 2023

The symptoms of the disease include delusions and hallucinations, associated with extremely disordered behavior and thinking, which may affect the daily lives of the patients. Advancements in technology have led to understanding the dynamics of the disease and the identification of the underlying causes.

Therapies 100

Therapies DNA Regulations Disease 100

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

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111

Disease Clinical Trials Therapies Science 111

SCIENMAG: Medicine & Health

JUNE 22, 2023

(June 22, 2023) – An article published today in the journal Lancet Neurology evaluates the risk of recurrence of active disease in older patients with multiple sclerosis after discontinuing disease-modifying therapies. Multiple sclerosis (MS) is a chronic illness, often presenting in young adulthood.

Therapies 70

Therapies Disease 70

Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.

Therapies 72

Therapies Clinical Supply Clinical Trials Molecular Biology 72

Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

Science 145

Science Disease FDA Approval Clinical Trials 145

Drug Target Review

JANUARY 2, 2024

Through a series of experiments, they successfully developed a personalised stem cell therapy using a data-driven, single-cell technique based on swift subcellular proteomic imaging. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.

RNA 128

RNA Immune Response Therapies Disease 128

Drug Target Review

JUNE 15, 2023

Interested in a more disease-focused training for his postdoc, he pursued work in the National Institutes of Health (NIH), particularly in the National Center for Advancing Translational Sciences (NCATS), US. The core of their platform aims to modulate disease-relevant mRNAs that have been previously thought to be undruggable.t.

RNA 96

RNA Small Molecule Disease Assay Development 96

Chemical Biology and Drug Design

APRIL 26, 2023

Figure created with BioRender.com Abstract IgA nephropathy (IgAN) is the most common glomerular autoimmune disease and has severe long-term consequences for patients, with 40% of the patients eventually progressing to end-stage renal disease. Despite the severity, no causal treatment is currently available.

Therapies 100

Therapies Disease Treatment Research 100

Fierce BioTech

SEPTEMBER 24, 2024

Unlocking European market access for rare disease therapies The European orphan drug market presents a significant opportunity for biotech firms. Learn more about early access programs (EAPs), and how they are emerging as a critical strategy for successfully launching rare disease therapies in this complex and diverse market.

Marketing 40

Marketing Therapies Disease Drugs 40

Drug Target Review

FEBRUARY 29, 2024

During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. AskBio) I continue to work to bring innovative gene therapies to patients in need.

Therapies 111

Therapies Disease Clinical Trials Science 111

Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

Disease 59

Disease Clinical Trials Clinical Pharmacology Trials 59

Drug Hunter

JUNE 25, 2023

Maze’s Oral GYS1 Inhibitor, MZE001 Sanofi is paying $150 million plus up to $600 million in milestones for Maze Therapeutics’ glycogen synthase 1 (GYS1) program, including MZE001, which aims to reduce glycogen substrate build-up in Pompe disease.

Targeted Protein Degradation 130

Targeted Protein Degradation Computational Chemistry Licensing Licensing 130

Drug Target Review

AUGUST 6, 2024

A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.

Therapies 117

Therapies Pharmaceutical Companies Disease Treatment 117

Drug Target Review

JANUARY 15, 2024

An important limitation of antibodies against tumour antigens is that these agents direct responses to molecular targets present on the surface of the cell. Figure 1: HLA Class I presentation of peptides derived from intracellular antigens. As described below, it also presents challenges and opportunities for TCR-based therapeutics.

Engineering 110

Engineering DNA Virus Immune Response 110

biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies.

Disease 59

Disease Medical Schools Pharma Companies Clinical Trials 59

Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? 1104 acts upstream in the inflammatory response, targeting one of the first key players: antigen-presenting cells (APCs). In allergic diseases, APCs identify and present unknown antigens to the immune system.

Disease 64

Disease Immune Response Clinical Trials Treatment 64

Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. These newly formed mutated proteins are called neoantigens and they are only present on tumour cells and are not found in normal, healthy cells. This allows the cancer to evolve and develop resistance to the therapy.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105

Drug Target Review

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 5 This has created a significant unmet need for new treatments that either prevent resistance or prolong the effectiveness of initial therapies while maintaining a tolerable safety profile.

Therapies 52

Therapies Treatment Clinical Trials Drugs 52

Drug Target Review

MAY 22, 2024

How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? As a first step, Circio is working on designing and validating circVec-AAV vectors for the treatment of genetic diseases, and testing these head-to-head against the mRNA-AAV equivalent in vivo.

Therapies 59

Therapies RNA Protein Expression DNA 59

Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

Clinical Research 72

Clinical Research Disease Research Clinical Trials 72