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 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.,

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Therapies Cell Biology Clinical Trials Trials 118

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

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

AUGUST 22, 2023

This Q&A explores how pre-clinical research is being used to identify potential therapies for Huntington’s disease, a devastating condition that currently lacks disease- modifying treatments. What is the current landscape of treatment options for Huntington’s disease, and how does this potential therapy fit in?

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Therapies Disease RNA 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.

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Therapies Engineering Molecular Biology Science 105

Drug Target Review

AUGUST 31, 2023

Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system. She expressed enthusiasm for the prospect of leveraging hematopoietic stem cell therapy to devise an innovative therapeutic approach for this devastating disorder.

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Therapies Disease Research Treatment 105

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

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

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 DNA 111

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

Broad Institute

JULY 19, 2023

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

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Therapies Research Engineering Virus 98

BioPharma Drive: Drug Pricing

JUNE 17, 2024

One expert described trial results presented at EULAR last week as “unprecedented.” ” But reports of relapses in some patients drew questions about the therapies’ ultimate potential.

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Disease Therapies Trials 139

ASPET

OCTOBER 12, 2023

It is evident from the current literature that the SNs of peptide for cancer treatment hold great promise for future cancer therapy, offering potential strategies for personalized medicine with improved patient outcomes. Different SNs approaches and recent literature reviews on peptide delivery are also presented to the readers.

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Therapies Small Molecule Treatment Drugs 100

BioPharma Drive: Drug Pricing

NOVEMBER 20, 2023

The regulator plans to convene an advisory panel to discuss an expanded indication for Abecma, presenting another hurdle for Bristol Myers and partner 2seventy bio.

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Therapies FDA Regulations 111

BioPharma Drive: Drug Pricing

MAY 3, 2024

Newly disclosed Phase 3 results presented Friday match findings the biotech disclosed last year, while offering a more comprehensive look at how its drug stacks up to rival therapies.

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Drugs Therapies 125

Chemical Biology and Drug Design

NOVEMBER 23, 2023

Nowadays, gene therapy is being implemented for its treatment as several of these genetic defects have been identified. The present article describes the basics regarding the disease, its causes and factors responsible, and the gene therapy solutions available to treat this disease.

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Therapies DNA Regulations Disease 100

Chemical Biology and Drug Design

DECEMBER 15, 2023

Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders. Abstract Neurodegenerative disorders, which affect millions worldwide, are marked by a steady decline of neurons that are selectively susceptible.

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Disease Therapies Treatment Research 189

Science Daily: Pharmacology News

JULY 5, 2023

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.

Protein Production 75

Protein Production Production Therapies RNA 75

Drug Discovery Today

SEPTEMBER 3, 2020

Funding will be used to explore scalable methods of human cell preservation, transport, and re-presentation for clinical applications

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Therapies 113

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.

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Therapies Clinical Supply Clinical Trials Molecular Biology 72

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.

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Therapies Disease 70

Drug Target Review

JULY 3, 2023

In considering the present and future of potential therapeutics for genetic diseases, gene editing emerges as a critical player.

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Disease Therapies DNA Treatment 98

Chemical Biology and Drug Design

APRIL 1, 2023

The summarized information presented in this review article may help and lead the researchers, scientists/industry persons to use berberine as a promising candidate against cancer. In addition to these actions, Berberine plays a role in, the regulation of reactive oxygen species and inflammatory cytokines in preventing cancer formation.

Therapies 100

Therapies RNA Regulations Research 100

Fierce BioTech

JULY 28, 2023

jsabatino Fri, 07/28/2023 - 11:29 In this white paper, we look at the past, present and future of antibodies as therapies, explaining how the modality became a key pillar of the biopharma industry—and looking forward to the methods and technologies that could unleash its full potential in the years to come. Download here!

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Therapies Drug Development Research Drugs 52

SCIENMAG: Medicine & Health

JUNE 18, 2023

A three-year-long retrospective cohort study of a single Atlanta hospital’s patient population found transgender and gender-diverse teenagers rarely chose to discontinue gender-affirming hormone therapy, according to a study being presented Sunday at ENDO 2023, the Endocrine Society’s annual meeting in Chicago, Ill.

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Therapies Hospitals 62

DrugBaron

JULY 19, 2022

Despite the current hype around so called “advanced therapies”, which range from gene editing to cell therapies, and the inexorable advance of biologic therapeutics such as monoclonal antibodies, even in 2022 the majority of drugs in development and reaching patients are still small organic molecules.

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Small Molecule Drugs DNA Pharma Companies 100

Fierce BioTech

JULY 17, 2024

The cell and gene therapy industry is experiencing rapid growth and evolution, presenting both exciting opportunities and unique challenges for researchers and logistics providers alike. | How is the cold chain adapting to the cell and gene therapy boom?

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Therapies Treatment Research 52

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

MAY 22, 2024

How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? There are three major reasons for why Circio has selected AATD as its lead gene therapy program. Firstly, there have been previous AAV gene therapy clinical studies in this indication that have shown promise.

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Therapies RNA Protein Expression DNA 59

Fierce BioTech

MAY 29, 2024

New Solutions for Aseptic Filling for Gene Therapy In the development of gene therapies, it is more important than ever to stay on top of the latest containment and aseptic filling solutions.

Therapies 52

Therapies Pharmaceuticals 52

Chemical Biology and Drug Design

APRIL 19, 2023

The present work identifies and explains compounds with considerable Acetylcholinesterase (AChE) inhibitory activities. This can be achieved by cholinergic neurotransmission through inhibition of the cholinesterase enzyme. The current research aims to find natural substances that can be used as drugs to treat AD.

Disease 100

Disease Therapies Research Drugs 100

BioPharma Drive: Drug Pricing

SEPTEMBER 16, 2024

Applying CAR T-cell therapy in new clinical settings presents unique challenges. Worldwide Clinical Trials is at the forefront, partnering with drug development teams to map this new path forward.

Therapies 52

Therapies Trials Clinical Trials Drug Development 52

Chemical Biology and Drug Design

FEBRUARY 2, 2024

Their use in combination therapy decreases the concentration of the reference drug used. This aim of this mini-review is to explore the most promising natural terpenes with anti-leishmanicidal, anti-schistosomicidal, and anti-echinococcal activity, their mechanisms of action, and their efficacy as combination therapy.

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Treatment Disease Immune Response Therapies 100

SCIENMAG: Medicine & Health

AUGUST 4, 2023

A multidisciplinary study led by scientists at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) presents a new method for assessing the structural and electrophysiological changes, called atrial remodeling, produced in the heart of patients with atrial fibrillation, one of the most frequent forms of cardiac arrhythmia.

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Therapies 96

Alta Sciences

JUNE 11, 2024

My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0

Therapies 52

Therapies Molecular Biology Immune Response Science 52

LifeSciVC

OCTOBER 10, 2024

This presentation marked the most significant clinical disclosure of any CBL-B inhibitor to date, an important moment not only for industry, but for us at HotSpot. That said, two presentations at ESMO in non-small cell lung cancer (NSCLC) were perhaps “green shoots” for the field. appeared first on LifeSciVC.

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Therapies Clinical Development Pharmacokinetics Treatment 63