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.

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Chemical Biology and Drug Design

MARCH 13, 2025

These findings position PTB derivatives as promising candidates for targeted breast cancer therapy. ABSTRACT This study presents the design, synthesis, and biological evaluation of a series of novel pyrrole-tethered bisbenzoxazole (PTB) derivatives as potential apoptosis-inducing agents targeting the MCF-7 human breast cancer cell line.

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Therapies Research 100

SugarCone Biotech

MARCH 2, 2025

Where TGF- is present there is no or limited IFN- secretion by T cells and that means no PD-L1 expression within the tumor microenvironment, aka the TME. VEGF is a secreted protein present in solution, but is also bound to VEGF receptor R2-positive endothelial cells within the TME.

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BioPharma Drive: Drug Pricing

DECEMBER 10, 2023

While new gene therapies for the blood disease grabbed headlines at ASH, Pfizer presented fresh data for an oral drug meant to build on its marketed therapy Oxbryta.

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Therapies Drugs Disease Marketing 190

Covalent Modifiers

MARCH 16, 2025

Here, we demonstrate the utility of a high-throughput in vitro screening platform along with a comprehensive panel to aid in the characterization of 15 Brutons tyrosine kinase (BTK) inhibitors that are either approved by the FDA or presently under clinical evaluation.

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FDA Therapies 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.

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Therapies Engineering Treatment Molecular Biology 116

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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Therapies Clinical Trials Clinical Research Trials 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.

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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 Clinical Trials Cell Biology Trials 118

ASPET

APRIL 19, 2024

Despite the severe, unmet, medical need; there is no literature regarding on-demand, rapid-onset, short-duration, drug-induced, voiding therapies. This article provides in depth discussion of recent discovery and development of two candidates for on-demand voiding therapies.

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Therapies Hospitals Disease Drugs 100

SCIENMAG: Medicine & Health

DECEMBER 22, 2023

Building on the momentum of the 6th World Congress on Targeting Phage Therapy, that gathered more than 150 attendees from over 30 countries and featured over 71 presentations, the highly anticipated Targeting Phage Therapy 2024 is set to unfold.

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Therapies 84

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 Treatment Research 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

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

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 69

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

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

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

Covalent Modifiers

DECEMBER 6, 2023

The conventional approach relies on covalent ligands, but our study presents an innovative method employing an N -sulfonyl pyridone warhead to selectively target tyrosine (Tyr) residues. The von Hippel–Lindau (VHL) moiety is transferred from the warhead to the exposed Tyr, allowing us to design a STING degrader (DC 50 0.53 μM, D max 56.65%).

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Targeted Protein Degradation Therapies 182

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 105

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

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

Drugs.com

JULY 13, 2023

WEDNESDAY, July 12, 2023 -- The centuries-old practice of mindfulness is having a moment in present times, and a new study finds the therapy can improve mental health for at least six months. Analyzing the results of 13 prior studies, U.K.

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Therapies 130

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

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 74

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

FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

While treating rare diseases presents significant challenges due to small patient populations and limited financial incentives, the Orphan Products Grants Program provides crucial financial support to help bridge the gap between early research and successful treatment development. Relative to other areas of medicine (e.g.,

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Clinical Development Production Clinical Trials Treatment 69

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

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.

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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.

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Disease Therapies Research Drugs 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

Chemical Biology and Drug Design

APRIL 26, 2023

The administration of synthetic polymers that present autoantigens in a multivalent manner have been established as promising therapeutic strategies in other autoimmune diseases and may be applied to IgAN.

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Therapies Disease Treatment Research 100

Alta Sciences

NOVEMBER 20, 2024

Now Available Online — Scientific Posters Presented at Eurotox 2024! EXPLORE Related resources you may be interested in: eBook: Nonclinical Studies in Cell and Gene Therapy Webinar: Best Practices to Reduce Animal Use in Toxicology Studies Webpage: The Use of Miniature Swine in Preclinical Studies Image alta-blog_4_2.jpg

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Therapies Drug Development Research Drugs 40