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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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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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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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.

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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.

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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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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.

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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.

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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.

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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?

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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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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? By encapsulating specific self-antigens within our biodegradable nanoparticles, we enable the targeted delivery of the CNPs to antigen-presenting cells (APCs) primarily in the spleen and liver.

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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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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.

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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.

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The Pharma Data

NOVEMBER 9, 2021

– More than 30 Abstracts Across HDV, HCV, HBV, NASH and PSC Reflect Gilead’s Ongoing Commitment to Addressing Challenges Facing People Living With Liver Diseases –. About Gilead Sciences in Liver Disease. For more than 20 years, Gilead has sought to address some of the biggest challenges in liver disease. Gilead Lores,Inc.

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Fierce BioTech

SEPTEMBER 11, 2023

Aligning cross-functional teams across the organization to ensure medical, marketing, and market access are all in lock-step is even more crucial when launching a therapy into a rare disease market. Click here to login. Listing Image HerspiegelConsulting_LogoListingNew_10262023.png On Demand Start Date Thu, 10/26/2023 - 11:00

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Elrig

APRIL 11, 2024

One of the great highlights of a:head’s work has been the ability to observe disease-specific phenotypes and profile clinically used therapeutics for their ability to alter neuronal network activity. The post How to develop drugs for neurological diseases | Insights from Josh Bagley first appeared on ELRIG.

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Drug Target Review

NOVEMBER 7, 2023

To target proteins involved in causing or spreading disease, typically a drug will obstruct a protein’s active site so it cannot function. Proteins can become part of a cancerous tumour and allow for its unrestricted growth, cause diseases like Alzheimer’s , and build up in the heart to impact how it pumps blood around the body.

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The Pharma Data

MAY 22, 2023

DUPIXENT® (DUPILUMAB) LATE-BREAKING PHASE 3 COPD RESULTS PRESENTED AT ATS AND SIMULTANEOUSLY PUBLISHED IN THE NEW ENGLAND JOURNAL OF MEDICINE Regeneron Pharmaceuticals, Inc. The Dupixent Phase 3 results were presented in the 2023 American Thoracic Society (ATS) International Conference session “New England Journal of Medicine and JAMA.

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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America. SATURDAY, Dec.

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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.

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The Pharma Data

MAY 26, 2023

Pfizer Presents Scientific Advancements from its Leading Oncology Portfolio at ASCO 2023 Annual Meeting will present data across its Oncology portfolio and growing pipeline, covering multiple tumor types and novel mechanisms of action at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago from June 2 through June 6.

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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.

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Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

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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.

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The Pharma Data

OCTOBER 14, 2021

The results, which demonstrate that patients treated with Biogen’s portfolio of MS therapies mount an effective antibody response to COVID-19 vaccination, are being presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting, October 13-15, 2021.

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The Pharma Data

JUNE 28, 2021

Food and Drug Administration (FDA) granted Breakthrough Therapy designation for donanemab, Eli Lilly and Company’s (NYSE: LLY) investigational antibody therapy for Alzheimer’s disease (AD). Dementia due to Alzheimer’s disease is the most common form of dementia, accounting for 60 to 80 percent of all cases 1.

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PPD

JANUARY 5, 2023

In an industry survey led by PPD, Thermo Fisher Scientific’s clinical research business, rare diseases emerged as a top therapeutic area in drug developers’ pipelines, with 39% of global biotech and biopharma leaders pursuing therapies in the broad rare disease space.

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The Pharma Data

SEPTEMBER 6, 2021

First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Breast Cancer Highlights. Lung Cancer Highlights.

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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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The Pharma Data

FEBRUARY 24, 2022

Designation is supported by Phase II efficacy and safety data that will be presented at ATS 2022 Boehringer Ingelheim plans to study this novel investigational therapy in patients with progressive fibrosing interstitial lung diseases. The FDA Breakthrough Therapy designation is supported by data collected to date.

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The Pharma Data

MARCH 5, 2021

Biogen (Nasdaq: BIIB) today announced it will host webcasts of its pre-recorded presentations and live discussions related to its Alzheimer’s disease investigational therapy, aducanumab, at the upcoming AD/PDTM 2021 Virtual Conference. An archived version of the webinar will be available following the presentation.

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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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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.

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The Pharma Data

OCTOBER 29, 2020

The first presentation of new data characterizing the anti-ChemR23 antibody was at the FOCIS Virtual Annual Meeting held October 28-31, 2020. The data presented show that OSE-230 is the first monoclonal antibody triggering the activation of specialized receptors of resolution to restor e tissue homeostasis, integrity and function s.

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