Chemical Biology and Drug Design

DECEMBER 15, 2023

The figure depicts the neuroprotective role of exendin-4 in Alzheimer's disease and Parkinson's disease. Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders.

Disease 189

Disease Therapies Treatment Research 189

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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Treatment Disease Immune Response Therapies 100

thought leadership

MAY 20, 2024

The Challenge: CAGT Treatments Require Time and Lack Availability Persons diagnosed with life threatening diseases which are now treatable with cell and gene therapies (CAGT) often needed to wait for a significant amount of time for treatment due to lack of availability.

Therapies 52

Therapies Treatment Disease Compliance 52

Chemical Biology and Drug Design

OCTOBER 6, 2024

Alzheimer's disease (AD) is a progressive age-related neurodegenerative disorder characterized by loss of memory and other cognitive functions. The problem of this disease is even more fundamental because the available therapies only provide symptomatic relief without addressing the basic factor of the disease.

Disease 100

Disease Treatment Therapies 100

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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Therapies Treatment Vaccine FDA Approval 64

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

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

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.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

Chemical Biology and Drug Design

APRIL 18, 2023

The severity of chagas disease remains a major concern in endemic areas and an emerging public health hazard in nonendemic countries. Abstract Twenty different infectious disorders induced by bacteria, viruses, and parasites are categorized as neglected tropical diseases (NTDs) by WHO.

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Treatment Disease Virus Therapies 100

SCIENMAG: Medicine & Health

JULY 26, 2023

FOR IMMEDIATE RELEASE ON WEDNESDAY, JULY 26, 2023 AAN Issues Guidance on New Treatments for Early Alzheimer’s Disease MINNEAPOLIS – New therapies for early Alzheimer’s disease, monoclonal antibodies that remove amyloid-β plaques in the brain, are bringing hope to people whose lives have been affected by the disease.

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Disease Treatment Therapies 68

SCIENMAG: Medicine & Health

JULY 13, 2023

New study suggests combination therapy to prevent resistance to treatment in hormone responsive metastatic breast cancer patients Credit: Gonçalo Ribeiro, iMM New study suggests combination therapy to prevent resistance to treatment in hormone responsive metastatic breast cancer patients Breast cancer is a heterogeneous disease, but 70% of cases (..)

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Therapies Treatment Disease 82

Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study.

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Therapies FDA Approval Engineering Disease 137

Science Daily: Pharmacology News

JULY 17, 2024

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies 121

Therapies Treatment Disease Research 121

Chemical Biology and Drug Design

FEBRUARY 13, 2024

Alzheimer's disease (AD) is a common age-related neurodegenerative brain disorder characterized by the impairment in memory and other cognitive functions. Abstract Alzheimer's disease (AD) is a common age-related neurodegenerative brain disorder characterized by the impairment in memory and other cognitive functions.

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Disease Treatment Therapies Research 100

Drugs.com

JUNE 18, 2024

TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.

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Therapies Trials Treatment Disease 105

Fierce BioTech

MAY 13, 2024

In this panel discussion, rare disease experts discuss the significant challenges with achieving a timely and accurate rare disease diagnosis and strategies to engage both patients and providers to expand understanding of and access to therapies that benefit patients. Click here to login.

Therapies 52

Therapies Disease Clinical Trials Trials 52

PLOS: DNA Science

FEBRUARY 1, 2024

Five people treated for pituitary dwarfism decades ago with human growth hormone (hGH) pooled from cadavers have shown cognitive decline reminiscent of early-onset Alzheimer’s disease. None had mutations that cause Alzheimer’s disease, ruling out genetics as a cause. More than 85 animal species develop prion diseases.

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Disease Treatment DNA Research 98

Drug Target Review

FEBRUARY 9, 2024

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? These characteristics position ADSCs as promising candidates for addressing a wide range of medical conditions, from degenerative diseases to traumatic injuries.

Therapies 105

Therapies Immune Response Clinical Trials Treatment 105

Drug Target Review

JULY 3, 2023

There is a significant unmet need for effective treatments to alleviate suffering and prevent premature death across the spectrum of genetic diseases. Unfortunately, the majority of genetic diseases lack a cure, leaving limited therapeutic options available.

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Disease Therapies DNA Treatment 98

FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. 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.

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Clinical Development Production Clinical Trials Treatment 69

Chemical Biology and Drug Design

AUGUST 15, 2024

The findings reveal the active constituents, key targets, and molecular mechanisms of myrrh in BC treatment, providing scientific evidence that supports the role of myrrh in BC therapy. ABSTRACT This study aimed to investigate the mechanism of action of myrrh in breast cancer (BC) treatment and identify its effective constituents.

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Treatment Therapies 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

Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

FDA Approval 124

FDA Approval Therapies FDA Treatment 124

Conversations in Drug Development Trends

AUGUST 14, 2024

By: Simran Padam, Medical Director, Medical Affairs Personalized treatment approaches have emerged as pivotal in improving outcomes for hematological cancers. Heterogeneity in Hematological Cancers Due to their disease heterogeneity, hematological cancers are well-suited for personalized approaches.

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Treatment Therapies Disease Protein Expression 78

ASPET

SEPTEMBER 16, 2024

Glioblastoma (GBM) is a disease of the whole brain, with infiltrative tumor cells protected by an intact BBB. GBM has a poor prognosis despite aggressive treatment, in part due to lack of adequate drug permeability at the BBB. Standard of care GBM therapies include radiation and cytotoxic chemotherapy that lead to DNA damage.

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Pharmacogenetics DNA Pharmacokinetics Treatment 100

Drugs.com

JUNE 14, 2024

FRIDAY, June 14, 2024 -- Animal studies are often considered a first step in finding new drugs and treatments for human diseases, but a new review has discovered that precious few actually produce real-world therapies. Only 5% of therapies tested.

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Treatment Therapies Disease Drugs 98

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.

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Therapies Virus DNA Clinical Trials 115

PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.

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Therapies Clinical Trials DNA Trials 98

Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing.

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Disease Clinical Trials Therapies Science 59

Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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Disease Clinical Trials Therapies Clinical Research 52

Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.

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FDA Approval Therapies FDA Treatment 98

Drug Target Review

OCTOBER 16, 2023

As we explore translatability, technological advancements and the integration of artificial intelligence (AI), we envision the potential role of PDOs throughout the entire medicine creation pipeline, from target identification to personalised clinical treatments, ultimately reducing costs and improving patient outcomes.

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Disease Immune Response Cell Biology Therapies 122

Drug Target Review

JUNE 27, 2023

This ebook delves into the intricate world of cancer therapies, immune system breakthroughs, therapeutic genetic engineering, and groundbreaking advancements in gene editing, featuring insightful interviews with renowned experts in the field.

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Therapies Immune Response Engineering Drugs 105

Drug Target Review

JANUARY 15, 2024

Cancer is a disease driven by variable genetic mutations. Could you provide insights into the driving factors behind exploring novel treatments for cancer types? Could you provide further details on how this phenomenon could impact broader treatment outcomes? What aspects prompted this research direction?

Treatment 105

Treatment Immune Response Therapies Clinical Trials 105

Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. So, what makes these therapies unique?

Therapies 98

Therapies Small Molecule Production Disease 98

Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

Treatment 105

Treatment Packaging Clinical Research Therapies 105

BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

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Therapies Disease Trials Treatment 74