Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Our team of chemists then uses this information to develop novel therapies.

Therapies 102

Therapies Disease RNA Engineering 102

Drug Target Review

AUGUST 31, 2023

In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system.

Therapies 105

Therapies Disease Treatment Research 105

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.

Therapies 137

Therapies FDA Approval Engineering Disease 137

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

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 116

Therapies Virus DNA Clinical Trials 116

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

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

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.

Treatment 100

Treatment Disease Virus Therapies 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

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.

Disease 122

Disease Immune Response Cell Biology Therapies 122

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.

Disease 97

Disease Treatment DNA Research 97

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.

Disease 98

Disease Therapies DNA Treatment 98

BioPharma Drive: Drug Pricing

JUNE 15, 2023

Lilly will pay the genetic medicine specialist $60 million upfront to work together on a treatment targeting a risk factor known as Lp(a).

Therapies 130

Therapies Disease Treatment 130

Drug Target Review

JANUARY 23, 2024

What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? Immuno-oncology, notably through checkpoint inhibitors, has significantly reshaped cancer treatment over the past decade.

Treatment 122

Treatment Immune Response Vaccine Therapies 122

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.

Therapies 104

Therapies Trials Treatment Disease 104

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.

Disease 100

Disease Treatment Therapies Research 100

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

Broad Institute

SEPTEMBER 6, 2023

Diabetes and cardiac fibrosis are two conditions in dire need of new therapies,” said Todd Golub, director of the Broad Institute. For both patient populations, there are no safe and effective therapies for reversing disease. The collaboration will focus on advancing three programs over the next three years.

Disease 136

Disease Research Therapies Treatment 136

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.

Therapies 105

Therapies Immune Response Engineering Drugs 105

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

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 102

Therapies Treatment Disease Research 102

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

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 RNA Engineering 98

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.

Therapies 95

Therapies Clinical Trials DNA Trials 95

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. Only three of the CAR-NK studies were for the treatment of solid tumours.

Therapies 118

Therapies Cell Biology Clinical Trials Trials 118

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.

Treatment 96

Treatment Therapies Disease Drugs 96

Drug Target Review

JULY 3, 2023

Dr David Baram from gene therapy company EmendoBio provides a snapshot of the history of therapeutic genetic engineering, explaining the early pitfalls and reasons for recent renewed optimism.

Therapies 98

Therapies Clinical Trials DNA Engineering 98

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

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? What is the ultimate impact of heterogeneity associated with these disorders and diseases? The lack of heterogeneity can introduce significant risk into mid- to late-stage studies.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

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.

FDA Approval 98

FDA Approval Therapies FDA Treatment 98

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

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

Therapies 98

Therapies Disease Clinical Development Hospitals 98

Drug Target Review

OCTOBER 5, 2023

This innovative approach involves the extraction of a patient’s own stem cells from either their bone marrow or blood , followed by a course of chemotherapy and antibody treatment. The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020.

Therapies 116

Therapies Treatment Clinical Trials Disease 116

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.

FDA 105

FDA Disease Clinical Pharmacology Science 105

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. In 2023, two gene-based treatments became available.

FDA Approval 93

FDA Approval Treatment FDA DNA 93

ASPET

DECEMBER 22, 2023

This study provides a unique translational research opportunity to help both humans and dogs diagnosed with diseases that carry dismal prognoses in both species: histiocytic sarcoma (HS), hemangiosarcoma (HSA), and disseminated mastocytosis/mast cell tumor (MCT). Standard-of-care therapeutics broadly synergize with PTL.

Clinical Trials 189

Clinical Trials Disease Drugs Therapies 189

Drugs.com

JULY 13, 2023

THURSDAY, July 13, 2023 -- A preclinical study offers a potential new therapy for treatment-resistant prostate cancer, offering new hope for men with the disease. The study used the chemotherapy drug cisplatin, administered orally, to disrupt the.

Treatment 98

Treatment Therapies Disease Drugs 98