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.

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Chemical Biology and Drug Design

NOVEMBER 4, 2024

Alzheimer's disease (AD) is a progressive age-related neurodegenerative brain disorder that destroys memory and other important mental functions. ABSTRACT Alzheimer's disease (AD) is a progressive chronic age-related neurodegenerative brain disorder characterized by the loss of memory and other cognitive functions.

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Drugs.com

JUNE 12, 2023

MONDAY, June 12, 2023 -- A clinical trial that’s attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients. Two of the patients were treated at Cleveland Clinic Children’s in.

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SCIENMAG: Medicine & Health

DECEMBER 6, 2023

Light therapy leads to significant improvements in sleep and psycho-behavioral symptoms for patients with Alzheimer’s disease, according to a new study published this week in the open-access journal PLOS ONE by Qinghui Meng of Weifang Medical University, China, and colleagues.

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Fierce BioTech

SEPTEMBER 24, 2024

Unlocking European market access for rare disease therapies The European orphan drug market presents a significant opportunity for biotech firms. Learn more about early access programs (EAPs), and how they are emerging as a critical strategy for successfully launching rare disease therapies in this complex and diverse market.

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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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Drugs.com

OCTOBER 10, 2024

10, 2024 -- Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new reports find.The 77 boys were treated for cerebral adrenoleukodystrophy (CALD), a formerly. THURSDAY, Oct.10,

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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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BioPharma Drive: Drug Pricing

JULY 1, 2024

Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy in hopes of riding a recent wave of investor interest in autoimmune disease research.

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BioPharma Drive: Drug Pricing

OCTOBER 10, 2024

Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.

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Fierce BioTech

MAY 14, 2024

Strategies for Expanding Access to Rare Disease Therapies Join us as we explore strategic approaches employed by industry leaders, who are bridging the diagnostic gap to improve patient outcomes and accelerate the development of precision therapies.

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SCIENMAG: Medicine & Health

AUGUST 28, 2023

military veterans with coronary artery disease, suboptimal lipid-lowering therapy was prevalent in the clinical setting. Optimization of statin therapy was projected to produce clinically relevant reductions in the risks of death and cardiovascular events. About The Study: In this study of 111,000 U.S.

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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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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.

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Drugs.com

NOVEMBER 2, 2023

1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. WEDNESDAY, Nov. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December. The FDA had already decided that the.

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BioPharma Drive: Drug Pricing

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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BioPharma Drive: Drug Pricing

JULY 18, 2024

The offering comes days after the publication of a paper showing what analysts said was early proof that “off-the-shelf” cell therapies can treat inflammatory diseases.

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Science Daily: Pharmacology News

AUGUST 29, 2024

For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. But a major hurdle has been finding a safe and efficient way to deliver those genes.

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SCIENMAG: Medicine & Health

OCTOBER 2, 2023

Researchers at the University of Cologne’s CECAD Cluster of Excellence for Aging Research and the CEPLAS Cluster of Excellence for Plant Sciences have found a promising synthetic plant biology approach for the development of a therapy to treat human neurodegenerative diseases, especially Huntington’s disease.

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Drugs.com

JULY 16, 2024

TUESDAY, July 16, 2024 -- Hormone therapy for breast cancer might reduce a woman’s later risk of dementia and Alzheimer’s disease, a new study finds.Overall, hormone therapy is associated with a 7% lower risk of developing Alzheimer’s or a related d.

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BioPharma Drive: Drug Pricing

JANUARY 30, 2024

Since a landmark paper in 2022, drugmakers have begun nearly a dozen trials of cell therapies for lupus, with more set to start. Here’s why their efforts are worth watching.

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Science Daily: Pharmacology News

AUGUST 9, 2023

Promising preclinical results show hematopoietic stem cell therapy was effective in rescuing memory loss, neuroinflammation and beta amyloid build-up in a mouse model of Alzheimer's disease.

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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.

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ASPET

APRIL 19, 2024

People with neurological injury (spinal cord injury, traumatic brain injury, stroke) or disease (multiple sclerosis, Parkinson's disease, spina bifida) and many elderly are unable to voluntarily initiate voiding. One cannot survive without regularly urinating and defecating.

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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.

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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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BioPharma Drive: Drug Pricing

JUNE 8, 2023

Two-year-old Kate Therapeutics is launching publicly with $51 million in funding and a licensing deal with Astellas Pharma for a neuromuscular disease gene therapy.

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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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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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Science Daily: Pharmacology News

MAY 16, 2024

In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

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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.

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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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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). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.

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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?

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Science Daily: Pharmacology News

AUGUST 30, 2023

Scientists have made a `paradigm shifting' discovery on the mechanisms required for learning and memory that could lead to new therapies for Alzheimer's disease and potentially Down syndrome.

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Science Daily: Pharmacology News

JULY 12, 2023

They also found that co-transplantation of neuronal cell therapy with host regulatory T cells resulted in effective suppression of needle trauma and significant improvement in the survival and recovery of grafts. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.

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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. We believe we are at a breakthrough moment in Alzheimer’s disease research.

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