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

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.

Therapies 131

Therapies Disease 131

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

Broad Institute

APRIL 2, 2024

Scientists link certain gut bacteria to lower heart disease risk By Allessandra DiCorato April 2, 2024 Breadcrumb Home Scientists link certain gut bacteria to lower heart disease risk Study finds several species of cholesterol-metabolizing bacteria in people with lower cholesterol levels. Online April 2, 2024. DOI:10.1016/j.cell.2024.03.014.

Disease 145

Disease Medical Schools Hospitals DNA 145

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.

Therapies 110

Therapies Disease 110

BioPharma Drive: Drug Pricing

DECEMBER 17, 2024

Phase 2 results in inflammatory bowel disease hinted the therapy could be more potent than similar medicines from Merck and Roche, each of which were acquired in multibillion-dollar deals.

Disease 111

Disease Therapies Drugs 111

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

OCTOBER 16, 2023

How can PDOs revolutionise drug discovery and deepen our understanding of disease? Patient-derived organoids (PDOs) are proliferative 3D cell structures derived from tissue samples of both healthy and diseased tissue. Improved translatability to the patient and the disease state.

Disease 122

Disease Immune Response Cell Biology Therapies 122

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

BioPharma Drive: Drug Pricing

OCTOBER 10, 2023

The pharma plans for the new Berkeley facility to produce clinical, and potentially commercial, supplies of a Parkinson’s disease cell therapy being tested by its subsidiary BlueRock.

Therapies 119

Therapies Disease 119

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

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 110

Therapies Treatment Disease Research 110

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.

Therapies 113

Therapies Engineering Disease Research 113

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.

Therapies 101

Therapies Disease Research 101

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,

Therapies 101

Therapies Disease 101

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

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

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

Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

Disease 113

Disease Treatment Small Molecule Therapies 113

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.

Therapies 78

Therapies Disease Research 78

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.

Therapies 100

Therapies Hospitals Disease Drugs 100

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

BioPharma Drive: Drug Pricing

SEPTEMBER 18, 2023

Three years after gaining European approval, Libmeldy is now under U.S. review with a deadline set for March.

Therapies 119

Therapies Disease FDA 119

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 102

Therapies Trials Treatment Disease 102

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

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.

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. developing immune stem cell therapies for treating ovarian and gastric cancer.

Therapies 118

Therapies Cell Biology Clinical Trials Trials 118

BioPharma Drive: Drug Pricing

JUNE 17, 2024

” But reports of relapses in some patients drew questions about the therapies’ ultimate potential. One expert described trial results presented at EULAR last week as “unprecedented.”

Disease 139

Disease Therapies Trials 139

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.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

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

Broad Institute

FEBRUARY 7, 2024

However, researchers lack efficient ways to explore how these genetic variants are molecularly connected to cardiovascular disease, limiting efforts to develop therapeutics. The team found that a key biological mechanism involved in a rare vascular disease may influence CAD risk.

Disease 105

Disease Hospitals Medical Schools Therapies 105

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.

Therapies 100

Therapies FDA Clinical Trials Disease 100

Broad Institute

JANUARY 25, 2024

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

Disease 124

Disease Research Science Medical Schools 124

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.

Disease 100

Disease Therapies Research 100

Drug Target Review

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates.

Therapies 86

Therapies Treatment Clinical Trials Disease 86

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

Fierce BioTech

SEPTEMBER 5, 2024

How can they engage providers and raise awareness of gene therapies for the patients who need them most? How can life sciences organizations effectively identify and recruit participants with specific genetic variants? How can life sciences organizations effectively identify and recruit participants with specific genetic variants?

Trials 87

Trials Disease Clinical Trials Therapies 87