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

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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Disease Therapies Trials 139

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

FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Therapies Trials FDA Clinical Trials 98

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

Quanticate

SEPTEMBER 25, 2024

In rheumatoid arthritis (RA) clinical trials, accurately measuring the effectiveness of treatments is critical for determining their value in managing this chronic and debilitating condition.

Clinical Trials 97

Clinical Trials Trials Therapies Treatment 97

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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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. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.

Therapies 118

Therapies Cell Biology Clinical Trials Trials 118

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

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 98

Therapies Treatment Clinical Trials Drugs 98

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.

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FDA Disease Clinical Pharmacology Science 105

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

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.

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Therapies FDA Clinical Trials Disease 100

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

BioPharma Drive: Drug Pricing

JUNE 22, 2023

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

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Therapies FDA Trials Disease 132

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

Advarra

MARCH 10, 2023

Having a one in four chance of developing sickle cell disease (SCD) is uniquely, and overwhelmingly, familiar to the African American population. Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations.

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Disease Clinical Trials Trials Doctors 98

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).

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Clinical Trials Disease Drugs Therapies 189

PPD

JUNE 7, 2023

Adults aren’t the only patients affected by the increasing prevalence of liver disease. The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations.

Trials 96

Trials Clinical Trials Clinical Research Disease 96

BioPharma Drive: Drug Pricing

DECEMBER 9, 2023

Study results disclosed at the American Society of Hematology meeting Saturday suggested potential for cell therapy in lupus and pointed out drug trial limitations.

Drug Trials 130

Drug Trials Disease Therapies Trials 130

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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Therapies Clinical Trials Clinical Research Trials 88

PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success. Historically, the available drugs and U.S. None offers a cure for PAH.

Clinical Trials 94

Clinical Trials Trials Clinical Research Disease 94

BioPharma Drive: Drug Pricing

DECEMBER 11, 2023

Editas had the tall task Monday of convincing ASH attendees its gene therapy for sickle cell disease could improve on Casgevy and Lyfgenia.

Clinical Trials 122

Clinical Trials Trials Therapies Disease 122

Drug Target Review

OCTOBER 5, 2023

This study sought to shed light on the safety and effectiveness of aHSCT in routine healthcare settings, moving beyond the confines of clinical trials. Around three years following their aHSCT, 20 patients (11 percent) were administered disease-modifying drugs.

Therapies 116

Therapies Treatment Clinical Trials Disease 116

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. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.

Therapies 103

Therapies Clinical Trials Immune Response Trials 103

BioPharma Drive: Drug Pricing

JUNE 27, 2023

A Duchenne gene therapy faces a crucial test, while highly anticipated study results are expected in lung cancer, obesity and heart disease.

Clinical Trials 119

Clinical Trials Trials Therapies Disease 119

Drug Target Review

DECEMBER 20, 2024

However, recent breakthroughs in AI, such as predictive modelling, clinical trial optimisation, and personalised medicine, have demonstrated its potential. We spoke with Aaron Smith, a mathematician-turned-machine learning scientist and the founder of Unlearn , a company leading the charge in applying AI to optimise clinical trial efficiency.

Clinical Trials 117

Clinical Trials Trials Pharmaceuticals Drug Development 117

Fierce BioTech

NOVEMBER 21, 2023

Cell and Gene Therapy (CGT) has the potential to transform the life trajectory of children with genetic conditions amendable to gene modification with advances in treatment and possible cures. Pediatric CGT trials have the potential to cure diseases and prolong lives.

Therapies 91

Therapies Research Trials Treatment 91

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.

Disease 100

Disease Research Clinical Trials Treatment 100

Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

Therapies 105

Therapies Research Small Molecule Clinical Trials 105

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.

Therapies 105

Therapies Engineering Production Clinical Trials 105

Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

Therapies 103

Therapies DNA Engineering Virus 103

Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111

Disease Clinical Trials Therapies Science 111

Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

Therapies 77

Therapies Clinical Trials Treatment Disease 77

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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Therapies Disease Clinical Trials Trials 52

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

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies 105

Therapies Engineering Molecular Biology Science 105