KIF1A

FEBRUARY 18, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Understanding the biology that links specific genetic mutations to patient symptoms could inform better therapies and interventions for patients with these disorders.

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Therapies DNA Clinical Trials Trials 52

Drug Discovery World

JULY 23, 2024

Dr Daniel Turner, Chief Scientific Officer, Enhanc3D Genomics, outlines how 3D genomics offers a radically novel approach to address challenges in drug target discovery and how it can affect change in drug target identification, cell therapy and personalised medicine.

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DNA Disease Regulations Molecular Biology 130

Drug Discovery World

JUNE 7, 2023

Hosted by DDW and sponsored by DNA Script, join us for this free-to-attend roundtable to learn about the biggest opportunities for the future of drug discovery and development. Panellists will discuss the following questions: Where will the opportunities be in 2024? What can we expect from the cell & gene therapy market?

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DNA Clinical Trials Drugs Vaccine 130

Drug Discovery World

JUNE 27, 2023

Hosted by DDW and supported by DNA Script, ‘Drug discovery and development trends – what will have most impact?’ Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer?

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DNA Drugs Clinical Trials Vaccine 130

Drug Discovery World

JUNE 22, 2023

Hosted by DDW and supported by DNA Script, ‘Drug discovery and development trends – what will have most impact?’ Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer?

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DNA Clinical Trials Drugs Vaccine 130

Drug Discovery World

JUNE 15, 2023

Hosted by DDW and sponsored by DNA Script, ‘Drug discovery and development trends – what will have most impact?’ Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer?

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DNA Drugs Clinical Trials Vaccine 130

Drug Discovery World

JUNE 8, 2023

Hosted by DDW and sponsored by DNA Script, ‘Drug discovery and development trends – what will have most impact?’ Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer?

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Drugs DNA Clinical Trials Vaccine 130

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. million DNA bases. million DNA bases.

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Broad Institute

OCTOBER 3, 2024

Largest-ever genetic study of epilepsy finds possible therapeutic targets By Ari Navetta October 3, 2024 Breadcrumb Home Largest-ever genetic study of epilepsy finds possible therapeutic targets Scientists have uncovered new genetic links to different types of epilepsy, which could lead to more tailored treatments.

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DNA Doctors Treatment Disease 72

Fierce BioTech

MAY 30, 2024

Quantify Cancer With Methylation-Based ctDNA Detection Download this resource to accelerate your oncology clinical trials, optimize patient enrollment, and monitor minimal residual disease and molecular response effectively.

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Clinical Trials DNA Trials Therapies 52

Broad Institute

JULY 10, 2024

We are hopeful that the use of prime editing to correct the predominant cause of cystic fibrosis might lead to a one-time, permanent treatment for this serious disease,” said Liu, the senior author on the study. There are more than 2,000 known variants of the CFTR gene, 700 of which cause disease. Online July 10, 2024.

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Drug Discovery World

APRIL 17, 2024

CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. For many diseases, only small increases in expression are needed to make the difference between sickness and health. What diseases are you currently targeting? Can you give some examples?

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Science RNA Disease Protein Expression 147

Broad Institute

JUNE 27, 2024

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. CHARMs are an elegant solution to the problem of silencing disease genes, and they have the potential to have an important position in the future of genetic medicines.”

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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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Drug Target Review

JULY 18, 2024

We believe liquid biopsy is very helpful for oncology drug development because the reduction and clearance of circulating DNA occurs quickly before radiographic imaging is available. The initial techniques were able to detect circulating tumour DNA in patients who already had metastatic spread. Nat Med 30, 531–542 (2024).

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Drug Development Clinical Trials Drugs Vaccine 59

Drug Discovery World

FEBRUARY 6, 2024

Auransa, which harnesses available data to discover highly intractable diseases with poorly understood biology. Pharos iBio, which utilises AI technology to develop treatments for refractory and rare diseases such as cancers and repurpose existing therapies.

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Drug Discovery World

FEBRUARY 28, 2024

Using a technique they’ve dubbed ‘cancer shredding’, the researchers at Gladstone Institutes, California, programmed CRISPR to zero-in on repeating DNA sequences present only in recurrent tumour cells – and then obliterate those cells by snipping away at them. Research into new cancer drugs remain strong.

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Drug Development Research Clinical Trials Drugs 162

PLOS: DNA Science

APRIL 18, 2024

Clues to combatting a devastating disease can come from identifying people who have gene variants – mutations – that protect them, by slowing the illness or lowering the risk that it develops in the first place. That’s what the researchers wanted to find out, publishing their findings in the January 2024 Cell.

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43 If the body fails to make the right protein, diseases such as cancer, type II diabetes, neurological conditions, and metabolic diseases can occur.

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The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. Occupancy of the new site is scheduled for 2024. Adams, vice president of genetic medicine at Lilly and co-director of the Institute. ” The Institute will be headquartered in 334,000 sq.

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RNA DNA Disease Therapies 52

Agency IQ

MARCH 29, 2024

FDA’s oncology advisory committee to consider MRD as a multiple myeloma endpoint Yesterday, the FDA announced that its Oncologic Drugs Advisory Committee would weigh in on the potential use of minimal residual disease (MRD) as an endpoint in multiple myeloma clinical trials. immune cells and other blood cell types) lack.

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The Pharma Data

JUNE 24, 2021

The study will evaluate the efficacy and safety of talazoparib, an oral poly (ADP-ribose) polymerase (PARP) inhibitor, in combination with enzalutamide, an androgen receptor inhibitor, compared with placebo plus enzalutamide in men with DNA damage response (DDR)-deficient metastatic castration-sensitive prostate cancer (mCSPC).

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DNA Clinical Trials Trials Biosimilars 52

KIF1A

APRIL 6, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. When a mutation causes a deletion or duplication of larger areas of DNA, we call it Copy Number Variation.

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DNA Medical Schools Therapies Research 111

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 118

KIF1A

JANUARY 13, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. As one cell turns into two, its DNA must be replicated and then pulled apart, so that when the cell divides each has all the DNA it needs to function.

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DNA Regulations Drug Development Disease 105

The Pharma Data

DECEMBER 14, 2020

It is considered an orphan disease for which there is no cure. billion by 2024, according to GlobalData, with the U.S. The current standard of care for GBM consists of de-bulking surgery followed by combined treatments with fractionated ionizing radiation (IR) and the DNA alkylating agent temozolomide (TMZ).

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DNA Clinical Trials Drugs Therapies 52

Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. DNA vectors compared to standard linear mRNA-based expression. “We

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RNA DNA Therapies Disease 130

Drug Discovery World

MAY 23, 2024

SD: The ability of RNA therapies to use the body’s own mechanisms to reprogram cells and tissues towards a healthy state offer a transformative approach to medicine, with early applications in the immunotherapy space. RA: Are multi-mechanistic or combination immunotherapies therapies better at fighting cancerous tumours?

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RNA Therapies Engineering Disease 147

KIF1A

JANUARY 27, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. The study found improved spasticity and movement in mice treated with ARL61P1 gene therapy; it also restored abnormalities observed in the brains of ARL61P1-deficient mice.

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Therapies Small Molecule DNA Treatment 111

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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Drug Development Treatment FDA Approval Drugs 52

Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Delivery dilemma Gene editing approaches promise to treat a range of diseases by precisely correcting genetic mutations that cause disease.

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Engineering Research Virus DNA 144

Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?

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Production FDA Therapies Biochemical Assays 40

Codon

JUNE 11, 2024

Biologists use nanopores for everything from diagnosing diseases and monitoring changes within rainforest ecosystems to discovering proteins from microbes frozen in Icelandic glaciers. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast. An Oxford Nanopore device.

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DNA Engineering RNA Small Molecule 73

KIF1A

FEBRUARY 4, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. But similar looking diseases won’t necessarily respond to the same treatments, which is why it’s so important to find accurate diagnoses. What is an allele specific ASO?

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RNA DNA Disease Trials 52

Codon

SEPTEMBER 29, 2024

2024 Statistical models of organisms have existed for decades. The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. By the end of 2024, interest in models of life had cooled.

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Engineering Virus DNA Therapies 125

Drug Discovery World

NOVEMBER 9, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’.

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Engineering Therapies Protein Production Science 130

Drug Discovery World

APRIL 25, 2024

The market for cell line development is currently rising due to an increased focus by the life sciences sector on biopharmaceuticals and targeted therapies. HEK 293 cells on the other hand are used within viral vector productions which have wide-scale applications for vaccines and therapies. Bacterial cell lines such as E.

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