Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

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Science Daily: Pharmacology News

JULY 27, 2023

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

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RNA Research Therapies Disease 183

Broad Institute

MARCH 12, 2025

By Leah Eisenstadt March 12, 2025 Credit: Broad Communications Scientists in the Spatial Technology Platform at the Broad develop, use, and share spatial -omics approaches, such as Perturb-FISH, that can reveal the roles of genes in various cell types and the impact of intercellular interactions on health and disease.

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Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.

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Drug Target Review

DECEMBER 6, 2024

This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. About the authors Dr Raminderpal Singh Dr Raminderpal Singh is a recognised visionary in the implementation of AI across technology and science-focused industries.

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RNA Drugs Research Science 52

Broad Institute

JANUARY 8, 2024

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. By Sarah C.P.

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Broad Institute

NOVEMBER 23, 2023

The work appears today in Science. The scientists found a surprising number and diversity of CRISPR systems, including ones that could make edits to DNA in human cells, others that can target RNA, and many with a variety of other functions. They studied several of the new systems in greater detail in the lab.

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RNA DNA Engineering Science 135

The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.

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RNA Small Molecule DNA Licensing 52

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

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DNA RNA Disease Hospitals 128

Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

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Therapies FDA Approval Engineering Disease 137

Broad Institute

JUNE 29, 2023

The method, described today in Science , provides a way for scientists to learn new details on how translation is regulated within individual cell types and how that changes in disease. By Sarah C.P. Williams June 29, 2023 Credit: Zeng H, Huang J, Ren J, et al. Paper cited Zeng, H. Online June 29, 2023. DOI: 10.1126/science.add3067

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Protein Production RNA Regulations Science 105

Broad Institute

AUGUST 31, 2023

Related News Next generation prime editing systems move closer to possible therapeutic applications New CRISPR genome editing system offers a wide range of versatility in human cells Prime editing technologies allow scientists to precisely edit the genome in a variety of ways and could one day be used to treat genetic diseases.

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Broad Institute

AUGUST 19, 2024

I’m so proud of this team and the partnership we formed to follow the science and set up some major positive impacts on women’s health,” Blainey said. Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Paper cited Zhu M et al.

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Hospitals Treatment Medical Schools Science 121

Broad Institute

AUGUST 31, 2023

We were surprised to see that just missing one copy of this gene can induce many changes at the RNA level, at the protein level, at the functional level, and at the behavioral level,” said Farsi. Tags: Stanley Center for Psychiatric Research Psychiatric Disease Paper(s) cited: Farsi Z, et al. Aug 31, 2023. DOI: 10.1016/j.neuron.2023.08.004.

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RNA Medical Schools Research Science 143

DrugBank

MAY 22, 2024

These tough nuts to crack in medical science—biological targets known to play roles in diseases but resistant to traditional drug design—are now seeing new strategies that shift the paradigm from "undruggable" to "druggable." Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

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Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications.

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Small Molecule Targeted Protein Degradation Science Disease 116

Broad Institute

OCTOBER 28, 2024

To speed up the annotation step, the Broad Institute’s Data Sciences Platform (DSP) has developed a new search engine that automates much of this process by using machine learning to search data on more than 50 million annotated single cells. Tags: Data Sciences Platform Machine Learning Single Cell Is CAS now available to use?

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Engineering Cell Biology Disease RNA 119

PLOS: DNA Science

JULY 13, 2023

Most studies on senescence are related to chronic inflammation, cancer, and age-related diseases. Analyzing RNA Revealed the Regeneration The researchers analyzed RNA to reconstruct the steps to regeneration. The researchers discovered RNA transcripts known to be involved in senescence in the disembodied heads.

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RNA DNA Research Disease 98

KIF1A

JANUARY 20, 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. Instead, this is what is called a splicing mutation; it occurs at the boundary between coding DNA that is read into RNA and protein, and noncoding DNA that is excluded.

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DNA RNA Disease Research 105

The Pharma Data

SEPTEMBER 28, 2020

This included compiling RNA data, as well as other data points such as patient demographics, clinical treatment arm and patient diagnosis. For years, patients diagnosed with a disease often received the same treatment. And for some people, that treatment worked.

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KIF1A

JULY 29, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A in New York: The 2023 KAND Family & Scientific Engagement Conference We won’t be posting next Saturday, but not for a lack of science!

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RNA Clinical Trials Disease Science 98

Broad Institute

MARCH 8, 2024

By Claire Hendershot March 8, 2024 Credit: National Institute of Allergy and Infectious Diseases, National Institutes of Health via U.S. Related news #WhyIScience Q&A: How a global collaboration is boosting science and public health in Senegal Why did you and your colleagues in Senegal decide to do this study?

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Research Disease Medical Schools RNA 111

DrugBank

MAY 15, 2024

This method was more about serendipity than science. Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. But as molecular biology has advanced, so too has our approach to finding new drugs.

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Small Molecule Molecular Biology Drugs Disease 98

The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets. About Novartis.

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Licensing Licensing Therapies Disease 52

Broad Institute

DECEMBER 13, 2023

Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. The researchers also discovered clues about cellular function and the potential roles of brain structures in disease. Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs.

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DNA Virus Research Engineering 140

Dark Matter Blog

JUNE 21, 2023

Since our company’s founding in 2015, we have taken the long view and been singularly focused on building an extremely flexible and broadly applicable platform that can develop a host of RNA‑targeted small molecules (rSMs) to deliver precision medicines for dozens of targets that have been out of reach for conventional approaches.

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Small Molecule RNA Pharma Companies Science 52

KIF1A

SEPTEMBER 16, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. #ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. What’s a pre-print?

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Regulations RNA Disease Research 105

Drug Target Review

SEPTEMBER 28, 2023

They had the science and the technology but not the business model. They needed reference materials for the disease in order to develop and validate diagnostic tests. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays. Twist was officially founded in 2013.

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The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We they can help many patients.”.

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The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma. doi: 10.1126/sciadv.abg9551. Source link: [link].

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Treatment RNA DNA Science 52

Broad Institute

SEPTEMBER 26, 2024

Luiz Mata Lopez By Rose Circeo September 26, 2024 Related Broad Summer Research Program Luiz Mata Lopez Luiz Mata Lopez is a junior studying computer science and mathematics at the University of Maryland, College Park. Morphological profiling of cells is commonly employed in drug discovery across many disease areas.

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RNA Science Disease Research 52

Dark Matter Blog

JANUARY 16, 2023

We are moving rapidly toward escape velocity in our RNA expedition at Arrakis. For those of you joining us for the first time, it’s worth a quick look back at why we decided to go all-in on the RNA world, why people thought we were crazy, and why we know we’re not. Our terra firma is small-molecule medicines.

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Drug Target Review

APRIL 3, 2024

Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Communication and networking are key in any career, with science being no exception.

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Drug Target Review

AUGUST 29, 2023

It’s also where I got exposed to career options beyond academia and learned about Flagship Pioneering, a venture creation firm that conceives, resources, and builds life science bio platform companies in health care and sustainability. One of my mentors asked me if I was ready to fail 90 percent of the time.

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Broad Institute

SEPTEMBER 26, 2024

Liya Mooradian By Rose Circeo September 26, 2024 Related Broad Summer Research Program Liya Mooradian Liya Mooradian is a senior studying Genetics and Science Communication at Iowa State University of Science and Technology. Non-coding genomic regions harbor 90% of disease-associated genetic variants.

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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. Although corticosteroids slow disease progression, weight gain and fragile bones develop with long-term use. Boys 6 years and older can take Duvystat, to slow the course of the illness.

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